'It's Transformed My Life': FDA Approves First Gene-Editing Treatment for Illness
UC Berkeley's Jennifer Doudna Shares Nobel Prize in Chemistry for CRISPR
China Scientist Who Edited Genes of Human Embryos Sentenced To Prison
What Two Sisters With a Rare Heart Condition Taught Doctors About Our Genes
CRISPR Wipes Out HIV in Some Mice
Here We Go Again: Feds Reopen Patent Dispute Between UC and Broad Institute
UC Partners With Pharmaceutical Giant on $67 Million CRISPR Lab
Making Sense of the CRISPR Patent Dispute Between the University of California and Broad
Science Summit Denounces Gene-Edited Babies Claim, But Not Future Research
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This brutal blood disorder has long been neglected by medical research.\u003c/p>\n\u003cp>The decisions are being hailed as milestones for treating sickle cell and for the rapidly advancing field of gene therapy, which is stirring excitement for the treatment of many diseases.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field, especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” says Dr. Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, in a statement. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”\u003c/p>\n\u003cp>“I’m elated, excited, in awe,” \u003ca href=\"https://vcresearch.berkeley.edu/faculty/jennifer-doudna\">Jennifer Doudna\u003c/a> of the University of California, Berkeley, who helped discover the gene-editing technique called \u003ca href=\"https://www.npr.org/series/773368439/the-crispr-revolution\">CRISPR\u003c/a> used in one of the sickle cell treatments, told NPR in an interview. “It’s an exciting day and the beginning of a new day in medicine.”\u003c/p>\n\u003cp>[pullquote align=\"right\" size=\"medium\" citation=\"Jennifer Doudna, biochemistry professor, UC Berkeley,\"]‘I’m elated, excited, in awe. It’s an exciting day and the beginning of a new day in medicine.’[/pullquote]For the CRISPR treatment, which was developed by \u003ca href=\"https://www.vrtx.com/\">Vertex Pharmaceuticals\u003c/a> and \u003ca href=\"https://crisprtx.com/\">CRISPR Therapeutics\u003c/a>, both in Boston, doctors remove cells from each patient’s bone marrow, edit a gene with CRISPR and then infuse billions of the modified cells back into patients.\u003c/p>\n\u003cp>The edited cells produce a form of hemoglobin known as fetal hemoglobin, restoring the normal function of red blood cells. While not a cure for the disease, the hope is the therapy, brand name Casgevy, is designed to be a one-time treatment that will alleviate symptoms for a lifetime.\u003c/p>\n\u003cp>In\u003ca href=\"https://www.fda.gov/media/173472/download\"> data presented to the FDA\u003c/a>, the treatment resolved the severe pain crises for at least 18 months for 29 of the subjects — 96.7%. The treatment has produced similar results for patients with a related condition known as \u003ca href=\"https://medlineplus.gov/genetics/condition/beta-thalassemia/\">beta thalassemia.\u003c/a>\u003c/p>\n\u003cp>The FDA approved another gene therapy called Lyfgenia, developed by \u003ca href=\"https://www.bluebirdbio.com/\">bluebird bio Inc\u003c/a>. of Somerville, Massachusetts, that doesn’t use CRISPR to treat sickle cell disease.\u003c/p>\n\u003ch2>Treatment comes with a high price\u003c/h2>\n\u003cp>However, the elation over the approvals was tempered by concerns the breakthrough treatments may not be accessible to many sickle cell patients.\u003c/p>\n\u003cp>They are both very expensive. Vertex says the wholesale price for Casgevy will be $2.2 million. Bluebird set the wholesale price of Lyfgenia at $3.1 million.\u003c/p>\n\u003cp>The treatments also require a complicated, arduous procedure many hospitals cannot provide. Many patients may find the treatment too physically and logistically daunting.\u003c/p>\n\u003cp>“We have a lot more work to do” to make gene-editing treatments widely available, Berkeley’s Doudna says.\u003c/p>\n\u003cp>Gene-editing, which allows scientists to manipulate the basic building blocks of life more easily than ever before, is being studied as a treatment for illnesses ranging from rare genetic disorders like muscular dystrophy to common ailments like cancer, heart disease, diabetes, AIDS and Alzheimer’s.\u003c/p>\n\u003cfigure id=\"attachment_1985712\" class=\"wp-caption alignnone\" style=\"max-width: 1024px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"size-full wp-image-1985712\" src=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920.jpg\" alt=\"A blond white woman in a lab coat stands looking to the camera with a smile and arms crossed in a laboratory as people work behind her.\" width=\"1024\" height=\"683\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920.jpg 1024w, https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920-800x534.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920-1020x680.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920-160x107.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920-768x512.jpg 768w\" sizes=\"(max-width: 1024px) 100vw, 1024px\">\u003cfigcaption class=\"wp-caption-text\">Jennifer Doudna, who helped discover the revolutionary gene-editing tool CRISPR, photographed in the Li Ka Shing Center on the Campus of UC Berkeley on Feb. 19, 2016. \u003ccite>(Nick Otto For The Washington Post via Getty Images)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>Sickle cell disease is caused by a genetic defect that produces an abnormal form of the protein hemoglobin, which red blood cells need to carry oxygen through the body. As a result, the red blood cells of sickle cell patients become misshapen sickle-shaped cells that get jammed inside blood vessels. That causes excruciating, unpredictable attacks of pain and damages vital organs, cutting patients’ lives short.\u003c/p>\n\u003cp>Sickle cell disproportionately occurs among people of African, Middle Eastern and Indian descent, affecting millions around the world and about 100,000 in the U.S. Although a rare disease, sickle cell is one of the most common genetic disorders.\u003c/p>\n\u003cp>Bone marrow transplants can cure some patients, but most can’t find a suitable donor. About 20,000 patients in the U.S. have the severe form of the disease the CRISPR treatment would initially be used to treat.\u003c/p>\n\u003cp>“I’m really excited,” Dr. \u003ca href=\"https://hospital.uillinois.edu/find-a-doctor/lewis-hsu\">Lewis Hsu\u003c/a>, a pediatric hematologist at the University of Illinois at Chicago who serves as the chief medical officer at the Sickle Cell Association of America, told NPR in an interview. “This is something that we’ve been waiting for in the sickle cell community for basically 70 years. This is a very big deal.”\u003c/p>\n\u003ch2>A life transformed\u003c/h2>\n\u003cp>The approval of the CRISPR gene-editing treatment was also welcomed by\u003ca href=\"https://www.npr.org/sections/health-shots/2019/12/25/784395525/a-young-mississippi-womans-journey-through-a-pioneering-gene-editing-experiment\"> Victoria Gray\u003c/a>, a Forest, Mississippi, sickle cell patient who was the first person to receive it in the U.S. NPR has had exclusive access to chronicle her experience since she was treated in 2019.\u003c/p>\n\u003cp>[pullquote align=\"right\" size=\"medium\" citation=\"Victoria Gray, sickle cell patient\"]‘Since I received the CRISPR treatment, I’ve had a new beginning. Most of all, I no longer have to fear dying and leaving my kids behind without a mother. My life is limitless now. I’m full of energy. I don’t have pain. It’s a real transformation.’[/pullquote]“I’m ecstatic. It’s a blessing that they approved this therapy. It’s a new beginning for people with sickle cell disease,” Gray told NPR in her latest interview with NPR.\u003c/p>\n\u003cp>Like many sickle cell patients, Gray was forced throughout her life to repeatedly rush to the hospital for powerful pain drugs and blood transfusions. She could not finish school, hold jobs or often even care for herself or her children.\u003c/p>\n\u003cp>“This has turned my life around. It gave me a new lease on life. It’s transformed my life more than I could have ever imagined,” Gray says.\u003c/p>\n\u003cp>Since the treatment, Gray has been much more energetic and able to start working full-time selling cosmetics at Walmart and spend more time with her four children, who are now teenagers.\u003c/p>\n\u003cp>“Since I received the CRISPR treatment, I’ve had a new beginning. Most of all, I no longer have to fear dying and leaving my kids behind without a mother,” Gray says. “My life is limitless now. I’m full of energy. I don’t have pain. It’s a real transformation.”\u003c/p>\n\u003ch2>Technical complexity and lengthy hospitalization\u003c/h2>\n\u003cp>Aside from the price of the treatments, another concern is the procedures are long, difficult and complex, requiring multiple trips to a hospital for testing, a grueling and potentially dangerous bone marrow transplant, and lengthy hospitalization. Those factors may put the treatment out of reach for those who need it most in the U.S., as well as in less affluent countries where the disease is most common.\u003c/p>\n\u003cp>[pullquote align=\"right\" size=\"medium\" citation=\"Melissa Creary, assistant professor, University of Michigan School of Public Health\"]‘I have a mixed reaction. … as this technology comes to market, it’s going to be really interesting to see the ways in which profit overtakes social justice.’[/pullquote]“I have a mixed reaction,” says \u003ca href=\"https://sph.umich.edu/faculty-profiles/creary-melissa.html\">Melissa Creary\u003c/a>, an assistant professor at the University of Michigan who studies sickle cell at the University of Michigan School of Public Health and has the disease herself. “I am excited about the promise that this technology has for those living with sickle cell disease. But as this technology comes to market, it’s going to be really interesting to see the ways in which profit overtakes social justice.”\u003c/p>\n\u003cp>Many of the countries where most sickle cell patients live don’t have enough sophisticated medical centers to provide complicated treatment. Even in the U.S., the treatment may not be widely available, making it difficult to access.\u003c/p>\n\u003cp>“Rural patients will likely to be at a disadvantage. And there might be whole states or regions with no gene-therapy options,” Hsu says.\u003c/p>\n\u003ch2>More gene-editing treatments are in the works\u003c/h2>\n\u003cp>Doudna heads a center at Berkeley to try to make gene-editing treatments simpler and, therefore, more accessible. The National Institutes of Health is also trying to address the problem.\u003c/p>\n\u003cp>The biotech companies say they are working with private and public insurers to cover the procedure. Advocates note that the high price could easily be offset by the savings of avoiding a lifetime of sickle cell complications.\u003c/p>\n\u003cp>Another concern is whether sufficient research had been done to spot “off-target” effects of the treatment — unintended editing errors that missed their mark in the DNA and that could potentially cause long-term health problems.\u003c/p>\n\u003cp>The companies plan to follow all the patients treated in the study for 15 years to see how long the benefits last, if the treatment actually helps patients live longer, and watch for any signs of long-term complications.\u003c/p>\n\u003cp>CRISPR-based treatments have also shown promise for treating a rare liver condition known as amyloidosis, as well as an inherited form of high cholesterol known as familial hypercholesterolemia.\u003c/p>\n\u003cp>“It’s only the beginning,” CRISPR researcher Doudna says.\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n","blocks":[],"excerpt":"The FDA approved two gene therapies for anyone 12 and older suffering from the most severe form of sickle cell disease, a brutal blood disorder long neglected by medical research.","status":"publish","parent":0,"modified":1704845806,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":36,"wordCount":1627},"headData":{"title":"'It's Transformed My Life': FDA Approves First Gene-Editing Treatment for Illness | KQED","description":"The FDA approved two gene therapies for anyone 12 and older suffering from the most severe form of sickle cell disease, a brutal blood disorder long neglected by medical research.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":""},"source":"NPR","sourceUrl":"https://www.kqed.org/arts/affiliate/npr","sticky":false,"nprByline":"\u003ca href=\"https://www.npr.org/people/146944972/rob-stein\">Rob Stein\u003c/a>","excludeFromSiteSearch":"Include","showOnAuthorArchivePages":"No","articleAge":"0","path":"/science/1985709/fda-approves-first-gene-editing-treatment-for-human-illness","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>In a landmark decision, the Food and Drug Administration on Friday approved the first gene-editing treatment to alleviate human illness.\u003c/p>\n\u003cp>The FDA approved two gene therapies for anyone 12 and older suffering from the most severe form of \u003ca href=\"https://www.nhlbi.nih.gov/health/sickle-cell-disease\">sickle cell disease\u003c/a>. This brutal blood disorder has long been neglected by medical research.\u003c/p>\n\u003cp>The decisions are being hailed as milestones for treating sickle cell and for the rapidly advancing field of gene therapy, which is stirring excitement for the treatment of many diseases.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field, especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” says Dr. Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research, in a statement. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”\u003c/p>\n\u003cp>“I’m elated, excited, in awe,” \u003ca href=\"https://vcresearch.berkeley.edu/faculty/jennifer-doudna\">Jennifer Doudna\u003c/a> of the University of California, Berkeley, who helped discover the gene-editing technique called \u003ca href=\"https://www.npr.org/series/773368439/the-crispr-revolution\">CRISPR\u003c/a> used in one of the sickle cell treatments, told NPR in an interview. “It’s an exciting day and the beginning of a new day in medicine.”\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"‘I’m elated, excited, in awe. It’s an exciting day and the beginning of a new day in medicine.’","name":"pullquote","attributes":{"named":{"align":"right","size":"medium","citation":"Jennifer Doudna, biochemistry professor, UC Berkeley,","label":""},"numeric":[]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>For the CRISPR treatment, which was developed by \u003ca href=\"https://www.vrtx.com/\">Vertex Pharmaceuticals\u003c/a> and \u003ca href=\"https://crisprtx.com/\">CRISPR Therapeutics\u003c/a>, both in Boston, doctors remove cells from each patient’s bone marrow, edit a gene with CRISPR and then infuse billions of the modified cells back into patients.\u003c/p>\n\u003cp>The edited cells produce a form of hemoglobin known as fetal hemoglobin, restoring the normal function of red blood cells. While not a cure for the disease, the hope is the therapy, brand name Casgevy, is designed to be a one-time treatment that will alleviate symptoms for a lifetime.\u003c/p>\n\u003cp>In\u003ca href=\"https://www.fda.gov/media/173472/download\"> data presented to the FDA\u003c/a>, the treatment resolved the severe pain crises for at least 18 months for 29 of the subjects — 96.7%. The treatment has produced similar results for patients with a related condition known as \u003ca href=\"https://medlineplus.gov/genetics/condition/beta-thalassemia/\">beta thalassemia.\u003c/a>\u003c/p>\n\u003cp>The FDA approved another gene therapy called Lyfgenia, developed by \u003ca href=\"https://www.bluebirdbio.com/\">bluebird bio Inc\u003c/a>. of Somerville, Massachusetts, that doesn’t use CRISPR to treat sickle cell disease.\u003c/p>\n\u003ch2>Treatment comes with a high price\u003c/h2>\n\u003cp>However, the elation over the approvals was tempered by concerns the breakthrough treatments may not be accessible to many sickle cell patients.\u003c/p>\n\u003cp>They are both very expensive. Vertex says the wholesale price for Casgevy will be $2.2 million. Bluebird set the wholesale price of Lyfgenia at $3.1 million.\u003c/p>\n\u003cp>The treatments also require a complicated, arduous procedure many hospitals cannot provide. Many patients may find the treatment too physically and logistically daunting.\u003c/p>\n\u003cp>“We have a lot more work to do” to make gene-editing treatments widely available, Berkeley’s Doudna says.\u003c/p>\n\u003cp>Gene-editing, which allows scientists to manipulate the basic building blocks of life more easily than ever before, is being studied as a treatment for illnesses ranging from rare genetic disorders like muscular dystrophy to common ailments like cancer, heart disease, diabetes, AIDS and Alzheimer’s.\u003c/p>\n\u003cfigure id=\"attachment_1985712\" class=\"wp-caption alignnone\" style=\"max-width: 1024px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"size-full wp-image-1985712\" src=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920.jpg\" alt=\"A blond white woman in a lab coat stands looking to the camera with a smile and arms crossed in a laboratory as people work behind her.\" width=\"1024\" height=\"683\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920.jpg 1024w, https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920-800x534.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920-1020x680.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920-160x107.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2023/12/GettyImages-528038920-768x512.jpg 768w\" sizes=\"(max-width: 1024px) 100vw, 1024px\">\u003cfigcaption class=\"wp-caption-text\">Jennifer Doudna, who helped discover the revolutionary gene-editing tool CRISPR, photographed in the Li Ka Shing Center on the Campus of UC Berkeley on Feb. 19, 2016. \u003ccite>(Nick Otto For The Washington Post via Getty Images)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>Sickle cell disease is caused by a genetic defect that produces an abnormal form of the protein hemoglobin, which red blood cells need to carry oxygen through the body. As a result, the red blood cells of sickle cell patients become misshapen sickle-shaped cells that get jammed inside blood vessels. That causes excruciating, unpredictable attacks of pain and damages vital organs, cutting patients’ lives short.\u003c/p>\n\u003cp>Sickle cell disproportionately occurs among people of African, Middle Eastern and Indian descent, affecting millions around the world and about 100,000 in the U.S. Although a rare disease, sickle cell is one of the most common genetic disorders.\u003c/p>\n\u003cp>Bone marrow transplants can cure some patients, but most can’t find a suitable donor. About 20,000 patients in the U.S. have the severe form of the disease the CRISPR treatment would initially be used to treat.\u003c/p>\n\u003cp>“I’m really excited,” Dr. \u003ca href=\"https://hospital.uillinois.edu/find-a-doctor/lewis-hsu\">Lewis Hsu\u003c/a>, a pediatric hematologist at the University of Illinois at Chicago who serves as the chief medical officer at the Sickle Cell Association of America, told NPR in an interview. “This is something that we’ve been waiting for in the sickle cell community for basically 70 years. This is a very big deal.”\u003c/p>\n\u003ch2>A life transformed\u003c/h2>\n\u003cp>The approval of the CRISPR gene-editing treatment was also welcomed by\u003ca href=\"https://www.npr.org/sections/health-shots/2019/12/25/784395525/a-young-mississippi-womans-journey-through-a-pioneering-gene-editing-experiment\"> Victoria Gray\u003c/a>, a Forest, Mississippi, sickle cell patient who was the first person to receive it in the U.S. NPR has had exclusive access to chronicle her experience since she was treated in 2019.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"‘Since I received the CRISPR treatment, I’ve had a new beginning. Most of all, I no longer have to fear dying and leaving my kids behind without a mother. My life is limitless now. I’m full of energy. I don’t have pain. It’s a real transformation.’","name":"pullquote","attributes":{"named":{"align":"right","size":"medium","citation":"Victoria Gray, sickle cell patient","label":""},"numeric":[]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>“I’m ecstatic. It’s a blessing that they approved this therapy. It’s a new beginning for people with sickle cell disease,” Gray told NPR in her latest interview with NPR.\u003c/p>\n\u003cp>Like many sickle cell patients, Gray was forced throughout her life to repeatedly rush to the hospital for powerful pain drugs and blood transfusions. She could not finish school, hold jobs or often even care for herself or her children.\u003c/p>\n\u003cp>“This has turned my life around. It gave me a new lease on life. It’s transformed my life more than I could have ever imagined,” Gray says.\u003c/p>\n\u003cp>Since the treatment, Gray has been much more energetic and able to start working full-time selling cosmetics at Walmart and spend more time with her four children, who are now teenagers.\u003c/p>\n\u003cp>“Since I received the CRISPR treatment, I’ve had a new beginning. Most of all, I no longer have to fear dying and leaving my kids behind without a mother,” Gray says. “My life is limitless now. I’m full of energy. I don’t have pain. It’s a real transformation.”\u003c/p>\n\u003ch2>Technical complexity and lengthy hospitalization\u003c/h2>\n\u003cp>Aside from the price of the treatments, another concern is the procedures are long, difficult and complex, requiring multiple trips to a hospital for testing, a grueling and potentially dangerous bone marrow transplant, and lengthy hospitalization. Those factors may put the treatment out of reach for those who need it most in the U.S., as well as in less affluent countries where the disease is most common.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"‘I have a mixed reaction. … as this technology comes to market, it’s going to be really interesting to see the ways in which profit overtakes social justice.’","name":"pullquote","attributes":{"named":{"align":"right","size":"medium","citation":"Melissa Creary, assistant professor, University of Michigan School of Public Health","label":""},"numeric":[]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>“I have a mixed reaction,” says \u003ca href=\"https://sph.umich.edu/faculty-profiles/creary-melissa.html\">Melissa Creary\u003c/a>, an assistant professor at the University of Michigan who studies sickle cell at the University of Michigan School of Public Health and has the disease herself. “I am excited about the promise that this technology has for those living with sickle cell disease. But as this technology comes to market, it’s going to be really interesting to see the ways in which profit overtakes social justice.”\u003c/p>\n\u003cp>Many of the countries where most sickle cell patients live don’t have enough sophisticated medical centers to provide complicated treatment. Even in the U.S., the treatment may not be widely available, making it difficult to access.\u003c/p>\n\u003cp>“Rural patients will likely to be at a disadvantage. And there might be whole states or regions with no gene-therapy options,” Hsu says.\u003c/p>\n\u003ch2>More gene-editing treatments are in the works\u003c/h2>\n\u003cp>Doudna heads a center at Berkeley to try to make gene-editing treatments simpler and, therefore, more accessible. The National Institutes of Health is also trying to address the problem.\u003c/p>\n\u003cp>The biotech companies say they are working with private and public insurers to cover the procedure. Advocates note that the high price could easily be offset by the savings of avoiding a lifetime of sickle cell complications.\u003c/p>\n\u003cp>Another concern is whether sufficient research had been done to spot “off-target” effects of the treatment — unintended editing errors that missed their mark in the DNA and that could potentially cause long-term health problems.\u003c/p>\n\u003cp>The companies plan to follow all the patients treated in the study for 15 years to see how long the benefits last, if the treatment actually helps patients live longer, and watch for any signs of long-term complications.\u003c/p>\n\u003cp>CRISPR-based treatments have also shown promise for treating a rare liver condition known as amyloidosis, as well as an inherited form of high cholesterol known as familial hypercholesterolemia.\u003c/p>\n\u003cp>“It’s only the beginning,” CRISPR researcher Doudna says.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"floatright"},"numeric":["floatright"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/science/1985709/fda-approves-first-gene-editing-treatment-for-human-illness","authors":["byline_science_1985709"],"categories":["science_39","science_3890","science_40","science_4450"],"tags":["science_1287","science_1050","science_4417","science_4414","science_327","science_190"],"featImg":"science_1985711","label":"source_science_1985709"},"science_1970166":{"type":"posts","id":"science_1970166","meta":{"index":"posts_1591205157","site":"science","id":"1970166","score":null,"sort":[1602072420000]},"guestAuthors":[],"slug":"pioneers-in-genetic-editing-win-nobel-prize-in-chemistry","title":"UC Berkeley's Jennifer Doudna Shares Nobel Prize in Chemistry for CRISPR","publishDate":1602072420,"format":"standard","headTitle":"UC Berkeley’s Jennifer Doudna Shares Nobel Prize in Chemistry for CRISPR | KQED","labelTerm":{},"content":"\u003cp>Two scientists won the Nobel Prize in chemistry on Wednesday for developing “molecular scissors” to edit genes, offering the promise of one day curing inherited diseases.\u003c/p>\n\u003cp>Working on opposite sides of the Atlantic, Frenchwoman Emmanuelle Charpentier and American Jennifer A. Doudna came up with a method known as CRISPR-cas9 that can be used to change the DNA of animals, plants and microorganisms. It was only the fourth time that a Nobel in the sciences was awarded exclusively to women, who have long received less recognition for their work than men in the prize’s 119-year history.\u003c/p>\n\u003cp>Charpentier and Doudna’s work allows for laser-sharp snips in the long strings of DNA that make up the “code of life,” enabling scientists to precisely edit specific genes to remove errors that lead to disease in humans — and is already being used for that purpose.\u003c/p>\n\u003cp>“There is enormous power in this genetic tool, which affects us all,” said Claes Gustafsson, chair of the Nobel Committee for Chemistry. “It has not only revolutionized basic science, but also resulted in innovative crops and will lead to groundbreaking new medical treatments.”\u003c/p>\n\u003cp>Gustafsson said that, as a result, any genome can now be edited “to fix genetic damage.”\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>Dr. Francis Collins, who led the drive to map the human genome, said the technology “has changed everything” about how to approach diseases with a genetic cause, such as sickle cell disease.\u003c/p>\n\u003cp>“You can draw a direct line from the success of the human genome project to the power of CRISPR-cas to make changes in the instruction book,” said Collins, director of the National Institutes of Health, which helped fund Doudna’s work.\u003c/p>\n\u003cp>But many also cautioned that the technology raises serious ethical questions and must be used carefully. Much of the world became more aware of CRISPR in 2018, when Chinese scientist He Jiankui revealed he had helped make the world’s first gene-edited babies, to try to engineer resistance to future infection with the AIDS virus. His work was denounced as unsafe human experimentation because of the risk of causing unintended changes that could pass to future generations, and he’s been sentenced to prison in China.\u003c/p>\n\u003cp>In September, an international panel of experts issued a report saying it’s still too soon to try to make genetically edited babies because the science isn’t advanced enough to ensure safety, but they mapped a pathway for countries that want to consider it.\u003c/p>\n\u003cp>“Being able to selectively edit genes means that you are playing God in a way,” said American Chemical Society President Luis Echegoyen, a chemistry professor at the University of Texas El Paso.\u003c/p>\n\u003cp>Charpentier, 51, spoke of the shock of winning.\u003c/p>\n\u003cp>“Strangely enough I was told a number of times (that I’d win), but when it happens you’re very surprised and you feel that it’s not real,” she told reporters by phone from Berlin after the award was announced in Stockholm by the Royal Swedish Academy of Sciences. “But obviously it’s real, so I have to get used to it now.”\u003c/p>\n\u003cp>When asked about the significance of two women winning, Charpentier said that while she considers herself first and foremost a scientist, “it’s reflective of the fact that science becomes more modern and involves more female leaders.”\u003c/p>\n\u003cp>“I do hope that it will remain and even develop more in this direction,” she said, adding that it’s “more cumbersome to be a woman in science than to be a man in science.”\u003c/p>\n\u003cp>Three times a woman has won a Nobel in the sciences by herself; this is the first time an all-female team won a science prize. In 1911, Marie Curie was the sole recipient of the chemistry award, as was Dorothy Crowfoot Hodgkin in 1964. In 1983, Barbara McClintock won the Nobel for medicine.\u003c/p>\n\u003cp>Doudna told The Associated Press of her own surprise — including that she learned she’d won from a reporter.\u003c/p>\n\u003cp>“I literally just found out, I’m in shock,” she said. “I was sound asleep.\u003c/p>\n\u003cp>“My greatest hope is that it’s used for good, to uncover new mysteries in biology and to benefit humankind,” said Doudna, who is affiliated with UC Berkeley and is paid by the Howard Hughes Medical Institute, which also supports AP’s Health and Science Department.\u003c/p>\n\u003cp>The breakthrough research done by Charpentier and Doudna was published in 2012, making the discovery very recent compared to much Nobel-wining research, which is often only honored after decades have passed.\u003c/p>\n\u003cp>Speaking to reporters from the Max Planck Unit for the Science of Pathogens in Berlin, which she leads, Charpentier said despite how recently it was developed, the method is now widely used by scientists researching diseases, developing drugs and engineering new plants.\u003c/p>\n\u003cp>Among the most promising therapies already being developed are for eye diseases and blood disorders, such as sickle cell disease and beta thalassemia, she said. It could also have applications in the growing field of cancer immunotherapy.\u003c/p>\n\u003cp>Developing hardy crops is another promising direction, said Charpentier. “I think this is very important considering the challenge we are facing of climate change,” she said.\u003c/p>\n\u003cp>The Broad Institute at Harvard and MIT have been in a long court fight over patents on CRISPR technology, and many other scientists did important work on it, but Doudna and Charpentier have been most consistently honored with prizes for turning it into an easily usable tool.\u003c/p>\n\u003cp>The prestigious award comes with a gold medal and prize money of 10 million kronor (more than $1.1 million), courtesy of a bequest left more than a century ago by the prize’s creator, Swedish inventor Alfred Nobel. The amount was increased recently to adjust for inflation.\u003c/p>\n\u003cp>On Monday, the Nobel Committee awarded the prize for physiology and medicine for discovering the liver-ravaging hepatitis C virus. Tuesday’s prize for physics honored breakthroughs in understanding the mysteries of cosmic black holes.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>The other prizes are for outstanding work in the fields of literature, peace and economics.\u003c/p>\n\n","blocks":[],"excerpt":"Jennifer Doudna and Emmanuelle Charpentier discovered a way to cut into broken genetic material, remove it and replace it. ","status":"publish","parent":0,"modified":1704847009,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":28,"wordCount":1058},"headData":{"title":"UC Berkeley's Jennifer Doudna Shares Nobel Prize in Chemistry for CRISPR | KQED","description":"Jennifer Doudna and Emmanuelle Charpentier discovered a way to cut into broken genetic material, remove it and replace it. ","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":""},"source":"Nobel Prize","sticky":false,"nprByline":"David Keyton, Christina Larson and Frank Jordans\u003cbr>Associated Press","path":"/science/1970166/pioneers-in-genetic-editing-win-nobel-prize-in-chemistry","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Two scientists won the Nobel Prize in chemistry on Wednesday for developing “molecular scissors” to edit genes, offering the promise of one day curing inherited diseases.\u003c/p>\n\u003cp>Working on opposite sides of the Atlantic, Frenchwoman Emmanuelle Charpentier and American Jennifer A. Doudna came up with a method known as CRISPR-cas9 that can be used to change the DNA of animals, plants and microorganisms. It was only the fourth time that a Nobel in the sciences was awarded exclusively to women, who have long received less recognition for their work than men in the prize’s 119-year history.\u003c/p>\n\u003cp>Charpentier and Doudna’s work allows for laser-sharp snips in the long strings of DNA that make up the “code of life,” enabling scientists to precisely edit specific genes to remove errors that lead to disease in humans — and is already being used for that purpose.\u003c/p>\n\u003cp>“There is enormous power in this genetic tool, which affects us all,” said Claes Gustafsson, chair of the Nobel Committee for Chemistry. “It has not only revolutionized basic science, but also resulted in innovative crops and will lead to groundbreaking new medical treatments.”\u003c/p>\n\u003cp>Gustafsson said that, as a result, any genome can now be edited “to fix genetic damage.”\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>Dr. Francis Collins, who led the drive to map the human genome, said the technology “has changed everything” about how to approach diseases with a genetic cause, such as sickle cell disease.\u003c/p>\n\u003cp>“You can draw a direct line from the success of the human genome project to the power of CRISPR-cas to make changes in the instruction book,” said Collins, director of the National Institutes of Health, which helped fund Doudna’s work.\u003c/p>\n\u003cp>But many also cautioned that the technology raises serious ethical questions and must be used carefully. Much of the world became more aware of CRISPR in 2018, when Chinese scientist He Jiankui revealed he had helped make the world’s first gene-edited babies, to try to engineer resistance to future infection with the AIDS virus. His work was denounced as unsafe human experimentation because of the risk of causing unintended changes that could pass to future generations, and he’s been sentenced to prison in China.\u003c/p>\n\u003cp>In September, an international panel of experts issued a report saying it’s still too soon to try to make genetically edited babies because the science isn’t advanced enough to ensure safety, but they mapped a pathway for countries that want to consider it.\u003c/p>\n\u003cp>“Being able to selectively edit genes means that you are playing God in a way,” said American Chemical Society President Luis Echegoyen, a chemistry professor at the University of Texas El Paso.\u003c/p>\n\u003cp>Charpentier, 51, spoke of the shock of winning.\u003c/p>\n\u003cp>“Strangely enough I was told a number of times (that I’d win), but when it happens you’re very surprised and you feel that it’s not real,” she told reporters by phone from Berlin after the award was announced in Stockholm by the Royal Swedish Academy of Sciences. “But obviously it’s real, so I have to get used to it now.”\u003c/p>\n\u003cp>When asked about the significance of two women winning, Charpentier said that while she considers herself first and foremost a scientist, “it’s reflective of the fact that science becomes more modern and involves more female leaders.”\u003c/p>\n\u003cp>“I do hope that it will remain and even develop more in this direction,” she said, adding that it’s “more cumbersome to be a woman in science than to be a man in science.”\u003c/p>\n\u003cp>Three times a woman has won a Nobel in the sciences by herself; this is the first time an all-female team won a science prize. In 1911, Marie Curie was the sole recipient of the chemistry award, as was Dorothy Crowfoot Hodgkin in 1964. In 1983, Barbara McClintock won the Nobel for medicine.\u003c/p>\n\u003cp>Doudna told The Associated Press of her own surprise — including that she learned she’d won from a reporter.\u003c/p>\n\u003cp>“I literally just found out, I’m in shock,” she said. “I was sound asleep.\u003c/p>\n\u003cp>“My greatest hope is that it’s used for good, to uncover new mysteries in biology and to benefit humankind,” said Doudna, who is affiliated with UC Berkeley and is paid by the Howard Hughes Medical Institute, which also supports AP’s Health and Science Department.\u003c/p>\n\u003cp>The breakthrough research done by Charpentier and Doudna was published in 2012, making the discovery very recent compared to much Nobel-wining research, which is often only honored after decades have passed.\u003c/p>\n\u003cp>Speaking to reporters from the Max Planck Unit for the Science of Pathogens in Berlin, which she leads, Charpentier said despite how recently it was developed, the method is now widely used by scientists researching diseases, developing drugs and engineering new plants.\u003c/p>\n\u003cp>Among the most promising therapies already being developed are for eye diseases and blood disorders, such as sickle cell disease and beta thalassemia, she said. It could also have applications in the growing field of cancer immunotherapy.\u003c/p>\n\u003cp>Developing hardy crops is another promising direction, said Charpentier. “I think this is very important considering the challenge we are facing of climate change,” she said.\u003c/p>\n\u003cp>The Broad Institute at Harvard and MIT have been in a long court fight over patents on CRISPR technology, and many other scientists did important work on it, but Doudna and Charpentier have been most consistently honored with prizes for turning it into an easily usable tool.\u003c/p>\n\u003cp>The prestigious award comes with a gold medal and prize money of 10 million kronor (more than $1.1 million), courtesy of a bequest left more than a century ago by the prize’s creator, Swedish inventor Alfred Nobel. The amount was increased recently to adjust for inflation.\u003c/p>\n\u003cp>On Monday, the Nobel Committee awarded the prize for physiology and medicine for discovering the liver-ravaging hepatitis C virus. Tuesday’s prize for physics honored breakthroughs in understanding the mysteries of cosmic black holes.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>The other prizes are for outstanding work in the fields of literature, peace and economics.\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/science/1970166/pioneers-in-genetic-editing-win-nobel-prize-in-chemistry","authors":["byline_science_1970166"],"categories":["science_29","science_39","science_3890","science_40"],"tags":["science_1287","science_4414","science_5181","science_1943"],"featImg":"science_1970169","label":"source_science_1970166"},"science_1954209":{"type":"posts","id":"science_1954209","meta":{"index":"posts_1591205157","site":"science","id":"1954209","score":null,"sort":[1577815447000]},"guestAuthors":[],"slug":"china-scientist-who-edited-genes-of-human-embryos-sentenced-to-prison","title":"China Scientist Who Edited Genes of Human Embryos Sentenced To Prison","publishDate":1577815447,"format":"standard","headTitle":"China Scientist Who Edited Genes of Human Embryos Sentenced To Prison | KQED","labelTerm":{},"content":"\u003cp>A Chinese scientist who shocked the medical community last year when he said he had illegally created the world’s first gene-edited babies has been sentenced to three years in prison by a court in southern China.\u003c/p>\n\u003cp>He Jiankui announced in November 2018 that he had used a powerful technique called CRISPR on a human embryo to edit the genes of twin girls. He said he modified a gene with the intention of protecting the girls against HIV, the virus that causes AIDS. Many scientists expressed concerns about possible unintended side effects of the genetic changes that could be passed down to future generations.\u003c/p>\n\u003cp>Last fall, He also indicated there might be another pregnancy involving a gene-edited embryo. The court indicated that three genetically edited babies have been born.\u003c/p>\n\u003cp>The closed court in Shenzhen found He and two colleagues guilty of illegal medical practice by knowingly violating the country’s regulations and ethical principles with their experiments, \u003ca href=\"http://www.xinhuanet.com/english/2019-12/30/c_138666754.htm\">Xinhua news agency\u003c/a> reported. It also ordered He to pay a fine of about $430,000.\u003c/p>\n\u003cp>He’s colleagues, Zhang Renli and Qin Jinzhou, were handed lesser sentences and fines.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>“None of the three defendants acquired doctor’s qualifications. [They] craved fame and fortune and deliberately went against the country’s regulations on scientific research and medical management. [They] went beyond the bottom lines of scientific research and medical ethics,” the court stated, according to the \u003ca href=\"https://www.scmp.com/news/china/science/article/3043894/chinas-gene-editing-frankenstein-jailed-3-years-modified-baby\">South China Morning Post\u003c/a>.\u003c/p>\n\u003cp>He has defended his controversial work by saying that it will help families. “I understand my work will be controversial,” he said, as \u003ca href=\"https://www.npr.org/sections/health-shots/2018/11/26/670752865/chinese-scientist-says-hes-first-to-genetically-edit-babies\">NPR’s Rob Stein reported\u003c/a>. “But I believe families need this technology. And I am willing to take the criticism for them.”\u003c/p>\n\u003cp>At the time, scientists had previously genetically modified human embryos, but none had publicly claimed to have implanted embryos in a woman’s womb in an experiment that resulted in human babies.\u003c/p>\n\u003cp>Chinese police detained He in January and, as the \u003cem>Post\u003c/em> reported, an initial investigation concluded that he “organised a project team that included foreign staff, which intentionally avoided surveillance and used technology of uncertain safety and effectiveness to perform human embryo gene-editing activity with the purpose of reproduction, which is officially banned in the country.”\u003c/p>\n\u003cp>The gene that He edited, CCR5, is known as a pathway for HIV to infect immune system cells. But \u003ca href=\"https://www.npr.org/sections/health-shots/2019/06/03/727957768/2-chinese-babies-with-edited-genes-may-face-higher-risk-of-premature-death\">as Stein notes\u003c/a>, research carried out since He’s stunning announcement has suggested that the genetic changes he made could cause more harm than good to the babies’ health.\u003c/p>\n\u003cp>A study \u003ca href=\"https://www.nature.com/articles/s41591-019-0459-6\">in Nature Medicine\u003c/a> analyzed the DNA of more than 400,000 people and found that the changes that He made could make people more vulnerable to viruses such as West Nile and influenza.\u003c/p>\n\u003cp>“This is a lesson in humility,” \u003ca href=\"https://daley.med.harvard.edu/\">George Daley\u003c/a>, the dean of the Harvard Medical School, told Stein. “Even when we think we know something about a gene, we can always be surprised and even startled, like in this case, to find out that a gene we thought was protective may actually be a problem.”\u003c/p>\n\u003cp>Marcy Darnovsky, the executive director of the Center for Genetics and Society, said in an email to NPR that He’s “reckless and self-serving acts should highlight the broader and deeper risks — and the pointlessness — of any proposal to use gene editing in human reproduction.”\u003c/p>\n\u003cp>William Hurlbut, a scientist and bioethicist at Stanford who had attempted to persuade He (who is nicknamed JK) not to do the experiment, called his arrest a “sad story.”\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>“Everyone lost in this (JK, his family, his colleagues, and his country), but the one gain is that the world is awakened to the seriousness of our advancing genetic technologies,” Hurlbut said in an emailed statement. “I feel sorry for JK’s little family though — I warned him things could end this way, but it was just too late.”\u003c/p>\n\u003cdiv class=\"fullattribution\">Copyright 2019 NPR. To see more, visit https://www.npr.org.\u003cimg decoding=\"async\" src=\"https://www.google-analytics.com/__utm.gif?utmac=UA-5828686-4&utmdt=Chinese+Researcher+Who+Created+Gene-Edited+Babies+Sentenced+To+3+Years+In+Prison&utme=8(APIKey)9(MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004)\">\u003c/div>\n\n","blocks":[],"excerpt":"He Jiankui announced in November 2018 that he had created the world's first gene-edited babies. Scientists are concerned about unintended side effects that could be passed down to future generations. ","status":"publish","parent":0,"modified":1704847955,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":17,"wordCount":690},"headData":{"title":"China Scientist Who Edited Genes of Human Embryos Sentenced To Prison | KQED","description":"He Jiankui announced in November 2018 that he had created the world's first gene-edited babies. Scientists are concerned about unintended side effects that could be passed down to future generations. ","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":""},"source":"CRISPR","sticky":false,"nprImageCredit":"Kin Cheung","nprByline":"Merrit Kennedy \u003cbr /> NPR \u003cbr>","nprImageAgency":"AP","nprStoryId":"792340177","nprApiLink":"http://api.npr.org/query?id=792340177&apiKey=MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004","nprHtmlLink":"https://www.npr.org/2019/12/30/792340177/chinese-researcher-who-created-gene-edited-babies-sentenced-to-3-years-in-prison?ft=nprml&f=792340177","nprRetrievedStory":"1","nprPubDate":"Mon, 30 Dec 2019 14:22:00 -0500","nprStoryDate":"Mon, 30 Dec 2019 12:07:05 -0500","nprLastModifiedDate":"Mon, 30 Dec 2019 14:22:45 -0500","path":"/science/1954209/china-scientist-who-edited-genes-of-human-embryos-sentenced-to-prison","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>A Chinese scientist who shocked the medical community last year when he said he had illegally created the world’s first gene-edited babies has been sentenced to three years in prison by a court in southern China.\u003c/p>\n\u003cp>He Jiankui announced in November 2018 that he had used a powerful technique called CRISPR on a human embryo to edit the genes of twin girls. He said he modified a gene with the intention of protecting the girls against HIV, the virus that causes AIDS. Many scientists expressed concerns about possible unintended side effects of the genetic changes that could be passed down to future generations.\u003c/p>\n\u003cp>Last fall, He also indicated there might be another pregnancy involving a gene-edited embryo. The court indicated that three genetically edited babies have been born.\u003c/p>\n\u003cp>The closed court in Shenzhen found He and two colleagues guilty of illegal medical practice by knowingly violating the country’s regulations and ethical principles with their experiments, \u003ca href=\"http://www.xinhuanet.com/english/2019-12/30/c_138666754.htm\">Xinhua news agency\u003c/a> reported. It also ordered He to pay a fine of about $430,000.\u003c/p>\n\u003cp>He’s colleagues, Zhang Renli and Qin Jinzhou, were handed lesser sentences and fines.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>“None of the three defendants acquired doctor’s qualifications. [They] craved fame and fortune and deliberately went against the country’s regulations on scientific research and medical management. [They] went beyond the bottom lines of scientific research and medical ethics,” the court stated, according to the \u003ca href=\"https://www.scmp.com/news/china/science/article/3043894/chinas-gene-editing-frankenstein-jailed-3-years-modified-baby\">South China Morning Post\u003c/a>.\u003c/p>\n\u003cp>He has defended his controversial work by saying that it will help families. “I understand my work will be controversial,” he said, as \u003ca href=\"https://www.npr.org/sections/health-shots/2018/11/26/670752865/chinese-scientist-says-hes-first-to-genetically-edit-babies\">NPR’s Rob Stein reported\u003c/a>. “But I believe families need this technology. And I am willing to take the criticism for them.”\u003c/p>\n\u003cp>At the time, scientists had previously genetically modified human embryos, but none had publicly claimed to have implanted embryos in a woman’s womb in an experiment that resulted in human babies.\u003c/p>\n\u003cp>Chinese police detained He in January and, as the \u003cem>Post\u003c/em> reported, an initial investigation concluded that he “organised a project team that included foreign staff, which intentionally avoided surveillance and used technology of uncertain safety and effectiveness to perform human embryo gene-editing activity with the purpose of reproduction, which is officially banned in the country.”\u003c/p>\n\u003cp>The gene that He edited, CCR5, is known as a pathway for HIV to infect immune system cells. But \u003ca href=\"https://www.npr.org/sections/health-shots/2019/06/03/727957768/2-chinese-babies-with-edited-genes-may-face-higher-risk-of-premature-death\">as Stein notes\u003c/a>, research carried out since He’s stunning announcement has suggested that the genetic changes he made could cause more harm than good to the babies’ health.\u003c/p>\n\u003cp>A study \u003ca href=\"https://www.nature.com/articles/s41591-019-0459-6\">in Nature Medicine\u003c/a> analyzed the DNA of more than 400,000 people and found that the changes that He made could make people more vulnerable to viruses such as West Nile and influenza.\u003c/p>\n\u003cp>“This is a lesson in humility,” \u003ca href=\"https://daley.med.harvard.edu/\">George Daley\u003c/a>, the dean of the Harvard Medical School, told Stein. “Even when we think we know something about a gene, we can always be surprised and even startled, like in this case, to find out that a gene we thought was protective may actually be a problem.”\u003c/p>\n\u003cp>Marcy Darnovsky, the executive director of the Center for Genetics and Society, said in an email to NPR that He’s “reckless and self-serving acts should highlight the broader and deeper risks — and the pointlessness — of any proposal to use gene editing in human reproduction.”\u003c/p>\n\u003cp>William Hurlbut, a scientist and bioethicist at Stanford who had attempted to persuade He (who is nicknamed JK) not to do the experiment, called his arrest a “sad story.”\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>“Everyone lost in this (JK, his family, his colleagues, and his country), but the one gain is that the world is awakened to the seriousness of our advancing genetic technologies,” Hurlbut said in an emailed statement. “I feel sorry for JK’s little family though — I warned him things could end this way, but it was just too late.”\u003c/p>\n\u003cdiv class=\"fullattribution\">Copyright 2019 NPR. To see more, visit https://www.npr.org.\u003cimg decoding=\"async\" src=\"https://www.google-analytics.com/__utm.gif?utmac=UA-5828686-4&utmdt=Chinese+Researcher+Who+Created+Gene-Edited+Babies+Sentenced+To+3+Years+In+Prison&utme=8(APIKey)9(MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004)\">\u003c/div>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/science/1954209/china-scientist-who-edited-genes-of-human-embryos-sentenced-to-prison","authors":["byline_science_1954209"],"categories":["science_3890","science_40"],"tags":["science_1287","science_3838","science_2936"],"featImg":"science_1954210","label":"source_science_1954209"},"science_1945458":{"type":"posts","id":"science_1945458","meta":{"index":"posts_1591205157","site":"science","id":"1945458","score":null,"sort":[1564066804000]},"guestAuthors":[],"slug":"what-two-sisters-with-a-rare-heart-condition-taught-doctors-about-our-genes","title":"What Two Sisters With a Rare Heart Condition Taught Doctors About Our Genes","publishDate":1564066804,"format":"standard","headTitle":"What Two Sisters With a Rare Heart Condition Taught Doctors About Our Genes | KQED","labelTerm":{"site":"science"},"content":"\u003cp>Early in February of 2008, just days after she was born, Tatiana Legkiy lay in a cardiac intensive care unit, her tiny body hooked up to a respirator. After crying for two hours, she was now briefly quiet, the tube in her throat helping her breathe but also preventing her from making any sound.\u003c/p>\n\u003cp>Tatiana’s heart was failing. A cardiologist, tipped off by a pediatrician who heard something strange in a routine checkup, had examined her earlier that day and grown worried. He sent Tatiana to a nearby hospital in Modesto, California, where she remained for only an hour before being whisked eighty miles west by ambulance to the UCSF Benioff Children’s Hospital.\u003c/p>\n\u003cp>Tatiana’s parents arrived at the hospital shortly afterwards. At the time, Lana and Andrey Legkiy lived in Manteca, a city in California’s Central Valley. Andrey worked at an animal supply company; Lana stayed home and took care of their four-year-old daughter, Anna. Weekends found the the family outside together, camping or fishing in the Delta where the San Joaquin and Sacramento rivers flow into the ocean.\u003c/p>\n\u003cp>Even before Tatiana’s birth, the Legkiys knew medical hardship well. A year earlier Lana had suffered a miscarriage, losing an unborn child, a boy, in her third trimester. At the time, physicians had ascribed his cause of death to pulmonary hypoplasia, or incomplete development of the lungs.\u003c/p>\n\u003cp>Given that medical history, when Tatiana arrived at Benioff in critical condition, the doctors requested slides from the unborn child’s autopsy. This time, taking a closer look at small samples of heart tissue, they noted the true cause of death — an extremely rare heart condition known as left ventricular noncompaction (LVNC), in which the heart muscle remains immature and cannot pump blood normally.\u003c/p>\n\u003cp>Visually, physicians identify LVNC by the fingerlike protrusions of muscle extending from the wall of the heart into the left ventricle, which supplies the body with oxygen-rich blood. An echocardiogram of Tatiana’s heart showed these same characteristics.\u003c/p>\n\u003cp>Gently, the physicians told Tatiana’s parents that there would be no surgery, but only because LVNC has no cure.\u003c/p>\n\u003cp>\u003cstrong>A Hunch\u003c/strong>\u003c/p>\n\u003cp>Immediately, Deepak Srivastava, then-attending physician at Benioff, suspected a genetic connection. He was a geneticist, after all, and Tatiana’s unborn sibling had shared the same disease.\u003c/p>\n\u003cp>Though he could not have foreseen it as Tatiana struggled for her life that day, proving this intuition would require over a decade of work. It would also require technology which was only just becoming usable, and the dedication of researchers he had not yet met.\u003c/p>\n\u003cp>In the coming years, Srivastava would move forward with his own career, juggling the roles of biology professor, pediatric cardiologist, and ultimately president of Gladstone Institutes, a nonprofit biomedical research institution in San Francisco.\u003c/p>\n\u003cp>But the story of the Legkiys would stay with him. For a decade, he wouldn’t be able to shake the desire to pinpoint a precise genetic cause of LVNC, a necessary first step towards finding a cure for the disease. With that knowledge, physicians could rapidly screen potential drugs using an accurate, personalized model for patients like Tatiana.\u003c/p>\n\u003cfigure id=\"attachment_1945469\" class=\"wp-caption alignnone\" style=\"max-width: 640px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"wp-image-1945469 size-large\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-1020x765.jpg\" alt=\"\" width=\"640\" height=\"480\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-1020x765.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-160x120.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-800x600.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-768x576.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-1200x900.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009.jpg 1600w\" sizes=\"(max-width: 640px) 100vw, 640px\">\u003cfigcaption class=\"wp-caption-text\">Anna and Tatiana Legkiy in 2009, just after Tatiana came off medication. \u003ccite>(Lana Legkiy)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>Adding to the urgency of the Legkiys’ situation: Tatiana was not their only child. Even as Tatiana received her diagnosis in 2008, her happy four-year-old sister, Anna, ran around the hospital waiting room. Blissfully oblivious, Anna charmed the physicians, who began to wonder: did all three Legkiy children share the same disease?\u003c/p>\n\u003cp>As Tatiana stabilized, thanks to medication helping her heart pump and a respirator helping her breathe, Srivastava’s team set about finding an answer. Two days after diagnosing Tatiana, the scientists took an echocardiogram, a kind of ultrasound, of her father’s heart. They observed that Andrey had a less severe, asymptomatic case of Tatiana’s condition.\u003c/p>\n\u003cp>[pullquote size='medium' align='right' citation='Deepak Srivastava, Gladstone Institutes']‘This is a family who had lost one child, their other child was in a life-threatening situation, and then they find out their other girl has the same thing… it was actually pretty devastating.’[/pullquote]\u003c/p>\n\u003cp>So by the time they tested Anna’s heart the following day, Lana Legkiy already knew more than a mother should about what LVNC looked like in an ultrasound. (The team would later examine Lana’s own heart, and find it normal.)\u003c/p>\n\u003cp>When she saw Anna’s heart up on the screen, she knew without being told that her daughters shared the same disease.\u003c/p>\n\u003cp>Today, Lana can’t remember precisely what she felt in that moment— so much happened at once — but she does recall a dream she had while in the hospital, after Tatiana’s diagnosis. Lana doesn’t consider herself a true believer of supernatural occurrences, but she thinks dreams are important. “I saw both of them… as grown women,” she said. “I think that settled me down a little bit, like, ‘Okay, we’re going to be okay.’”\u003c/p>\n\u003cp>Srivastava, however, remembers the Legkiys’ distress acutely. “It was actually very sad,” he said. “This is a family who had lost one child, their other child was in a life-threatening situation, and then they find out their other girl has the same thing… it was actually pretty devastating.”\u003c/p>\n\u003cp>\u003cstrong>The Long Search for Proof\u003c/strong>\u003c/p>\n\u003cp>As the physicians told the Legkiys, there was and is still no cure or surgical procedure that can remedy LVNC. In cases such as Anna’s, the heart compensates for its left ventricle and swells, allowing it to pump more blood through the body. Anna, like others with enlarged hearts, did not display typical symptoms of heart dysfunction such as fatigue or rapid breathing.\u003c/p>\n\u003cp>Srivastava, however, told the Legkiys that both girls’ hearts would need to be monitored with yearly ultrasounds. Tatiana was sent home with more of the medication that helped her heart pump; she would take the medicine for a year before her heart muscles improved and the medication was no longer necessary. The family resumed what was, for the most part, a normal life.\u003c/p>\n\u003cp>[pullquote size='small' align='left' citation='Lea Starita, University of Washington']‘For years, I think we’ve known… that not all pathogenicities were going to be caused by single genes. I don’t think there’s ever been a study that so well defined the interaction between three human variants on a phenotype like this.’[/pullquote]As Anna and Tatiana grew into young women, now 15 and 11, Srivastava and his coworkers set about pinpointing the precise causes of the family’s congenital heart disease. The team sequenced the family’s DNA, combing through their genes in search of the variants all three children shared, screening against frequency of occurrence in the general population and for association with the heart.\u003c/p>\n\u003cp>The scientists quickly found three mutations in three different genes shared by the three siblings that they suspected to be the cause of the disease, two inherited from Andrey (\u003cem>MKL2\u003c/em> and \u003cem>MYH7\u003c/em>) and one from Lana (\u003cem>NKX2-5\u003c/em>). If not for the advent of a few key technologies, the investigation would have stopped here, the Legkiys left to wonder what was hidden in their genes. Srivastava’s search for proof, however, was just beginning.\u003c/p>\n\u003cp>\u003cstrong>CRISPR’s Perfect Timing\u003c/strong>\u003c/p>\n\u003cp>By 2014, the Legkiys were living in Seattle; Tatiana and Anna were six and 10, both medication-free. Tatiana’s hospital experience seemed like a distant memory.\u003c/p>\n\u003cp>In San Francisco, meanwhile, Casey Gifford, a recent PhD from Harvard, had joined Srivastava’s research team. New to both human genomics and heart disease, she and Srivastava spent a four-hour car ride to a conference in Lake Tahoe discussing what they thought would be the next big questions in medicine. Srivastava brought up the family he couldn’t forget — and realized, as he did so, that the tools they would need to prove which genetic mutations caused LVNC in the Legkiys might finally exist.\u003c/p>\n\u003cp>\u003ca href=\"https://www.kqed.org/futureofyou/436872/explainer-the-new-gene-editing-tool-significantly-more-precise-than-crispr\">CRISPR/Cas9\u003c/a>, with which scientists can selectively remove and replace portions of the genome, had just been used in mammalian cells for the first time the year before. Its advent, Srivastava knew, meant that creating a mouse model with a desired genetic condition, which used to take a year, could now be done in three weeks.\u003c/p>\n\u003cfigure id=\"attachment_1945465\" class=\"wp-caption alignleft\" style=\"max-width: 319px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"wp-image-1945465 size-full\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/07/for_kqed_mouse_lv.jpg\" alt=\"\" width=\"319\" height=\"241\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/for_kqed_mouse_lv.jpg 319w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/for_kqed_mouse_lv-160x121.jpg 160w\" sizes=\"(max-width: 319px) 100vw, 319px\">\u003cfigcaption class=\"wp-caption-text\">Fingerlike protrusions, a hallmark of LVNC, extend into the cavity of a three day old mouse’s left ventricle. Cell nuclei are marked in blue, and the lining of the cavity is shown in red. \u003ccite>(Casey Gifford/Gladstone Institutes)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>Armed with CRISPR/Cas9, Srivastava and Gifford decided to engineer mice with the same three mutations that Srivastava suspected had caused the Legkiy siblings’ heart condition. They hypothesized that the mutations would result in phenotypes, or observable physical expressions of genes, that resembled the phenotype of the human disease. As predicted, the mice with all three mutations showed the same finger-like protrusions in their hearts, demonstrating that, at least in mice, the mutations were enough to cause LVNC.\u003c/p>\n\u003cp>\u003cstrong>A Patient’s Beating Heart (Cells)\u003c/strong>\u003c/p>\n\u003cp>The mouse heart tissue that resembled the Legkiy childrens’ was an exciting breakthrough, but Srivastava and Gifford knew that to persuade the scientific community of the genes’ importance, they’d need to design an equally convincing experiment in human cells. As Srivastava realized on that car ride, their timing could not have been better: in 2012, Gladstone researchers had won the Nobel Prize for \u003ca href=\"https://www.nobelprize.org/prizes/medicine/2012/press-release/\">induced pluripotent stem cells\u003c/a>, cells that are genetically taken back in time to behave like embryonic stem cells, which scientists can then turn into any cell type in the body.\u003c/p>\n\u003cp>The scientists took skin grafts from the family and reprogrammed the cells to grow petri dishes full of heart cells genetically identical to Lana, Andrey, Anna and Tatiana.\u003c/p>\n\u003cp>In the lab, these cells pulsed (literally; heart cells \u003ca href=\"https://www.youtube.com/watch?v=bLiMUqYp3Jk\">pulse\u003c/a> with a heartbeat all their own) in confirmation of the team’s diagnosis. Even these cardiomyocytes, the one kind of heart cell the researchers had decided to test, displayed hallmarks of LVNC.\u003c/p>\n\u003cp>Each family member had their own petri dish of cells. Lana’s cells spread across her petri dish the way normal cells do, as if they were stars in the sky. Anna’s, by contrast, clumped together and did not stick well to the dish. Together with RNA sequencing data, the results showed that cell adhesion, long suspected to play a role in LVNC, had been affected.\u003c/p>\n\u003cfigure id=\"attachment_1945466\" class=\"wp-caption alignright\" style=\"max-width: 640px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"wp-image-1945466 size-large\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-1020x574.jpg\" alt=\"\" width=\"640\" height=\"360\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-1020x574.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-160x90.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-800x450.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-768x432.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-1200x675.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-1920x1080.jpg 1920w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford.jpg 2048w\" sizes=\"(max-width: 640px) 100vw, 640px\">\u003cfigcaption class=\"wp-caption-text\">Casey Gifford, lead author, and Deepak Srivastava, senior author, examine sections of heart tissue for signs of LVNC. \u003ccite>(Gladstone Institutes)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>In conjunction with the CRISPR/Cas9 experiments, the cell study showed that the three mutations Srivastava’s team had identified had been enough to cause Anna and Tatiana to display symptoms of LVNC. \u003ca href=\"https://gladstone.org/about-us/news/combination-three-gene-mutations-results-deadly-human-heart-disease\">This result\u003c/a>, published in \u003cem>Science\u003c/em> this May, gave proof to a phenomenon that had long been suspected by the medical community: that multiple genetic mutations could work together to cause a disease.\u003c/p>\n\u003cp>Lea Starita, a research assistant professor in the Department of Genome Sciences at the University of Washington, calls this proof “extremely important” for the field. She points out that the results hinged on the advent of cutting-edge technology and a single cardiologist paying the case a lot of attention.\u003c/p>\n\u003cp>“For years, I think we’ve known… that not all pathogenicities were going to be caused by single genes,” she said. “I don’t think there’s ever been a study that so well defined the interaction between three human variants on a phenotype like this.”\u003c/p>\n\u003cp>\u003cstrong>Looking Forward\u003c/strong>\u003c/p>\n\u003cp>Tatiana and Anna now live happy, normal lives: Anna is introverted and creative, a walking encyclopedia, her mother says; Tatiana is more outgoing. By now, their hearts seem to have developed normally, at last catching up to their bodies. Neither takes heart medication, though Srivastava says their hearts will need to be monitored for the rest of their lives, especially in moments of stress and when they get older. “It’s my hope that before they get to that point, we’ll have a better way to treat them,” he said.\u003c/p>\n\u003cfigure id=\"attachment_1945467\" class=\"wp-caption aligncenter\" style=\"max-width: 550px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"wp-image-1945467\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-800x600.jpg\" alt=\"\" width=\"550\" height=\"413\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-800x600.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-160x120.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-768x576.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-1020x765.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-1200x900.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o.jpg 1280w\" sizes=\"(max-width: 550px) 100vw, 550px\">\u003cfigcaption class=\"wp-caption-text\">Tatiana, Anna and Lana Legkiy. \u003ccite>(Lana Legkiy)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>With precise knowledge of which genetic mutations caused Anna and Tatiana’s rare heart condition, scientists now have taken the first steps toward identifying a cure. Gifford says that their combination of CRISPR/Cas9 models and use of stem cells illustrates the true power of genome editing. “We can [now] try to create much more faithful models of disease,” she said — models which could accelerate drug screening for a wide range of diseases, including other heart conditions and breast cancer.[pullquote size='medium' align='right' citation='Deepak Srivastava, Gladstone Institutes']‘These cases are the kinds that don’t come along all that often, but they’re the ones that can help us treat other diseases better.’[/pullquote]\u003c/p>\n\u003cp>The private sector has also been watching the Legkiys’ case, and is enthusiastic about what it could mean for how other genetic cases are studied. Tim Behrens, senior VP of a start-up company called \u003ca href=\"https://mazetx.com/\">Maze Therapeutics\u003c/a> (not associated with the study), commends the work as “a terrific story of how genetics and functional genomics really [help] us understand the underlying biology driving disease.”\u003c/p>\n\u003cp>Behrens does point out that diseases influenced by many mutations would be harder to tackle. Informed by large-scale screening of genes for mutations, Maze is on its way to drug development with the hope that patients like Anna and Tatiana can receive treatment someday.\u003c/p>\n\u003cp>Srivastava, meanwhile, is still pushing the limits of what technology can do for patients. Advances in single cell assays from the past year are allowing his team to use CRISPR/Cas9 to test a hundred suspected mutations in patients’ cells at once. He says that they’re at work on the genomes of thousands of children with heart disease, all thanks to their experience with the Legkiys. “These cases are the kinds that don’t come along all that often,” he said, “but they’re the ones that can help us treat other diseases better.”\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n","blocks":[],"excerpt":"Tatiana and Anna Legkiy have LVNC, a rare heart condition. After eleven years, their cardiologist finally knows why.","status":"publish","parent":0,"modified":1704848472,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":45,"wordCount":2445},"headData":{"title":"What Two Sisters With a Rare Heart Condition Taught Doctors About Our Genes | KQED","description":"Tatiana and Anna Legkiy have LVNC, a rare heart condition. After eleven years, their cardiologist finally knows why.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":""},"sticky":false,"path":"/science/1945458/what-two-sisters-with-a-rare-heart-condition-taught-doctors-about-our-genes","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Early in February of 2008, just days after she was born, Tatiana Legkiy lay in a cardiac intensive care unit, her tiny body hooked up to a respirator. After crying for two hours, she was now briefly quiet, the tube in her throat helping her breathe but also preventing her from making any sound.\u003c/p>\n\u003cp>Tatiana’s heart was failing. A cardiologist, tipped off by a pediatrician who heard something strange in a routine checkup, had examined her earlier that day and grown worried. He sent Tatiana to a nearby hospital in Modesto, California, where she remained for only an hour before being whisked eighty miles west by ambulance to the UCSF Benioff Children’s Hospital.\u003c/p>\n\u003cp>Tatiana’s parents arrived at the hospital shortly afterwards. At the time, Lana and Andrey Legkiy lived in Manteca, a city in California’s Central Valley. Andrey worked at an animal supply company; Lana stayed home and took care of their four-year-old daughter, Anna. Weekends found the the family outside together, camping or fishing in the Delta where the San Joaquin and Sacramento rivers flow into the ocean.\u003c/p>\n\u003cp>Even before Tatiana’s birth, the Legkiys knew medical hardship well. A year earlier Lana had suffered a miscarriage, losing an unborn child, a boy, in her third trimester. At the time, physicians had ascribed his cause of death to pulmonary hypoplasia, or incomplete development of the lungs.\u003c/p>\n\u003cp>Given that medical history, when Tatiana arrived at Benioff in critical condition, the doctors requested slides from the unborn child’s autopsy. This time, taking a closer look at small samples of heart tissue, they noted the true cause of death — an extremely rare heart condition known as left ventricular noncompaction (LVNC), in which the heart muscle remains immature and cannot pump blood normally.\u003c/p>\n\u003cp>Visually, physicians identify LVNC by the fingerlike protrusions of muscle extending from the wall of the heart into the left ventricle, which supplies the body with oxygen-rich blood. An echocardiogram of Tatiana’s heart showed these same characteristics.\u003c/p>\n\u003cp>Gently, the physicians told Tatiana’s parents that there would be no surgery, but only because LVNC has no cure.\u003c/p>\n\u003cp>\u003cstrong>A Hunch\u003c/strong>\u003c/p>\n\u003cp>Immediately, Deepak Srivastava, then-attending physician at Benioff, suspected a genetic connection. He was a geneticist, after all, and Tatiana’s unborn sibling had shared the same disease.\u003c/p>\n\u003cp>Though he could not have foreseen it as Tatiana struggled for her life that day, proving this intuition would require over a decade of work. It would also require technology which was only just becoming usable, and the dedication of researchers he had not yet met.\u003c/p>\n\u003cp>In the coming years, Srivastava would move forward with his own career, juggling the roles of biology professor, pediatric cardiologist, and ultimately president of Gladstone Institutes, a nonprofit biomedical research institution in San Francisco.\u003c/p>\n\u003cp>But the story of the Legkiys would stay with him. For a decade, he wouldn’t be able to shake the desire to pinpoint a precise genetic cause of LVNC, a necessary first step towards finding a cure for the disease. With that knowledge, physicians could rapidly screen potential drugs using an accurate, personalized model for patients like Tatiana.\u003c/p>\n\u003cfigure id=\"attachment_1945469\" class=\"wp-caption alignnone\" style=\"max-width: 640px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"wp-image-1945469 size-large\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-1020x765.jpg\" alt=\"\" width=\"640\" height=\"480\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-1020x765.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-160x120.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-800x600.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-768x576.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009-1200x900.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/AnnaTatiana2009.jpg 1600w\" sizes=\"(max-width: 640px) 100vw, 640px\">\u003cfigcaption class=\"wp-caption-text\">Anna and Tatiana Legkiy in 2009, just after Tatiana came off medication. \u003ccite>(Lana Legkiy)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>Adding to the urgency of the Legkiys’ situation: Tatiana was not their only child. Even as Tatiana received her diagnosis in 2008, her happy four-year-old sister, Anna, ran around the hospital waiting room. Blissfully oblivious, Anna charmed the physicians, who began to wonder: did all three Legkiy children share the same disease?\u003c/p>\n\u003cp>As Tatiana stabilized, thanks to medication helping her heart pump and a respirator helping her breathe, Srivastava’s team set about finding an answer. Two days after diagnosing Tatiana, the scientists took an echocardiogram, a kind of ultrasound, of her father’s heart. They observed that Andrey had a less severe, asymptomatic case of Tatiana’s condition.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"‘This is a family who had lost one child, their other child was in a life-threatening situation, and then they find out their other girl has the same thing… it was actually pretty devastating.’","name":"pullquote","attributes":{"named":{"size":"medium","align":"right","citation":"Deepak Srivastava, Gladstone Institutes","label":""},"numeric":[]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>So by the time they tested Anna’s heart the following day, Lana Legkiy already knew more than a mother should about what LVNC looked like in an ultrasound. (The team would later examine Lana’s own heart, and find it normal.)\u003c/p>\n\u003cp>When she saw Anna’s heart up on the screen, she knew without being told that her daughters shared the same disease.\u003c/p>\n\u003cp>Today, Lana can’t remember precisely what she felt in that moment— so much happened at once — but she does recall a dream she had while in the hospital, after Tatiana’s diagnosis. Lana doesn’t consider herself a true believer of supernatural occurrences, but she thinks dreams are important. “I saw both of them… as grown women,” she said. “I think that settled me down a little bit, like, ‘Okay, we’re going to be okay.’”\u003c/p>\n\u003cp>Srivastava, however, remembers the Legkiys’ distress acutely. “It was actually very sad,” he said. “This is a family who had lost one child, their other child was in a life-threatening situation, and then they find out their other girl has the same thing… it was actually pretty devastating.”\u003c/p>\n\u003cp>\u003cstrong>The Long Search for Proof\u003c/strong>\u003c/p>\n\u003cp>As the physicians told the Legkiys, there was and is still no cure or surgical procedure that can remedy LVNC. In cases such as Anna’s, the heart compensates for its left ventricle and swells, allowing it to pump more blood through the body. Anna, like others with enlarged hearts, did not display typical symptoms of heart dysfunction such as fatigue or rapid breathing.\u003c/p>\n\u003cp>Srivastava, however, told the Legkiys that both girls’ hearts would need to be monitored with yearly ultrasounds. Tatiana was sent home with more of the medication that helped her heart pump; she would take the medicine for a year before her heart muscles improved and the medication was no longer necessary. The family resumed what was, for the most part, a normal life.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"‘For years, I think we’ve known… that not all pathogenicities were going to be caused by single genes. I don’t think there’s ever been a study that so well defined the interaction between three human variants on a phenotype like this.’","name":"pullquote","attributes":{"named":{"size":"small","align":"left","citation":"Lea Starita, University of Washington","label":""},"numeric":[]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>As Anna and Tatiana grew into young women, now 15 and 11, Srivastava and his coworkers set about pinpointing the precise causes of the family’s congenital heart disease. The team sequenced the family’s DNA, combing through their genes in search of the variants all three children shared, screening against frequency of occurrence in the general population and for association with the heart.\u003c/p>\n\u003cp>The scientists quickly found three mutations in three different genes shared by the three siblings that they suspected to be the cause of the disease, two inherited from Andrey (\u003cem>MKL2\u003c/em> and \u003cem>MYH7\u003c/em>) and one from Lana (\u003cem>NKX2-5\u003c/em>). If not for the advent of a few key technologies, the investigation would have stopped here, the Legkiys left to wonder what was hidden in their genes. Srivastava’s search for proof, however, was just beginning.\u003c/p>\n\u003cp>\u003cstrong>CRISPR’s Perfect Timing\u003c/strong>\u003c/p>\n\u003cp>By 2014, the Legkiys were living in Seattle; Tatiana and Anna were six and 10, both medication-free. Tatiana’s hospital experience seemed like a distant memory.\u003c/p>\n\u003cp>In San Francisco, meanwhile, Casey Gifford, a recent PhD from Harvard, had joined Srivastava’s research team. New to both human genomics and heart disease, she and Srivastava spent a four-hour car ride to a conference in Lake Tahoe discussing what they thought would be the next big questions in medicine. Srivastava brought up the family he couldn’t forget — and realized, as he did so, that the tools they would need to prove which genetic mutations caused LVNC in the Legkiys might finally exist.\u003c/p>\n\u003cp>\u003ca href=\"https://www.kqed.org/futureofyou/436872/explainer-the-new-gene-editing-tool-significantly-more-precise-than-crispr\">CRISPR/Cas9\u003c/a>, with which scientists can selectively remove and replace portions of the genome, had just been used in mammalian cells for the first time the year before. Its advent, Srivastava knew, meant that creating a mouse model with a desired genetic condition, which used to take a year, could now be done in three weeks.\u003c/p>\n\u003cfigure id=\"attachment_1945465\" class=\"wp-caption alignleft\" style=\"max-width: 319px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"wp-image-1945465 size-full\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/07/for_kqed_mouse_lv.jpg\" alt=\"\" width=\"319\" height=\"241\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/for_kqed_mouse_lv.jpg 319w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/for_kqed_mouse_lv-160x121.jpg 160w\" sizes=\"(max-width: 319px) 100vw, 319px\">\u003cfigcaption class=\"wp-caption-text\">Fingerlike protrusions, a hallmark of LVNC, extend into the cavity of a three day old mouse’s left ventricle. Cell nuclei are marked in blue, and the lining of the cavity is shown in red. \u003ccite>(Casey Gifford/Gladstone Institutes)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>Armed with CRISPR/Cas9, Srivastava and Gifford decided to engineer mice with the same three mutations that Srivastava suspected had caused the Legkiy siblings’ heart condition. They hypothesized that the mutations would result in phenotypes, or observable physical expressions of genes, that resembled the phenotype of the human disease. As predicted, the mice with all three mutations showed the same finger-like protrusions in their hearts, demonstrating that, at least in mice, the mutations were enough to cause LVNC.\u003c/p>\n\u003cp>\u003cstrong>A Patient’s Beating Heart (Cells)\u003c/strong>\u003c/p>\n\u003cp>The mouse heart tissue that resembled the Legkiy childrens’ was an exciting breakthrough, but Srivastava and Gifford knew that to persuade the scientific community of the genes’ importance, they’d need to design an equally convincing experiment in human cells. As Srivastava realized on that car ride, their timing could not have been better: in 2012, Gladstone researchers had won the Nobel Prize for \u003ca href=\"https://www.nobelprize.org/prizes/medicine/2012/press-release/\">induced pluripotent stem cells\u003c/a>, cells that are genetically taken back in time to behave like embryonic stem cells, which scientists can then turn into any cell type in the body.\u003c/p>\n\u003cp>The scientists took skin grafts from the family and reprogrammed the cells to grow petri dishes full of heart cells genetically identical to Lana, Andrey, Anna and Tatiana.\u003c/p>\n\u003cp>In the lab, these cells pulsed (literally; heart cells \u003ca href=\"https://www.youtube.com/watch?v=bLiMUqYp3Jk\">pulse\u003c/a> with a heartbeat all their own) in confirmation of the team’s diagnosis. Even these cardiomyocytes, the one kind of heart cell the researchers had decided to test, displayed hallmarks of LVNC.\u003c/p>\n\u003cp>Each family member had their own petri dish of cells. Lana’s cells spread across her petri dish the way normal cells do, as if they were stars in the sky. Anna’s, by contrast, clumped together and did not stick well to the dish. Together with RNA sequencing data, the results showed that cell adhesion, long suspected to play a role in LVNC, had been affected.\u003c/p>\n\u003cfigure id=\"attachment_1945466\" class=\"wp-caption alignright\" style=\"max-width: 640px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"wp-image-1945466 size-large\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-1020x574.jpg\" alt=\"\" width=\"640\" height=\"360\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-1020x574.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-160x90.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-800x450.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-768x432.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-1200x675.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford-1920x1080.jpg 1920w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/Gladstone_SrivastavaGifford.jpg 2048w\" sizes=\"(max-width: 640px) 100vw, 640px\">\u003cfigcaption class=\"wp-caption-text\">Casey Gifford, lead author, and Deepak Srivastava, senior author, examine sections of heart tissue for signs of LVNC. \u003ccite>(Gladstone Institutes)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>In conjunction with the CRISPR/Cas9 experiments, the cell study showed that the three mutations Srivastava’s team had identified had been enough to cause Anna and Tatiana to display symptoms of LVNC. \u003ca href=\"https://gladstone.org/about-us/news/combination-three-gene-mutations-results-deadly-human-heart-disease\">This result\u003c/a>, published in \u003cem>Science\u003c/em> this May, gave proof to a phenomenon that had long been suspected by the medical community: that multiple genetic mutations could work together to cause a disease.\u003c/p>\n\u003cp>Lea Starita, a research assistant professor in the Department of Genome Sciences at the University of Washington, calls this proof “extremely important” for the field. She points out that the results hinged on the advent of cutting-edge technology and a single cardiologist paying the case a lot of attention.\u003c/p>\n\u003cp>“For years, I think we’ve known… that not all pathogenicities were going to be caused by single genes,” she said. “I don’t think there’s ever been a study that so well defined the interaction between three human variants on a phenotype like this.”\u003c/p>\n\u003cp>\u003cstrong>Looking Forward\u003c/strong>\u003c/p>\n\u003cp>Tatiana and Anna now live happy, normal lives: Anna is introverted and creative, a walking encyclopedia, her mother says; Tatiana is more outgoing. By now, their hearts seem to have developed normally, at last catching up to their bodies. Neither takes heart medication, though Srivastava says their hearts will need to be monitored for the rest of their lives, especially in moments of stress and when they get older. “It’s my hope that before they get to that point, we’ll have a better way to treat them,” he said.\u003c/p>\n\u003cfigure id=\"attachment_1945467\" class=\"wp-caption aligncenter\" style=\"max-width: 550px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"wp-image-1945467\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-800x600.jpg\" alt=\"\" width=\"550\" height=\"413\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-800x600.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-160x120.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-768x576.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-1020x765.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o-1200x900.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/07/413223_443577799016188_1715831936_o.jpg 1280w\" sizes=\"(max-width: 550px) 100vw, 550px\">\u003cfigcaption class=\"wp-caption-text\">Tatiana, Anna and Lana Legkiy. \u003ccite>(Lana Legkiy)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>With precise knowledge of which genetic mutations caused Anna and Tatiana’s rare heart condition, scientists now have taken the first steps toward identifying a cure. Gifford says that their combination of CRISPR/Cas9 models and use of stem cells illustrates the true power of genome editing. “We can [now] try to create much more faithful models of disease,” she said — models which could accelerate drug screening for a wide range of diseases, including other heart conditions and breast cancer.\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"‘These cases are the kinds that don’t come along all that often, but they’re the ones that can help us treat other diseases better.’","name":"pullquote","attributes":{"named":{"size":"medium","align":"right","citation":"Deepak Srivastava, Gladstone Institutes","label":""},"numeric":[]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>The private sector has also been watching the Legkiys’ case, and is enthusiastic about what it could mean for how other genetic cases are studied. Tim Behrens, senior VP of a start-up company called \u003ca href=\"https://mazetx.com/\">Maze Therapeutics\u003c/a> (not associated with the study), commends the work as “a terrific story of how genetics and functional genomics really [help] us understand the underlying biology driving disease.”\u003c/p>\n\u003cp>Behrens does point out that diseases influenced by many mutations would be harder to tackle. Informed by large-scale screening of genes for mutations, Maze is on its way to drug development with the hope that patients like Anna and Tatiana can receive treatment someday.\u003c/p>\n\u003cp>Srivastava, meanwhile, is still pushing the limits of what technology can do for patients. Advances in single cell assays from the past year are allowing his team to use CRISPR/Cas9 to test a hundred suspected mutations in patients’ cells at once. He says that they’re at work on the genomes of thousands of children with heart disease, all thanks to their experience with the Legkiys. “These cases are the kinds that don’t come along all that often,” he said, “but they’re the ones that can help us treat other diseases better.”\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"floatright"},"numeric":["floatright"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/science/1945458/what-two-sisters-with-a-rare-heart-condition-taught-doctors-about-our-genes","authors":["11615"],"categories":["science_30","science_39"],"tags":["science_1287","science_3370","science_3832"],"featImg":"science_1945478","label":"science"},"science_1944497":{"type":"posts","id":"science_1944497","meta":{"index":"posts_1591205157","site":"science","id":"1944497","score":null,"sort":[1562112271000]},"guestAuthors":[],"slug":"crispr-wipes-out-hiv-in-some-mice","title":"CRISPR Wipes Out HIV in Some Mice","publishDate":1562112271,"format":"standard","headTitle":"CRISPR Wipes Out HIV in Some Mice | KQED","labelTerm":{},"content":"\u003cp>For the millions of people infected with HIV, the best way to manage the disease is antiretroviral therapy, which can lower the amount of HIV replicating in the body to undetectable levels.\u003c/p>\n\u003carticle>\n\u003cdiv class=\"body-text\">\n\u003cp>But antiretroviral therapy (ART) can only manage HIV, never eliminate it — leading people to rely on the \u003ca href=\"https://aidsinfo.nih.gov/guidelines/html/1/adult-and-adolescent-arv/459/cost-considerations-and-antiretroviral-therapy\">expensive drugs\u003c/a> for decades. Plus if ART is halted by these patients, HIV bounces back and its levels rise in a matter of weeks. In 2017 the World Health Organization \u003ca href=\"https://www.who.int/gho/hiv/en/\">reported that of\u003c/a> 36.9 million people living with HIV, 21.7 million were taking ART.\u003c/p>\n\u003cp>A new study of mice charts a preliminary path to eliminating this medication dependency, through an injection of gene-editing \u003ca href=\"https://ghr.nlm.nih.gov/primer/genomicresearch/genomeediting\">CRISPR\u003c/a>.\u003c/p>\n\u003cp>Removing HIV from a person’s body is so hard because “once the virus infects cells [the] viral genome integrates into the host genome and then becomes part of our DNA,” said Kamel Khalili, a neurovirologist at Temple University who co-led the study.\u003c/p>\n\u003cp>Once this integration happens, ART can’t reverse it — at least not on its own. But Khalili and his colleagues found combining CRISPR, which can alter DNA, with ART can remove the lingering signs of HIV in a mouse model of the disease. The collaborative effort between researchers at Temple University and the University of Nebraska Medical Center was \u003ca href=\"https://www.nature.com/articles/s41467-019-10366-y\">reported today in Nature Communications\u003c/a>.\u003c/p>\n\u003cp>Results in mouse models do not always translate into effective treatments in humans, but Fyodor Urnov says this study is an “important next step” in addressing a disease like HIV with a technique.\u003c/p>\n\u003cp>“Gene editing is the first-ever technology we have had to address disease at the DNA level,” said Urnov, who works at the Innovative Genomics Institute at the University of California Berkeley and was not involved in the study.\u003c/p>\n\u003cp>CRISPR, which scientists can program to cut DNA at specific sites, has garnered attention as a potential cure for genetic diseases, such as \u003ca href=\"https://cysticfibrosisnewstoday.com/crisprcas9-approach-for-cystic-fibrosis/\">cystic fibrosis\u003c/a> and \u003ca href=\"https://directorsblog.nih.gov/2019/02/26/more-progress-toward-gene-editing-for-kids-with-muscular-dystrophy/\">muscular dystrophy\u003c/a>. Using CRISPR and ART to develop an HIV cure could eventually eliminate the cost and distribution barriers that come with current treatments. To date, \u003ca href=\"https://www.theguardian.com/society/2019/mar/05/london-patient-becomes-second-man-to-be-cleared-of-aids-virus\">only two people have been cured of HIV\u003c/a>after receiving stem cell therapy to treat cancer. This expensive and invasive treatment has failed in every other HIV patient.\u003c/p>\n\u003cp>But CRISPR comes with its own caveats — namely, at the moment it isn’t as precise as many in the general public might think.\u003c/p>\n\u003ch2>What the Study Did\u003c/h2>\n\u003cp>The researchers used “humanized” mice, meaning mice were infused with the type of human immune cells that HIV infects.\u003c/p>\n\u003cp>In three separate trials, HIV-infected mice were given one of three injection therapies: a slow-release version of the common HIV drug ART, the CRISPR protein that finds and cuts genome-incorporated HIV, or a combination of the two.\u003c/p>\n\u003cp>After four weeks of ART treatment, the researchers dispensed CRISPR into the mice by packaging the protein inside of a completely separate virus, called adeno-associated virus, or AAV. AAV doesn’t cause illness, but it acts like a mail carrier, increasing the circulation of CRISPR. CRISPR then becomes more likely to find and cut out all of the HIV DNA, an essential step to prevent the disease from rebounding.\u003c/p>\n\u003cp>The researchers waited eight weeks after all the mice were taken off of ART, then searched for traces of HIV genetic material in blood, spleen, kidneys, gut, lungs, bone marrow and the brain.\u003c/p>\n\u003cp>After eight ART-free weeks, more than 30 percent of the mice that received ART and CRISPR lacked detectable amounts of HIV in any of the tissues the researchers studied.\u003c/p>\n\u003cp>In contrast, HIV was still detectable in all of the other mice, including those that were treated with ART or CRISPR alone.\u003c/p>\n\u003ch2>A Cautious Way Forward\u003c/h2>\n\u003cp>Though the researchers used “humanized mice,” the technology has a long way to go. “Clinical trials…will not start tonight. It will take a few years,” said Urnov. However, Khalili revealed that early data from preliminary trials in monkeys looks promising.\u003c/p>\n\u003cp>Khalili and his colleagues have applied to the U.S. Food and Drug Administration for permission to start human clinical trials as early as next year. Khalili has also launched a for-profit company that is developing CRISPR-based technology to treat disease.\u003c/p>\n\u003cp>That the technology is moving this fast concerns Zandrea Ambrose, a retrovirologist at the University of Pittsburgh, who was not involved in the study. CRISPR comes with a downside, in that it sometimes accidentally cuts the wrong gene.\u003c/p>\n\u003cp>Relative to other gene editors, CRISPR has fewer of these “off-target effects” as they’re called, but even a single error is worrisome. If CRISPR makes a hazardous mutation, it could disrupt any cell’s normal behavior and cause an inadvertent disease.\u003c/p>\n\u003cp>With billions of letters in the genome, it’s going to take a lot of work to avoid all possible off-target effects, Ambrose said. This study is promising, she added, but scientists in the CRISPR field need to get better at detecting these potential accidents.\u003c/p>\n\u003cp>Before clinical trials can begin, she said, researchers need to be sure these accidents won’t occur in humans by first testing them in animals. When researchers scanned the DNA from the “cured” mice, they didn’t find any signs that CRISPR had made cuts outside of the HIV DNA. But they could tell that CRISPR had made cuts within the HIV DNA, meaning the instructions for making new virus were broken.\u003c/p>\n\u003cp>Along with possible off-target effects, Ambrose worries that CRISPR, enclosed in an AAV virus, will remain in the body long after its job is done. If that’s the case, she says “there’s no way to turn” the CRISPR editing off, increasing the chances of an off-target cut.\u003c/p>\n\u003cp>But Khalili is confident that CRISPR is only cutting the DNA where it’s supposed to, based on searches for the “footprints” CRISPR leaves on DNA when it cuts. And CRISPR \u003ca href=\"https://www.pbs.org/newshour/science/jumping-genes-could-help-crispr-replace-disease-causing-dna-study-finds\" target=\"_blank\" rel=\"noopener\">continues to improve everyday\u003c/a>.\u003c/p>\n\u003cp>While a comprehensive, single-shot cure for HIV may be years away, Khalili is encouraged that this milestone “will give those affected hope.”\u003c/p>\n\u003cp>\u003cem>\u003ca href=\"https://www.pbs.org/newshour/science/crispr-gene-editing-eliminates-hiv-in-some-mice-what-does-it-mean-for-humans\">This story\u003c/a> was originally published on PBS Newshour.\u003c/em>\u003c/p>\n\u003c/div>\n\u003c/article>\n\u003cp>[ad fullwidth]\u003c/p>\u003cp>[ad floatright]\u003c/p>\n","blocks":[],"excerpt":"The new study could be an “important next step” in developing a pathway for addressing HIV with a gene-editing technique. ","status":"publish","parent":0,"modified":1704848541,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":27,"wordCount":1074},"headData":{"title":"CRISPR Wipes Out HIV in Some Mice | KQED","description":"The new study could be an “important next step” in developing a pathway for addressing HIV with a gene-editing technique. ","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":""},"source":"PBS Newshour","sticky":false,"nprByline":"Berly McCoy \u003cbr/> PBS Newshour \u003cbr>","path":"/science/1944497/crispr-wipes-out-hiv-in-some-mice","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>For the millions of people infected with HIV, the best way to manage the disease is antiretroviral therapy, which can lower the amount of HIV replicating in the body to undetectable levels.\u003c/p>\n\u003carticle>\n\u003cdiv class=\"body-text\">\n\u003cp>But antiretroviral therapy (ART) can only manage HIV, never eliminate it — leading people to rely on the \u003ca href=\"https://aidsinfo.nih.gov/guidelines/html/1/adult-and-adolescent-arv/459/cost-considerations-and-antiretroviral-therapy\">expensive drugs\u003c/a> for decades. Plus if ART is halted by these patients, HIV bounces back and its levels rise in a matter of weeks. In 2017 the World Health Organization \u003ca href=\"https://www.who.int/gho/hiv/en/\">reported that of\u003c/a> 36.9 million people living with HIV, 21.7 million were taking ART.\u003c/p>\n\u003cp>A new study of mice charts a preliminary path to eliminating this medication dependency, through an injection of gene-editing \u003ca href=\"https://ghr.nlm.nih.gov/primer/genomicresearch/genomeediting\">CRISPR\u003c/a>.\u003c/p>\n\u003cp>Removing HIV from a person’s body is so hard because “once the virus infects cells [the] viral genome integrates into the host genome and then becomes part of our DNA,” said Kamel Khalili, a neurovirologist at Temple University who co-led the study.\u003c/p>\n\u003cp>Once this integration happens, ART can’t reverse it — at least not on its own. But Khalili and his colleagues found combining CRISPR, which can alter DNA, with ART can remove the lingering signs of HIV in a mouse model of the disease. The collaborative effort between researchers at Temple University and the University of Nebraska Medical Center was \u003ca href=\"https://www.nature.com/articles/s41467-019-10366-y\">reported today in Nature Communications\u003c/a>.\u003c/p>\n\u003cp>Results in mouse models do not always translate into effective treatments in humans, but Fyodor Urnov says this study is an “important next step” in addressing a disease like HIV with a technique.\u003c/p>\n\u003cp>“Gene editing is the first-ever technology we have had to address disease at the DNA level,” said Urnov, who works at the Innovative Genomics Institute at the University of California Berkeley and was not involved in the study.\u003c/p>\n\u003cp>CRISPR, which scientists can program to cut DNA at specific sites, has garnered attention as a potential cure for genetic diseases, such as \u003ca href=\"https://cysticfibrosisnewstoday.com/crisprcas9-approach-for-cystic-fibrosis/\">cystic fibrosis\u003c/a> and \u003ca href=\"https://directorsblog.nih.gov/2019/02/26/more-progress-toward-gene-editing-for-kids-with-muscular-dystrophy/\">muscular dystrophy\u003c/a>. Using CRISPR and ART to develop an HIV cure could eventually eliminate the cost and distribution barriers that come with current treatments. To date, \u003ca href=\"https://www.theguardian.com/society/2019/mar/05/london-patient-becomes-second-man-to-be-cleared-of-aids-virus\">only two people have been cured of HIV\u003c/a>after receiving stem cell therapy to treat cancer. This expensive and invasive treatment has failed in every other HIV patient.\u003c/p>\n\u003cp>But CRISPR comes with its own caveats — namely, at the moment it isn’t as precise as many in the general public might think.\u003c/p>\n\u003ch2>What the Study Did\u003c/h2>\n\u003cp>The researchers used “humanized” mice, meaning mice were infused with the type of human immune cells that HIV infects.\u003c/p>\n\u003cp>In three separate trials, HIV-infected mice were given one of three injection therapies: a slow-release version of the common HIV drug ART, the CRISPR protein that finds and cuts genome-incorporated HIV, or a combination of the two.\u003c/p>\n\u003cp>After four weeks of ART treatment, the researchers dispensed CRISPR into the mice by packaging the protein inside of a completely separate virus, called adeno-associated virus, or AAV. AAV doesn’t cause illness, but it acts like a mail carrier, increasing the circulation of CRISPR. CRISPR then becomes more likely to find and cut out all of the HIV DNA, an essential step to prevent the disease from rebounding.\u003c/p>\n\u003cp>The researchers waited eight weeks after all the mice were taken off of ART, then searched for traces of HIV genetic material in blood, spleen, kidneys, gut, lungs, bone marrow and the brain.\u003c/p>\n\u003cp>After eight ART-free weeks, more than 30 percent of the mice that received ART and CRISPR lacked detectable amounts of HIV in any of the tissues the researchers studied.\u003c/p>\n\u003cp>In contrast, HIV was still detectable in all of the other mice, including those that were treated with ART or CRISPR alone.\u003c/p>\n\u003ch2>A Cautious Way Forward\u003c/h2>\n\u003cp>Though the researchers used “humanized mice,” the technology has a long way to go. “Clinical trials…will not start tonight. It will take a few years,” said Urnov. However, Khalili revealed that early data from preliminary trials in monkeys looks promising.\u003c/p>\n\u003cp>Khalili and his colleagues have applied to the U.S. Food and Drug Administration for permission to start human clinical trials as early as next year. Khalili has also launched a for-profit company that is developing CRISPR-based technology to treat disease.\u003c/p>\n\u003cp>That the technology is moving this fast concerns Zandrea Ambrose, a retrovirologist at the University of Pittsburgh, who was not involved in the study. CRISPR comes with a downside, in that it sometimes accidentally cuts the wrong gene.\u003c/p>\n\u003cp>Relative to other gene editors, CRISPR has fewer of these “off-target effects” as they’re called, but even a single error is worrisome. If CRISPR makes a hazardous mutation, it could disrupt any cell’s normal behavior and cause an inadvertent disease.\u003c/p>\n\u003cp>With billions of letters in the genome, it’s going to take a lot of work to avoid all possible off-target effects, Ambrose said. This study is promising, she added, but scientists in the CRISPR field need to get better at detecting these potential accidents.\u003c/p>\n\u003cp>Before clinical trials can begin, she said, researchers need to be sure these accidents won’t occur in humans by first testing them in animals. When researchers scanned the DNA from the “cured” mice, they didn’t find any signs that CRISPR had made cuts outside of the HIV DNA. But they could tell that CRISPR had made cuts within the HIV DNA, meaning the instructions for making new virus were broken.\u003c/p>\n\u003cp>Along with possible off-target effects, Ambrose worries that CRISPR, enclosed in an AAV virus, will remain in the body long after its job is done. If that’s the case, she says “there’s no way to turn” the CRISPR editing off, increasing the chances of an off-target cut.\u003c/p>\n\u003cp>But Khalili is confident that CRISPR is only cutting the DNA where it’s supposed to, based on searches for the “footprints” CRISPR leaves on DNA when it cuts. And CRISPR \u003ca href=\"https://www.pbs.org/newshour/science/jumping-genes-could-help-crispr-replace-disease-causing-dna-study-finds\" target=\"_blank\" rel=\"noopener\">continues to improve everyday\u003c/a>.\u003c/p>\n\u003cp>While a comprehensive, single-shot cure for HIV may be years away, Khalili is encouraged that this milestone “will give those affected hope.”\u003c/p>\n\u003cp>\u003cem>\u003ca href=\"https://www.pbs.org/newshour/science/crispr-gene-editing-eliminates-hiv-in-some-mice-what-does-it-mean-for-humans\">This story\u003c/a> was originally published on PBS Newshour.\u003c/em>\u003c/p>\n\u003c/div>\n\u003c/article>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"floatright"},"numeric":["floatright"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/science/1944497/crispr-wipes-out-hiv-in-some-mice","authors":["byline_science_1944497"],"categories":["science_2874","science_39","science_3890","science_40"],"tags":["science_1287","science_660"],"featImg":"science_1944503","label":"source_science_1944497"},"science_1943941":{"type":"posts","id":"science_1943941","meta":{"index":"posts_1591205157","site":"science","id":"1943941","score":null,"sort":[1561495618000]},"guestAuthors":[],"slug":"here-we-go-again-feds-reopen-patent-dispute-between-uc-and-broad-institute","title":"Here We Go Again: Feds Reopen Patent Dispute Between UC and Broad Institute","publishDate":1561495618,"format":"aside","headTitle":"Here We Go Again: Feds Reopen Patent Dispute Between UC and Broad Institute | KQED","labelTerm":{},"content":"\u003cp>The U.S. patent office has \u003ca href=\"https://www.broadinstitute.org/files/news/pdfs/106115-NoticeDeclaringInterference.pdf\" target=\"_blank\" rel=\"noopener\">declared an interference\u003c/a> between a dozen key patents awarded to the Broad Institute on the genome-editing technology CRISPR and 10 CRISPR patent applications submitted by the University of California and its partners, according to documents posted by the U.S. Patent and Trademark Office.\u003c/p>\n\u003cp>\u003cimg loading=\"lazy\" decoding=\"async\" class=\"alignnone size-medium wp-image-1943947\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-800x531.jpg\" alt=\"\" width=\"800\" height=\"531\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-800x531.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-160x106.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-768x510.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-1020x677.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-1200x797.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1.jpg 1280w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003c/p>\n\u003cp>The declaration of an interference means that the patent office has determined that one or more patent applications describe inventions that are substantially the same as those for which patents have already been issued. In this case, the patents awarded to the Broad, beginning in 2014, describe the use of CRISPR-Cas9 to edit the genomes of eukaryotes — organisms whose genomes are enclosed within a cell nucleus, including all plants and animals — based on the research of Broad biologist Feng Zhang. UC’s patent applications also cover the use of CRISPR in eukaryotes, based on the work of UC Berkeley biochemist Jennifer Doudna and her collaborator Emmanuelle Charpentier.\u003c/p>\n\u003cp>UC and the Broad already went through an interference proceeding that went all the way to federal appeals court, with the Broad \u003ca href=\"https://www.statnews.com/2018/09/10/appeals-court-upholds-crispr-patents-awarded-to-broad-institute/\" target=\"_blank\" rel=\"noopener\">prevailing\u003c/a>.\u003c/p>\n\u003cp>That history made patent experts react almost identically to this latest development. “Here we are again,” said attorney Kevin Noonan of the Chicago law firm McDonnell Boehnen Hulbert & Berghoff LLP, who specializes in biotech patents. “I can only imagine that this will go on, and on, and on.”\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>Both the Doudna and Zhang teams did their research under a system that awarded patents based on who was the first to invent (the current system, in place since 2013, awards patents based on who was the first to file). The interference proceeding will entail motions filed with the patent office, which will likely take a year, and then possibly a hearing. At some point, the patent office will therefore have to determine who was the inventor of CRISPR genome editing in higher organisms — not bacteria, and not DNA floating freely in a test tube.\u003c/p>\n\u003cp>“Now we’re having the fight over who invented CRISPR in eukaryotes,” said Eldora Ellison of Sterne Kessler Goldstein & Fox, who represents UC. The declaration of interference, she said, “means that the patent office has recognized that it has a duty to determine who invented this important invention. The fact that the Broad has patents does not resolve that question.”\u003c/p>\n\u003cp>The answer to that question would reverberate well beyond the potentially billion-dollar market for CRISPR therapies. Those are being developed by at least three companies, including Editas Medicine, CRISPR Therapeutics, and Intellia Therapeutics. The outcome could also affect who the science record books, to say nothing of the Nobel Prize committee, recognizes as the inventors of this revolutionary technology.\u003c/p>\n\u003cp>In a statement, the Broad said, “We welcome this action by the [patent office], which has previously ruled that the claims of the Broad patents, issued for methods for eukaryotic genome editing, were properly granted.”\u003c/p>\n\u003cp>Unlike the last interference, which UC requested, neither party asked for this one. But that can be done “indirectly,” Noonan said.\u003c/p>\n\u003cp>“The interesting thing in terms of the [University of California] strategy is that they seem to have filed a bunch of patent applications intended to provoke an interference,” by describing the use of CRISPR in eukaryotes even though the UC team was not the first to achieve that, Noonan said.\u003c/p>\n\u003cp>“If you write the [patent] claim the right way, and the patent examiner is aware that the Broad’s patents [on that invention] exist, it wouldn’t take a genius examiner to say, aha,” he said.\u003c/p>\n\u003cp>The patent office has designated the Broad as the “senior party” in the interference and UC as the “junior party.” That means the Broad, with patents in hand since 2014, is presumed to be the rightful, first inventor. UC therefore has to prove its case to the patent office.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>\u003cem>This \u003ca href=\"https://www.statnews.com/2019/05/01/from-protegee-to-whistleblower-a-former-theranos-scientist-says-elizabeth-holmes-should-come-forward-and-apologize/\">story\u003c/a> was originally published by \u003ca href=\"https://www.statnews.com/\">STAT\u003c/a>, an online publication of Boston Globe Media that covers health, medicine, and scientific discovery.\u003c/em>\u003c/p>\n\n","blocks":[],"excerpt":"The U.S. patent office declared an interference between CRISPR patents awarded to the Broad Institute and the University of California. ","status":"publish","parent":0,"modified":1704848566,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":16,"wordCount":694},"headData":{"title":"Here We Go Again: Feds Reopen Patent Dispute Between UC and Broad Institute | KQED","description":"The U.S. patent office declared an interference between CRISPR patents awarded to the Broad Institute and the University of California. ","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":""},"source":"STAT News","sticky":false,"nprByline":"Sharon Begley \u003cbr/> STAT News \u003cbr>","path":"/science/1943941/here-we-go-again-feds-reopen-patent-dispute-between-uc-and-broad-institute","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>The U.S. patent office has \u003ca href=\"https://www.broadinstitute.org/files/news/pdfs/106115-NoticeDeclaringInterference.pdf\" target=\"_blank\" rel=\"noopener\">declared an interference\u003c/a> between a dozen key patents awarded to the Broad Institute on the genome-editing technology CRISPR and 10 CRISPR patent applications submitted by the University of California and its partners, according to documents posted by the U.S. Patent and Trademark Office.\u003c/p>\n\u003cp>\u003cimg loading=\"lazy\" decoding=\"async\" class=\"alignnone size-medium wp-image-1943947\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-800x531.jpg\" alt=\"\" width=\"800\" height=\"531\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-800x531.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-160x106.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-768x510.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-1020x677.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1-1200x797.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1.jpg 1280w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003c/p>\n\u003cp>The declaration of an interference means that the patent office has determined that one or more patent applications describe inventions that are substantially the same as those for which patents have already been issued. In this case, the patents awarded to the Broad, beginning in 2014, describe the use of CRISPR-Cas9 to edit the genomes of eukaryotes — organisms whose genomes are enclosed within a cell nucleus, including all plants and animals — based on the research of Broad biologist Feng Zhang. UC’s patent applications also cover the use of CRISPR in eukaryotes, based on the work of UC Berkeley biochemist Jennifer Doudna and her collaborator Emmanuelle Charpentier.\u003c/p>\n\u003cp>UC and the Broad already went through an interference proceeding that went all the way to federal appeals court, with the Broad \u003ca href=\"https://www.statnews.com/2018/09/10/appeals-court-upholds-crispr-patents-awarded-to-broad-institute/\" target=\"_blank\" rel=\"noopener\">prevailing\u003c/a>.\u003c/p>\n\u003cp>That history made patent experts react almost identically to this latest development. “Here we are again,” said attorney Kevin Noonan of the Chicago law firm McDonnell Boehnen Hulbert & Berghoff LLP, who specializes in biotech patents. “I can only imagine that this will go on, and on, and on.”\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>Both the Doudna and Zhang teams did their research under a system that awarded patents based on who was the first to invent (the current system, in place since 2013, awards patents based on who was the first to file). The interference proceeding will entail motions filed with the patent office, which will likely take a year, and then possibly a hearing. At some point, the patent office will therefore have to determine who was the inventor of CRISPR genome editing in higher organisms — not bacteria, and not DNA floating freely in a test tube.\u003c/p>\n\u003cp>“Now we’re having the fight over who invented CRISPR in eukaryotes,” said Eldora Ellison of Sterne Kessler Goldstein & Fox, who represents UC. The declaration of interference, she said, “means that the patent office has recognized that it has a duty to determine who invented this important invention. The fact that the Broad has patents does not resolve that question.”\u003c/p>\n\u003cp>The answer to that question would reverberate well beyond the potentially billion-dollar market for CRISPR therapies. Those are being developed by at least three companies, including Editas Medicine, CRISPR Therapeutics, and Intellia Therapeutics. The outcome could also affect who the science record books, to say nothing of the Nobel Prize committee, recognizes as the inventors of this revolutionary technology.\u003c/p>\n\u003cp>In a statement, the Broad said, “We welcome this action by the [patent office], which has previously ruled that the claims of the Broad patents, issued for methods for eukaryotic genome editing, were properly granted.”\u003c/p>\n\u003cp>Unlike the last interference, which UC requested, neither party asked for this one. But that can be done “indirectly,” Noonan said.\u003c/p>\n\u003cp>“The interesting thing in terms of the [University of California] strategy is that they seem to have filed a bunch of patent applications intended to provoke an interference,” by describing the use of CRISPR in eukaryotes even though the UC team was not the first to achieve that, Noonan said.\u003c/p>\n\u003cp>“If you write the [patent] claim the right way, and the patent examiner is aware that the Broad’s patents [on that invention] exist, it wouldn’t take a genius examiner to say, aha,” he said.\u003c/p>\n\u003cp>The patent office has designated the Broad as the “senior party” in the interference and UC as the “junior party.” That means the Broad, with patents in hand since 2014, is presumed to be the rightful, first inventor. UC therefore has to prove its case to the patent office.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>\u003cem>This \u003ca href=\"https://www.statnews.com/2019/05/01/from-protegee-to-whistleblower-a-former-theranos-scientist-says-elizabeth-holmes-should-come-forward-and-apologize/\">story\u003c/a> was originally published by \u003ca href=\"https://www.statnews.com/\">STAT\u003c/a>, an online publication of Boston Globe Media that covers health, medicine, and scientific discovery.\u003c/em>\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/science/1943941/here-we-go-again-feds-reopen-patent-dispute-between-uc-and-broad-institute","authors":["byline_science_1943941"],"categories":["science_30","science_29","science_39","science_40"],"tags":["science_5178","science_1287","science_3838"],"featImg":"science_1943946","label":"source_science_1943941"},"science_1943302":{"type":"posts","id":"science_1943302","meta":{"index":"posts_1591205157","site":"science","id":"1943302","score":null,"sort":[1560420917000]},"guestAuthors":[],"slug":"uc-partners-with-pharmaceutical-giant-on-67-million-crispr-lab","title":"UC Partners With Pharmaceutical Giant on $67 Million CRISPR Lab","publishDate":1560420917,"format":"aside","headTitle":"UC Partners With Pharmaceutical Giant on $67 Million CRISPR Lab | KQED","labelTerm":{},"content":"\u003cp>The University of California and the British pharmaceutical giant GlaxoSmithKline on Thursday announced plans to build a new $67 million genetics laboratory focused on the gene-editing technology CRISPR.\u003c/p>\n\u003cp>Scientists at what will be called the Laboratory for Genomics Research, to be built over the next five years in San Francisco’s Mission Bay, will explore how genetic mutations cause disease, while developing new gene therapies and other treatments.\u003c/p>\n\u003cp>The study of human genetics has exploded in the last decade, and scientists can now identify mutations in DNA that cause a wide range of disease, from cancer to Huntington’s to muscular dystrophy.\u003c/p>\n\u003cp>“But turning that into an actionable item where you can develop a therapy has been challenging,” said Jonathan Weissman, a biochemist at UCSF. Weissman is designing the laboratory with UC Berkeley’s Jennifer Doudna, a CRISPR pioneer, and Hal Barron, chief science officer and president of GSK.\u003c/p>\n\u003cp>Weissman hopes the lab will enable researchers to more fully understand genetic differences through the advancement of functional genomics — the study of gene relationships and interactions — that relies on CRISPR technology.\u003c/p>\n\u003cp>CRISPR is so powerful because it targets specific genes with precise edits in DNA.\u003c/p>\n\u003cp>\u003cimg loading=\"lazy\" decoding=\"async\" class=\"size-medium wp-image-1943305 alignright\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-800x531.jpg\" alt=\"\" width=\"800\" height=\"531\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-800x531.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-160x106.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-768x510.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1020x677.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1200x797.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115.jpg 1280w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003c/p>\n\u003cp>“CRISPR is a great discovery tool that lets us understand why changes in our DNA can cause disease and then give us clues as to how we might be able to intervene to prevent it,” said Weissman.\u003c/p>\n\u003cp>Recent advances in machine learning have given scientists the ability to use powerful computers to analyze the massive amount of data generated by CRISPR applications. The hope is that these supercomputers can help unlock the mysteries of cell biology and rapidly accelerate the discovery of new treatments.\u003c/p>\n\u003cp>“We think that human genetics, functional genomics and machine learning will allow us to identify novel targets that will result in medicines that will have a profound effect ,” said Barron.\u003c/p>\n\u003cp>GSK’s Barron said he hopes the lab will spur advancements in gene therapy at “a pace previously thought impossible.”\u003c/p>\n\u003cp>The lab will employ about 40 people, GSK and UC said, and will be located between UCSF’s Mission Bay campus and the new Warriors \u003ca href=\"http://www.gswconstruction.com/webcam/\">stadium\u003c/a>.\u003c/p>\n\u003cp>\u003cstrong>CRISPR and Ethics\u003c/strong>\u003c/p>\n\u003cp>With CRISPR technology, scientists can modify or add entirely new genes.\u003c/p>\n\u003cp>CRISPR is different from other gene-editing tools in that its applications can alter the DNA of somatic cells and germ cells.\u003c/p>\n\u003cp>Somatic cells are found in organs and tissues and are not passed on through reproduction. Germ cells hold genes that are heritable.\u003c/p>\n\u003cp>Doudna said the focus of the new lab will be on fundamental discovery science, and not on germ cell editing.\u003c/p>\n\u003cfigure id=\"attachment_1943306\" class=\"wp-caption alignright\" style=\"max-width: 800px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"size-medium wp-image-1943306\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-800x565.jpg\" alt=\"\" width=\"800\" height=\"565\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-800x565.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-160x113.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-768x543.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-1020x721.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-1200x848.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-1920x1357.jpg 1920w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758.jpg 2048w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003cfigcaption class=\"wp-caption-text\">Jennifer Doudna will spearhead a new CRISPR lab in the Mission Bay neighborhood of San Francisco. speaks onstage at WIRED Business Conference Presented By Visa At Spring Studios In New York City on June 7, 2017 in New York City. (Photo by Brian Ach/Getty \u003ccite>(Brian Ach/Getty Images)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>“I don’t think there’s any intention right now to be editing human embryos in the center,” she said. “I think our goal is actually to work on various kinds of disease-related questions that would be addressable using primary cells and tissues.”\u003c/p>\n\u003cp>Last year, controversy swarmed around He Jiankui, a biochemist with Southern University of Science and Technology in Shenzhen, China, who used CRISPR to perform germline editing to modify genes in a human embryo.\u003c/p>\n\u003cp>At the time, Doudna was one of the scientists who quickly criticized Jiankui, telling NPR that his work is a “break from the cautious and transparent approach of the global scientific community’s application of CRISPR-Cas9 for human germline editing.”\u003c/p>\n\u003cp>Doudna has declared a need to confine the use of gene-editing in human embryos to situations in which there is a clear medical need with zero alternative viable approach.\u003c/p>\n\u003cp>Additionally, Doudna says there’s a lot of fundamental research that needs to be done prior to any use of genome editing for clinical purposes in human embryos. “In that regard, the [lab] will play a very important role in stimulating fundamental, curiosity-driven research that needs to be done,” she said. “It will both advance our understanding of the human genome and we’ll also advance the potential in the power of the technology.”\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>\u003c/p>\n","blocks":[],"excerpt":"The new Laboratory for Genomics Research will focus on studying how genetic mutations cause disease and searching for treatments. ","status":"publish","parent":0,"modified":1704848601,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":24,"wordCount":738},"headData":{"title":"UC Partners With Pharmaceutical Giant on $67 Million CRISPR Lab | KQED","description":"The new Laboratory for Genomics Research will focus on studying how genetic mutations cause disease and searching for treatments. ","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":""},"source":"CRISPR","audioUrl":"https://www.kqed.org/.stream/anon/radio/science/2019/06/StarkUCCrisprLab.mp3","sticky":false,"path":"/science/1943302/uc-partners-with-pharmaceutical-giant-on-67-million-crispr-lab","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>The University of California and the British pharmaceutical giant GlaxoSmithKline on Thursday announced plans to build a new $67 million genetics laboratory focused on the gene-editing technology CRISPR.\u003c/p>\n\u003cp>Scientists at what will be called the Laboratory for Genomics Research, to be built over the next five years in San Francisco’s Mission Bay, will explore how genetic mutations cause disease, while developing new gene therapies and other treatments.\u003c/p>\n\u003cp>The study of human genetics has exploded in the last decade, and scientists can now identify mutations in DNA that cause a wide range of disease, from cancer to Huntington’s to muscular dystrophy.\u003c/p>\n\u003cp>“But turning that into an actionable item where you can develop a therapy has been challenging,” said Jonathan Weissman, a biochemist at UCSF. Weissman is designing the laboratory with UC Berkeley’s Jennifer Doudna, a CRISPR pioneer, and Hal Barron, chief science officer and president of GSK.\u003c/p>\n\u003cp>Weissman hopes the lab will enable researchers to more fully understand genetic differences through the advancement of functional genomics — the study of gene relationships and interactions — that relies on CRISPR technology.\u003c/p>\n\u003cp>CRISPR is so powerful because it targets specific genes with precise edits in DNA.\u003c/p>\n\u003cp>\u003cimg loading=\"lazy\" decoding=\"async\" class=\"size-medium wp-image-1943305 alignright\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-800x531.jpg\" alt=\"\" width=\"800\" height=\"531\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-800x531.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-160x106.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-768x510.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1020x677.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115-1200x797.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/DESKTOP_CRISPR_171115.jpg 1280w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003c/p>\n\u003cp>“CRISPR is a great discovery tool that lets us understand why changes in our DNA can cause disease and then give us clues as to how we might be able to intervene to prevent it,” said Weissman.\u003c/p>\n\u003cp>Recent advances in machine learning have given scientists the ability to use powerful computers to analyze the massive amount of data generated by CRISPR applications. The hope is that these supercomputers can help unlock the mysteries of cell biology and rapidly accelerate the discovery of new treatments.\u003c/p>\n\u003cp>“We think that human genetics, functional genomics and machine learning will allow us to identify novel targets that will result in medicines that will have a profound effect ,” said Barron.\u003c/p>\n\u003cp>GSK’s Barron said he hopes the lab will spur advancements in gene therapy at “a pace previously thought impossible.”\u003c/p>\n\u003cp>The lab will employ about 40 people, GSK and UC said, and will be located between UCSF’s Mission Bay campus and the new Warriors \u003ca href=\"http://www.gswconstruction.com/webcam/\">stadium\u003c/a>.\u003c/p>\n\u003cp>\u003cstrong>CRISPR and Ethics\u003c/strong>\u003c/p>\n\u003cp>With CRISPR technology, scientists can modify or add entirely new genes.\u003c/p>\n\u003cp>CRISPR is different from other gene-editing tools in that its applications can alter the DNA of somatic cells and germ cells.\u003c/p>\n\u003cp>Somatic cells are found in organs and tissues and are not passed on through reproduction. Germ cells hold genes that are heritable.\u003c/p>\n\u003cp>Doudna said the focus of the new lab will be on fundamental discovery science, and not on germ cell editing.\u003c/p>\n\u003cfigure id=\"attachment_1943306\" class=\"wp-caption alignright\" style=\"max-width: 800px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"size-medium wp-image-1943306\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-800x565.jpg\" alt=\"\" width=\"800\" height=\"565\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-800x565.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-160x113.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-768x543.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-1020x721.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-1200x848.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758-1920x1357.jpg 1920w, https://cdn.kqed.org/wp-content/uploads/sites/35/2019/06/GettyImages-693524758.jpg 2048w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003cfigcaption class=\"wp-caption-text\">Jennifer Doudna will spearhead a new CRISPR lab in the Mission Bay neighborhood of San Francisco. speaks onstage at WIRED Business Conference Presented By Visa At Spring Studios In New York City on June 7, 2017 in New York City. (Photo by Brian Ach/Getty \u003ccite>(Brian Ach/Getty Images)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>“I don’t think there’s any intention right now to be editing human embryos in the center,” she said. “I think our goal is actually to work on various kinds of disease-related questions that would be addressable using primary cells and tissues.”\u003c/p>\n\u003cp>Last year, controversy swarmed around He Jiankui, a biochemist with Southern University of Science and Technology in Shenzhen, China, who used CRISPR to perform germline editing to modify genes in a human embryo.\u003c/p>\n\u003cp>At the time, Doudna was one of the scientists who quickly criticized Jiankui, telling NPR that his work is a “break from the cautious and transparent approach of the global scientific community’s application of CRISPR-Cas9 for human germline editing.”\u003c/p>\n\u003cp>Doudna has declared a need to confine the use of gene-editing in human embryos to situations in which there is a clear medical need with zero alternative viable approach.\u003c/p>\n\u003cp>Additionally, Doudna says there’s a lot of fundamental research that needs to be done prior to any use of genome editing for clinical purposes in human embryos. “In that regard, the [lab] will play a very important role in stimulating fundamental, curiosity-driven research that needs to be done,” she said. “It will both advance our understanding of the human genome and we’ll also advance the potential in the power of the technology.”\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>\u003c/p>\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/science/1943302/uc-partners-with-pharmaceutical-giant-on-67-million-crispr-lab","authors":["11608"],"categories":["science_30","science_29","science_39","science_3890","science_40"],"tags":["science_1287","science_3840","science_190","science_5155"],"featImg":"science_1943306","label":"source_science_1943302"},"science_1938007":{"type":"posts","id":"science_1938007","meta":{"index":"posts_1591205157","site":"science","id":"1938007","score":null,"sort":[1550687407000]},"guestAuthors":[],"slug":"making-sense-of-the-crispr-patent-dispute-between-the-university-of-california-and-broad","title":"Making Sense of the CRISPR Patent Dispute Between the University of California and Broad","publishDate":1550687407,"format":"aside","headTitle":"Making Sense of the CRISPR Patent Dispute Between the University of California and Broad | KQED","labelTerm":{"site":"science"},"content":"\u003cp>Anyone fond of intricate intellectual property disputes (and really, who isn’t?) is going to have their cup runnething over when it comes to the long-running battle between the University of California and Harvard/MIT’s Broad Institute to determine who will get to cash in the most on their seminal CRISPR/Cas 9 discoveries.\u003c/p>\n\u003cp>CRISPR/Cas9 is a precise gene-editing technology. Scientists can use it to home in on specific locations within the human genetic code to swap out a problematic section of DNA with a corrected segment. The advance has created enormous excitement over its potential for curing or preventing genetic diseases, and galvanized an \u003ca href=\"https://www.kqed.org/futureofyou/435096/as-human-gene-editing-advances-doudna-says-ethical-discussions-cant-wait\" target=\"_blank\" rel=\"noopener\">ethical debate\u003c/a> over \u003ca href=\"https://www.kqed.org/science/1934926/trying-to-understand-the-crispr-baby-five-things-to-read\" target=\"_blank\" rel=\"noopener\">human genome editing\u003c/a>.\u003c/p>\n\u003cp>\u003ca href=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115.jpg\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"aligncenter size-large wp-image-1938166\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-1020x677.jpg\" alt=\"\" width=\"640\" height=\"425\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-1020x677.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-160x106.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-800x531.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-768x510.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-1200x797.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1.jpg 1280w\" sizes=\"(max-width: 640px) 100vw, 640px\">\u003c/a>\u003c/p>\n\u003cp>UC and Broad have been slugging it out in administrative hearings and the courts since 2014, when Broad, which had paid for an expedited review, received a key patent while UC still waited for approval on its own filing. UC eventually \u003ca href=\"https://www.kqed.org/futureofyou/444391/uc-loses-appeal-on-crispr-patent\" target=\"_blank\" rel=\"noopener\">lost\u003c/a> the legal fight, but on Feb. 8, \u003ca href=\"https://www.reuters.com/article/us-ucberkeley-ip-crispr/university-of-california-to-be-granted-pioneering-crispr-patent-idUSKCN1PX25K\" target=\"_blank\" rel=\"noopener\">news broke\u003c/a> that the U.S. Patent and Trademark Office will finally issue UC’s foundational CRISPR/Cas9 patent. Not everyone expected the decision, and it has created a potentially even bigger muddle over who will get paid for what should the considerable hopes for the technology come to fruition.\u003c/p>\n\u003cp>To sort out the complexities, KQED spoke with two people following the case, reporter \u003ca href=\"https://www.statnews.com/2019/02/08/the-university-of-california-gets-its-key-crispr-patent/\" target=\"_blank\" rel=\"noopener\">Sharon Begley\u003c/a> of the health news website \u003ca href=\"https://www.statnews.com/\" target=\"_blank\" rel=\"noopener\">STAT,\u003c/a> and \u003ca href=\"https://www.nyls.edu/faculty/faculty-profiles/faculty_profiles/jacob-s-sherkow/\" target=\"_blank\" rel=\"noopener\">Professor Jacob Sherkow\u003c/a>, of the Innovation Center for Law and Technology at New York Law School. Some key points …\u003c/p>\n\u003cp>\u003cstrong>What’s the history of the dispute between Broad and UC? \u003c/strong>\u003c/p>\n\u003cp>In 2012, University of California Berkeley biochemist \u003ca href=\"https://chemistry.berkeley.edu/faculty/chem/doudna\" target=\"_blank\" rel=\"noopener\">Jennifer Doudna\u003c/a> and colleagues made a seminal discovery about how to use CRISPR/Cas9 for gene editing, performing their experiments on DNA in a test tube. UC subsequently filed with the United States Patent and Trademark Office for a patent on the process.\u003c/p>\n\u003cp>In 2013, a group led by molecular biologist \u003ca href=\"https://www.broadinstitute.org/bios/feng-zhang\" target=\"_blank\" rel=\"noopener\">Feng Zhang\u003c/a> at the Broad Institute in Boston also edited genes using CRISPR. This team was able to edit the DNA inside of actual mouse and human cells. Broad filed for a patent on its process, and in 2014 the United States Patent and Trademark Office granted the patents.\u003c/p>\n\u003cp>At that point, UC claimed the Broad patents “interfered” with the patent for which UC had applied. “That’s called an interference proceeding,” Begley said of the legal maneuver. “Lawyers make gazillions of dollars on it.”\u003c/p>\n\u003cp>But the tactic didn’t work. In February 2017, the Patent office ruled that Broad’s patents were sufficiently different from the one UC claimed, so no interference had occurred. UC then took Broad to court, and in September 2018, the U.S. Court of Appeals for the Federal Circuit affirmed the patent office’s decision to award Broad its patents.\u003c/p>\n\u003cp>“So at this point, and again, we’re six years later, UC’s (2013) patent application is still sitting there at the Patent and Trademark Office,” said Begley. “And what happened on February 8 is that the patent office finally said, ‘UC, we are going to give you this patent that you applied for on the basic use of CRISPR in all kinds of cells.’ And that should be very good news for UC.”\u003c/p>\n\u003cp>Jacob Sherkow, a patent law expert who has been following the case closely, wrote in an email that he was surprised at the news that UC is going to be awarded the patent for which it filed in 2013. He thought UC would run into trouble given the filing of an earlier patent from a Lithuanian researcher named Virginijus Šikšnys, who won the prestigious Kavli Prize last year for “seminal” CRISPR advances. Science magazine \u003ca href=\"https://www.sciencemag.org/news/2018/06/prestigious-prize-overshadowed-crispr-researcher-wins-spotlight\" target=\"_blank\" rel=\"noopener\">called\u003c/a> Šikšnys an “overshadowed” researcher in the field.\u003c/p>\n\u003cp>“Surprisingly — and I wasn’t the only one surprised — the patent office assigned the patent to a new examiner, who simply allowed the patent with only minimal comment,” Sherkow said.\u003c/p>\n\u003cp>\u003cstrong>How much are these patents worth?\u003c/strong>\u003c/p>\n\u003cp>A lot of companies are trying to produce human therapies using CRISPR. If they succeed, “that market almost certainly will be worth tens of billions of dollars per year,” said Begley. But the holder of the patent that enables these advances won’t get a huge slice. Despite early reports that the patents might be worth billions of dollars, she said the figure will “almost certainly be in the millions of dollars.”\u003c/p>\n\u003cp>Sherkow’s back-of-the-envelope calculation puts that figure at “easily tens of millions” per year, or $100 million over the life of the patent. Developments over the last several years, he says, have diminished the stakes.\u003c/p>\n\u003cp>“When the case was first filed, it was unclear whether enzymes other than Cas9 would have the same efficacy. Now, \u003ca href=\"https://www.latimes.com/science/sciencenow/la-sci-sn-crisper-casx-cas12b-20190204-story.html\" target=\"_blank\" rel=\"noopener\">we’ve got a riot\u003c/a>: Cas12, CasX, CasY, etc. ”\u003c/p>\n\u003cp>Begley said the scientists she’s spoken to “have sort of been rooting for UC because it’s a public institution,” whereas Harvard and MIT are private universities. With the funding difficulties UC has had over the years, “if a significant revenue stream can come to UC as a result of [CRISPR patents], there’s just a lot of people who are hoping that happens,” Begley said.\u003c/p>\n\u003cp>\u003cstrong>Is the dispute over? \u003c/strong>\u003c/p>\n\u003cp>The Broad Institute could challenge UC’s patents in a procedure called a post-grant review, said Sherkow. He said Broad has nine months from the date the UC patent is issued to do that.\u003c/p>\n\u003cp>“Alternatively, there may be an opportunity to bring a lawsuit in federal court challenging the UC patent,” he said.\u003c/p>\n\u003cp>For now, it appears UC and Broad may both need to be compensated for any applications using CRISPR in humans. Begley said we’re “years away” from knowing the final outcome of the dispute. “Not until there’s an actual commercialized therapy.”\u003c/p>\n\u003cp>And there could be one more complication down the road: Another expert in intellectual property and innovation, Stanford Law Professor Lisa Larrimore Ouellette, \u003ca href=\"https://www.statnews.com/2019/02/08/the-university-of-california-gets-its-key-crispr-patent/\" target=\"_blank\" rel=\"noopener\">told Begley\u003c/a> that UC’s patent could be vulnerable to challenges based on something called the “enablement clause,” which requires that patents allow for anyone to follow a series of steps to carry out the invention. Sherkow agreed UC’s patent may fall short in this regard.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>\u003cem>Brian Watt and Danielle Venton contributed to this post.\u003c/em>\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n","blocks":[],"excerpt":"Recent news that the U.S. Patent and Trademark Office will award a key CRISPR/Cas9 patent to the University of California may create even more complications in its long-running dispute with the Broad Institute.","status":"publish","parent":0,"modified":1704848841,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":26,"wordCount":1136},"headData":{"title":"Making Sense of the CRISPR Patent Dispute Between the University of California and Broad | KQED","description":"Recent news that the U.S. Patent and Trademark Office will award a key CRISPR/Cas9 patent to the University of California may create even more complications in its long-running dispute with the Broad Institute.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":""},"audioUrl":"https://www.kqed.org/.stream/anon/radio/science/2019/02/WattCrisprPatent.mp3","sticky":false,"audioTrackLength":159,"path":"/science/1938007/making-sense-of-the-crispr-patent-dispute-between-the-university-of-california-and-broad","audioDuration":182000,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Anyone fond of intricate intellectual property disputes (and really, who isn’t?) is going to have their cup runnething over when it comes to the long-running battle between the University of California and Harvard/MIT’s Broad Institute to determine who will get to cash in the most on their seminal CRISPR/Cas 9 discoveries.\u003c/p>\n\u003cp>CRISPR/Cas9 is a precise gene-editing technology. Scientists can use it to home in on specific locations within the human genetic code to swap out a problematic section of DNA with a corrected segment. The advance has created enormous excitement over its potential for curing or preventing genetic diseases, and galvanized an \u003ca href=\"https://www.kqed.org/futureofyou/435096/as-human-gene-editing-advances-doudna-says-ethical-discussions-cant-wait\" target=\"_blank\" rel=\"noopener\">ethical debate\u003c/a> over \u003ca href=\"https://www.kqed.org/science/1934926/trying-to-understand-the-crispr-baby-five-things-to-read\" target=\"_blank\" rel=\"noopener\">human genome editing\u003c/a>.\u003c/p>\n\u003cp>\u003ca href=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115.jpg\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"aligncenter size-large wp-image-1938166\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-1020x677.jpg\" alt=\"\" width=\"640\" height=\"425\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-1020x677.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-160x106.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-800x531.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-768x510.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1-1200x797.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2039/02/DESKTOP_CRISPR_171115-1.jpg 1280w\" sizes=\"(max-width: 640px) 100vw, 640px\">\u003c/a>\u003c/p>\n\u003cp>UC and Broad have been slugging it out in administrative hearings and the courts since 2014, when Broad, which had paid for an expedited review, received a key patent while UC still waited for approval on its own filing. UC eventually \u003ca href=\"https://www.kqed.org/futureofyou/444391/uc-loses-appeal-on-crispr-patent\" target=\"_blank\" rel=\"noopener\">lost\u003c/a> the legal fight, but on Feb. 8, \u003ca href=\"https://www.reuters.com/article/us-ucberkeley-ip-crispr/university-of-california-to-be-granted-pioneering-crispr-patent-idUSKCN1PX25K\" target=\"_blank\" rel=\"noopener\">news broke\u003c/a> that the U.S. Patent and Trademark Office will finally issue UC’s foundational CRISPR/Cas9 patent. Not everyone expected the decision, and it has created a potentially even bigger muddle over who will get paid for what should the considerable hopes for the technology come to fruition.\u003c/p>\n\u003cp>To sort out the complexities, KQED spoke with two people following the case, reporter \u003ca href=\"https://www.statnews.com/2019/02/08/the-university-of-california-gets-its-key-crispr-patent/\" target=\"_blank\" rel=\"noopener\">Sharon Begley\u003c/a> of the health news website \u003ca href=\"https://www.statnews.com/\" target=\"_blank\" rel=\"noopener\">STAT,\u003c/a> and \u003ca href=\"https://www.nyls.edu/faculty/faculty-profiles/faculty_profiles/jacob-s-sherkow/\" target=\"_blank\" rel=\"noopener\">Professor Jacob Sherkow\u003c/a>, of the Innovation Center for Law and Technology at New York Law School. Some key points …\u003c/p>\n\u003cp>\u003cstrong>What’s the history of the dispute between Broad and UC? \u003c/strong>\u003c/p>\n\u003cp>In 2012, University of California Berkeley biochemist \u003ca href=\"https://chemistry.berkeley.edu/faculty/chem/doudna\" target=\"_blank\" rel=\"noopener\">Jennifer Doudna\u003c/a> and colleagues made a seminal discovery about how to use CRISPR/Cas9 for gene editing, performing their experiments on DNA in a test tube. UC subsequently filed with the United States Patent and Trademark Office for a patent on the process.\u003c/p>\n\u003cp>In 2013, a group led by molecular biologist \u003ca href=\"https://www.broadinstitute.org/bios/feng-zhang\" target=\"_blank\" rel=\"noopener\">Feng Zhang\u003c/a> at the Broad Institute in Boston also edited genes using CRISPR. This team was able to edit the DNA inside of actual mouse and human cells. Broad filed for a patent on its process, and in 2014 the United States Patent and Trademark Office granted the patents.\u003c/p>\n\u003cp>At that point, UC claimed the Broad patents “interfered” with the patent for which UC had applied. “That’s called an interference proceeding,” Begley said of the legal maneuver. “Lawyers make gazillions of dollars on it.”\u003c/p>\n\u003cp>But the tactic didn’t work. In February 2017, the Patent office ruled that Broad’s patents were sufficiently different from the one UC claimed, so no interference had occurred. UC then took Broad to court, and in September 2018, the U.S. Court of Appeals for the Federal Circuit affirmed the patent office’s decision to award Broad its patents.\u003c/p>\n\u003cp>“So at this point, and again, we’re six years later, UC’s (2013) patent application is still sitting there at the Patent and Trademark Office,” said Begley. “And what happened on February 8 is that the patent office finally said, ‘UC, we are going to give you this patent that you applied for on the basic use of CRISPR in all kinds of cells.’ And that should be very good news for UC.”\u003c/p>\n\u003cp>Jacob Sherkow, a patent law expert who has been following the case closely, wrote in an email that he was surprised at the news that UC is going to be awarded the patent for which it filed in 2013. He thought UC would run into trouble given the filing of an earlier patent from a Lithuanian researcher named Virginijus Šikšnys, who won the prestigious Kavli Prize last year for “seminal” CRISPR advances. Science magazine \u003ca href=\"https://www.sciencemag.org/news/2018/06/prestigious-prize-overshadowed-crispr-researcher-wins-spotlight\" target=\"_blank\" rel=\"noopener\">called\u003c/a> Šikšnys an “overshadowed” researcher in the field.\u003c/p>\n\u003cp>“Surprisingly — and I wasn’t the only one surprised — the patent office assigned the patent to a new examiner, who simply allowed the patent with only minimal comment,” Sherkow said.\u003c/p>\n\u003cp>\u003cstrong>How much are these patents worth?\u003c/strong>\u003c/p>\n\u003cp>A lot of companies are trying to produce human therapies using CRISPR. If they succeed, “that market almost certainly will be worth tens of billions of dollars per year,” said Begley. But the holder of the patent that enables these advances won’t get a huge slice. Despite early reports that the patents might be worth billions of dollars, she said the figure will “almost certainly be in the millions of dollars.”\u003c/p>\n\u003cp>Sherkow’s back-of-the-envelope calculation puts that figure at “easily tens of millions” per year, or $100 million over the life of the patent. Developments over the last several years, he says, have diminished the stakes.\u003c/p>\n\u003cp>“When the case was first filed, it was unclear whether enzymes other than Cas9 would have the same efficacy. Now, \u003ca href=\"https://www.latimes.com/science/sciencenow/la-sci-sn-crisper-casx-cas12b-20190204-story.html\" target=\"_blank\" rel=\"noopener\">we’ve got a riot\u003c/a>: Cas12, CasX, CasY, etc. ”\u003c/p>\n\u003cp>Begley said the scientists she’s spoken to “have sort of been rooting for UC because it’s a public institution,” whereas Harvard and MIT are private universities. With the funding difficulties UC has had over the years, “if a significant revenue stream can come to UC as a result of [CRISPR patents], there’s just a lot of people who are hoping that happens,” Begley said.\u003c/p>\n\u003cp>\u003cstrong>Is the dispute over? \u003c/strong>\u003c/p>\n\u003cp>The Broad Institute could challenge UC’s patents in a procedure called a post-grant review, said Sherkow. He said Broad has nine months from the date the UC patent is issued to do that.\u003c/p>\n\u003cp>“Alternatively, there may be an opportunity to bring a lawsuit in federal court challenging the UC patent,” he said.\u003c/p>\n\u003cp>For now, it appears UC and Broad may both need to be compensated for any applications using CRISPR in humans. Begley said we’re “years away” from knowing the final outcome of the dispute. “Not until there’s an actual commercialized therapy.”\u003c/p>\n\u003cp>And there could be one more complication down the road: Another expert in intellectual property and innovation, Stanford Law Professor Lisa Larrimore Ouellette, \u003ca href=\"https://www.statnews.com/2019/02/08/the-university-of-california-gets-its-key-crispr-patent/\" target=\"_blank\" rel=\"noopener\">told Begley\u003c/a> that UC’s patent could be vulnerable to challenges based on something called the “enablement clause,” which requires that patents allow for anyone to follow a series of steps to carry out the invention. Sherkow agreed UC’s patent may fall short in this regard.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>\u003cem>Brian Watt and Danielle Venton contributed to this post.\u003c/em>\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"floatright"},"numeric":["floatright"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/science/1938007/making-sense-of-the-crispr-patent-dispute-between-the-university-of-california-and-broad","authors":["80"],"categories":["science_30","science_3890","science_40"],"tags":["science_3841","science_1287","science_3370","science_327","science_3830"],"featImg":"science_1938166","label":"science"},"science_1935019":{"type":"posts","id":"science_1935019","meta":{"index":"posts_1591205157","site":"science","id":"1935019","score":null,"sort":[1543528310000]},"guestAuthors":[],"slug":"science-summit-denounces-gene-edited-babies-claim-but-rejects-moratorium","title":"Science Summit Denounces Gene-Edited Babies Claim, But Not Future Research","publishDate":1543528310,"format":"standard","headTitle":"Science Summit Denounces Gene-Edited Babies Claim, But Not Future Research | KQED","labelTerm":{},"content":"\u003cp>A Chinese scientist’s claims that he created the world’s first gene-edited babies is a “deeply disturbing” and “irresponsible” violation of international scientific norms, according to a formal conclusion issued Thursday by organizers of the \u003ca href=\"http://www.nationalacademies.org/gene-editing/2nd_summit/index.htm\">Second International Summit on Human Genome Editing\u003c/a> in Hong Kong.\u003c/p>\n\u003cp>But the summit rejected calls for a blanket moratorium on such research, saying that the work could eventually lead to new ways to prevent a long list of serious genetic diseases.\u003c/p>\n\u003cp>“Making changes in the DNA of embryos could allow parents carrying disease-causing mutation have healthy genetically related children,” said \u003ca href=\"https://www.broadinstitute.org/what-broad/history-leadership/board-scientific-counselors/bios/david-baltimore-phd\">David Baltimore\u003c/a>, a Nobel-prize winning U.S. biologist who chaired the summit.\u003c/p>\n\u003cp>The summit was jolted by scientist \u003ca href=\"http://www.sustc-genome.org.cn/\">He Jiankui’s \u003c/a>\u003ca href=\"https://www.npr.org/sections/health-shots/2018/11/26/670752865/chinese-scientist-says-hes-first-to-genetically-edit-babies\">surprise and unverified claims\u003c/a> earlier this week that he had edited the genes of twin girls who were born last month.\u003c/p>\n\u003cp>\u003ca href=\"http://www.sustc-genome.org.cn/\">He, \u003c/a>of the Southern University of Science and Technology in Shenzhen, China, claims he modified the embryos of the twins with the gene-editing technique \u003ca href=\"https://www.npr.org/tags/419142387/crispr\">CRISPR\u003c/a> so that they would be immune to the AIDS virus. His claims remain unproven.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>Nevertheless, hundreds of scientists from dozens of countries were engrossed by his claims as they gathered for the three-day summit, which was organized by the Academy of Sciences of Hong Kong, the Royal Society of London, the U.S. National Academy of Sciences and the U.S. National Academy of Medicine.\u003c/p>\n\u003cp>The goal was to reach a global scientific consensus on how scientists might some day ethically use powerful new gene-editing techniques such as \u003ca href=\"https://www.npr.org/tags/419142387/crispr\">CRISPR\u003c/a> to edit the human genetic blueprint.\u003c/p>\n\u003cp>\u003cstrong>A question of ethics\u003c/strong>\u003c/p>\n\u003cp>In the summit’s closing \u003ca href=\"http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID=11282018b\">statement\u003c/a> released early Thursday, the organizers called for an investigation to verify or refute He’s claims. But regardless of whether it is true, the organizers said the researcher’s experiment was premature, deeply flawed and unethical.\u003c/p>\n\u003cfigure id=\"attachment_1935027\" class=\"wp-caption alignright\" style=\"max-width: 800px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"size-medium wp-image-1935027\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-800x533.jpg\" alt=\"\" width=\"800\" height=\"533\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-800x533.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-160x107.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-768x512.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-1020x680.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-1200x800.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-1920x1280.jpg 1920w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-1180x787.jpg 1180w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-960x640.jpg 960w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-240x160.jpg 240w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-375x250.jpg 375w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-520x347.jpg 520w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003cfigcaption class=\"wp-caption-text\">Chinese scientist He Jiankui speaks at the Second International Summit on Human Genome Editing in Hong Kong on November 28, 2018. \u003ccite>(ANTHONY WALLACE/AFP/Getty Images)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>“Its flaws include an inadequate medical indication, a poorly designed study protocol, a failure to meet ethical standards for protecting the welfare of research subjects, and a lack of transparency in the development, review, and conduct of the clinical procedures,” said Baltimore.\u003c/p>\n\u003cp>Much more research is needed before anyone tries to prevent diseases by editing human embryos, the organizers concluded.\u003c/p>\n\u003cp>“Making changes in the DNA of embryos … could allow parents who carry disease-causing mutations to have healthy, genetically related children,” Baltimore said. “However, heritable genome editing of … embryos … poses risks that remain difficult to evaluate.”\u003c/p>\n\u003cp>But enough scientific advances have been made since the last summit in 2015 to begin plotting a course for how that could happen some day, according to the statement.\u003c/p>\n\u003cp>“Progress over the last three years and the discussions at the current summit, … suggest that it is time to define a rigorous, responsible … pathway toward such trials,” said Baltimore, a Nobel-prize winning U.S. biologist.\u003c/p>\n\u003cp>In doing this, the organizers rejected calls for a moratorium on such research.\u003c/p>\n\u003cp>Baltimore said “draconian bans would be antithetical to the goals of science,” and unnecessarily hinder the advancement of science.\u003c/p>\n\u003cp>\u003ca href=\"https://law.wisc.edu/profiles/racharo\">R. Alta Charo\u003c/a>, a University of Wisconsin bioethicist who helped organize the summit, argued that just because one scientist violated scientific norms, doesn’t necessarily mean the scientific system is flawed.\u003c/p>\n\u003cp>“I think the failure was his, not the failure of the scientific community,” Charo said. “You can’t expect to have perfection. What you can try to do is minimize these incidents with the constant effort of conversation and oversight and ultimately enforcement measures that will discourage rogue behavior that goes outside international norms.”\u003c/p>\n\u003cp>\u003cstrong>Concerns and possible benefits\u003c/strong>\u003c/p>\n\u003cp>Making changes to the DNA in human embryos has long been considered taboo because of safety concerns and fears it could lead to “designer babies” — children whose traits are picked to make supposedly genetically superior people.\u003c/p>\n\u003cp>But many scientists have now become convinced that it may be ethical someday to edit human embryos to prevent genetic disorders, such as Huntington’s disease, cystic fibrosis, muscular dystrophy and hemophilia. And several scientists have \u003ca href=\"https://www.npr.org/sections/health-shots/2017/08/18/543769759/a-first-look-inside-the-lab-where-scientists-are-editing-dna-in-human-embryos\">already edited\u003c/a> human embryos in their labs to try to determine the safety and effectiveness of the procedure.\u003c/p>\n\u003cp>Most scientist and bioethicists agree that it is far too early to try to make babies from edited human embryos — primarily because safety protocols for the technique remain unclear.\u003c/p>\n\u003cp>DNA editing may inadvertently cause genetic mutations that could cause health problems for any babies created this way and cause new health problems that would then be passed down for generations.\u003c/p>\n\u003cp>Some oppose all efforts to create genetically modified babies, saying it will be extremely difficult to draw a clear line between medical uses and attempts to create genetically enhanced individuals. And that could lead to a world of genetic haves and have-nots.\u003c/p>\n\u003cp>\u003cstrong>Varied regulations\u003c/strong>\u003c/p>\n\u003cp>While gene-editing experiments on human embryos is \u003ca href=\"https://rbej.biomedcentral.com/articles/10.1186/1477-7827-12-108\">prohibited\u003c/a> in many countries, it has not been barred in many others. And scientist have long relied on self-regulation to prevent new technologies from being abused.\u003c/p>\n\u003cp>The summit statement came amid a growing call for governments around the world to impose enforceable moratoriums on any future experiments. While such experiments are prohibited in some countries, previous scientific policing has largely relied on scientists to follow guidelines.\u003c/p>\n\u003cp>As the summit opened, \u003ca href=\"https://www.broadinstitute.org/bios/feng-zhang\">Feng Zhang\u003c/a>, an MIT scientist who helped develop CRISPR, immediately \u003ca href=\"https://www.broadinstitute.org/news/crispr-pioneers-feng-zhang-and-david-liu-respond-report-embryo-editing-china\">called\u003c/a> for a moratorium on such experiments.\u003c/p>\n\u003cp>“Given the current early state of genome editing technology, I’m in favor of a moratorium on implantation of edited embryos … until we have come up with a thoughtful set of safety requirements first,” Zhang wrote in a statement.\u003c/p>\n\u003cp>But after the summit, Zhang said he agreed with the outcome.\u003c/p>\n\u003cp>“Rather than focusing on criticizing what has happened, we should learn the lessons that it has taught us,” Feng wrote in an email. “There is a lot of potential for using gene editing to alleviate disease suffering, and providing a productive path forward is the best way to ensure that patient’s hopes will get realized.”\u003c/p>\n\u003cp>As the last day of the summit was getting underway, more than 100 activists, bioethicists, scientists and other released a joint \u003ca href=\"https://www.geneticsandsociety.org/internal-content/civil-society-statement-organizers-second-international-summit-human-genome\">statement \u003c/a>calling for the summit to call on governments and the United Nations to adopt moratoriums.\u003c/p>\n\u003cp>“If the organizers of this week’s summit in Hong Kong wish to demonstrate that science is not out of control, and is worthy of public trust, now is the time for them and the rest of the international scientific community to act,” the statement said.\u003c/p>\n\u003cp>It noted that when He \u003ca href=\"https://www.npr.org/sections/health-shots/2018/11/28/671375070/facing-backlash-chinese-scientist-defends-gene-editing-research-on-babies\">defended his experiment\u003c/a> at the summit, he justified his experiment in part on a 2017 \u003ca href=\"https://www.nap.edu/catalog/24623/human-genome-editing-science-ethics-and-governance\">report\u003c/a> from the National Academies of Sciences. That report concluded that clinical trials “might be permitted” after laboratory studies show it would be safe and then only for “compelling medical reasons in the absence of reasonable alternatives.”\u003c/p>\n\u003cp>That sentiment was echoed by the Berkeley, Calif., based Center for Genetics and Society, which accused the summit organizers of “complicity” in He’s rogue research, saying the recommendations of the National Academies and the \u003ca href=\"http://nuffieldbioethics.org/about\">Nuttfield Council of Bioethics\u003c/a> had been interpreted as a “green light” by He.\u003c/p>\n\u003cp>In their closing statement, the summit organizers “all but said outright that nothing will get in their way: not laws in dozens of countries or an international treaty, not widespread public and civil society opposition, not deep concern among their own scientific community, and not a grandstanding researcher,” CGS said in a statement.\u003c/p>\n\u003cp>\u003ca href=\"http://www.hgalert.org/topics/hge/threat.htm\">David King\u003c/a> of Human Genetics Alert, brought up the specter of “[the] horrifying history of eugenics in the 20th century,” and warned of the “disastrous consequences of going down this path.”\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>“It should act immediately to prohibit such experiments, and ensure that He Jiankui is prosecuted as a warning to others,” King said in a statement.\u003c/p>\n\u003cdiv class=\"fullattribution\">\u003cem>Copyright 2018 \u003ca href=\"https://www.npr.org\">NPR\u003c/a>.\u003c/em>\u003c/div>\n\n","blocks":[],"excerpt":"The Second International Summit on Human Genome Editing issues a consensus on how scientists might responsibly move forward to create gene-edited babies in the wake of a rogue scientist's claims.","status":"publish","parent":0,"modified":1704927276,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":40,"wordCount":1386},"headData":{"title":"Science Summit Denounces Gene-Edited Babies Claim, But Not Future Research | KQED","description":"The Second International Summit on Human Genome Editing issues a consensus on how scientists might responsibly move forward to create gene-edited babies in the wake of a rogue scientist's claims.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":""},"source":"NPR","sticky":false,"nprImageCredit":"China News Service","nprByline":"Rob Stein\u003cbr />NPR","nprImageAgency":"VCG via Getty Images","nprStoryId":"671657301","nprApiLink":"http://api.npr.org/query?id=671657301&apiKey=MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004","nprHtmlLink":"https://www.npr.org/sections/health-shots/2018/11/29/671657301/international-science-summit-denounces-gene-edited-babies-but-rejects-moratorium?ft=nprml&f=671657301","nprRetrievedStory":"1","nprPubDate":"Thu, 29 Nov 2018 11:30:00 -0500","nprStoryDate":"Thu, 29 Nov 2018 02:06:00 -0500","nprLastModifiedDate":"Thu, 29 Nov 2018 11:30:02 -0500","path":"/science/1935019/science-summit-denounces-gene-edited-babies-claim-but-rejects-moratorium","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>A Chinese scientist’s claims that he created the world’s first gene-edited babies is a “deeply disturbing” and “irresponsible” violation of international scientific norms, according to a formal conclusion issued Thursday by organizers of the \u003ca href=\"http://www.nationalacademies.org/gene-editing/2nd_summit/index.htm\">Second International Summit on Human Genome Editing\u003c/a> in Hong Kong.\u003c/p>\n\u003cp>But the summit rejected calls for a blanket moratorium on such research, saying that the work could eventually lead to new ways to prevent a long list of serious genetic diseases.\u003c/p>\n\u003cp>“Making changes in the DNA of embryos could allow parents carrying disease-causing mutation have healthy genetically related children,” said \u003ca href=\"https://www.broadinstitute.org/what-broad/history-leadership/board-scientific-counselors/bios/david-baltimore-phd\">David Baltimore\u003c/a>, a Nobel-prize winning U.S. biologist who chaired the summit.\u003c/p>\n\u003cp>The summit was jolted by scientist \u003ca href=\"http://www.sustc-genome.org.cn/\">He Jiankui’s \u003c/a>\u003ca href=\"https://www.npr.org/sections/health-shots/2018/11/26/670752865/chinese-scientist-says-hes-first-to-genetically-edit-babies\">surprise and unverified claims\u003c/a> earlier this week that he had edited the genes of twin girls who were born last month.\u003c/p>\n\u003cp>\u003ca href=\"http://www.sustc-genome.org.cn/\">He, \u003c/a>of the Southern University of Science and Technology in Shenzhen, China, claims he modified the embryos of the twins with the gene-editing technique \u003ca href=\"https://www.npr.org/tags/419142387/crispr\">CRISPR\u003c/a> so that they would be immune to the AIDS virus. His claims remain unproven.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>Nevertheless, hundreds of scientists from dozens of countries were engrossed by his claims as they gathered for the three-day summit, which was organized by the Academy of Sciences of Hong Kong, the Royal Society of London, the U.S. National Academy of Sciences and the U.S. National Academy of Medicine.\u003c/p>\n\u003cp>The goal was to reach a global scientific consensus on how scientists might some day ethically use powerful new gene-editing techniques such as \u003ca href=\"https://www.npr.org/tags/419142387/crispr\">CRISPR\u003c/a> to edit the human genetic blueprint.\u003c/p>\n\u003cp>\u003cstrong>A question of ethics\u003c/strong>\u003c/p>\n\u003cp>In the summit’s closing \u003ca href=\"http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID=11282018b\">statement\u003c/a> released early Thursday, the organizers called for an investigation to verify or refute He’s claims. But regardless of whether it is true, the organizers said the researcher’s experiment was premature, deeply flawed and unethical.\u003c/p>\n\u003cfigure id=\"attachment_1935027\" class=\"wp-caption alignright\" style=\"max-width: 800px\">\u003cimg loading=\"lazy\" decoding=\"async\" class=\"size-medium wp-image-1935027\" src=\"https://ww2.kqed.org/science/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-800x533.jpg\" alt=\"\" width=\"800\" height=\"533\" srcset=\"https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-800x533.jpg 800w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-160x107.jpg 160w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-768x512.jpg 768w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-1020x680.jpg 1020w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-1200x800.jpg 1200w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-1920x1280.jpg 1920w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-1180x787.jpg 1180w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-960x640.jpg 960w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-240x160.jpg 240w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-375x250.jpg 375w, https://cdn.kqed.org/wp-content/uploads/sites/35/2018/11/GettyImages-1066011752-520x347.jpg 520w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003cfigcaption class=\"wp-caption-text\">Chinese scientist He Jiankui speaks at the Second International Summit on Human Genome Editing in Hong Kong on November 28, 2018. \u003ccite>(ANTHONY WALLACE/AFP/Getty Images)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>“Its flaws include an inadequate medical indication, a poorly designed study protocol, a failure to meet ethical standards for protecting the welfare of research subjects, and a lack of transparency in the development, review, and conduct of the clinical procedures,” said Baltimore.\u003c/p>\n\u003cp>Much more research is needed before anyone tries to prevent diseases by editing human embryos, the organizers concluded.\u003c/p>\n\u003cp>“Making changes in the DNA of embryos … could allow parents who carry disease-causing mutations to have healthy, genetically related children,” Baltimore said. “However, heritable genome editing of … embryos … poses risks that remain difficult to evaluate.”\u003c/p>\n\u003cp>But enough scientific advances have been made since the last summit in 2015 to begin plotting a course for how that could happen some day, according to the statement.\u003c/p>\n\u003cp>“Progress over the last three years and the discussions at the current summit, … suggest that it is time to define a rigorous, responsible … pathway toward such trials,” said Baltimore, a Nobel-prize winning U.S. biologist.\u003c/p>\n\u003cp>In doing this, the organizers rejected calls for a moratorium on such research.\u003c/p>\n\u003cp>Baltimore said “draconian bans would be antithetical to the goals of science,” and unnecessarily hinder the advancement of science.\u003c/p>\n\u003cp>\u003ca href=\"https://law.wisc.edu/profiles/racharo\">R. Alta Charo\u003c/a>, a University of Wisconsin bioethicist who helped organize the summit, argued that just because one scientist violated scientific norms, doesn’t necessarily mean the scientific system is flawed.\u003c/p>\n\u003cp>“I think the failure was his, not the failure of the scientific community,” Charo said. “You can’t expect to have perfection. What you can try to do is minimize these incidents with the constant effort of conversation and oversight and ultimately enforcement measures that will discourage rogue behavior that goes outside international norms.”\u003c/p>\n\u003cp>\u003cstrong>Concerns and possible benefits\u003c/strong>\u003c/p>\n\u003cp>Making changes to the DNA in human embryos has long been considered taboo because of safety concerns and fears it could lead to “designer babies” — children whose traits are picked to make supposedly genetically superior people.\u003c/p>\n\u003cp>But many scientists have now become convinced that it may be ethical someday to edit human embryos to prevent genetic disorders, such as Huntington’s disease, cystic fibrosis, muscular dystrophy and hemophilia. And several scientists have \u003ca href=\"https://www.npr.org/sections/health-shots/2017/08/18/543769759/a-first-look-inside-the-lab-where-scientists-are-editing-dna-in-human-embryos\">already edited\u003c/a> human embryos in their labs to try to determine the safety and effectiveness of the procedure.\u003c/p>\n\u003cp>Most scientist and bioethicists agree that it is far too early to try to make babies from edited human embryos — primarily because safety protocols for the technique remain unclear.\u003c/p>\n\u003cp>DNA editing may inadvertently cause genetic mutations that could cause health problems for any babies created this way and cause new health problems that would then be passed down for generations.\u003c/p>\n\u003cp>Some oppose all efforts to create genetically modified babies, saying it will be extremely difficult to draw a clear line between medical uses and attempts to create genetically enhanced individuals. And that could lead to a world of genetic haves and have-nots.\u003c/p>\n\u003cp>\u003cstrong>Varied regulations\u003c/strong>\u003c/p>\n\u003cp>While gene-editing experiments on human embryos is \u003ca href=\"https://rbej.biomedcentral.com/articles/10.1186/1477-7827-12-108\">prohibited\u003c/a> in many countries, it has not been barred in many others. And scientist have long relied on self-regulation to prevent new technologies from being abused.\u003c/p>\n\u003cp>The summit statement came amid a growing call for governments around the world to impose enforceable moratoriums on any future experiments. While such experiments are prohibited in some countries, previous scientific policing has largely relied on scientists to follow guidelines.\u003c/p>\n\u003cp>As the summit opened, \u003ca href=\"https://www.broadinstitute.org/bios/feng-zhang\">Feng Zhang\u003c/a>, an MIT scientist who helped develop CRISPR, immediately \u003ca href=\"https://www.broadinstitute.org/news/crispr-pioneers-feng-zhang-and-david-liu-respond-report-embryo-editing-china\">called\u003c/a> for a moratorium on such experiments.\u003c/p>\n\u003cp>“Given the current early state of genome editing technology, I’m in favor of a moratorium on implantation of edited embryos … until we have come up with a thoughtful set of safety requirements first,” Zhang wrote in a statement.\u003c/p>\n\u003cp>But after the summit, Zhang said he agreed with the outcome.\u003c/p>\n\u003cp>“Rather than focusing on criticizing what has happened, we should learn the lessons that it has taught us,” Feng wrote in an email. “There is a lot of potential for using gene editing to alleviate disease suffering, and providing a productive path forward is the best way to ensure that patient’s hopes will get realized.”\u003c/p>\n\u003cp>As the last day of the summit was getting underway, more than 100 activists, bioethicists, scientists and other released a joint \u003ca href=\"https://www.geneticsandsociety.org/internal-content/civil-society-statement-organizers-second-international-summit-human-genome\">statement \u003c/a>calling for the summit to call on governments and the United Nations to adopt moratoriums.\u003c/p>\n\u003cp>“If the organizers of this week’s summit in Hong Kong wish to demonstrate that science is not out of control, and is worthy of public trust, now is the time for them and the rest of the international scientific community to act,” the statement said.\u003c/p>\n\u003cp>It noted that when He \u003ca href=\"https://www.npr.org/sections/health-shots/2018/11/28/671375070/facing-backlash-chinese-scientist-defends-gene-editing-research-on-babies\">defended his experiment\u003c/a> at the summit, he justified his experiment in part on a 2017 \u003ca href=\"https://www.nap.edu/catalog/24623/human-genome-editing-science-ethics-and-governance\">report\u003c/a> from the National Academies of Sciences. That report concluded that clinical trials “might be permitted” after laboratory studies show it would be safe and then only for “compelling medical reasons in the absence of reasonable alternatives.”\u003c/p>\n\u003cp>That sentiment was echoed by the Berkeley, Calif., based Center for Genetics and Society, which accused the summit organizers of “complicity” in He’s rogue research, saying the recommendations of the National Academies and the \u003ca href=\"http://nuffieldbioethics.org/about\">Nuttfield Council of Bioethics\u003c/a> had been interpreted as a “green light” by He.\u003c/p>\n\u003cp>In their closing statement, the summit organizers “all but said outright that nothing will get in their way: not laws in dozens of countries or an international treaty, not widespread public and civil society opposition, not deep concern among their own scientific community, and not a grandstanding researcher,” CGS said in a statement.\u003c/p>\n\u003cp>\u003ca href=\"http://www.hgalert.org/topics/hge/threat.htm\">David King\u003c/a> of Human Genetics Alert, brought up the specter of “[the] horrifying history of eugenics in the 20th century,” and warned of the “disastrous consequences of going down this path.”\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"floatright"},"numeric":["floatright"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>“It should act immediately to prohibit such experiments, and ensure that He Jiankui is prosecuted as a warning to others,” King said in a statement.\u003c/p>\n\u003cdiv class=\"fullattribution\">\u003cem>Copyright 2018 \u003ca href=\"https://www.npr.org\">NPR\u003c/a>.\u003c/em>\u003c/div>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/science/1935019/science-summit-denounces-gene-edited-babies-claim-but-rejects-moratorium","authors":["byline_science_1935019"],"categories":["science_30","science_39","science_40"],"tags":["science_5196","science_1287","science_5181"],"featImg":"science_1935034","label":"source_science_1935019"}},"programsReducer":{"possible":{"id":"possible","title":"Possible","info":"Possible is hosted by entrepreneur Reid Hoffman and writer Aria Finger. 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You ask the questions. You decide what Bay Curious investigates. And you join us on the journey to find the answers.","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Bay-Curious-Podcast-Tile-703x703-1.jpg","imageAlt":"\"KQED Bay Curious","officialWebsiteLink":"/news/series/baycurious","meta":{"site":"news","source":"kqed","order":"4"},"link":"/podcasts/baycurious","subscribe":{"apple":"https://podcasts.apple.com/us/podcast/bay-curious/id1172473406","npr":"https://www.npr.org/podcasts/500557090/bay-curious","rss":"https://ww2.kqed.org/news/category/bay-curious-podcast/feed/podcast","google":"https://podcasts.google.com/feed/aHR0cHM6Ly93dzIua3FlZC5vcmcvbmV3cy9jYXRlZ29yeS9iYXktY3VyaW91cy1wb2RjYXN0L2ZlZWQvcG9kY2FzdA","stitcher":"https://www.stitcher.com/podcast/kqed/bay-curious","spotify":"https://open.spotify.com/show/6O76IdmhixfijmhTZLIJ8k"}},"bbc-world-service":{"id":"bbc-world-service","title":"BBC World Service","info":"The day's top stories from BBC News compiled twice daily in the week, once at weekends.","airtime":"MON-FRI 9pm-10pm, TUE-FRI 1am-2am","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/BBC-World-Service-Podcast-Tile-360x360-1.jpg","officialWebsiteLink":"https://www.bbc.co.uk/sounds/play/live:bbc_world_service","meta":{"site":"news","source":"BBC World Service"},"link":"/radio/program/bbc-world-service","subscribe":{"apple":"https://itunes.apple.com/us/podcast/global-news-podcast/id135067274?mt=2","tuneIn":"https://tunein.com/radio/BBC-World-Service-p455581/","rss":"https://podcasts.files.bbci.co.uk/p02nq0gn.rss"}},"code-switch-life-kit":{"id":"code-switch-life-kit","title":"Code Switch / Life Kit","info":"\u003cem>Code Switch\u003c/em>, which listeners will hear in the first part of the hour, has fearless and much-needed conversations about race. Hosted by journalists of color, the show tackles the subject of race head-on, exploring how it impacts every part of society — from politics and pop culture to history, sports and more.\u003cbr />\u003cbr />\u003cem>Life Kit\u003c/em>, which will be in the second part of the hour, guides you through spaces and feelings no one prepares you for — from finances to mental health, from workplace microaggressions to imposter syndrome, from relationships to parenting. The show features experts with real world experience and shares their knowledge. Because everyone needs a little help being human.\u003cbr />\u003cbr />\u003ca href=\"https://www.npr.org/podcasts/510312/codeswitch\">\u003cem>Code Switch\u003c/em> offical site and podcast\u003c/a>\u003cbr />\u003ca href=\"https://www.npr.org/lifekit\">\u003cem>Life Kit\u003c/em> offical site and podcast\u003c/a>\u003cbr />","airtime":"SUN 9pm-10pm","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Code-Switch-Life-Kit-Podcast-Tile-360x360-1.jpg","meta":{"site":"radio","source":"npr"},"link":"/radio/program/code-switch-life-kit","subscribe":{"apple":"https://podcasts.apple.com/podcast/1112190608?mt=2&at=11l79Y&ct=nprdirectory","google":"https://podcasts.google.com/feed/aHR0cHM6Ly93d3cubnByLm9yZy9yc3MvcG9kY2FzdC5waHA_aWQ9NTEwMzEy","spotify":"https://open.spotify.com/show/3bExJ9JQpkwNhoHvaIIuyV","rss":"https://feeds.npr.org/510312/podcast.xml"}},"commonwealth-club":{"id":"commonwealth-club","title":"Commonwealth Club of California Podcast","info":"The Commonwealth Club of California is the nation's oldest and largest public affairs forum. As a non-partisan forum, The Club brings to the public airwaves diverse viewpoints on important topics. The Club's weekly radio broadcast - the oldest in the U.S., dating back to 1924 - is carried across the nation on public radio stations and is now podcasting. Our website archive features audio of our recent programs, as well as selected speeches from our long and distinguished history. This podcast feed is usually updated twice a week and is always un-edited.","airtime":"THU 10pm, FRI 1am","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Commonwealth-Club-Podcast-Tile-360x360-1.jpg","officialWebsiteLink":"https://www.commonwealthclub.org/podcasts","meta":{"site":"news","source":"Commonwealth Club of California"},"link":"/radio/program/commonwealth-club","subscribe":{"apple":"https://itunes.apple.com/us/podcast/commonwealth-club-of-california-podcast/id976334034?mt=2","google":"https://podcasts.google.com/feed/aHR0cDovL3d3dy5jb21tb253ZWFsdGhjbHViLm9yZy9hdWRpby9wb2RjYXN0L3dlZWtseS54bWw","tuneIn":"https://tunein.com/radio/Commonwealth-Club-of-California-p1060/"}},"considerthis":{"id":"considerthis","title":"Consider This","tagline":"Make sense of the day","info":"Make sense of the day. Every weekday afternoon, Consider This helps you consider the major stories of the day in less than 15 minutes, featuring the reporting and storytelling resources of NPR. Plus, KQED’s Bianca Taylor brings you the local KQED news you need to know.","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Consider-This-Podcast-Tile-703x703-1.jpg","imageAlt":"Consider This from NPR and KQED","officialWebsiteLink":"/podcasts/considerthis","meta":{"site":"news","source":"kqed","order":"7"},"link":"/podcasts/considerthis","subscribe":{"apple":"https://podcasts.apple.com/podcast/id1503226625?mt=2&at=11l79Y&ct=nprdirectory","npr":"https://rpb3r.app.goo.gl/coronavirusdaily","google":"https://podcasts.google.com/feed/aHR0cHM6Ly9mZWVkcy5ucHIub3JnLzUxMDM1NS9wb2RjYXN0LnhtbA","spotify":"https://open.spotify.com/show/3Z6JdCS2d0eFEpXHKI6WqH"}},"forum":{"id":"forum","title":"Forum","tagline":"The conversation starts here","info":"KQED’s live call-in program discussing local, state, national and international issues, as well as in-depth interviews.","airtime":"MON-FRI 9am-11am, 10pm-11pm","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Forum-Podcast-Tile-703x703-1.jpg","imageAlt":"KQED Forum with Mina Kim and Alexis Madrigal","officialWebsiteLink":"/forum","meta":{"site":"news","source":"kqed","order":"8"},"link":"/forum","subscribe":{"apple":"https://podcasts.apple.com/us/podcast/kqeds-forum/id73329719","google":"https://podcasts.google.com/feed/aHR0cHM6Ly9mZWVkcy5tZWdhcGhvbmUuZm0vS1FJTkM5NTU3MzgxNjMz","npr":"https://www.npr.org/podcasts/432307980/forum","stitcher":"https://www.stitcher.com/podcast/kqedfm-kqeds-forum-podcast","rss":"https://feeds.megaphone.fm/KQINC9557381633"}},"freakonomics-radio":{"id":"freakonomics-radio","title":"Freakonomics Radio","info":"Freakonomics Radio is a one-hour award-winning podcast and public-radio project hosted by Stephen Dubner, with co-author Steve Levitt as a regular guest. 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Hosts Ki Sung and Katrina Schwartz introduce listeners to educators, researchers, parents and students who are developing effective ways to improve how kids learn. We cover topics like how fed-up administrators are developing surprising tactics to deal with classroom disruptions; how listening to podcasts are helping kids develop reading skills; the consequences of overparenting; and why interdisciplinary learning can engage students on all ends of the traditional achievement spectrum. This podcast is part of the MindShift education site, a division of KQED News. KQED is an NPR/PBS member station based in San Francisco. You can also visit the MindShift website for episodes and supplemental blog posts or tweet us \u003ca href=\"https://twitter.com/MindShiftKQED\">@MindShiftKQED\u003c/a> or visit us at \u003ca href=\"/mindshift\">MindShift.KQED.org\u003c/a>","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Mindshift-Podcast-Tile-703x703-1.jpg","imageAlt":"KQED MindShift: How We Will Learn","officialWebsiteLink":"/mindshift/","meta":{"site":"news","source":"kqed","order":"2"},"link":"/podcasts/mindshift","subscribe":{"apple":"https://podcasts.apple.com/us/podcast/mindshift-podcast/id1078765985","google":"https://podcasts.google.com/feed/aHR0cHM6Ly9mZWVkcy5tZWdhcGhvbmUuZm0vS1FJTkM1NzY0NjAwNDI5","npr":"https://www.npr.org/podcasts/464615685/mind-shift-podcast","stitcher":"https://www.stitcher.com/podcast/kqed/stories-teachers-share","spotify":"https://open.spotify.com/show/0MxSpNYZKNprFLCl7eEtyx"}},"morning-edition":{"id":"morning-edition","title":"Morning Edition","info":"\u003cem>Morning Edition\u003c/em> takes listeners around the country and the world with multi-faceted stories and commentaries every weekday. 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