Historic Gene Editing Attempt in First Human Off to a Positive Start
Major CRISPR Hurdle: Edited Cells Might Cause Cancer, Find Studies
Trying to Slow Lou Gehrig's Disease With CRISPR-Cas9
In First, Scientists Try to Cure Man's Disease by Editing DNA Inside Body
Even More Precise CRISPR Rearranges Atoms to Edit Genes
As Human Gene-Editing Advances, Doudna Says Ethical Discussions Can't Wait
Scientists Precisely Edit DNA in Human Embryos to Fix a Disease Gene
Scientists in US Edit Human Embryos With CRISPR for First Time
UC Berkeley Renews Legal Fight Over CRISPR-Cas9 Patent Rights
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She lives in Sonoma County and enjoys backpacking.","avatar":"https://secure.gravatar.com/avatar/ebaf11ee6cfb7bb40329a143d463829e?s=600&d=blank&r=g","twitter":"DanielleVenton","facebook":null,"instagram":null,"linkedin":null,"sites":[{"site":"arts","roles":["contributor"]},{"site":"news","roles":["editor"]},{"site":"futureofyou","roles":["editor"]},{"site":"science","roles":["editor"]}],"headData":{"title":"Danielle Venton | KQED","description":"Science reporter","ogImgSrc":"https://secure.gravatar.com/avatar/ebaf11ee6cfb7bb40329a143d463829e?s=600&d=blank&r=g","twImgSrc":"https://secure.gravatar.com/avatar/ebaf11ee6cfb7bb40329a143d463829e?s=600&d=blank&r=g"},"isLoading":false,"link":"/author/dventon"}},"breakingNewsReducer":{},"campaignFinanceReducer":{},"firebase":{"requesting":{},"requested":{},"timestamps":{},"data":{},"ordered":{},"auth":{"isLoaded":false,"isEmpty":true},"authError":null,"profile":{"isLoaded":false,"isEmpty":true},"listeners":{"byId":{},"allIds":[]},"isInitializing":false,"errors":[]},"navBarReducer":{"navBarId":"home","fullView":true,"showPlayer":false},"navMenuReducer":{"menus":[{"key":"menu1","items":[{"name":"News","link":"/","type":"title"},{"name":"Politics","link":"/politics"},{"name":"Science","link":"/science"},{"name":"Education","link":"/educationnews"},{"name":"Housing","link":"/housing"},{"name":"Immigration","link":"/immigration"},{"name":"Criminal Justice","link":"/criminaljustice"},{"name":"Silicon Valley","link":"/siliconvalley"},{"name":"Forum","link":"/forum"},{"name":"The California Report","link":"/californiareport"}]},{"key":"menu2","items":[{"name":"Arts & Culture","link":"/arts","type":"title"},{"name":"Critics’ Picks","link":"/thedolist"},{"name":"Cultural Commentary","link":"/artscommentary"},{"name":"Food & Drink","link":"/food"},{"name":"Bay Area Hip-Hop","link":"/bayareahiphop"},{"name":"Rebel Girls","link":"/rebelgirls"},{"name":"Arts Video","link":"/artsvideos"}]},{"key":"menu3","items":[{"name":"Podcasts","link":"/podcasts","type":"title"},{"name":"Bay Curious","link":"/podcasts/baycurious"},{"name":"Rightnowish","link":"/podcasts/rightnowish"},{"name":"The Bay","link":"/podcasts/thebay"},{"name":"On Our Watch","link":"/podcasts/onourwatch"},{"name":"Mindshift","link":"/podcasts/mindshift"},{"name":"Consider This","link":"/podcasts/considerthis"},{"name":"Political Breakdown","link":"/podcasts/politicalbreakdown"}]},{"key":"menu4","items":[{"name":"Live Radio","link":"/radio","type":"title"},{"name":"TV","link":"/tv","type":"title"},{"name":"Events","link":"/events","type":"title"},{"name":"For Educators","link":"/education","type":"title"},{"name":"Support KQED","link":"/support","type":"title"},{"name":"About","link":"/about","type":"title"},{"name":"Help Center","link":"https://kqed-helpcenter.kqed.org/s","type":"title"}]}]},"pagesReducer":{},"postsReducer":{"stream_live":{"type":"live","id":"stream_live","audioUrl":"https://streams.kqed.org/kqedradio","title":"Live Stream","excerpt":"Live Stream information currently unavailable.","link":"/radio","featImg":"","label":{"name":"KQED Live","link":"/"}},"stream_kqedNewscast":{"type":"posts","id":"stream_kqedNewscast","audioUrl":"https://www.kqed.org/.stream/anon/radio/RDnews/newscast.mp3?_=1","title":"KQED Newscast","featImg":"","label":{"name":"88.5 FM","link":"/"}},"futureofyou_444281":{"type":"posts","id":"futureofyou_444281","meta":{"index":"posts_1591205157","site":"futureofyou","id":"444281","score":null,"sort":[1536178859000]},"guestAuthors":[],"slug":"historic-gene-editing-attempt-in-first-human-off-to-a-positive-start","title":"Historic Gene Editing Attempt in First Human Off to a Positive Start","publishDate":1536178859,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp>Early, partial results from a historic gene editing study give encouraging signs that the treatment may be safe and having at least some of its hoped-for effect, but it’s too soon to know whether it ultimately will succeed.[contextly_sidebar id=\"nLB0LaskunDkFSzX8O5LVSxfj3Ayt7CZ\"]\u003c/p>\n\u003cp>The results announced Wednesday are from the first human \u003ca href=\"//mps2study.com/study-overview/?pn=\">test\u003c/a> of gene editing in the body, an attempt to permanently change someone’s DNA to cure a disease — in this case, a genetic disorder called \u003ca href=\"https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Mucopolysaccharidoses-Fact-Sheet\">Hunter syndrome\u003c/a> that often kills people in their teens.\u003c/p>\n\u003cp>In two patients who got a medium dose of the treatment, urine levels of large sugar compounds that are hallmarks of Hunter syndrome had fallen by half, on average, four months later — a possible sign the treatment is working. Two others who got a low dose have seen little change in these sugars so far.\u003c/p>\n\u003cp>There’s no way to know yet whether the change in the middle-dose patients is due to the gene editing or something else, but the fact their sugars have declined consistently since treatment suggests it might be.\u003c/p>\n\u003cp>“I cannot absolutely say it’s a treatment effect” but the drop is “really encouraging,” said the study leader, Dr. Joseph Muenzer of the University of North Carolina, Chapel Hill. The main goal of early treatment studies is to test safety, though researchers also look for hints that the therapy is working. Muenzer gave the results at a conference in Greece and consults for the treatment’s maker, California-based Sangamo Therapeutics.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>The company’s president, Dr. Sandy Macrae, said tests in about five months will reveal more, but the change in the middle-dose group so far “looks really good.”[contextly_sidebar id=\"FMPymRVSamCP4f8RpmzlwSdu0yioj0rt\"]\u003c/p>\n\u003cp>“The most rational explanation for this is that what we hoped was going to happen has happened,” he said.\u003c/p>\n\u003cp>Several independent experts agreed.\u003c/p>\n\u003cp>“The results are exciting” and suggest that the gene editing is working to some degree, without safety concerns so far, said Dr. Howard Kaufman, a Boston scientist and member of a National Institutes of Health panel that reviewed the study before it began.\u003c/p>\n\u003cp>Dr. Matthew Porteus, a genetics expert at Stanford University who consults for two other companies developing gene therapies, said more time is needed to see how the patients’ immune system continues to react to the treatment and whether the effects last, but added, “I would be excited about continuing to push along” based on these results.\u003c/p>\n\u003cp>\u003cstrong>How It Works\u003c/strong>\u003c/p>\n\u003cp>Gene editing is intended as a more precise way to do gene therapy, to knock out a bad gene or supply a good one that’s missing. Doctors hope it will give a way to address a host of diseases that can’t be treated well now.\u003c/p>\n\u003cp>In November, a Phoenix-area man with Hunter syndrome, Brian Madeux, became the first person to test this inside the body. He lacks a gene that makes an enzyme that breaks down certain large sugar compounds called GAGs. These build up in cells and cause havoc throughout the body.\u003c/p>\n\u003cp>Through an IV, Madeux received many copies of a corrective gene and a gene-editing tool called zinc finger nucleases to help put it in a precise spot in his DNA. He was one of the two patients given a very low dose of the treatment, because this first-in-human testing called for extreme caution.\u003c/p>\n\u003cp>\u003cstrong>Early Results\u003c/strong>\u003c/p>\n\u003cp>In Madeux and the other low-dose patient, levels of the tell-tale sugar compounds in urine rose 9 percent on average after four months. Muenzer said it’s hard to know whether this is a significant change; little is known about the biology of these compounds, including whether they fluctuate during the day or before or after meals.[contextly_sidebar id=\"g6IAb2xjtv0z0Te9cBhCwQAn8pi68Yxx\"]\u003c/p>\n\u003cp>A liver biopsy on one patient given a low dose of the therapy found no evidence that the gene editing had occurred, but Sangamo scientists said this dose is far below the level at which such signs had been detected in research on primates.\u003c/p>\n\u003cp>Two other patients were given a middle dose that was twice what the first two patients received. Their GAG levels declined by 51 percent after four months, on average. Two of the main types of these sugars that accumulate in tissues declined 32 percent and 61 percent, respectively.\u003c/p>\n\u003cp>It is not yet known if declines like these can improve patients’ health or slow the progression of the disease.\u003c/p>\n\u003cp>“This is not proof that this is a successful therapy yet, that these patients had enough gene editing to now supply them with the enzyme they need for the rest of their life,” Muenzer said.\u003c/p>\n\u003cp>But he said an important goal was met: the treatment seems safe. There were two serious side effects — one patient was hospitalized for bronchitis and another for an irregular heartbeat — but those were deemed due to their disease and pre-existing conditions, not the gene treatment.\u003c/p>\n\u003cp>Blood tests did not detect the missing enzyme. Company scientists said this could be because any that was being made was rapidly used by cells rather than getting into the bloodstream — an explanation some outside experts agreed with. What counts, they said, was seeing the result of enzyme activity, the drop in sugars.\u003c/p>\n\u003cp>\u003cstrong>Next Steps\u003c/strong>\u003c/p>\n\u003cp>Two more patients have been given the highest dose being tested — 10 times the starting dose — for a total of six patients in the study. The next step is to start taking patients off the weekly enzyme treatments they’ve been receiving to see if the gene therapy has changed their bodies so they make enough of the enzyme themselves.\u003c/p>\n\u003cp>More results are expected at a medical meeting in February.\u003c/p>\n\u003cp>“We need to see sustained levels for this to be practical. If this only works for six months, that’s not very beneficial,” Muenzer said. “Time’s going to tell.”\u003c/p>\n\u003cp>In an interview at his home in Arizona last month, Madeux, 45, told The Associated Press he volunteered for the study in hope of being able to stop the weekly, three-hour enzyme infusions, but also to help find a treatment for future generations with the disease.\u003c/p>\n\u003cp>“I’m old and having Hunter’s has done a lot of damage to my body,” Madeux said. “I’m actually pretty lucky I’ve lived this long.”\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp> \u003c/p>\n\n","blocks":[],"excerpt":"Partial results for gene editing study give promising signs that the treatment may be safe and have at least some of its hoped - for effect.","status":"publish","parent":0,"modified":1536177185,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":30,"wordCount":1104},"headData":{"title":"Historic Gene Editing Attempt in First Human Off to a Positive Start | KQED","description":"Partial results for gene editing study give promising signs that the treatment may be safe and have at least some of its hoped - for effect.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Historic Gene Editing Attempt in First Human Off to a Positive Start","datePublished":"2018-09-05T20:20:59.000Z","dateModified":"2018-09-05T19:53:05.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"444281 https://ww2.kqed.org/futureofyou/?p=444281","disqusUrl":"https://ww2.kqed.org/futureofyou/2018/09/05/historic-gene-editing-attempt-in-first-human-off-to-a-positive-start/","disqusTitle":"Historic Gene Editing Attempt in First Human Off to a Positive Start","source":"Your Genes","nprByline":"Marilynn Marchione\u003cbr />The Associated Press","path":"/futureofyou/444281/historic-gene-editing-attempt-in-first-human-off-to-a-positive-start","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Early, partial results from a historic gene editing study give encouraging signs that the treatment may be safe and having at least some of its hoped-for effect, but it’s too soon to know whether it ultimately will succeed.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>The results announced Wednesday are from the first human \u003ca href=\"//mps2study.com/study-overview/?pn=\">test\u003c/a> of gene editing in the body, an attempt to permanently change someone’s DNA to cure a disease — in this case, a genetic disorder called \u003ca href=\"https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Mucopolysaccharidoses-Fact-Sheet\">Hunter syndrome\u003c/a> that often kills people in their teens.\u003c/p>\n\u003cp>In two patients who got a medium dose of the treatment, urine levels of large sugar compounds that are hallmarks of Hunter syndrome had fallen by half, on average, four months later — a possible sign the treatment is working. Two others who got a low dose have seen little change in these sugars so far.\u003c/p>\n\u003cp>There’s no way to know yet whether the change in the middle-dose patients is due to the gene editing or something else, but the fact their sugars have declined consistently since treatment suggests it might be.\u003c/p>\n\u003cp>“I cannot absolutely say it’s a treatment effect” but the drop is “really encouraging,” said the study leader, Dr. Joseph Muenzer of the University of North Carolina, Chapel Hill. The main goal of early treatment studies is to test safety, though researchers also look for hints that the therapy is working. Muenzer gave the results at a conference in Greece and consults for the treatment’s maker, California-based Sangamo Therapeutics.\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>The company’s president, Dr. Sandy Macrae, said tests in about five months will reveal more, but the change in the middle-dose group so far “looks really good.”\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>“The most rational explanation for this is that what we hoped was going to happen has happened,” he said.\u003c/p>\n\u003cp>Several independent experts agreed.\u003c/p>\n\u003cp>“The results are exciting” and suggest that the gene editing is working to some degree, without safety concerns so far, said Dr. Howard Kaufman, a Boston scientist and member of a National Institutes of Health panel that reviewed the study before it began.\u003c/p>\n\u003cp>Dr. Matthew Porteus, a genetics expert at Stanford University who consults for two other companies developing gene therapies, said more time is needed to see how the patients’ immune system continues to react to the treatment and whether the effects last, but added, “I would be excited about continuing to push along” based on these results.\u003c/p>\n\u003cp>\u003cstrong>How It Works\u003c/strong>\u003c/p>\n\u003cp>Gene editing is intended as a more precise way to do gene therapy, to knock out a bad gene or supply a good one that’s missing. Doctors hope it will give a way to address a host of diseases that can’t be treated well now.\u003c/p>\n\u003cp>In November, a Phoenix-area man with Hunter syndrome, Brian Madeux, became the first person to test this inside the body. He lacks a gene that makes an enzyme that breaks down certain large sugar compounds called GAGs. These build up in cells and cause havoc throughout the body.\u003c/p>\n\u003cp>Through an IV, Madeux received many copies of a corrective gene and a gene-editing tool called zinc finger nucleases to help put it in a precise spot in his DNA. He was one of the two patients given a very low dose of the treatment, because this first-in-human testing called for extreme caution.\u003c/p>\n\u003cp>\u003cstrong>Early Results\u003c/strong>\u003c/p>\n\u003cp>In Madeux and the other low-dose patient, levels of the tell-tale sugar compounds in urine rose 9 percent on average after four months. Muenzer said it’s hard to know whether this is a significant change; little is known about the biology of these compounds, including whether they fluctuate during the day or before or after meals.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>A liver biopsy on one patient given a low dose of the therapy found no evidence that the gene editing had occurred, but Sangamo scientists said this dose is far below the level at which such signs had been detected in research on primates.\u003c/p>\n\u003cp>Two other patients were given a middle dose that was twice what the first two patients received. Their GAG levels declined by 51 percent after four months, on average. Two of the main types of these sugars that accumulate in tissues declined 32 percent and 61 percent, respectively.\u003c/p>\n\u003cp>It is not yet known if declines like these can improve patients’ health or slow the progression of the disease.\u003c/p>\n\u003cp>“This is not proof that this is a successful therapy yet, that these patients had enough gene editing to now supply them with the enzyme they need for the rest of their life,” Muenzer said.\u003c/p>\n\u003cp>But he said an important goal was met: the treatment seems safe. There were two serious side effects — one patient was hospitalized for bronchitis and another for an irregular heartbeat — but those were deemed due to their disease and pre-existing conditions, not the gene treatment.\u003c/p>\n\u003cp>Blood tests did not detect the missing enzyme. Company scientists said this could be because any that was being made was rapidly used by cells rather than getting into the bloodstream — an explanation some outside experts agreed with. What counts, they said, was seeing the result of enzyme activity, the drop in sugars.\u003c/p>\n\u003cp>\u003cstrong>Next Steps\u003c/strong>\u003c/p>\n\u003cp>Two more patients have been given the highest dose being tested — 10 times the starting dose — for a total of six patients in the study. The next step is to start taking patients off the weekly enzyme treatments they’ve been receiving to see if the gene therapy has changed their bodies so they make enough of the enzyme themselves.\u003c/p>\n\u003cp>More results are expected at a medical meeting in February.\u003c/p>\n\u003cp>“We need to see sustained levels for this to be practical. If this only works for six months, that’s not very beneficial,” Muenzer said. “Time’s going to tell.”\u003c/p>\n\u003cp>In an interview at his home in Arizona last month, Madeux, 45, told The Associated Press he volunteered for the study in hope of being able to stop the weekly, three-hour enzyme infusions, but also to help find a treatment for future generations with the disease.\u003c/p>\n\u003cp>“I’m old and having Hunter’s has done a lot of damage to my body,” Madeux said. “I’m actually pretty lucky I’ve lived this long.”\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> \u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/444281/historic-gene-editing-attempt-in-first-human-off-to-a-positive-start","authors":["byline_futureofyou_444281"],"categories":["futureofyou_1062","futureofyou_1","futureofyou_73","futureofyou_1064"],"tags":["futureofyou_647","futureofyou_103","futureofyou_295"],"collections":["futureofyou_1097","futureofyou_1094"],"featImg":"futureofyou_444292","label":"source_futureofyou_444281"},"futureofyou_442526":{"type":"posts","id":"futureofyou_442526","meta":{"index":"posts_1591205157","site":"futureofyou","id":"442526","score":null,"sort":[1528743626000]},"guestAuthors":[],"slug":"major-hurdle-for-crispr-edited-cells-might-cause-cancer-find-two-studies","title":"Major CRISPR Hurdle: Edited Cells Might Cause Cancer, Find Studies","publishDate":1528743626,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp>Editing cells’ genomes with CRISPR-Cas9 might increase the risk that the altered cells, intended to treat disease, will trigger cancer, two studies published on Monday warn — a potential game-changer for the companies developing CRISPR-based therapies.[contextly_sidebar id=\"Pbm72hAOjQOHNUgNeQaKv0l2d5rX0APO\"]\u003c/p>\n\u003cp>In the studies, published in Nature Medicine, scientists found that cells whose genomes are successfully edited by CRISPR-Cas9 have the potential to seed tumors inside a patient. That could make some CRISPR’d cells ticking time bombs, according to researchers from Sweden’s Karolinska Institute and, separately, Novartis.\u003c/p>\n\u003cp class=\"danger-zone\">\u003ca href=\"https://www.statnews.com/feature/crispr/tracker/\">CRISPR\u003c/a> has already dodged two potentially fatal bullets — a 2017 \u003ca href=\"https://www.nature.com/articles/nmeth.4293.epdf\" target=\"_blank\" rel=\"noopener\">claim\u003c/a> that it causes sky-high numbers of off-target effects was \u003ca href=\"https://www.nature.com/articles/nmeth.4664\" target=\"_blank\" rel=\"noopener\">retracted\u003c/a> in March, and a \u003ca href=\"https://www.biorxiv.org/content/early/2018/01/05/243345\" target=\"_blank\" rel=\"noopener\">report \u003c/a>of human immunity to Cas9 was largely shrugged off as \u003ca href=\"https://www.statnews.com/2018/01/08/immunity-crispr-cas9/\">solvable\u003c/a>. But experts are taking the cancer-risk finding seriously.\u003c/p>\n\u003cp class=\"danger-zone\">The CEO of CRISPR Therapeutics, Sam Kulkarni, told STAT the results are “plausible.” Although they likely apply to one of the main ways that CRISPR edits genomes (replacing disease-causing DNA with healthy versions) more than another (just excising DNA), he said, “it’s something we need to pay attention to, especially as CRISPR expands to more diseases. We need to do the work and make sure edited cells returned to patients don’t become cancerous.”\u003c/p>\n\u003cp class=\"\">Another leading CRISPR scientist, who asked not to be named because of involvement with genome-editing companies, called the new data “pretty striking,” and raised concerns that a potential fatal flaw in some uses of CRISPR had “been missed.”\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>On the other hand, the Novartis paper has been \u003ca href=\"https://www.biorxiv.org/content/early/2017/07/26/168443\" target=\"_blank\" rel=\"noopener\">available\u003c/a> in preliminary form since last summer, and CRISPR experts “haven’t freaked out,” said Erik Sontheimer of the University of Massachusetts Medical School, whose CRISPR research \u003ca href=\"https://www.statnews.com/2018/03/05/crispr-off-target-editing/\">centers\u003c/a> on novel enzymes and off-target effects. “This is something that bears paying attention to, but I don’t think it’s a deal-breaker” for CRISPR therapies.\u003c/p>\n\u003cp>The Karolinska and Novartis groups tested CRISPR on different kinds of human cells — retinal cells and pluripotent stem cells, respectively. But they found essentially the same phenomenon. Standard CRISPR-Cas9 works by cutting both strands of the DNA double helix. That injury causes a cell to activate a biochemical first-aid kit orchestrated by a gene called \u003ca href=\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4235614/\" target=\"_blank\" rel=\"noopener\">p53,\u003c/a> which either mends the DNA break or makes the cell self-destruct.[contextly_sidebar id=\"X1qu0ifjesXey9YHHpOinoS4KNdNuyur\"]\u003c/p>\n\u003cp>Whichever action p53 takes, the consequence is the same: CRISPR doesn’t work, either because the genome edit is stitched up or the cell is dead. (The Novartis team calculated that p53 reduces CRISPR efficiency in pluripotent stem cells seventeenfold.) That might explain something found over and over: CRISPR is woefully inefficient, with only a small minority of cells into which CRISPR is introduced, usually by a virus, actually having their genomes edited as intended.\u003c/p>\n\u003cp>“We found that cutting the genome with CRISPR-Cas9 induced the activation of … p53,” said Emma Haapaniemi, the lead author of the \u003ca href=\"https://www.nature.com/articles/s41591-018-0049-z\" target=\"_blank\" rel=\"noopener\">Karolinska study\u003c/a>. That “makes editing much more difficult.”\u003c/p>\n\u003cp>The flip side of p53 repairing CRISPR edits, or killing cells that accept the edits, is that cells that survive with the edits do so precisely because they have a dysfunctional p53 and therefore lack this fix-it-or-kill-it mechanism.\u003c/p>\n\u003cp>The reason why that could be a problem is that p53 dysfunction can cause cancer. And not just occasionally. P53 mutations are \u003ca href=\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2827900/figure/A001008F1/\" target=\"_blank\" rel=\"noopener\">responsible for\u003c/a> nearly half of ovarian cancers; 43 percent of colorectal cancers; 38 percent of lung cancers; nearly one-third of pancreatic, stomach, and liver cancers; and one-quarter of breast cancers, among others.\u003c/p>\n\u003cp>The Novartis team was trying to see how it could increase the efficiency of CRISPR editing of pluripotent stem cells. Because this kind of stem cell can morph into virtually any kind of cell, it might be able to treat a variety of diseases. Neuroscientist Ajamete Kaykas of the company’s Institutes for BioMedical Research in Cambridge, Mass., got CRISPR’s efficiency at inserting or deleting chunks of DNA up to 80 percent. Unfortunately, when CRISPR worked, it was because p53 didn’t, which raises cancer concerns.\u003c/p>\n\u003cp>As a result, the Novartis \u003ca href=\"https://www.nature.com/articles/s41591-018-0050-6\" target=\"_blank\" rel=\"noopener\">paper\u003c/a> concludes that “it will be critical to ensure that [genome-edited cells] have a functional p53 before and after [genome] engineering.” The Karolinska team warns that p53 and related genes “should be monitored when developing cell-based therapies utilizing CRISPR-Cas9.”[contextly_sidebar id=\"f80ZxqMXgIrsBguckwJZNJRkL6wI6fMd\"]\u003c/p>\n\u003cp>The p53 finding doesn’t mean CRISPR is toast. For one thing, “the two papers present preliminary results,” biochemist Bernhard Schmierer of the Karolinska, co-leader of its study, told STAT. “It is unclear if the findings translate into cells actually used in current clinical studies.”\u003c/p>\n\u003cp>For another, the p53 problem might be worse with Cas9 than with other DNA-cutting enzymes used in CRISPR. And, crucially, it probably affects only one avenue of genome-editing.\u003c/p>\n\u003cp>CRISPR edits genomes in either of two ways. It slices out a chunk of disease-causing DNA, in a process called non-homologous end joining (NHEJ), or gene disruption. That’s how CRISPR Therapeutics is going after sickle cell disease. Alternatively, CRISPR both cuts out a disease-causing stretch of DNA and replaces it with healthy nucleotides, in homology-directed repair (HDR), or gene correction. Several university labs are investigating HDR to treat Duchenne \u003ca href=\"https://www.nature.com/articles/s41551-017-0137-2\" target=\"_blank\" rel=\"noopener\">muscular dystrophy,\u003c/a> among many other diseases.\u003c/p>\n\u003cp>In the normal, mature cells she and her team studied, Haapaniemi said, gene disruption “can happen even when p53 is activated.”\u003c/p>\n\u003cp>That’s good news for CRISPR Therapeutics’ sickle-cell and thalassemia \u003ca href=\"http://www.crisprtx.com/our-programs/our-pipeline.php\" target=\"_blank\" rel=\"noopener\">programs \u003c/a>as well as for Editas Medicine’s lead product, targeting a form of blindness, and others in its \u003ca href=\"http://www.editasmedicine.com/pipeline\" target=\"_blank\" rel=\"noopener\">pipeline\u003c/a>, all of which use NHEJ gene disruption. It also should not affect the gene-disruption approach that Intellia Therapeutics and Regeneron are \u003ca href=\"https://www.intelliatx.com/pipeline/\" target=\"_blank\" rel=\"noopener\">taking\u003c/a> to \u003ca href=\"https://ghr.nlm.nih.gov/condition/transthyretin-amyloidosis\" target=\"_blank\" rel=\"noopener\">transthyretin amyloidosis\u003c/a>.\u003c/p>\n\u003cp>CRISPR-based editing of T cells to treat cancer, as scientists at the University of Pennsylvania are studying in a \u003ca href=\"https://www.statnews.com/2016/06/21/crispr-human-trials/\">clinical trial,\u003c/a> should also not have a p53 problem. Nor should any therapy developed with CRISPR base editing, which does not make the double-stranded breaks that trigger p53. Developed by Harvard’s David Liu, base editing replaces a wrong DNA “letter” with the right one, without cutting, and is the basis for startup \u003ca href=\"https://www.statnews.com/2018/05/14/crispr-editas-beam-base-editing/\" target=\"_blank\" rel=\"noopener\">Beam Therapeutics\u003c/a>.\u003c/p>\n\u003cp>The p53 problem, however, might affect other products that companies hope to develop via gene correction, including glycogen storage disease, cystic fibrosis, and severe combined immunodeficiency.\u003c/p>\n\u003cp>It’s also a potential problem for stem cells. There, the Novartis team showed, p53 inactivation seems to be necessary for both NHEJ disruption and HDR correction. (Novartis’ Kaykas said he could not speak to a reporter without clearance from the company’s communications office.) That could be an issue for therapies using CRISPR’d stem cells: The same dysfunctional p53 that allows CRISPR to work its magic also makes cells likely to become cancerous.\u003c/p>\n\u003cp>Which raises an obvious question — if successfully CRISPR’d cells can seed cancers, why hasn’t this been seen before, and why haven’t the many CRISPR’d mice developed tumors?\u003c/p>\n\u003cp>Karolinska’s Haapaniemi said the effect shows up in large-scale experiments like hers and Novartis’ “but can be missed in small-scale studies where people only focus on editing one gene in one cell type.” In speaking to other scientists, she said, “it seems that other teams have noticed the effect of p53 on editing,” but have not highlighted it.\u003c/p>\n\u003cp>Jacob Corn of the University of California, Berkeley, said that although his lab has seen evidence of p53 activation in a few cases, they have “looked hard for growth effects after editing in [human stem cells] and found nothing.”\u003c/p>\n\u003cp>As for why no one has reported CRISPR’d mice getting cancer, Haapaniemi said, “This is a good question.” One reason might be that “laboratory mice are killed early,” perhaps leaving too little time for them to develop cancer.\u003c/p>\n\u003cp>But Corn said he and others “have all been looking for the possibility of cancer. So far, no one has seen evidence of [it] based on p53 status or induced by editing.”\u003c/p>\n\u003cp>Nevertheless, he called the two papers “important, since they remind everyone that genome editing isn’t magic.”\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>\u003cem>This\u003ca href=\"https://www.statnews.com/2018/06/11/crispr-hurdle-edited-cells-might-cause-cancer/\" target=\"_blank\" rel=\"noopener\"> story\u003c/a> was originally published by STAT, an online publication of Boston Globe Media that covers health, medicine, and scientific discovery.\u003c/em>\u003c/p>\n\n","blocks":[],"excerpt":null,"status":"publish","parent":0,"modified":1528739244,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":27,"wordCount":1502},"headData":{"title":"Major CRISPR Hurdle: Edited Cells Might Cause Cancer, Find Studies | KQED","description":"Editing cells’ genomes with CRISPR-Cas9 might increase the risk that the altered cells, intended to treat disease, will trigger cancer, two studies published on Monday warn — a potential game-changer for the companies developing CRISPR-based therapies. In the studies, published in Nature Medicine, scientists found that cells whose genomes are successfully edited by CRISPR-Cas9 have the","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Major CRISPR Hurdle: Edited Cells Might Cause Cancer, Find Studies","datePublished":"2018-06-11T19:00:26.000Z","dateModified":"2018-06-11T17:47:24.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"442526 https://ww2.kqed.org/futureofyou/?p=442526","disqusUrl":"https://ww2.kqed.org/futureofyou/2018/06/11/major-hurdle-for-crispr-edited-cells-might-cause-cancer-find-two-studies/","disqusTitle":"Major CRISPR Hurdle: Edited Cells Might Cause Cancer, Find Studies","source":"Health","nprByline":"Sharon Begley\u003cbr />STAT","path":"/futureofyou/442526/major-hurdle-for-crispr-edited-cells-might-cause-cancer-find-two-studies","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Editing cells’ genomes with CRISPR-Cas9 might increase the risk that the altered cells, intended to treat disease, will trigger cancer, two studies published on Monday warn — a potential game-changer for the companies developing CRISPR-based therapies.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>In the studies, published in Nature Medicine, scientists found that cells whose genomes are successfully edited by CRISPR-Cas9 have the potential to seed tumors inside a patient. That could make some CRISPR’d cells ticking time bombs, according to researchers from Sweden’s Karolinska Institute and, separately, Novartis.\u003c/p>\n\u003cp class=\"danger-zone\">\u003ca href=\"https://www.statnews.com/feature/crispr/tracker/\">CRISPR\u003c/a> has already dodged two potentially fatal bullets — a 2017 \u003ca href=\"https://www.nature.com/articles/nmeth.4293.epdf\" target=\"_blank\" rel=\"noopener\">claim\u003c/a> that it causes sky-high numbers of off-target effects was \u003ca href=\"https://www.nature.com/articles/nmeth.4664\" target=\"_blank\" rel=\"noopener\">retracted\u003c/a> in March, and a \u003ca href=\"https://www.biorxiv.org/content/early/2018/01/05/243345\" target=\"_blank\" rel=\"noopener\">report \u003c/a>of human immunity to Cas9 was largely shrugged off as \u003ca href=\"https://www.statnews.com/2018/01/08/immunity-crispr-cas9/\">solvable\u003c/a>. But experts are taking the cancer-risk finding seriously.\u003c/p>\n\u003cp class=\"danger-zone\">The CEO of CRISPR Therapeutics, Sam Kulkarni, told STAT the results are “plausible.” Although they likely apply to one of the main ways that CRISPR edits genomes (replacing disease-causing DNA with healthy versions) more than another (just excising DNA), he said, “it’s something we need to pay attention to, especially as CRISPR expands to more diseases. We need to do the work and make sure edited cells returned to patients don’t become cancerous.”\u003c/p>\n\u003cp class=\"\">Another leading CRISPR scientist, who asked not to be named because of involvement with genome-editing companies, called the new data “pretty striking,” and raised concerns that a potential fatal flaw in some uses of CRISPR had “been missed.”\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>On the other hand, the Novartis paper has been \u003ca href=\"https://www.biorxiv.org/content/early/2017/07/26/168443\" target=\"_blank\" rel=\"noopener\">available\u003c/a> in preliminary form since last summer, and CRISPR experts “haven’t freaked out,” said Erik Sontheimer of the University of Massachusetts Medical School, whose CRISPR research \u003ca href=\"https://www.statnews.com/2018/03/05/crispr-off-target-editing/\">centers\u003c/a> on novel enzymes and off-target effects. “This is something that bears paying attention to, but I don’t think it’s a deal-breaker” for CRISPR therapies.\u003c/p>\n\u003cp>The Karolinska and Novartis groups tested CRISPR on different kinds of human cells — retinal cells and pluripotent stem cells, respectively. But they found essentially the same phenomenon. Standard CRISPR-Cas9 works by cutting both strands of the DNA double helix. That injury causes a cell to activate a biochemical first-aid kit orchestrated by a gene called \u003ca href=\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4235614/\" target=\"_blank\" rel=\"noopener\">p53,\u003c/a> which either mends the DNA break or makes the cell self-destruct.\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>Whichever action p53 takes, the consequence is the same: CRISPR doesn’t work, either because the genome edit is stitched up or the cell is dead. (The Novartis team calculated that p53 reduces CRISPR efficiency in pluripotent stem cells seventeenfold.) That might explain something found over and over: CRISPR is woefully inefficient, with only a small minority of cells into which CRISPR is introduced, usually by a virus, actually having their genomes edited as intended.\u003c/p>\n\u003cp>“We found that cutting the genome with CRISPR-Cas9 induced the activation of … p53,” said Emma Haapaniemi, the lead author of the \u003ca href=\"https://www.nature.com/articles/s41591-018-0049-z\" target=\"_blank\" rel=\"noopener\">Karolinska study\u003c/a>. That “makes editing much more difficult.”\u003c/p>\n\u003cp>The flip side of p53 repairing CRISPR edits, or killing cells that accept the edits, is that cells that survive with the edits do so precisely because they have a dysfunctional p53 and therefore lack this fix-it-or-kill-it mechanism.\u003c/p>\n\u003cp>The reason why that could be a problem is that p53 dysfunction can cause cancer. And not just occasionally. P53 mutations are \u003ca href=\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2827900/figure/A001008F1/\" target=\"_blank\" rel=\"noopener\">responsible for\u003c/a> nearly half of ovarian cancers; 43 percent of colorectal cancers; 38 percent of lung cancers; nearly one-third of pancreatic, stomach, and liver cancers; and one-quarter of breast cancers, among others.\u003c/p>\n\u003cp>The Novartis team was trying to see how it could increase the efficiency of CRISPR editing of pluripotent stem cells. Because this kind of stem cell can morph into virtually any kind of cell, it might be able to treat a variety of diseases. Neuroscientist Ajamete Kaykas of the company’s Institutes for BioMedical Research in Cambridge, Mass., got CRISPR’s efficiency at inserting or deleting chunks of DNA up to 80 percent. Unfortunately, when CRISPR worked, it was because p53 didn’t, which raises cancer concerns.\u003c/p>\n\u003cp>As a result, the Novartis \u003ca href=\"https://www.nature.com/articles/s41591-018-0050-6\" target=\"_blank\" rel=\"noopener\">paper\u003c/a> concludes that “it will be critical to ensure that [genome-edited cells] have a functional p53 before and after [genome] engineering.” The Karolinska team warns that p53 and related genes “should be monitored when developing cell-based therapies utilizing CRISPR-Cas9.”\u003c/p>\u003cp>\u003c/p>\u003cp>\u003c/p>\n\u003cp>The p53 finding doesn’t mean CRISPR is toast. For one thing, “the two papers present preliminary results,” biochemist Bernhard Schmierer of the Karolinska, co-leader of its study, told STAT. “It is unclear if the findings translate into cells actually used in current clinical studies.”\u003c/p>\n\u003cp>For another, the p53 problem might be worse with Cas9 than with other DNA-cutting enzymes used in CRISPR. And, crucially, it probably affects only one avenue of genome-editing.\u003c/p>\n\u003cp>CRISPR edits genomes in either of two ways. It slices out a chunk of disease-causing DNA, in a process called non-homologous end joining (NHEJ), or gene disruption. That’s how CRISPR Therapeutics is going after sickle cell disease. Alternatively, CRISPR both cuts out a disease-causing stretch of DNA and replaces it with healthy nucleotides, in homology-directed repair (HDR), or gene correction. Several university labs are investigating HDR to treat Duchenne \u003ca href=\"https://www.nature.com/articles/s41551-017-0137-2\" target=\"_blank\" rel=\"noopener\">muscular dystrophy,\u003c/a> among many other diseases.\u003c/p>\n\u003cp>In the normal, mature cells she and her team studied, Haapaniemi said, gene disruption “can happen even when p53 is activated.”\u003c/p>\n\u003cp>That’s good news for CRISPR Therapeutics’ sickle-cell and thalassemia \u003ca href=\"http://www.crisprtx.com/our-programs/our-pipeline.php\" target=\"_blank\" rel=\"noopener\">programs \u003c/a>as well as for Editas Medicine’s lead product, targeting a form of blindness, and others in its \u003ca href=\"http://www.editasmedicine.com/pipeline\" target=\"_blank\" rel=\"noopener\">pipeline\u003c/a>, all of which use NHEJ gene disruption. It also should not affect the gene-disruption approach that Intellia Therapeutics and Regeneron are \u003ca href=\"https://www.intelliatx.com/pipeline/\" target=\"_blank\" rel=\"noopener\">taking\u003c/a> to \u003ca href=\"https://ghr.nlm.nih.gov/condition/transthyretin-amyloidosis\" target=\"_blank\" rel=\"noopener\">transthyretin amyloidosis\u003c/a>.\u003c/p>\n\u003cp>CRISPR-based editing of T cells to treat cancer, as scientists at the University of Pennsylvania are studying in a \u003ca href=\"https://www.statnews.com/2016/06/21/crispr-human-trials/\">clinical trial,\u003c/a> should also not have a p53 problem. Nor should any therapy developed with CRISPR base editing, which does not make the double-stranded breaks that trigger p53. Developed by Harvard’s David Liu, base editing replaces a wrong DNA “letter” with the right one, without cutting, and is the basis for startup \u003ca href=\"https://www.statnews.com/2018/05/14/crispr-editas-beam-base-editing/\" target=\"_blank\" rel=\"noopener\">Beam Therapeutics\u003c/a>.\u003c/p>\n\u003cp>The p53 problem, however, might affect other products that companies hope to develop via gene correction, including glycogen storage disease, cystic fibrosis, and severe combined immunodeficiency.\u003c/p>\n\u003cp>It’s also a potential problem for stem cells. There, the Novartis team showed, p53 inactivation seems to be necessary for both NHEJ disruption and HDR correction. (Novartis’ Kaykas said he could not speak to a reporter without clearance from the company’s communications office.) That could be an issue for therapies using CRISPR’d stem cells: The same dysfunctional p53 that allows CRISPR to work its magic also makes cells likely to become cancerous.\u003c/p>\n\u003cp>Which raises an obvious question — if successfully CRISPR’d cells can seed cancers, why hasn’t this been seen before, and why haven’t the many CRISPR’d mice developed tumors?\u003c/p>\n\u003cp>Karolinska’s Haapaniemi said the effect shows up in large-scale experiments like hers and Novartis’ “but can be missed in small-scale studies where people only focus on editing one gene in one cell type.” In speaking to other scientists, she said, “it seems that other teams have noticed the effect of p53 on editing,” but have not highlighted it.\u003c/p>\n\u003cp>Jacob Corn of the University of California, Berkeley, said that although his lab has seen evidence of p53 activation in a few cases, they have “looked hard for growth effects after editing in [human stem cells] and found nothing.”\u003c/p>\n\u003cp>As for why no one has reported CRISPR’d mice getting cancer, Haapaniemi said, “This is a good question.” One reason might be that “laboratory mice are killed early,” perhaps leaving too little time for them to develop cancer.\u003c/p>\n\u003cp>But Corn said he and others “have all been looking for the possibility of cancer. So far, no one has seen evidence of [it] based on p53 status or induced by editing.”\u003c/p>\n\u003cp>Nevertheless, he called the two papers “important, since they remind everyone that genome editing isn’t magic.”\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>\u003cem>This\u003ca href=\"https://www.statnews.com/2018/06/11/crispr-hurdle-edited-cells-might-cause-cancer/\" target=\"_blank\" rel=\"noopener\"> story\u003c/a> was originally published by STAT, 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":"/futureofyou/442526/major-hurdle-for-crispr-edited-cells-might-cause-cancer-find-two-studies","authors":["byline_futureofyou_442526"],"categories":["futureofyou_1","futureofyou_1064"],"tags":["futureofyou_1521","futureofyou_103","futureofyou_1077","futureofyou_94","futureofyou_17","futureofyou_295"],"collections":["futureofyou_1093","futureofyou_1094"],"featImg":"futureofyou_442536","label":"source_futureofyou_442526"},"futureofyou_437942":{"type":"posts","id":"futureofyou_437942","meta":{"index":"posts_1591205157","site":"futureofyou","id":"437942","score":null,"sort":[1513974354000]},"guestAuthors":[],"slug":"trying-to-slow-lou-gehrigs-disease-with-crispr-cas9","title":"Trying to Slow Lou Gehrig's Disease With CRISPR-Cas9","publishDate":1513974354,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{"site":"futureofyou"},"content":"\u003cp>Researchers have taken the first step toward a possible cure for \u003cspan class=\"example\">Lou Gehrig's disease \u003c/span>based on gene editing. The study, though limited in scope, provides a possible way forward to address some genetically driven cases of the disease.\u003c/p>\n\u003cp>[contextly_sidebar id=\"Zj5jMJb0LrYIQhKiETUb8gCvIGVfUWhp\"]An estimated 20,000 Americans live with the disease, which is also called amyotrophic lateral sclerosis, or ALS. The condition causes muscles to waste away and is fatal. Eventually sufferers lose use of muscles essential for breathing. Two drugs have been approved by U.S. health officials, but they are minimally effective, increasing survival by \u003ca href=\"https://link.springer.com/article/10.1007/s00415-003-1026-z\" target=\"_blank\" rel=\"noopener\">only a few months\u003c/a>.\u003c/p>\n\u003cp>The researchers, at UC Berkeley, the University of Illinois and the University of Pittsburgh, are working on a new approach: altering a mutated gene that contributes to some cases of the disease. In mice, the technique increased average life span by about 25 percent and delayed onset of the disease by nearly 40 percent. The \u003ca href=\"http://advances.sciencemag.org/content/3/12/eaar3952\" target=\"_blank\" rel=\"noopener\">study\u003c/a> is published the Dec. 20 issue of\u003cem> Science Advances\u003c/em>.\u003c/p>\n\u003cp>\"If this were to translate to humans it would be clinically very meaningful for patients,\" says co-author David Schaffer, of UC Berkeley.\u003c/p>\n\u003cp>[contextly_sidebar id=\"gi0jm5XjbAQNm096mmQndorYwApHqftD\"]The mice used in the study were genetically engineered to express a mutated human gene, known as SOD1, that causes 1 in 5 inherited cases of ALS. To disable the mutation, the researchers engineered a virus carrying a pair of molecular scissors in the form of a Cas9 protein, designed to knock out the SOD1 gene. The scientists targeted the neurons in the spinal cord, which the SOD1 mutation cause to prematurely die.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>Schaffer and others are now at work optimizing the technique for humans.\u003c/p>\n\n","blocks":[],"excerpt":"Researchers at UC Berkeley have taken the first step toward a possible cure for Lou Gehrig's disease based on gene editing. ","status":"publish","parent":0,"modified":1513976057,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":8,"wordCount":282},"headData":{"title":"Trying to Slow Lou Gehrig's Disease With CRISPR-Cas9 | KQED","description":"Researchers at UC Berkeley have taken the first step toward a possible cure for Lou Gehrig's disease based on gene editing. ","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Trying to Slow Lou Gehrig's Disease With CRISPR-Cas9","datePublished":"2017-12-22T20:25:54.000Z","dateModified":"2017-12-22T20:54:17.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"437942 https://ww2.kqed.org/futureofyou/?p=437942","disqusUrl":"https://ww2.kqed.org/futureofyou/2017/12/22/trying-to-slow-lou-gehrigs-disease-with-crispr-cas9/","disqusTitle":"Trying to Slow Lou Gehrig's Disease With CRISPR-Cas9","path":"/futureofyou/437942/trying-to-slow-lou-gehrigs-disease-with-crispr-cas9","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Researchers have taken the first step toward a possible cure for \u003cspan class=\"example\">Lou Gehrig's disease \u003c/span>based on gene editing. The study, though limited in scope, provides a possible way forward to address some genetically driven cases of the disease.\u003c/p>\n\u003cp>\u003c/p>\u003cp>\u003c/p>\u003cp>An estimated 20,000 Americans live with the disease, which is also called amyotrophic lateral sclerosis, or ALS. The condition causes muscles to waste away and is fatal. Eventually sufferers lose use of muscles essential for breathing. Two drugs have been approved by U.S. health officials, but they are minimally effective, increasing survival by \u003ca href=\"https://link.springer.com/article/10.1007/s00415-003-1026-z\" target=\"_blank\" rel=\"noopener\">only a few months\u003c/a>.\u003c/p>\n\u003cp>The researchers, at UC Berkeley, the University of Illinois and the University of Pittsburgh, are working on a new approach: altering a mutated gene that contributes to some cases of the disease. In mice, the technique increased average life span by about 25 percent and delayed onset of the disease by nearly 40 percent. The \u003ca href=\"http://advances.sciencemag.org/content/3/12/eaar3952\" target=\"_blank\" rel=\"noopener\">study\u003c/a> is published the Dec. 20 issue of\u003cem> Science Advances\u003c/em>.\u003c/p>\n\u003cp>\"If this were to translate to humans it would be clinically very meaningful for patients,\" says co-author David Schaffer, of UC Berkeley.\u003c/p>\n\u003cp>\u003c/p>\u003cp>\u003c/p>\u003cp>The mice used in the study were genetically engineered to express a mutated human gene, known as SOD1, that causes 1 in 5 inherited cases of ALS. To disable the mutation, the researchers engineered a virus carrying a pair of molecular scissors in the form of a Cas9 protein, designed to knock out the SOD1 gene. The scientists targeted the neurons in the spinal cord, which the SOD1 mutation cause to prematurely die.\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\u003cp>Schaffer and others are now at work optimizing the technique for humans.\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/437942/trying-to-slow-lou-gehrigs-disease-with-crispr-cas9","authors":["11088"],"categories":["futureofyou_1","futureofyou_1064"],"tags":["futureofyou_983","futureofyou_94","futureofyou_295"],"featImg":"futureofyou_437949","label":"futureofyou"},"futureofyou_437054":{"type":"posts","id":"futureofyou_437054","meta":{"index":"posts_1591205157","site":"futureofyou","id":"437054","score":null,"sort":[1510765041000]},"guestAuthors":[],"slug":"first-attempt-to-edit-gene-inside-body-in-hopes-of-curing-mans-disease","title":"In First, Scientists Try to Cure Man's Disease by Editing DNA Inside Body","publishDate":1510765041,"format":"aside","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp>https://www.youtube.com/watch?v=W-gUUB0ofVA&ab_channel=AssociatedPress\u003c/p>\n\u003cp>Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person's DNA to cure a disease.\u003c/p>\n\u003cp>The experiment was done Monday in California on 44-year-old Brian Madeux. Through an IV, he received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot.\u003c/p>\n\u003cp>\"It's kind of humbling\" to be the first to test this, said Madeux, who has a metabolic disease called Hunter syndrome. \"I'm willing to take that risk. Hopefully it will help me and other people.\"\u003c/p>\n\u003cp>Signs of whether it's working may come in a month; tests will show for sure in three months.\u003c/p>\n\u003caside class=\"pullquote alignright\">'You’re really toying with Mother Nature.'\u003ccite>Dr. Eric Topol, Scripps Translational Science Institute\u003c/cite>\u003c/aside>\n\u003cp>If it's successful, it could give a major boost to the fledgling field of gene therapy . Scientists have edited people's genes before, altering cells in the lab that are then returned to patients. There also are gene therapies that don't involve editing DNA.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>But these methods can only be used for a few types of diseases. Some give results that may not last. Some others supply a new gene like a spare part, but can't control where it inserts in the DNA, possibly causing a new problem like cancer.\u003c/p>\n\u003cp>This time, the gene tinkering is happening in a precise way inside the body. It's like sending a mini surgeon along to place the new gene in exactly the right location.\u003c/p>\n\u003cp>\"We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending,\" said Dr. Sandy Macrae, president of Sangamo Therapeutics, the California company testing this for two metabolic diseases and hemophilia. \"It becomes part of your DNA and is there for the rest of your life.\"\u003c/p>\n\u003cp>That also means there's no going back, no way to erase any mistakes the editing might cause.\u003c/p>\n\u003caside class=\"aligncenter\">\u003cem>Also from KQED Future of You:\u003c/em>\n\u003cul>\n\u003cli>\u003ca href=\"https://ww2.kqed.org/futureofyou/2017/10/31/the-crippling-ransomware-attack-on-kqed-the-inside-story/\" target=\"_blank\" rel=\"noopener\">The Crippling Ransomware Attack on an NPR Member Station\u003c/a>\u003c/li>\n\u003cli>\u003ca href=\"https://ww2.kqed.org/futureofyou/2017/11/13/the-difference-between-a-bully-and-a-true-alpha-male/\" target=\"_blank\" rel=\"noopener\">The Difference Between a Bully and a True Alpha Male\u003c/a>\u003c/li>\n\u003c/ul>\n\u003c/aside>\n\u003cp>\"You're really toying with Mother Nature\" and the risks can't be fully known, but the studies should move forward because these are incurable diseases, said one independent expert, Dr. Eric Topol of the Scripps Translational Science Institute in San Diego.\u003c/p>\n\u003cp>Protections are in place to help ensure safety, and animal tests were very encouraging, said Dr. Howard Kaufman, a Boston scientist on the National Institutes of Health panel that approved the studies.\u003c/p>\n\u003cp>He said gene editing's promise is too great to ignore. \"So far there's been no evidence that this is going to be dangerous,\" he said. \"Now is not the time to get scared.\"\u003c/p>\n\u003cp>\u003cstrong>Woe From Head to Toe\u003c/strong>\u003c/p>\n\u003cp>Fewer than 10,000 people worldwide have these metabolic diseases, partly because many die very young. Those with Madeux's condition, Hunter syndrome, lack a gene that makes an enzyme that breaks down certain carbohydrates. These build up in cells and cause havoc throughout the body.\u003c/p>\n\u003cp>Patients may have frequent colds and ear infections, distorted facial features, hearing loss, heart problems, breathing trouble, skin and eye problems, bone and joint flaws, bowel issues and brain and thinking problems.\u003c/p>\n\u003cp>\"Many are in wheelchairs ... dependent on their parents until they die,\" said Dr. Chester Whitley, a University of Minnesota genetics expert who plans to enroll patients in the studies.\u003c/p>\n\u003cp>Weekly IV doses of the missing enzyme can ease some symptoms, but cost $100,000 to $400,000 a year and don't prevent brain damage.\u003c/p>\n\u003cp>Madeux, who now lives near Phoenix, is engaged to a nurse, Marcie Humphrey. He met her 15 years ago in a study that tested this enzyme therapy at UCSF Benioff Children's Hospital Oakland, where the gene editing experiment took place.\u003c/p>\n\u003cp>He has had 26 operations for hernias, bunions, bones pinching his spinal column, and ear, eye and gall bladder problems.\u003c/p>\n\u003cp>\"It seems like I had a surgery every other year of my life\" and many procedures in between, he said. Last year he nearly died from a bronchitis and pneumonia attack. The disease had warped his airway, and \"I was drowning in my secretions, I couldn't cough it out.\"\u003cbr>\nMadeux has a chef's degree and was part owner of two restaurants in Utah, cooking for U.S. ski teams and celebrities, but now can't work in a kitchen or ride horses as he used to.\u003c/p>\n\u003cp>Gene editing won't fix damage he's already suffered, but he hopes it will stop the need for weekly enzyme treatments.\u003c/p>\n\u003cp>Initial studies will involve up to 30 adults to test safety, but the ultimate goal is to treat children very young, before much damage occurs.\u003c/p>\n\u003cp>\u003cstrong>How It Works\u003c/strong>\u003c/p>\n\u003cp>A gene-editing tool called CRISPR has gotten a lot of recent attention, but this study used a different one called zinc finger nucleases. They're like molecular scissors that seek and cut a specific piece of DNA.\u003c/p>\n\u003cp>The therapy has three parts: The new gene and two zinc finger proteins. DNA instructions for each part are placed in a virus that's been altered to not cause infection but to ferry them into cells. Billions of copies of these are given through a vein.\u003c/p>\n\u003cp>They travel to the liver, where cells use the instructions to make the zinc fingers and prepare the corrective gene. The fingers cut the DNA, allowing the new gene to slip in. The new gene then directs the cell to make the enzyme the patient lacked.\u003c/p>\n\u003cp>Only 1 percent of liver cells would have to be corrected to successfully treat the disease, said Madeux's physician and study leader, Dr. Paul Harmatz at the Oakland hospital.\u003c/p>\n\u003cp>\"How bulletproof is the technology? We're just learning,\" but safety tests have been very good, said Dr. Carl June, a University of Pennsylvania scientist who has done other gene therapy work but was not involved in this study.\u003c/p>\n\u003cp>\u003cstrong>What Could Go Wrong\u003c/strong>\u003c/p>\n\u003cp>Safety issues plagued some earlier gene therapies. One worry is that the virus might provoke an immune system attack. In 1999, 18-year-old Jesse Gelsinger died in a gene therapy study from that problem, but the new studies use a different virus that's proved much safer in other experiments.\u003c/p>\n\u003cp>Another worry is that inserting a new gene might have unforeseen effects on other genes. That happened years ago, when researchers used gene therapy to cure some cases of the immune system disorder called \"bubble boy\" disease. Several patients later developed leukemia because the new gene inserted into a place in the native DNA where it unintentionally activated a cancer gene.\u003c/p>\n\u003cp>\"When you stick a chunk of DNA in randomly, sometimes it works well, sometimes it does nothing and sometimes it causes harm,\" said Hank Greely, a Stanford University bioethicist. \"The advantage with gene editing is you can put the gene in where you want it.\"\u003c/p>\n\u003cp>Finally, some fear that the virus could get into other places like the heart, or eggs and sperm where it could affect future generations. Doctors say built-in genetic safeguards prevent the therapy from working anywhere but the liver, like a seed that only germinates in certain conditions.\u003c/p>\n\u003cp>This experiment is not connected to other, more controversial work being debated to try to edit genes in human embryos to prevent diseases before birth — changes that would be passed down from generation to generation.\u003c/p>\n\u003cp>\u003cstrong>Making History\u003c/strong>\u003c/p>\n\u003cp>Madeux's treatment was to have happened a week earlier, but a small glitch prevented it.\u003c/p>\n\u003cp>He and his fiancee returned to Arizona, but nearly didn't make it back to Oakland in time for the second attempt because their Sunday flight was canceled and no others were available until Monday, after the treatment was to take place.\u003c/p>\n\u003cp>Scrambling, they finally got a flight to Monterey, California, and a car service took them just over 100 miles north to Oakland.\u003c/p>\n\u003cp>On Monday he had the three-hour infusion, surrounded by half a dozen doctors, nurses and others wearing head-to-toe protective garb to lower the risk of giving him any germs. His doctor, Harmatz, spent the night at the hospital to help ensure his patient stayed well.\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>\"I'm nervous and excited,\" Madeux said as he prepared to leave the hospital. \"I've been waiting for this my whole life, something that can potentially cure me.\"\u003c/p>\n\n","blocks":[],"excerpt":"In Oakland, Monday, 44-year-old Brian Madeux received billions of copies of a corrective gene, plus a genetic tool to cut his DNA in a precise spot.","status":"publish","parent":0,"modified":1510789638,"stats":{"hasAudio":false,"hasVideo":true,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":42,"wordCount":1419},"headData":{"title":"In First, Scientists Try to Cure Man's Disease by Editing DNA Inside Body | KQED","description":"In Oakland, Monday, 44-year-old Brian Madeux received billions of copies of a corrective gene, plus a genetic tool to cut his DNA in a precise spot.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"In First, Scientists Try to Cure Man's Disease by Editing DNA Inside Body","datePublished":"2017-11-15T16:57:21.000Z","dateModified":"2017-11-15T23:47:18.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"437054 https://ww2.kqed.org/futureofyou/?p=437054","disqusUrl":"https://ww2.kqed.org/futureofyou/2017/11/15/first-attempt-to-edit-gene-inside-body-in-hopes-of-curing-mans-disease/","disqusTitle":"In First, Scientists Try to Cure Man's Disease by Editing DNA Inside Body","source":"KQED Future of You","nprByline":"Marilynn Marchione\u003cbr />Associated Press","path":"/futureofyou/437054/first-attempt-to-edit-gene-inside-body-in-hopes-of-curing-mans-disease","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\u003cp>\u003cspan class='utils-parseShortcode-shortcodes-__youtubeShortcode__embedYoutube'>\n \u003cspan class='utils-parseShortcode-shortcodes-__youtubeShortcode__embedYoutubeInside'>\n \u003ciframe\n loading='lazy'\n class='utils-parseShortcode-shortcodes-__youtubeShortcode__youtubePlayer'\n type='text/html'\n src='//www.youtube.com/embed/W-gUUB0ofVA'\n title='//www.youtube.com/embed/W-gUUB0ofVA'\n allowfullscreen='true'\n style='border:0;'>\u003c/iframe>\n \u003c/span>\n \u003c/span>\u003c/p>\u003cp>\u003cp>Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person's DNA to cure a disease.\u003c/p>\n\u003cp>The experiment was done Monday in California on 44-year-old Brian Madeux. Through an IV, he received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot.\u003c/p>\n\u003cp>\"It's kind of humbling\" to be the first to test this, said Madeux, who has a metabolic disease called Hunter syndrome. \"I'm willing to take that risk. Hopefully it will help me and other people.\"\u003c/p>\n\u003cp>Signs of whether it's working may come in a month; tests will show for sure in three months.\u003c/p>\n\u003caside class=\"pullquote alignright\">'You’re really toying with Mother Nature.'\u003ccite>Dr. Eric Topol, Scripps Translational Science Institute\u003c/cite>\u003c/aside>\n\u003cp>If it's successful, it could give a major boost to the fledgling field of gene therapy . Scientists have edited people's genes before, altering cells in the lab that are then returned to patients. There also are gene therapies that don't involve editing DNA.\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>But these methods can only be used for a few types of diseases. Some give results that may not last. Some others supply a new gene like a spare part, but can't control where it inserts in the DNA, possibly causing a new problem like cancer.\u003c/p>\n\u003cp>This time, the gene tinkering is happening in a precise way inside the body. It's like sending a mini surgeon along to place the new gene in exactly the right location.\u003c/p>\n\u003cp>\"We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending,\" said Dr. Sandy Macrae, president of Sangamo Therapeutics, the California company testing this for two metabolic diseases and hemophilia. \"It becomes part of your DNA and is there for the rest of your life.\"\u003c/p>\n\u003cp>That also means there's no going back, no way to erase any mistakes the editing might cause.\u003c/p>\n\u003caside class=\"aligncenter\">\u003cem>Also from KQED Future of You:\u003c/em>\n\u003cul>\n\u003cli>\u003ca href=\"https://ww2.kqed.org/futureofyou/2017/10/31/the-crippling-ransomware-attack-on-kqed-the-inside-story/\" target=\"_blank\" rel=\"noopener\">The Crippling Ransomware Attack on an NPR Member Station\u003c/a>\u003c/li>\n\u003cli>\u003ca href=\"https://ww2.kqed.org/futureofyou/2017/11/13/the-difference-between-a-bully-and-a-true-alpha-male/\" target=\"_blank\" rel=\"noopener\">The Difference Between a Bully and a True Alpha Male\u003c/a>\u003c/li>\n\u003c/ul>\n\u003c/aside>\n\u003cp>\"You're really toying with Mother Nature\" and the risks can't be fully known, but the studies should move forward because these are incurable diseases, said one independent expert, Dr. Eric Topol of the Scripps Translational Science Institute in San Diego.\u003c/p>\n\u003cp>Protections are in place to help ensure safety, and animal tests were very encouraging, said Dr. Howard Kaufman, a Boston scientist on the National Institutes of Health panel that approved the studies.\u003c/p>\n\u003cp>He said gene editing's promise is too great to ignore. \"So far there's been no evidence that this is going to be dangerous,\" he said. \"Now is not the time to get scared.\"\u003c/p>\n\u003cp>\u003cstrong>Woe From Head to Toe\u003c/strong>\u003c/p>\n\u003cp>Fewer than 10,000 people worldwide have these metabolic diseases, partly because many die very young. Those with Madeux's condition, Hunter syndrome, lack a gene that makes an enzyme that breaks down certain carbohydrates. These build up in cells and cause havoc throughout the body.\u003c/p>\n\u003cp>Patients may have frequent colds and ear infections, distorted facial features, hearing loss, heart problems, breathing trouble, skin and eye problems, bone and joint flaws, bowel issues and brain and thinking problems.\u003c/p>\n\u003cp>\"Many are in wheelchairs ... dependent on their parents until they die,\" said Dr. Chester Whitley, a University of Minnesota genetics expert who plans to enroll patients in the studies.\u003c/p>\n\u003cp>Weekly IV doses of the missing enzyme can ease some symptoms, but cost $100,000 to $400,000 a year and don't prevent brain damage.\u003c/p>\n\u003cp>Madeux, who now lives near Phoenix, is engaged to a nurse, Marcie Humphrey. He met her 15 years ago in a study that tested this enzyme therapy at UCSF Benioff Children's Hospital Oakland, where the gene editing experiment took place.\u003c/p>\n\u003cp>He has had 26 operations for hernias, bunions, bones pinching his spinal column, and ear, eye and gall bladder problems.\u003c/p>\n\u003cp>\"It seems like I had a surgery every other year of my life\" and many procedures in between, he said. Last year he nearly died from a bronchitis and pneumonia attack. The disease had warped his airway, and \"I was drowning in my secretions, I couldn't cough it out.\"\u003cbr>\nMadeux has a chef's degree and was part owner of two restaurants in Utah, cooking for U.S. ski teams and celebrities, but now can't work in a kitchen or ride horses as he used to.\u003c/p>\n\u003cp>Gene editing won't fix damage he's already suffered, but he hopes it will stop the need for weekly enzyme treatments.\u003c/p>\n\u003cp>Initial studies will involve up to 30 adults to test safety, but the ultimate goal is to treat children very young, before much damage occurs.\u003c/p>\n\u003cp>\u003cstrong>How It Works\u003c/strong>\u003c/p>\n\u003cp>A gene-editing tool called CRISPR has gotten a lot of recent attention, but this study used a different one called zinc finger nucleases. They're like molecular scissors that seek and cut a specific piece of DNA.\u003c/p>\n\u003cp>The therapy has three parts: The new gene and two zinc finger proteins. DNA instructions for each part are placed in a virus that's been altered to not cause infection but to ferry them into cells. Billions of copies of these are given through a vein.\u003c/p>\n\u003cp>They travel to the liver, where cells use the instructions to make the zinc fingers and prepare the corrective gene. The fingers cut the DNA, allowing the new gene to slip in. The new gene then directs the cell to make the enzyme the patient lacked.\u003c/p>\n\u003cp>Only 1 percent of liver cells would have to be corrected to successfully treat the disease, said Madeux's physician and study leader, Dr. Paul Harmatz at the Oakland hospital.\u003c/p>\n\u003cp>\"How bulletproof is the technology? We're just learning,\" but safety tests have been very good, said Dr. Carl June, a University of Pennsylvania scientist who has done other gene therapy work but was not involved in this study.\u003c/p>\n\u003cp>\u003cstrong>What Could Go Wrong\u003c/strong>\u003c/p>\n\u003cp>Safety issues plagued some earlier gene therapies. One worry is that the virus might provoke an immune system attack. In 1999, 18-year-old Jesse Gelsinger died in a gene therapy study from that problem, but the new studies use a different virus that's proved much safer in other experiments.\u003c/p>\n\u003cp>Another worry is that inserting a new gene might have unforeseen effects on other genes. That happened years ago, when researchers used gene therapy to cure some cases of the immune system disorder called \"bubble boy\" disease. Several patients later developed leukemia because the new gene inserted into a place in the native DNA where it unintentionally activated a cancer gene.\u003c/p>\n\u003cp>\"When you stick a chunk of DNA in randomly, sometimes it works well, sometimes it does nothing and sometimes it causes harm,\" said Hank Greely, a Stanford University bioethicist. \"The advantage with gene editing is you can put the gene in where you want it.\"\u003c/p>\n\u003cp>Finally, some fear that the virus could get into other places like the heart, or eggs and sperm where it could affect future generations. Doctors say built-in genetic safeguards prevent the therapy from working anywhere but the liver, like a seed that only germinates in certain conditions.\u003c/p>\n\u003cp>This experiment is not connected to other, more controversial work being debated to try to edit genes in human embryos to prevent diseases before birth — changes that would be passed down from generation to generation.\u003c/p>\n\u003cp>\u003cstrong>Making History\u003c/strong>\u003c/p>\n\u003cp>Madeux's treatment was to have happened a week earlier, but a small glitch prevented it.\u003c/p>\n\u003cp>He and his fiancee returned to Arizona, but nearly didn't make it back to Oakland in time for the second attempt because their Sunday flight was canceled and no others were available until Monday, after the treatment was to take place.\u003c/p>\n\u003cp>Scrambling, they finally got a flight to Monterey, California, and a car service took them just over 100 miles north to Oakland.\u003c/p>\n\u003cp>On Monday he had the three-hour infusion, surrounded by half a dozen doctors, nurses and others wearing head-to-toe protective garb to lower the risk of giving him any germs. His doctor, Harmatz, spent the night at the hospital to help ensure his patient stayed well.\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>\"I'm nervous and excited,\" Madeux said as he prepared to leave the hospital. \"I've been waiting for this my whole life, something that can potentially cure me.\"\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/437054/first-attempt-to-edit-gene-inside-body-in-hopes-of-curing-mans-disease","authors":["byline_futureofyou_437054"],"categories":["futureofyou_452","futureofyou_1062","futureofyou_1","futureofyou_73","futureofyou_1064"],"tags":["futureofyou_1275","futureofyou_295","futureofyou_1209","futureofyou_80","futureofyou_1396"],"featImg":"futureofyou_437058","label":"source_futureofyou_437054"},"futureofyou_436444":{"type":"posts","id":"futureofyou_436444","meta":{"index":"posts_1591205157","site":"futureofyou","id":"436444","score":null,"sort":[1508963960000]},"guestAuthors":[],"slug":"even-more-precise-crispr-rearranges-atoms-to-edit-genes","title":"Even More Precise CRISPR Rearranges Atoms to Edit Genes","publishDate":1508963960,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{"site":"futureofyou"},"content":"\u003cp>The acronyms might not be quite as catchy as CRISPR — since, really, what is? — but what new genetic tools dubbed REPAIR and ABE lack in whimsy they promise to make up in utility. These advances announced Wednesday solve two of the problems hobbling CRISPR, the revolutionary genome-editing technique: that its idea of “editing” is often like 1,000 monkeys editing a Word document, and that making permanent changes to DNA might not be the best approach.\u003c/p>\n\u003caside class=\"pullquote alignright\">New gene-editing tools, both announced Wednesday, solve two problems associated with 'classic' CRISPR.\u003c/aside>\n\u003cp>Together, the discoveries, described in separate studies, show that five years after scientists \u003ca href=\"https://www.ncbi.nlm.nih.gov/pubmed/22745249\" target=\"_blank\" rel=\"noopener\">demonstrated\u003c/a> that CRISPR can edit DNA, bioengineers are still racing to develop the most efficient, precise, versatile — and therefore lucrative — genome-editing tools possible.\u003c/p>\n\u003cp>“One reason these are so exciting is, they show the CRISPR toolbox is still growing,” said chemical engineer Gene Yeo of the University of California, San Diego. “There are going to be a lot more, and it’s not going to stop anytime soon.” His lab has been working on one of the CRISPR advances but was not involved in either of the two new studies — it’s “personally frustrating” to get beaten, he added.\u003c/p>\n\u003cp>One discovery, led by biochemist David Liu of Harvard University, extends his 2016 \u003ca href=\"https://www.statnews.com/2016/04/20/clever-crispr-advance-unveiled/\">invention\u003c/a> of a way to change a single DNA “letter,” or base, on the 3-billion-letter-long human genome. Classic CRISPR cuts DNA with a molecular scissors and leaves the cell to repair the breach willy nilly, introducing the problem of 1,000 monkeys editing away. In contrast, Liu’s “base editor” replaces the molecular scissors with something like a pencil wielded by an expert forger: It is an enzyme that literally rearranges atoms — cleanly and without collateral damage that the cell needs to fix.\u003c/p>\n\u003carticle>\u003c/article>\n\u003cp>As in classic CRISPR, this version finds its way to a target on the genome via a molecule that acts like a bloodhound. Attached to the bloodhound is the atom-rearranger, which in Liu’s 2016 version turned the DNA letter G into A. Thousands of genetic diseases arise because a gene has a G where it should have an A,\u003cstrong> \u003c/strong>so the edit might one day treat or prevent them.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>But other inherited disorders need different alphabetical magic. That’s what Liu, postdoctoral fellows Nicole Gaudelli and Alexis Komor, and their colleagues report in a \u003ca href=\"https://www.nature.com/articles/doi:10.1038/nature24644\" target=\"_blank\" rel=\"noopener\">paper\u003c/a> in Nature: Their new “ABE” (adenine base editor) can turn A into G. Attached to CRISPR’s bloodhound molecule, ABE works “at virtually any target site in genomic DNA,” Liu said.\u003c/p>\n\u003cp>In tests so far, it changed DNA in more of the lab-grown human cells that it was slipped into than standard CRISPR (for all its fame, CRISPR often bungles the job). ABE also seems to make fewer off-target edits: In one test, it mistakenly hit four of the 12 off-target sites, compared to CRISPR’s nine, and made that mistake in 1.3 percent of cases compared to 14 percent for CRISPR, Liu said.\u003c/p>\n\u003cp>About half of the 32,000 known disease-causing, single-letter mutations have one of the misspellings that ABE can fix, Liu said. They include \u003ca href=\"https://www.statnews.com/2017/09/18/sickle-cell-pain-treatment/\">sickle cell\u003c/a>, Tay-Sachs, and cystic fibrosis, raising hopes that ABE could be used to treat these diseases, or (in early embryos) prevent them. In tests of cells growing in lab dishes, ABE reversed the mutation that causes hereditary hemochromatosis in about 30 percent of the cells, and changed another gene into a form that prevents sickle cell disease even in people who have its disease-causing mutation.\u003c/p>\n\u003cp>As with all forms of CRISPR, before ABE helps any patients, scientists will have to test whether it’s safe and effective. “But having the molecular machine is a good start,” said Liu, a co-founder of the CRISPR company Editas Medicine. He and colleagues have filed for patents on ABE.\u003c/p>\n\u003cp>Harvard biologist George Church, who \u003ca href=\"https://www.ncbi.nlm.nih.gov/pubmed/23287722\" target=\"_blank\" rel=\"noopener\">tied\u003c/a> for first in the race to make CRISPR work in human cells, called base editing “especially interesting.” Changing a single DNA letter, he said, means “fewer worries about the editing enzyme [in classic CRISPR] later going rogue or silent.” He also expects that crops with a single base change will not be designated as “transgenic,” reducing regulatory barriers to commercialization.\u003c/p>\n\u003cp>In a separate study, CRISPR pioneer Feng Zhang of the Broad Institute and his colleagues discovered a new version of CRISPR that can edit RNA, DNA’s friskier cousin. While DNA mostly sits sedately in cells and issues orders to make proteins that keep life living, RNA zips around the cell carrying out those orders, and then disappears. That makes RNA a tantalizing target: By editing the errant orders (RNA) rather than their issuer (DNA), scientists might be able to make temporary, reversible genetic edits, rather than CRISPR’s permanent ones.\u003c/p>\n\u003cp>“Editing DNA is hard to reverse, but once you stop providing the RNA-editing system, the changes will disappear over time,” said Zhang, also a co-founder of Editas. “That might make it possible to treat conditions where you don’t need a permanent edit,” such as when the immune system is in overdrive and causing inflammation.\u003c/p>\n\u003cp>To create what Zhang and his colleagues call REPAIR (“RNA editing for programmable A to I [G] replacement”), they fused an enzyme that binds to RNA with one that changes the RNA letter A (adenosine) to inosine, a molecule similar to the RNA letter G (guanosine), they \u003ca href=\"http://science.sciencemag.org/content/early/2017/10/24/science.aaq0180\" target=\"_blank\" rel=\"noopener\">report\u003c/a> in Science. Other labs, including that of CRISPR developer Jennifer Doudna of the University of California, Berkeley, have also developed \u003ca href=\"https://www.ncbi.nlm.nih.gov/pubmed/28475872?dopt=Abstract\" target=\"_blank\" rel=\"noopener\">RNA editors\u003c/a>, including \u003ca href=\"https://www.ncbi.nlm.nih.gov/pubmed/28892041?dopt=Abstract\" target=\"_blank\" rel=\"noopener\">one\u003c/a> using the same Cas13 enzyme. But REPAIR’s creators say theirs is more efficient and less error-prone.\u003c/p>\n\u003cp>In tests on human cells growing in the lab, REPAIR corrected misspellings in the RNA that was made by disease-causing DNA — in this case, Fanconi anemia, an inherited and devastating bone marrow disease, or nephrogenic diabetes insipidus, a serious inborn kidney disease. Although the DNA still had its disease-causing mutations, 23 percent and 35 percent, respectively, of the RNA made by those defective genes was REPAIR’ed. Those levels might be high enough to treat the diseases. Some 5,800 inherited diseases are the result of the G-to-A glitch that REPAIR can fix, including epilepsy and Duchenne muscular dystrophy.\u003c/p>\n\u003cp>Both REPAIR and ABE might venture where CRISPR stumbles: in mature cells, like neurons, that don’t divide. In unpublished research, Liu said, he and his team have shown that ABE can edit genes in neurons, raising the possibility of treating devastating neurological diseases with ABE.\u003c/p>\n\u003cp>The furious race to improve CRISPR, via ABE or REPAIR or whatever comes next, Church said, are “potent reminders of how far CRISPR is from precise genome-editing in humans.”\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>\u003cem>\u003cspan style=\"font-weight: 400\"> This\u003ca href=\"https://www.statnews.com/2017/10/25/crispr-gene-editing-advances/\" target=\"_blank\" rel=\"noopener\"> story\u003c/a> was originally published by STAT, an online publication of Boston Globe Media that covers health, medicine, and scientific discovery. \u003c/span>\u003c/em>\u003c/p>\n\n","blocks":[],"excerpt":"Two separate discoveries announced Wednesday show that bioengineers are still racing to develop the most efficient, precise and versatile genome-editing tools possible.","status":"publish","parent":0,"modified":1508965165,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":19,"wordCount":1236},"headData":{"title":"Even More Precise CRISPR Rearranges Atoms to Edit Genes | KQED","description":"Two separate discoveries announced Wednesday show that bioengineers are still racing to develop the most efficient, precise and versatile genome-editing tools possible.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Even More Precise CRISPR Rearranges Atoms to Edit Genes","datePublished":"2017-10-25T20:39:20.000Z","dateModified":"2017-10-25T20:59:25.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"436444 https://ww2.kqed.org/futureofyou/?p=436444","disqusUrl":"https://ww2.kqed.org/futureofyou/2017/10/25/even-more-precise-crispr-rearranges-atoms-to-edit-genes/","disqusTitle":"Even More Precise CRISPR Rearranges Atoms to Edit Genes","nprByline":"Sharon Begley\u003cbr / >\u003ca href=\"https://www.statnews.com/\">STAT\u003c/a>","path":"/futureofyou/436444/even-more-precise-crispr-rearranges-atoms-to-edit-genes","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>The acronyms might not be quite as catchy as CRISPR — since, really, what is? — but what new genetic tools dubbed REPAIR and ABE lack in whimsy they promise to make up in utility. These advances announced Wednesday solve two of the problems hobbling CRISPR, the revolutionary genome-editing technique: that its idea of “editing” is often like 1,000 monkeys editing a Word document, and that making permanent changes to DNA might not be the best approach.\u003c/p>\n\u003caside class=\"pullquote alignright\">New gene-editing tools, both announced Wednesday, solve two problems associated with 'classic' CRISPR.\u003c/aside>\n\u003cp>Together, the discoveries, described in separate studies, show that five years after scientists \u003ca href=\"https://www.ncbi.nlm.nih.gov/pubmed/22745249\" target=\"_blank\" rel=\"noopener\">demonstrated\u003c/a> that CRISPR can edit DNA, bioengineers are still racing to develop the most efficient, precise, versatile — and therefore lucrative — genome-editing tools possible.\u003c/p>\n\u003cp>“One reason these are so exciting is, they show the CRISPR toolbox is still growing,” said chemical engineer Gene Yeo of the University of California, San Diego. “There are going to be a lot more, and it’s not going to stop anytime soon.” His lab has been working on one of the CRISPR advances but was not involved in either of the two new studies — it’s “personally frustrating” to get beaten, he added.\u003c/p>\n\u003cp>One discovery, led by biochemist David Liu of Harvard University, extends his 2016 \u003ca href=\"https://www.statnews.com/2016/04/20/clever-crispr-advance-unveiled/\">invention\u003c/a> of a way to change a single DNA “letter,” or base, on the 3-billion-letter-long human genome. Classic CRISPR cuts DNA with a molecular scissors and leaves the cell to repair the breach willy nilly, introducing the problem of 1,000 monkeys editing away. In contrast, Liu’s “base editor” replaces the molecular scissors with something like a pencil wielded by an expert forger: It is an enzyme that literally rearranges atoms — cleanly and without collateral damage that the cell needs to fix.\u003c/p>\n\u003carticle>\u003c/article>\n\u003cp>As in classic CRISPR, this version finds its way to a target on the genome via a molecule that acts like a bloodhound. Attached to the bloodhound is the atom-rearranger, which in Liu’s 2016 version turned the DNA letter G into A. Thousands of genetic diseases arise because a gene has a G where it should have an A,\u003cstrong> \u003c/strong>so the edit might one day treat or prevent them.\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>But other inherited disorders need different alphabetical magic. That’s what Liu, postdoctoral fellows Nicole Gaudelli and Alexis Komor, and their colleagues report in a \u003ca href=\"https://www.nature.com/articles/doi:10.1038/nature24644\" target=\"_blank\" rel=\"noopener\">paper\u003c/a> in Nature: Their new “ABE” (adenine base editor) can turn A into G. Attached to CRISPR’s bloodhound molecule, ABE works “at virtually any target site in genomic DNA,” Liu said.\u003c/p>\n\u003cp>In tests so far, it changed DNA in more of the lab-grown human cells that it was slipped into than standard CRISPR (for all its fame, CRISPR often bungles the job). ABE also seems to make fewer off-target edits: In one test, it mistakenly hit four of the 12 off-target sites, compared to CRISPR’s nine, and made that mistake in 1.3 percent of cases compared to 14 percent for CRISPR, Liu said.\u003c/p>\n\u003cp>About half of the 32,000 known disease-causing, single-letter mutations have one of the misspellings that ABE can fix, Liu said. They include \u003ca href=\"https://www.statnews.com/2017/09/18/sickle-cell-pain-treatment/\">sickle cell\u003c/a>, Tay-Sachs, and cystic fibrosis, raising hopes that ABE could be used to treat these diseases, or (in early embryos) prevent them. In tests of cells growing in lab dishes, ABE reversed the mutation that causes hereditary hemochromatosis in about 30 percent of the cells, and changed another gene into a form that prevents sickle cell disease even in people who have its disease-causing mutation.\u003c/p>\n\u003cp>As with all forms of CRISPR, before ABE helps any patients, scientists will have to test whether it’s safe and effective. “But having the molecular machine is a good start,” said Liu, a co-founder of the CRISPR company Editas Medicine. He and colleagues have filed for patents on ABE.\u003c/p>\n\u003cp>Harvard biologist George Church, who \u003ca href=\"https://www.ncbi.nlm.nih.gov/pubmed/23287722\" target=\"_blank\" rel=\"noopener\">tied\u003c/a> for first in the race to make CRISPR work in human cells, called base editing “especially interesting.” Changing a single DNA letter, he said, means “fewer worries about the editing enzyme [in classic CRISPR] later going rogue or silent.” He also expects that crops with a single base change will not be designated as “transgenic,” reducing regulatory barriers to commercialization.\u003c/p>\n\u003cp>In a separate study, CRISPR pioneer Feng Zhang of the Broad Institute and his colleagues discovered a new version of CRISPR that can edit RNA, DNA’s friskier cousin. While DNA mostly sits sedately in cells and issues orders to make proteins that keep life living, RNA zips around the cell carrying out those orders, and then disappears. That makes RNA a tantalizing target: By editing the errant orders (RNA) rather than their issuer (DNA), scientists might be able to make temporary, reversible genetic edits, rather than CRISPR’s permanent ones.\u003c/p>\n\u003cp>“Editing DNA is hard to reverse, but once you stop providing the RNA-editing system, the changes will disappear over time,” said Zhang, also a co-founder of Editas. “That might make it possible to treat conditions where you don’t need a permanent edit,” such as when the immune system is in overdrive and causing inflammation.\u003c/p>\n\u003cp>To create what Zhang and his colleagues call REPAIR (“RNA editing for programmable A to I [G] replacement”), they fused an enzyme that binds to RNA with one that changes the RNA letter A (adenosine) to inosine, a molecule similar to the RNA letter G (guanosine), they \u003ca href=\"http://science.sciencemag.org/content/early/2017/10/24/science.aaq0180\" target=\"_blank\" rel=\"noopener\">report\u003c/a> in Science. Other labs, including that of CRISPR developer Jennifer Doudna of the University of California, Berkeley, have also developed \u003ca href=\"https://www.ncbi.nlm.nih.gov/pubmed/28475872?dopt=Abstract\" target=\"_blank\" rel=\"noopener\">RNA editors\u003c/a>, including \u003ca href=\"https://www.ncbi.nlm.nih.gov/pubmed/28892041?dopt=Abstract\" target=\"_blank\" rel=\"noopener\">one\u003c/a> using the same Cas13 enzyme. But REPAIR’s creators say theirs is more efficient and less error-prone.\u003c/p>\n\u003cp>In tests on human cells growing in the lab, REPAIR corrected misspellings in the RNA that was made by disease-causing DNA — in this case, Fanconi anemia, an inherited and devastating bone marrow disease, or nephrogenic diabetes insipidus, a serious inborn kidney disease. Although the DNA still had its disease-causing mutations, 23 percent and 35 percent, respectively, of the RNA made by those defective genes was REPAIR’ed. Those levels might be high enough to treat the diseases. Some 5,800 inherited diseases are the result of the G-to-A glitch that REPAIR can fix, including epilepsy and Duchenne muscular dystrophy.\u003c/p>\n\u003cp>Both REPAIR and ABE might venture where CRISPR stumbles: in mature cells, like neurons, that don’t divide. In unpublished research, Liu said, he and his team have shown that ABE can edit genes in neurons, raising the possibility of treating devastating neurological diseases with ABE.\u003c/p>\n\u003cp>The furious race to improve CRISPR, via ABE or REPAIR or whatever comes next, Church said, are “potent reminders of how far CRISPR is from precise genome-editing in humans.”\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>\u003cem>\u003cspan style=\"font-weight: 400\"> This\u003ca href=\"https://www.statnews.com/2017/10/25/crispr-gene-editing-advances/\" target=\"_blank\" rel=\"noopener\"> story\u003c/a> was originally published by STAT, an online publication of Boston Globe Media that covers health, medicine, and scientific discovery. \u003c/span>\u003c/em>\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/436444/even-more-precise-crispr-rearranges-atoms-to-edit-genes","authors":["byline_futureofyou_436444"],"categories":["futureofyou_1","futureofyou_1064"],"tags":["futureofyou_1383","futureofyou_94","futureofyou_1275","futureofyou_295","futureofyou_1382"],"featImg":"futureofyou_436449","label":"futureofyou"},"futureofyou_435096":{"type":"posts","id":"futureofyou_435096","meta":{"index":"posts_1591205157","site":"futureofyou","id":"435096","score":null,"sort":[1504197017000]},"guestAuthors":[],"slug":"as-human-gene-editing-advances-doudna-says-ethical-discussions-cant-wait","title":"As Human Gene-Editing Advances, Doudna Says Ethical Discussions Can't Wait","publishDate":1504197017,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp>If you want to have a role in shaping the near and coming future of biotechnology, the time is now. A science degree is not required, but a sense of urgency is.\u003c/p>\n\u003cp>This was the primary takeaway from Jennifer Doudna's recent public remarks at\u003ca href=\"http://crisprcon.org/\" target=\"_blank\" rel=\"noopener noreferrer\"> CRISPRcon\u003c/a>, a two day event at UC Berkeley, intended to get nonspecialists talking about the promise and potential peril of the fast-moving biotech landscape.\u003c/p>\n\u003cp>\"Decisions about gene editing have an impact on all of us,\" said Michael Krasney, host of KQED's \"Forum\" radio program and the CRISPRcon emcee. \"That's why there are no outsiders in discussing this topic.\" (For a primer on how CRISPR gene editing works, check out our story \"\u003ca href=\"https://ww2.kqed.org/futureofyou/2015/12/30/a-crispr-solution-to-bubble-boy-disease/\" target=\"_blank\" rel=\"noopener noreferrer\">CRISPR: What You Need to Know About the Medical Science ‘Breakthrough of the Year\u003c/a>.\")\u003c/p>\n\u003caside class=\"pullquote alignright\">\"We can no longer say, well there's a lot of technology development to be done before we have to worry about that application, it's now a question of 'We know this can work, are we willing to go there or not?'\" \u003ccite>Jennifer Doudna, one of the inventors of CRISPR/Cas9 gene-editing technology\u003c/cite>\u003c/aside>\n\u003cp>The National Academies of Sciences, Engineering and Medicine wants public input, as well. The rationale is, if it affects the public, the public should have a say.\u003c/p>\n\u003cp>\u003cstrong>What's at Stake\u003c/strong>\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>In her new book, \"\u003ca href=\"http://www.acrackincreation.com/\" target=\"_blank\" rel=\"noopener noreferrer\">A Crack in Creation: Gene Editing and the Unthinkable Power to Control Evolution\u003c/a>,\" Doudna (who co-authored the book with former student Samuel Sternberg) writes humans are \"on the cusp of a new age in genetic engineering and biological mastery.\"\u003c/p>\n\u003cp>\"It won’t be long before CRISPR allows us to bend nature to our will.”\u003c/p>\n\u003cp>CRISPR applications could bring us \u003ca href=\"https://www.sciencedaily.com/releases/2017/05/170518140335.htm\" target=\"_blank\" rel=\"noopener noreferrer\">higher tomato yields\u003c/a> or cattle \u003ca href=\"https://geneticliteracyproject.org/2016/05/11/gene-edited-hornless-cow-improve-animal-welfare-regulatory-fate-unclear/\" target=\"_blank\" rel=\"noopener noreferrer\">born without horns\u003c/a> (allowing livestock to avoid the potentially painful procedure of having them removed). Ongoing medical research using CRISPR includes \u003ca href=\"https://www.nature.com/news/first-crispr-clinical-trial-gets-green-light-from-us-panel-1.20137\" target=\"_blank\" rel=\"noopener noreferrer\">gene editing to target immune response\u003c/a> in cancer patients and to \u003ca href=\"https://www.nature.com/news/crispr-deployed-to-combat-sickle-cell-anaemia-1.20782\" target=\"_blank\" rel=\"noopener noreferrer\">correct sickle-cell anemia\u003c/a>, a painful blood disorder.\u003c/p>\n\u003cp>But the $10 million question at CRISPRcon loomed large:\u003c/p>\n\u003cp>\"How do you decide which genes are appropriate for germline editing?\" asked a woman in the audience, during a question and answer session with Doudna. Germline editing means that the changes made affect sperm or egg cells, and therefore can be inherited.\u003c/p>\n\u003cp>[contextly_sidebar id=\"HfCYio1aAJwJdg2Ip7LwMBF1sWeN5NFY\"]\"That's a question that could be debated and discussed for the entire time of the conference,\" said Doudna. But her short answer, referencing a \u003ca href=\"http://www.nationalacademies.org/gene-editing/consensus-study/index.htm\" target=\"_blank\" rel=\"noopener noreferrer\">recent report with recommendations\u003c/a> from the National Academies, was, \"We look for situations where there would be no other reasonable way to deal with a genetic disease other than gene editing. And when you think about it that way, those situations are rather rare.\"\u003c/p>\n\u003cp>Preimplantation genetic diagnosis (for parents using in vitro fertilization) and genetic counseling can offer routes to avoid serious genetic mutations without gene editing. This latter technique would, however, require parents to decide if they are willing to terminate a pregnancy.\u003c/p>\n\u003cp>Doudna believes that in most cases genetic counseling and preimplantation genetic diagnosis are sufficient to deal with disorders caused by a single gene. But some couples might not have any other option to conceive a healthy embryo outside of editing their genes. For example, if both parents both have the same disease-causing gene, \"That's an issue where gene editing could be relevant in the future.\"\u003c/p>\n\u003cp>For any parents who want to select for green-eyed, athletic geniuses, their best bet is still a random roll of the dice, not gene-editing, if the National Academies has anything to do with it. \"Do not proceed at this time with human genome editing for purposes other than treatment or prevention of disease and disability,\" the study recommends.\u003c/p>\n\u003cp>But the question of whether embryos should be edited to avoid disease and disability has reached new urgency, says Doudna, with the publication several weeks ago of the first \u003ca href=\"https://ww2.kqed.org/futureofyou/2017/07/27/scientists-in-us-edit-human-embryos-with-crispr-for-first-time-reports-suggest/\" target=\"_blank\" rel=\"noopener noreferrer\">paper\u003c/a> that lays out a good protocol (a written procedure) for targeting a gene linked to disease. It has the potential of being useful in clinical applications.\u003c/p>\n\u003cp>\"What they showed, importantly,\" said Doudna, \"was that there was very few off-target effects [unintended changes] and also they could avoid something called 'mosaicism'. \" That arises when the desired edit occurs in only some of the cells in the developing embryo. Both edited and non-editing cells replicate, resulting in an organism with a genetic patchwork, which may or may not be harmful.\u003c/p>\n\u003cp>\"We now understand that, when applied in certain ways this technology can be very robust, in viable human embryos. We can no longer say, 'Well, there's a lot of technology development to be done before we have to worry about that application.' It's now a question of, 'We know this can work, are we willing to go there or not?' \"\u003c/p>\n\u003cp>Since CRISPRcon, the paper in question has come in for some \u003ca href=\"https://ipscell.com/2017/08/doubts-raised-on-key-points-of-nature-paper-on-crispr-gene-editing-of-human-embryos/\" target=\"_blank\" rel=\"noopener noreferrer\">recent criticism from scientists\u003c/a> not involved in the research. They call into question the main conclusions of the paper and say more definitive studies are needed. That said, most scientists believe precise editing in embryos will be possible someday, and likely someday soon.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>For members of the public eager to be part of the debate, events like CRISPRcon provide a rare forum with scientists like Doudna who have \u003ca href=\"https://ipscell.com/2015/06/doudnacongress/\" target=\"_blank\" rel=\"noopener noreferrer\">testified before Congress\u003c/a> regarding the science and ethics of gene editing. But for the average person on the street, the most direct route to voicing opinions and concerns may be \u003ca href=\"http://www.nationalacademies.org/gene-editing/consensus-study/index.htm\" target=\"_blank\" rel=\"noopener noreferrer\">submitting comments\u003c/a> on the homepage of the National Academies' Human Gene-Editing Initiative. Of course, there is also the tried and true method of \u003ca href=\"https://callyourrep.co/\" target=\"_blank\" rel=\"noopener noreferrer\">calling your representatives\u003c/a>.\u003c/p>\n\n","blocks":[],"excerpt":"Will debating the ethics of gene editing lead to more agreement? Some scientists and policy makers are hoping so. \r\n","status":"publish","parent":0,"modified":1504203620,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":21,"wordCount":959},"headData":{"title":"As Human Gene-Editing Advances, Doudna Says Ethical Discussions Can't Wait | KQED","description":"Will debating the ethics of gene editing lead to more agreement? Some scientists and policy makers are hoping so. \r\n","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"As Human Gene-Editing Advances, Doudna Says Ethical Discussions Can't Wait","datePublished":"2017-08-31T16:30:17.000Z","dateModified":"2017-08-31T18:20:20.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"435096 https://ww2.kqed.org/futureofyou/?p=435096","disqusUrl":"https://ww2.kqed.org/futureofyou/2017/08/31/as-human-gene-editing-advances-doudna-says-ethical-discussions-cant-wait/","disqusTitle":"As Human Gene-Editing Advances, Doudna Says Ethical Discussions Can't Wait","source":"Future of You","path":"/futureofyou/435096/as-human-gene-editing-advances-doudna-says-ethical-discussions-cant-wait","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>If you want to have a role in shaping the near and coming future of biotechnology, the time is now. A science degree is not required, but a sense of urgency is.\u003c/p>\n\u003cp>This was the primary takeaway from Jennifer Doudna's recent public remarks at\u003ca href=\"http://crisprcon.org/\" target=\"_blank\" rel=\"noopener noreferrer\"> CRISPRcon\u003c/a>, a two day event at UC Berkeley, intended to get nonspecialists talking about the promise and potential peril of the fast-moving biotech landscape.\u003c/p>\n\u003cp>\"Decisions about gene editing have an impact on all of us,\" said Michael Krasney, host of KQED's \"Forum\" radio program and the CRISPRcon emcee. \"That's why there are no outsiders in discussing this topic.\" (For a primer on how CRISPR gene editing works, check out our story \"\u003ca href=\"https://ww2.kqed.org/futureofyou/2015/12/30/a-crispr-solution-to-bubble-boy-disease/\" target=\"_blank\" rel=\"noopener noreferrer\">CRISPR: What You Need to Know About the Medical Science ‘Breakthrough of the Year\u003c/a>.\")\u003c/p>\n\u003caside class=\"pullquote alignright\">\"We can no longer say, well there's a lot of technology development to be done before we have to worry about that application, it's now a question of 'We know this can work, are we willing to go there or not?'\" \u003ccite>Jennifer Doudna, one of the inventors of CRISPR/Cas9 gene-editing technology\u003c/cite>\u003c/aside>\n\u003cp>The National Academies of Sciences, Engineering and Medicine wants public input, as well. The rationale is, if it affects the public, the public should have a say.\u003c/p>\n\u003cp>\u003cstrong>What's at Stake\u003c/strong>\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>In her new book, \"\u003ca href=\"http://www.acrackincreation.com/\" target=\"_blank\" rel=\"noopener noreferrer\">A Crack in Creation: Gene Editing and the Unthinkable Power to Control Evolution\u003c/a>,\" Doudna (who co-authored the book with former student Samuel Sternberg) writes humans are \"on the cusp of a new age in genetic engineering and biological mastery.\"\u003c/p>\n\u003cp>\"It won’t be long before CRISPR allows us to bend nature to our will.”\u003c/p>\n\u003cp>CRISPR applications could bring us \u003ca href=\"https://www.sciencedaily.com/releases/2017/05/170518140335.htm\" target=\"_blank\" rel=\"noopener noreferrer\">higher tomato yields\u003c/a> or cattle \u003ca href=\"https://geneticliteracyproject.org/2016/05/11/gene-edited-hornless-cow-improve-animal-welfare-regulatory-fate-unclear/\" target=\"_blank\" rel=\"noopener noreferrer\">born without horns\u003c/a> (allowing livestock to avoid the potentially painful procedure of having them removed). Ongoing medical research using CRISPR includes \u003ca href=\"https://www.nature.com/news/first-crispr-clinical-trial-gets-green-light-from-us-panel-1.20137\" target=\"_blank\" rel=\"noopener noreferrer\">gene editing to target immune response\u003c/a> in cancer patients and to \u003ca href=\"https://www.nature.com/news/crispr-deployed-to-combat-sickle-cell-anaemia-1.20782\" target=\"_blank\" rel=\"noopener noreferrer\">correct sickle-cell anemia\u003c/a>, a painful blood disorder.\u003c/p>\n\u003cp>But the $10 million question at CRISPRcon loomed large:\u003c/p>\n\u003cp>\"How do you decide which genes are appropriate for germline editing?\" asked a woman in the audience, during a question and answer session with Doudna. Germline editing means that the changes made affect sperm or egg cells, and therefore can be inherited.\u003c/p>\n\u003cp>\u003c/p>\u003cp>\u003c/p>\u003cp>\"That's a question that could be debated and discussed for the entire time of the conference,\" said Doudna. But her short answer, referencing a \u003ca href=\"http://www.nationalacademies.org/gene-editing/consensus-study/index.htm\" target=\"_blank\" rel=\"noopener noreferrer\">recent report with recommendations\u003c/a> from the National Academies, was, \"We look for situations where there would be no other reasonable way to deal with a genetic disease other than gene editing. And when you think about it that way, those situations are rather rare.\"\u003c/p>\n\u003cp>Preimplantation genetic diagnosis (for parents using in vitro fertilization) and genetic counseling can offer routes to avoid serious genetic mutations without gene editing. This latter technique would, however, require parents to decide if they are willing to terminate a pregnancy.\u003c/p>\n\u003cp>Doudna believes that in most cases genetic counseling and preimplantation genetic diagnosis are sufficient to deal with disorders caused by a single gene. But some couples might not have any other option to conceive a healthy embryo outside of editing their genes. For example, if both parents both have the same disease-causing gene, \"That's an issue where gene editing could be relevant in the future.\"\u003c/p>\n\u003cp>For any parents who want to select for green-eyed, athletic geniuses, their best bet is still a random roll of the dice, not gene-editing, if the National Academies has anything to do with it. \"Do not proceed at this time with human genome editing for purposes other than treatment or prevention of disease and disability,\" the study recommends.\u003c/p>\n\u003cp>But the question of whether embryos should be edited to avoid disease and disability has reached new urgency, says Doudna, with the publication several weeks ago of the first \u003ca href=\"https://ww2.kqed.org/futureofyou/2017/07/27/scientists-in-us-edit-human-embryos-with-crispr-for-first-time-reports-suggest/\" target=\"_blank\" rel=\"noopener noreferrer\">paper\u003c/a> that lays out a good protocol (a written procedure) for targeting a gene linked to disease. It has the potential of being useful in clinical applications.\u003c/p>\n\u003cp>\"What they showed, importantly,\" said Doudna, \"was that there was very few off-target effects [unintended changes] and also they could avoid something called 'mosaicism'. \" That arises when the desired edit occurs in only some of the cells in the developing embryo. Both edited and non-editing cells replicate, resulting in an organism with a genetic patchwork, which may or may not be harmful.\u003c/p>\n\u003cp>\"We now understand that, when applied in certain ways this technology can be very robust, in viable human embryos. We can no longer say, 'Well, there's a lot of technology development to be done before we have to worry about that application.' It's now a question of, 'We know this can work, are we willing to go there or not?' \"\u003c/p>\n\u003cp>Since CRISPRcon, the paper in question has come in for some \u003ca href=\"https://ipscell.com/2017/08/doubts-raised-on-key-points-of-nature-paper-on-crispr-gene-editing-of-human-embryos/\" target=\"_blank\" rel=\"noopener noreferrer\">recent criticism from scientists\u003c/a> not involved in the research. They call into question the main conclusions of the paper and say more definitive studies are needed. That said, most scientists believe precise editing in embryos will be possible someday, and likely someday soon.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>For members of the public eager to be part of the debate, events like CRISPRcon provide a rare forum with scientists like Doudna who have \u003ca href=\"https://ipscell.com/2015/06/doudnacongress/\" target=\"_blank\" rel=\"noopener noreferrer\">testified before Congress\u003c/a> regarding the science and ethics of gene editing. But for the average person on the street, the most direct route to voicing opinions and concerns may be \u003ca href=\"http://www.nationalacademies.org/gene-editing/consensus-study/index.htm\" target=\"_blank\" rel=\"noopener noreferrer\">submitting comments\u003c/a> on the homepage of the National Academies' Human Gene-Editing Initiative. Of course, there is also the tried and true method of \u003ca href=\"https://callyourrep.co/\" target=\"_blank\" rel=\"noopener noreferrer\">calling your representatives\u003c/a>.\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/435096/as-human-gene-editing-advances-doudna-says-ethical-discussions-cant-wait","authors":["11088"],"categories":["futureofyou_452","futureofyou_1062","futureofyou_1","futureofyou_73"],"tags":["futureofyou_1308","futureofyou_94","futureofyou_1275","futureofyou_295","futureofyou_953"],"featImg":"futureofyou_435099","label":"source_futureofyou_435096"},"futureofyou_434297":{"type":"posts","id":"futureofyou_434297","meta":{"index":"posts_1591205157","site":"futureofyou","id":"434297","score":null,"sort":[1501703229000]},"guestAuthors":[],"slug":"scientists-precisely-edit-dna-in-human-embryos-to-fix-a-disease-gene","title":"Scientists Precisely Edit DNA in Human Embryos to Fix a Disease Gene","publishDate":1501703229,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{"site":"futureofyou"},"content":"\u003cp>Scientists have been tinkering with the DNA in humans and other living things for decades. But one thing has long been considered off-limits: modifying human DNA in any way that could be passed down for generations.\u003c/p>\n\u003cp>Now, an international team of scientists \u003ca href=\"http://nature.com/articles/doi:10.1038/nature23305\">reports\u003c/a> they have, for the first time, figured out a way to successfully edit the DNA in human embryos — without introducing the harmful mutations that were a problem in previous attempts elsewhere. The work was published online Wednesday in the journal \u003cem>Nature.\u003c/em>\u003c/p>\n\u003cp>\"It's a pretty exciting piece of science,\" says \u003ca href=\"https://daley.med.harvard.edu/\">George Daley\u003c/a>, dean of the Harvard Medical School, who was not involved in the research. \"It's a technical tour de force. It's really remarkable.\"\u003c/p>\n\u003cp>[contextly_sidebar id=\"7SHcMEHgm6xBjgR2ZgdXf4oHbyU10mYY\"]The research is ultimately aimed at helping families plagued by genetic diseases. The new experiment used a powerful new gene-editing technique to correct a genetic defect behind a heart disorder that can cause seemingly healthy young people to suddenly die from heart failure.\u003c/p>\n\u003cp>The experiment corrected the defect in nearly two-thirds of several dozen embryos, without causing potentially dangerous mutations elsewhere in the DNA.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>None of the embryos were used to try to create a baby. But if future experiments confirm the techniques are safe and effective, the scientists say the same approach could be used to prevent a long list of inheritable diseases.\u003c/p>\n\u003cp>\"Potentially, we're talking about thousands of genes and thousands of patients,\" says \u003ca href=\"https://www.ohsu.edu/providers/paula-amato/42A6A8A8BA74496A98855052CA32BFAD\">Paula Amato\u003c/a>, an associate professor of obstetrics and gynecology at Oregon Health & Science University in Portland. She was a member of the scientific team from the U.S., China and South Korea.\u003c/p>\n\u003cp>Other diseases that might ultimately benefit from such an approach include \u003ca href=\"https://medlineplus.gov/huntingtonsdisease.html\">Huntington's disease\u003c/a>, \u003ca href=\"https://ghr.nlm.nih.gov/condition/cystic-fibrosis\">cystic fibrosis\u003c/a>, perhaps an inherited form of \u003ca href=\"https://medlineplus.gov/alzheimersdisease.html\">Alzheimer's disease\u003c/a> and cases of breast and ovarian cancer caused by mutations in the \u003ca href=\"https://www.cancer.gov/about-cancer/causes-prevention/genetics/brca-fact-sheet\">BRCA genes\u003c/a>.\u003c/p>\n\u003cp>Nonetheless, the work is setting off alarm bells among critics around the world.\u003c/p>\n\u003cfigure id=\"attachment_434299\" class=\"wp-caption aligncenter\" style=\"max-width: 800px\">\u003cimg class=\"size-medium wp-image-434299\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-800x599.jpg\" alt=\"\" width=\"800\" height=\"599\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-800x599.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-160x120.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-768x575.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-1020x764.jpg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-1180x884.jpg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-960x719.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-240x180.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-375x281.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-520x389.jpg 520w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29.jpg 1773w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003cfigcaption class=\"wp-caption-text\">Shoukhrat Mitalipov, principal investigator for the embryo editing study, directs the Center for Embryonic Cell and Gene Therapy at Oregon Health \u003ccite>(Courtesy of Kristyna Wentz-Graff/OHSU)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>\"I think it's extraordinarily disturbing,\" says \u003ca href=\"https://www.geneticsandsociety.org/article/marcy-darnovsky\">Marcy Darnovsky\u003c/a>, who directs the Center for Genetics and Society, a genetics watchdog group in Berkeley, Calif. \"It's a flagrant disregard of calls for a broad societal consensus in decisions about a really momentous technology that could be used good, but in this case is being used in preparation for an extraordinarily risky application.\"\u003c/p>\n\u003cp>\"If irresponsible scientists are not stopped, the world may soon be presented with a fait accompli of the first [genetically modified] baby,\" says \u003ca href=\"http://www.hgalert.org/topics/hge/threat.htm\">David King\u003c/a>, who heads the U.K-based group Human Genetics Alert. \"We call on governments and international organizations to wake up and pass an immediate global ban on creating cloned or GM babies, before it is too late.\"\u003c/p>\n\u003cp>Amato and others stress that their work is aimed at preventing terrible diseases, not creating genetically enhanced people. And they note that much more research is needed to confirm the technique is safe and effective before anyone tries to make a baby this way.\u003c/p>\n\u003cp>But scientists hoping to continue the work in the U.S. face many regulatory obstacles. The National Institutes of Health will not fund any research involving human embryos (the new work was funded by Oregon Health & Science University). And the Food and Drug Administration is prohibited by Congress from considering any experiments that involve genetically modified human embryos.\u003c/p>\n\u003caside class=\"pullquote alignright\">'This is a strong statement that we can do genome editing. The question that remains is: \u003cem>Should we?\u003c/em>'\u003ccite>George Daley, dean of the Harvard Medical School\u003c/cite>\u003c/aside>\n\u003cp>Nevertheless, the researchers say they're hopeful about continuing the work, perhaps in Britain. The United Kingdom has \u003ca href=\"http://www.npr.org/2016/02/01/465180953/british-scientists-gain-approval-to-edit-dna-in-human-embryos\">permitted\u003c/a> \u003ca href=\"http://www.npr.org/2015/02/03/383578221/u-k-lawmakers-allow-scientists-to-attempt-dna-transplants\">genetic experiments \u003c/a>involving human embryos forbidden in the United States.\u003c/p>\n\u003cp>\"If other countries would be interested, we would be happy to work with their regulatory bodies,\" says \u003ca href=\"http://www.ohsu.edu/xd/research/centers-institutes/embryonic-cell-gene-therapy-center/people.cfm\">Shoukhrat Mitalipov\u003c/a>, director of the Oregon Health & Science University's Center for Embryonic Cell and Gene Therapy.\u003c/p>\n\u003cp>One major concern is safety to a developing embryo — whether genetically modified human embryos would indeed produce healthy babies. But on a broader level, any changes made in the DNA of an embryo would be passed down for generations. That raises fears that any mistakes in the editing that inadvertently caused new diseases could become a permanent part of that family's genetic blueprint.\u003c/p>\n\u003cp>Darnovsky and others also worry that modifying human DNA in an embryo could give rise to \"designer babies.\" That's when parents pick and choose the traits of their children to try to make them smarter, taller, stronger or have other traits that make them seem superior. That's not yet technically possible. But critics fear scientists are already moving in that direction.\u003c/p>\n\u003cp>\"The scenario is that you would have fertility clinics advertising to people who wanted to engineer their future children so that they could be presented as 'enhanced' — as biologically better than everyone else,\" Darnovsky says. \"It's not a world we want to build. It's not a world we want to live in.\"\u003c/p>\n\u003cp>Unscrupulous researchers could also rush the technology into fertility clinics to try to start creating babies they can bill as genetically enhanced before the technology has even been proved safe, and before a societal consensus has been reached about what applications should be permitted.\u003c/p>\n\u003cp>\"This is a strong statement that we can do genome editing,\" says Harvard's Daley. \"The question that remains is: 'Should we?' We need a deeper public discourse around the ethical implications of this technology.\"\u003c/p>\n\u003cp>Darnovsky and some scientists argue that many couples who carry genetic diseases already have safer alternatives to this sort of gene editing. Couples carrying genetic diseases can go through in vitro fertilization (\u003ca href=\"http://www.sart.org/SART_Success_Rates/\">IVF\u003c/a>) and have their embryos tested before being implanted in the womb.\u003c/p>\n\u003cp>\"I will admit to experiencing a sense of puzzlement,\" says \u003ca href=\"http://www.altius.org/#organization\">Fyodor Urnov\u003c/a>, an associate director at the Altius Institute for Biomedical Sciences, a nonprofit research institute in Seattle.\u003c/p>\n\u003cp>\"The question I have is: 'Why did you folks bother, given that there is a safe, effective, approved and ethical way to attain exactly the goal you have set out to do without any of the significant logical and ethic hurdles of having to edit a human embryo?\" Urnov says.\u003c/p>\n\u003cp>Amato and the other scientists on the international team say their approach could offer an alternative for couples for whom those standard options won't work or are less desirable. But they agree the work should only move forward with careful regulatory oversight to prevent abuse.\u003c/p>\n\u003cp>\"Anytime there's a new technology there's a potential for misuse. We have to acknowledge that,\" Amato says. \"Personally I don't feel that's a reason not to pursue the research if you think there's a potential benefit that outweighs that risk. And I think if you can prevent serious disease in future generations, that makes it worthwhile to pursue this.\"\u003c/p>\n\u003cp>The advance was first \u003ca href=\"https://www.technologyreview.com/s/608350/first-human-embryos-edited-in-us/\">reported\u003c/a> last week in \u003cem>Technology Review\u003c/em>, a magazine published by the Massachusetts Institute of Technology. But the details were withheld and the researchers did not elaborate until the scientific paper had finished being vetted by other scientists for publication in \u003cem>Nature.\u003c/em>\u003c/p>\n\u003cp>For their experiments, the scientists obtained sperm from a donor carrying a mutation for the heart disorder \u003ca href=\"http://www.heart.org/HEARTORG/Conditions/More/Cardiomyopathy/What-Is-Cardiomyopathy-in-Adults_UCM_444168_Article.jsp#.WYESnoTyvIU\">cardiomyopathy\u003c/a>. They then used that sperm to fertilize dozens of eggs obtained from healthy women.\u003c/p>\n\u003cp>At the same time as fertilization, the researchers injected a powerful, microscopic gene-editing tool known as \u003ca href=\"http://www.npr.org/tags/419142387/crispr\">CRISPR-Cas9\u003c/a>. The new technique makes it much easier than previous approaches to make very precise changes in DNA.\u003c/p>\n\u003cp>Several scientists likened the approach to doing surgery on fetuses when they are in the womb. But this takes that idea much further and involves repairing damaged DNA at a molecular level in the womb.\u003c/p>\n\u003cp>\"This is nano-surgery,\" says \u003ca href=\"http://arep.med.harvard.edu/gmc/\">George Church\u003c/a>, a prominent Harvard geneticist who also was not involved in the research. \"You're doing it with the finest possible scalpel.\"\u003c/p>\n\u003cp>The editing tool very accurately cut into a mutated gene known as MYBPC3, which causes cardiomyopathy. To the researchers' surprise, the cut triggered the embryos to repair the defective gene on their own. This is a process that had previously been unknown, the scientists say.\u003c/p>\n\u003cp>\"The most exciting moment was when we realized the mechanism of repair,\" Amato says. \"It was fixing itself.\"\u003c/p>\n\u003cp>The researchers then let the embryos develop for several days so they could analyze them to see how well the experiments worked. In one part of the experiments involving 58 embryos, the approach corrected the mutation in more than 70 percent of the embryos, the researchers reported.\u003c/p>\n\u003cp>\"The gene defect was corrected with high efficiency,\" Amato says.\u003c/p>\n\u003cp>In addition, a detailed genetic analysis of the embryos concluded that the gene editing had not caused safety problems.\u003c/p>\n\u003cp>\"I think this is a significant advance,\" Church says. \"This is important.\"\u003c/p>\n\u003cp>In 2015, Chinese scientists \u003ca href=\"http://www.npr.org/sections/health-shots/2015/04/23/401655818/critics-lash-out-at-chinese-scientists-who-edited-dna-in-human-embryos\">reported\u003c/a> trying to edit the DNA of embryos for the first time, also using CRISPR-Cas9. But that experiment involved embryos that could never develop normally. And while those researchers did succeed in editing the targeted defect, it also produced unintended defects elsewhere in the embryos' DNA.\u003c/p>\n\u003cp>The scientists who conducted the new experiments say they think they avoided those problems by injecting CRISPR at the same time the eggs were being fertilized by sperm.\u003c/p>\n\u003cp>\"That was key,\" Mitalipov says.\u003c/p>\n\u003cp>\u003ca href=\"https://law.wisc.edu/profiles/racharo@wisc.edu\">Alta Charo\u003c/a>, a bioethicist at the University of Wisconsin, dismissed concerns about the work leading to designer babies.\u003c/p>\n\u003cp>\"This is not the dawn of the era of the designer baby,\" says Charo, who co-chaired a committee formed by the National Academies of Sciences and the National Academy of Medicine to determine whether such experiments should be permissible. The committee \u003ca href=\"http://www.npr.org/sections/health-shots/2017/02/14/514580162/scientific-panel-says-editing-heritable-human-genes-could-be-ok-in-the-future\">concluded\u003c/a> earlier this year that gene editing of human embryos could be allowed in rare cases when no other options are available — but only to treat diseases.\u003c/p>\n\u003cp>\"I do not think that the constant drumbeat about the fear of designer baby is warranted, Charo says. \"What this is, is a possible step toward being able to edit the DNA in human embryos that's reliable and precise.\"\u003c/p>\n\u003cp>In the meantime, scientists in Britain have won \u003ca href=\"http://www.npr.org/2016/02/01/465180953/british-scientists-gain-approval-to-edit-dna-in-human-embryos\">approval \u003c/a>to use CRISPR to edit the DNA in healthy human embryos to learn more about normal human development. A \u003ca href=\"http://www.npr.org/about-npr/494863809/npr-exclusive-report-stockholm-lab-first-to-try-to-edit-dna-of-healthy-human-emb\">team in Sweden\u003c/a> has started similar experiments.\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>\"I think this needs to be tightly regulated,\" says \u003ca href=\"http://ki.se/en/people/frelan\">Fredrik Lanner\u003c/a>, a geneticist at the Karolinska Institute in Stockholm who is conducting those experiments. \"This is very exciting. But it also could be a double-edged sword. So I think we really have to be extra cautious with this technology.\"\u003c/p>\n\u003cdiv class=\"fullattribution\">Copyright 2017 NPR. To see more, visit http://www.npr.org/.\u003cimg src=\"https://www.google-analytics.com/__utm.gif?utmac=UA-5828686-4&utmdt=Scientists+Precisely+Edit+DNA+In+Human+Embryos+To+Fix+A+Disease+Gene&utme=8(APIKey)9(MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004)\">\u003c/div>\n\n","blocks":[],"excerpt":"In experimental embryos, scientists were able to repair the gene that causes a serious heart disorder. More research is needed to confirm the method would produce healthy babies, they say.","status":"publish","parent":0,"modified":1501711262,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":46,"wordCount":1804},"headData":{"title":"Scientists Precisely Edit DNA in Human Embryos to Fix a Disease Gene | KQED","description":"In experimental embryos, scientists were able to repair the gene that causes a serious heart disorder. More research is needed to confirm the method would produce healthy babies, they say.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Scientists Precisely Edit DNA in Human Embryos to Fix a Disease Gene","datePublished":"2017-08-02T19:47:09.000Z","dateModified":"2017-08-02T22:01:02.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"434297 https://ww2.kqed.org/futureofyou/?p=434297","disqusUrl":"https://ww2.kqed.org/futureofyou/2017/08/02/scientists-precisely-edit-dna-in-human-embryos-to-fix-a-disease-gene/","disqusTitle":"Scientists Precisely Edit DNA in Human Embryos to Fix a Disease Gene","nprByline":"Rob Stein\u003c/br>NPR","nprImageAgency":"Courtesy of OHSU","nprStoryId":"540975224","nprApiLink":"http://api.npr.org/query?id=540975224&apiKey=MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004","nprHtmlLink":"http://www.npr.org/sections/health-shots/2017/08/02/540975224/scientists-precisely-edit-dna-in-human-embryos-to-fix-a-disease-gene?ft=nprml&f=540975224","nprRetrievedStory":"1","nprPubDate":"Wed, 02 Aug 2017 14:53:00 -0400","nprStoryDate":"Wed, 02 Aug 2017 13:09:00 -0400","nprLastModifiedDate":"Wed, 02 Aug 2017 14:53:14 -0400","path":"/futureofyou/434297/scientists-precisely-edit-dna-in-human-embryos-to-fix-a-disease-gene","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Scientists have been tinkering with the DNA in humans and other living things for decades. But one thing has long been considered off-limits: modifying human DNA in any way that could be passed down for generations.\u003c/p>\n\u003cp>Now, an international team of scientists \u003ca href=\"http://nature.com/articles/doi:10.1038/nature23305\">reports\u003c/a> they have, for the first time, figured out a way to successfully edit the DNA in human embryos — without introducing the harmful mutations that were a problem in previous attempts elsewhere. The work was published online Wednesday in the journal \u003cem>Nature.\u003c/em>\u003c/p>\n\u003cp>\"It's a pretty exciting piece of science,\" says \u003ca href=\"https://daley.med.harvard.edu/\">George Daley\u003c/a>, dean of the Harvard Medical School, who was not involved in the research. \"It's a technical tour de force. It's really remarkable.\"\u003c/p>\n\u003cp>\u003c/p>\u003cp>\u003c/p>\u003cp>The research is ultimately aimed at helping families plagued by genetic diseases. The new experiment used a powerful new gene-editing technique to correct a genetic defect behind a heart disorder that can cause seemingly healthy young people to suddenly die from heart failure.\u003c/p>\n\u003cp>The experiment corrected the defect in nearly two-thirds of several dozen embryos, without causing potentially dangerous mutations elsewhere in the DNA.\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 embryos were used to try to create a baby. But if future experiments confirm the techniques are safe and effective, the scientists say the same approach could be used to prevent a long list of inheritable diseases.\u003c/p>\n\u003cp>\"Potentially, we're talking about thousands of genes and thousands of patients,\" says \u003ca href=\"https://www.ohsu.edu/providers/paula-amato/42A6A8A8BA74496A98855052CA32BFAD\">Paula Amato\u003c/a>, an associate professor of obstetrics and gynecology at Oregon Health & Science University in Portland. She was a member of the scientific team from the U.S., China and South Korea.\u003c/p>\n\u003cp>Other diseases that might ultimately benefit from such an approach include \u003ca href=\"https://medlineplus.gov/huntingtonsdisease.html\">Huntington's disease\u003c/a>, \u003ca href=\"https://ghr.nlm.nih.gov/condition/cystic-fibrosis\">cystic fibrosis\u003c/a>, perhaps an inherited form of \u003ca href=\"https://medlineplus.gov/alzheimersdisease.html\">Alzheimer's disease\u003c/a> and cases of breast and ovarian cancer caused by mutations in the \u003ca href=\"https://www.cancer.gov/about-cancer/causes-prevention/genetics/brca-fact-sheet\">BRCA genes\u003c/a>.\u003c/p>\n\u003cp>Nonetheless, the work is setting off alarm bells among critics around the world.\u003c/p>\n\u003cfigure id=\"attachment_434299\" class=\"wp-caption aligncenter\" style=\"max-width: 800px\">\u003cimg class=\"size-medium wp-image-434299\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-800x599.jpg\" alt=\"\" width=\"800\" height=\"599\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-800x599.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-160x120.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-768x575.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-1020x764.jpg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-1180x884.jpg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-960x719.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-240x180.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-375x281.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29-520x389.jpg 520w, https://ww2.kqed.org/app/uploads/sites/13/2017/08/mitalia-1_custom-57c3c6b2b7ed2aac34d5c34fba0ea89317aa2d29.jpg 1773w\" sizes=\"(max-width: 800px) 100vw, 800px\">\u003cfigcaption class=\"wp-caption-text\">Shoukhrat Mitalipov, principal investigator for the embryo editing study, directs the Center for Embryonic Cell and Gene Therapy at Oregon Health \u003ccite>(Courtesy of Kristyna Wentz-Graff/OHSU)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>\"I think it's extraordinarily disturbing,\" says \u003ca href=\"https://www.geneticsandsociety.org/article/marcy-darnovsky\">Marcy Darnovsky\u003c/a>, who directs the Center for Genetics and Society, a genetics watchdog group in Berkeley, Calif. \"It's a flagrant disregard of calls for a broad societal consensus in decisions about a really momentous technology that could be used good, but in this case is being used in preparation for an extraordinarily risky application.\"\u003c/p>\n\u003cp>\"If irresponsible scientists are not stopped, the world may soon be presented with a fait accompli of the first [genetically modified] baby,\" says \u003ca href=\"http://www.hgalert.org/topics/hge/threat.htm\">David King\u003c/a>, who heads the U.K-based group Human Genetics Alert. \"We call on governments and international organizations to wake up and pass an immediate global ban on creating cloned or GM babies, before it is too late.\"\u003c/p>\n\u003cp>Amato and others stress that their work is aimed at preventing terrible diseases, not creating genetically enhanced people. And they note that much more research is needed to confirm the technique is safe and effective before anyone tries to make a baby this way.\u003c/p>\n\u003cp>But scientists hoping to continue the work in the U.S. face many regulatory obstacles. The National Institutes of Health will not fund any research involving human embryos (the new work was funded by Oregon Health & Science University). And the Food and Drug Administration is prohibited by Congress from considering any experiments that involve genetically modified human embryos.\u003c/p>\n\u003caside class=\"pullquote alignright\">'This is a strong statement that we can do genome editing. The question that remains is: \u003cem>Should we?\u003c/em>'\u003ccite>George Daley, dean of the Harvard Medical School\u003c/cite>\u003c/aside>\n\u003cp>Nevertheless, the researchers say they're hopeful about continuing the work, perhaps in Britain. The United Kingdom has \u003ca href=\"http://www.npr.org/2016/02/01/465180953/british-scientists-gain-approval-to-edit-dna-in-human-embryos\">permitted\u003c/a> \u003ca href=\"http://www.npr.org/2015/02/03/383578221/u-k-lawmakers-allow-scientists-to-attempt-dna-transplants\">genetic experiments \u003c/a>involving human embryos forbidden in the United States.\u003c/p>\n\u003cp>\"If other countries would be interested, we would be happy to work with their regulatory bodies,\" says \u003ca href=\"http://www.ohsu.edu/xd/research/centers-institutes/embryonic-cell-gene-therapy-center/people.cfm\">Shoukhrat Mitalipov\u003c/a>, director of the Oregon Health & Science University's Center for Embryonic Cell and Gene Therapy.\u003c/p>\n\u003cp>One major concern is safety to a developing embryo — whether genetically modified human embryos would indeed produce healthy babies. But on a broader level, any changes made in the DNA of an embryo would be passed down for generations. That raises fears that any mistakes in the editing that inadvertently caused new diseases could become a permanent part of that family's genetic blueprint.\u003c/p>\n\u003cp>Darnovsky and others also worry that modifying human DNA in an embryo could give rise to \"designer babies.\" That's when parents pick and choose the traits of their children to try to make them smarter, taller, stronger or have other traits that make them seem superior. That's not yet technically possible. But critics fear scientists are already moving in that direction.\u003c/p>\n\u003cp>\"The scenario is that you would have fertility clinics advertising to people who wanted to engineer their future children so that they could be presented as 'enhanced' — as biologically better than everyone else,\" Darnovsky says. \"It's not a world we want to build. It's not a world we want to live in.\"\u003c/p>\n\u003cp>Unscrupulous researchers could also rush the technology into fertility clinics to try to start creating babies they can bill as genetically enhanced before the technology has even been proved safe, and before a societal consensus has been reached about what applications should be permitted.\u003c/p>\n\u003cp>\"This is a strong statement that we can do genome editing,\" says Harvard's Daley. \"The question that remains is: 'Should we?' We need a deeper public discourse around the ethical implications of this technology.\"\u003c/p>\n\u003cp>Darnovsky and some scientists argue that many couples who carry genetic diseases already have safer alternatives to this sort of gene editing. Couples carrying genetic diseases can go through in vitro fertilization (\u003ca href=\"http://www.sart.org/SART_Success_Rates/\">IVF\u003c/a>) and have their embryos tested before being implanted in the womb.\u003c/p>\n\u003cp>\"I will admit to experiencing a sense of puzzlement,\" says \u003ca href=\"http://www.altius.org/#organization\">Fyodor Urnov\u003c/a>, an associate director at the Altius Institute for Biomedical Sciences, a nonprofit research institute in Seattle.\u003c/p>\n\u003cp>\"The question I have is: 'Why did you folks bother, given that there is a safe, effective, approved and ethical way to attain exactly the goal you have set out to do without any of the significant logical and ethic hurdles of having to edit a human embryo?\" Urnov says.\u003c/p>\n\u003cp>Amato and the other scientists on the international team say their approach could offer an alternative for couples for whom those standard options won't work or are less desirable. But they agree the work should only move forward with careful regulatory oversight to prevent abuse.\u003c/p>\n\u003cp>\"Anytime there's a new technology there's a potential for misuse. We have to acknowledge that,\" Amato says. \"Personally I don't feel that's a reason not to pursue the research if you think there's a potential benefit that outweighs that risk. And I think if you can prevent serious disease in future generations, that makes it worthwhile to pursue this.\"\u003c/p>\n\u003cp>The advance was first \u003ca href=\"https://www.technologyreview.com/s/608350/first-human-embryos-edited-in-us/\">reported\u003c/a> last week in \u003cem>Technology Review\u003c/em>, a magazine published by the Massachusetts Institute of Technology. But the details were withheld and the researchers did not elaborate until the scientific paper had finished being vetted by other scientists for publication in \u003cem>Nature.\u003c/em>\u003c/p>\n\u003cp>For their experiments, the scientists obtained sperm from a donor carrying a mutation for the heart disorder \u003ca href=\"http://www.heart.org/HEARTORG/Conditions/More/Cardiomyopathy/What-Is-Cardiomyopathy-in-Adults_UCM_444168_Article.jsp#.WYESnoTyvIU\">cardiomyopathy\u003c/a>. They then used that sperm to fertilize dozens of eggs obtained from healthy women.\u003c/p>\n\u003cp>At the same time as fertilization, the researchers injected a powerful, microscopic gene-editing tool known as \u003ca href=\"http://www.npr.org/tags/419142387/crispr\">CRISPR-Cas9\u003c/a>. The new technique makes it much easier than previous approaches to make very precise changes in DNA.\u003c/p>\n\u003cp>Several scientists likened the approach to doing surgery on fetuses when they are in the womb. But this takes that idea much further and involves repairing damaged DNA at a molecular level in the womb.\u003c/p>\n\u003cp>\"This is nano-surgery,\" says \u003ca href=\"http://arep.med.harvard.edu/gmc/\">George Church\u003c/a>, a prominent Harvard geneticist who also was not involved in the research. \"You're doing it with the finest possible scalpel.\"\u003c/p>\n\u003cp>The editing tool very accurately cut into a mutated gene known as MYBPC3, which causes cardiomyopathy. To the researchers' surprise, the cut triggered the embryos to repair the defective gene on their own. This is a process that had previously been unknown, the scientists say.\u003c/p>\n\u003cp>\"The most exciting moment was when we realized the mechanism of repair,\" Amato says. \"It was fixing itself.\"\u003c/p>\n\u003cp>The researchers then let the embryos develop for several days so they could analyze them to see how well the experiments worked. In one part of the experiments involving 58 embryos, the approach corrected the mutation in more than 70 percent of the embryos, the researchers reported.\u003c/p>\n\u003cp>\"The gene defect was corrected with high efficiency,\" Amato says.\u003c/p>\n\u003cp>In addition, a detailed genetic analysis of the embryos concluded that the gene editing had not caused safety problems.\u003c/p>\n\u003cp>\"I think this is a significant advance,\" Church says. \"This is important.\"\u003c/p>\n\u003cp>In 2015, Chinese scientists \u003ca href=\"http://www.npr.org/sections/health-shots/2015/04/23/401655818/critics-lash-out-at-chinese-scientists-who-edited-dna-in-human-embryos\">reported\u003c/a> trying to edit the DNA of embryos for the first time, also using CRISPR-Cas9. But that experiment involved embryos that could never develop normally. And while those researchers did succeed in editing the targeted defect, it also produced unintended defects elsewhere in the embryos' DNA.\u003c/p>\n\u003cp>The scientists who conducted the new experiments say they think they avoided those problems by injecting CRISPR at the same time the eggs were being fertilized by sperm.\u003c/p>\n\u003cp>\"That was key,\" Mitalipov says.\u003c/p>\n\u003cp>\u003ca href=\"https://law.wisc.edu/profiles/racharo@wisc.edu\">Alta Charo\u003c/a>, a bioethicist at the University of Wisconsin, dismissed concerns about the work leading to designer babies.\u003c/p>\n\u003cp>\"This is not the dawn of the era of the designer baby,\" says Charo, who co-chaired a committee formed by the National Academies of Sciences and the National Academy of Medicine to determine whether such experiments should be permissible. The committee \u003ca href=\"http://www.npr.org/sections/health-shots/2017/02/14/514580162/scientific-panel-says-editing-heritable-human-genes-could-be-ok-in-the-future\">concluded\u003c/a> earlier this year that gene editing of human embryos could be allowed in rare cases when no other options are available — but only to treat diseases.\u003c/p>\n\u003cp>\"I do not think that the constant drumbeat about the fear of designer baby is warranted, Charo says. \"What this is, is a possible step toward being able to edit the DNA in human embryos that's reliable and precise.\"\u003c/p>\n\u003cp>In the meantime, scientists in Britain have won \u003ca href=\"http://www.npr.org/2016/02/01/465180953/british-scientists-gain-approval-to-edit-dna-in-human-embryos\">approval \u003c/a>to use CRISPR to edit the DNA in healthy human embryos to learn more about normal human development. A \u003ca href=\"http://www.npr.org/about-npr/494863809/npr-exclusive-report-stockholm-lab-first-to-try-to-edit-dna-of-healthy-human-emb\">team in Sweden\u003c/a> has started similar experiments.\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>\"I think this needs to be tightly regulated,\" says \u003ca href=\"http://ki.se/en/people/frelan\">Fredrik Lanner\u003c/a>, a geneticist at the Karolinska Institute in Stockholm who is conducting those experiments. \"This is very exciting. But it also could be a double-edged sword. So I think we really have to be extra cautious with this technology.\"\u003c/p>\n\u003cdiv class=\"fullattribution\">Copyright 2017 NPR. To see more, visit http://www.npr.org/.\u003cimg src=\"https://www.google-analytics.com/__utm.gif?utmac=UA-5828686-4&utmdt=Scientists+Precisely+Edit+DNA+In+Human+Embryos+To+Fix+A+Disease+Gene&utme=8(APIKey)9(MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004)\">\u003c/div>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/434297/scientists-precisely-edit-dna-in-human-embryos-to-fix-a-disease-gene","authors":["byline_futureofyou_434297"],"categories":["futureofyou_1"],"tags":["futureofyou_94","futureofyou_1275","futureofyou_295"],"featImg":"futureofyou_434298","label":"futureofyou"},"futureofyou_432213":{"type":"posts","id":"futureofyou_432213","meta":{"index":"posts_1591205157","site":"futureofyou","id":"432213","score":null,"sort":[1501185602000]},"guestAuthors":[],"slug":"scientists-in-us-edit-human-embryos-with-crispr-for-first-time-reports-suggest","title":"Scientists in US Edit Human Embryos With CRISPR for First Time","publishDate":1501185602,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{"site":"futureofyou"},"content":"\u003cp>In a step that some of the nation’s leading scientists have long warned against and that has never before been accomplished, biologists in Oregon have edited the DNA of viable human embryos efficiently and apparently with few mistakes, according to a \u003ca href=\"https://www.technologyreview.com/s/608350/first-human-embryos-edited-in-us/\" target=\"_blank\" rel=\"noopener noreferrer\">report\u003c/a> in Technology Review.\u003c/p>\n\u003cp>The experiment, using the revolutionary genome-editing technique \u003ca href=\"https://www.statnews.com/2017/06/11/crispr-jennifer-doudna-book/\" target=\"_blank\" rel=\"noopener noreferrer\">CRISPR-Cas9\u003c/a>, was led by \u003ca href=\"https://www.statnews.com/2016/07/08/fertility-gene-therapy-mitalipov/\" target=\"_blank\" rel=\"noopener noreferrer\">Shoukhrat Mitalipov\u003c/a> of Oregon Health & Science University. It went beyond previous experiments using CRISPR to alter the DNA of human embryos, all of which were conducted in China, in that it edited the genomes of many more embryos and targeted a gene associated with a significant human disease.\u003c/p>\n\u003cp>“This is the kind of research that is essential if we are to know if it’s possible to safely and precisely make corrections” in embryos’ DNA to repair disease-causing genes,” legal scholar and bioethicist R. Alta Charo of the University of Wisconsin, Madison, told STAT. “While there will be time for the public to decide if they want to get rid of regulatory obstacles to these studies, I do not find them inherently unethical.” Those regulatory barriers include a \u003ca href=\"https://www.nih.gov/about-nih/who-we-are/nih-director/statements/statement-nih-funding-research-using-gene-editing-technologies-human-embryos\">ban\u003c/a> on using National Institutes of Health funding for experiments that use genome-editing technologies in human embryos.\u003c/p>\n\u003cp>The \u003ca href=\"https://link.springer.com/article/10.1007%2Fs13238-015-0153-5\">first \u003c/a>experiment using CRISPR to alter the DNA of human embryos, in 2015, used embryos obtained from fertility clinics that had such serious genetic defects they could never have developed. In the new work, Technology Review reported, Mitalipov and his colleagues created human embryos using sperm donated by men with the genetic mutation that they planned to try to repair with CRISPR. The embryos are described as “clinical quality.” A 2017 \u003ca href=\"https://link.springer.com/article/10.1007%2Fs00438-017-1299-z\">experiment\u003c/a>, also in China, used CRISPR to edit DNA in normal, presumably viable fertilized eggs, or one-cell human embryos.\u003c/p>\n\u003cp>Also in contrast to the experiments in China, those led by Mitalipov reportedly produced very few “\u003ca href=\"https://www.statnews.com/2016/07/18/crispr-off-target-effects/\" target=\"_blank\" rel=\"noopener noreferrer\">off-target\u003c/a>” effects, or editing of genes that CRISPR was supposed to leave alone. And the experiment avoided what is called “mosaicism,” in which only some cells of an embryo have the intended DNA changes. The embryos were not allowed to develop beyond a very early stage.\u003c/p>\n\u003caside class=\"pullquote alignright\">If the embryo is born and grows to adulthood, any children he or she has will inherit the genetic alteration. That has led to fears that such manipulations could alter the course of human evolution.\u003c/aside>\n\u003cp>Because changing the DNA of an early embryo results in changes to cells that will eventually produce sperm and eggs, if the embryo is born and grows to adulthood, any children he or she has will inherit the genetic alteration, which is called germline editing. That has led to fears that such manipulations could alter the course of human evolution.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>It has also triggered warnings about “designer babies,” in which parents customize their IVF embryos by adding, removing, or changing genes for certain traits.\u003c/p>\n\u003cp>A recent \u003ca href=\"https://www.statnews.com/2017/02/14/national-academy-crispr-report/\">report\u003c/a> on genome editing from the National Academies did not call for a moratorium on research into germline editing, arguing that it might one day be a way for some parents to have healthy, biological children, such as when both mother and father carry genetic mutations that cause severe diseases.\u003c/p>\n\u003cp>“But we anticipated that there would need to be a lot of research to see if you could make these changes without any unintentional effects,”said Charo, who co-chaired the Academies committee. Mitalipov, who did not respond to requests for comment, has now shown that the answer to that might be yes.\u003c/p>\n\u003cp>Some scholars questioned how important the new study is, however. Stanford University law professor and bioethicist Hank Greely \u003ca href=\"https://twitter.com/HankGreelyLSJU/status/890370419651366912\">tweeted\u003c/a> that “the key point” is that no one has tried to implant any edited embryos. “Research embryos” that are “not to be transferred for possible implantation” are “not a big deal,” he \u003ca href=\"https://twitter.com/HankGreelyLSJU/status/890371791947833344\">argued\u003c/a>.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>\u003cem>This \u003ca href=\"https://www.statnews.com/2017/07/26/human-embryos-edited/\" target=\"_blank\" rel=\"noopener noreferrer\">story\u003c/a> has been updated with additional comments by experts and details of similar experiments. It \u003c/em>\u003cem>was originally published by STAT, an online publication of Boston Globe Media that covers health, medicine and scientific discovery.\u003c/em>\u003c/p>\n\n","blocks":[],"excerpt":"Researchers have altered DNA in human embryos for the first time in the U.S. and made few errors, reports suggest. \r\n","status":"publish","parent":0,"modified":1501191109,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":13,"wordCount":716},"headData":{"title":"Scientists in US Edit Human Embryos With CRISPR for First Time | KQED","description":"Researchers have altered DNA in human embryos for the first time in the U.S. and made few errors, reports suggest. \r\n","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Scientists in US Edit Human Embryos With CRISPR for First Time","datePublished":"2017-07-27T20:00:02.000Z","dateModified":"2017-07-27T21:31:49.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"432213 https://ww2.kqed.org/futureofyou/?p=432213","disqusUrl":"https://ww2.kqed.org/futureofyou/2017/07/27/scientists-in-us-edit-human-embryos-with-crispr-for-first-time-reports-suggest/","disqusTitle":"Scientists in US Edit Human Embryos With CRISPR for First Time","nprByline":"Sharon Begley\u003c/br>STAT","path":"/futureofyou/432213/scientists-in-us-edit-human-embryos-with-crispr-for-first-time-reports-suggest","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>In a step that some of the nation’s leading scientists have long warned against and that has never before been accomplished, biologists in Oregon have edited the DNA of viable human embryos efficiently and apparently with few mistakes, according to a \u003ca href=\"https://www.technologyreview.com/s/608350/first-human-embryos-edited-in-us/\" target=\"_blank\" rel=\"noopener noreferrer\">report\u003c/a> in Technology Review.\u003c/p>\n\u003cp>The experiment, using the revolutionary genome-editing technique \u003ca href=\"https://www.statnews.com/2017/06/11/crispr-jennifer-doudna-book/\" target=\"_blank\" rel=\"noopener noreferrer\">CRISPR-Cas9\u003c/a>, was led by \u003ca href=\"https://www.statnews.com/2016/07/08/fertility-gene-therapy-mitalipov/\" target=\"_blank\" rel=\"noopener noreferrer\">Shoukhrat Mitalipov\u003c/a> of Oregon Health & Science University. It went beyond previous experiments using CRISPR to alter the DNA of human embryos, all of which were conducted in China, in that it edited the genomes of many more embryos and targeted a gene associated with a significant human disease.\u003c/p>\n\u003cp>“This is the kind of research that is essential if we are to know if it’s possible to safely and precisely make corrections” in embryos’ DNA to repair disease-causing genes,” legal scholar and bioethicist R. Alta Charo of the University of Wisconsin, Madison, told STAT. “While there will be time for the public to decide if they want to get rid of regulatory obstacles to these studies, I do not find them inherently unethical.” Those regulatory barriers include a \u003ca href=\"https://www.nih.gov/about-nih/who-we-are/nih-director/statements/statement-nih-funding-research-using-gene-editing-technologies-human-embryos\">ban\u003c/a> on using National Institutes of Health funding for experiments that use genome-editing technologies in human embryos.\u003c/p>\n\u003cp>The \u003ca href=\"https://link.springer.com/article/10.1007%2Fs13238-015-0153-5\">first \u003c/a>experiment using CRISPR to alter the DNA of human embryos, in 2015, used embryos obtained from fertility clinics that had such serious genetic defects they could never have developed. In the new work, Technology Review reported, Mitalipov and his colleagues created human embryos using sperm donated by men with the genetic mutation that they planned to try to repair with CRISPR. The embryos are described as “clinical quality.” A 2017 \u003ca href=\"https://link.springer.com/article/10.1007%2Fs00438-017-1299-z\">experiment\u003c/a>, also in China, used CRISPR to edit DNA in normal, presumably viable fertilized eggs, or one-cell human embryos.\u003c/p>\n\u003cp>Also in contrast to the experiments in China, those led by Mitalipov reportedly produced very few “\u003ca href=\"https://www.statnews.com/2016/07/18/crispr-off-target-effects/\" target=\"_blank\" rel=\"noopener noreferrer\">off-target\u003c/a>” effects, or editing of genes that CRISPR was supposed to leave alone. And the experiment avoided what is called “mosaicism,” in which only some cells of an embryo have the intended DNA changes. The embryos were not allowed to develop beyond a very early stage.\u003c/p>\n\u003caside class=\"pullquote alignright\">If the embryo is born and grows to adulthood, any children he or she has will inherit the genetic alteration. That has led to fears that such manipulations could alter the course of human evolution.\u003c/aside>\n\u003cp>Because changing the DNA of an early embryo results in changes to cells that will eventually produce sperm and eggs, if the embryo is born and grows to adulthood, any children he or she has will inherit the genetic alteration, which is called germline editing. That has led to fears that such manipulations could alter the course of human evolution.\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>It has also triggered warnings about “designer babies,” in which parents customize their IVF embryos by adding, removing, or changing genes for certain traits.\u003c/p>\n\u003cp>A recent \u003ca href=\"https://www.statnews.com/2017/02/14/national-academy-crispr-report/\">report\u003c/a> on genome editing from the National Academies did not call for a moratorium on research into germline editing, arguing that it might one day be a way for some parents to have healthy, biological children, such as when both mother and father carry genetic mutations that cause severe diseases.\u003c/p>\n\u003cp>“But we anticipated that there would need to be a lot of research to see if you could make these changes without any unintentional effects,”said Charo, who co-chaired the Academies committee. Mitalipov, who did not respond to requests for comment, has now shown that the answer to that might be yes.\u003c/p>\n\u003cp>Some scholars questioned how important the new study is, however. Stanford University law professor and bioethicist Hank Greely \u003ca href=\"https://twitter.com/HankGreelyLSJU/status/890370419651366912\">tweeted\u003c/a> that “the key point” is that no one has tried to implant any edited embryos. “Research embryos” that are “not to be transferred for possible implantation” are “not a big deal,” he \u003ca href=\"https://twitter.com/HankGreelyLSJU/status/890371791947833344\">argued\u003c/a>.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>\u003cem>This \u003ca href=\"https://www.statnews.com/2017/07/26/human-embryos-edited/\" target=\"_blank\" rel=\"noopener noreferrer\">story\u003c/a> has been updated with additional comments by experts and details of similar experiments. It \u003c/em>\u003cem>was originally published by STAT, 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":"/futureofyou/432213/scientists-in-us-edit-human-embryos-with-crispr-for-first-time-reports-suggest","authors":["byline_futureofyou_432213"],"categories":["futureofyou_1"],"tags":["futureofyou_94","futureofyou_1275","futureofyou_295","futureofyou_744"],"featImg":"futureofyou_432218","label":"futureofyou"},"futureofyou_372137":{"type":"posts","id":"futureofyou_372137","meta":{"index":"posts_1591205157","site":"futureofyou","id":"372137","score":null,"sort":[1492113669000]},"guestAuthors":[],"slug":"uc-berkeley-renews-legal-fight-over-crispr-cas9-patent-rights","title":"UC Berkeley Renews Legal Fight Over CRISPR-Cas9 Patent Rights","publishDate":1492113669,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp>Scientists at the University of California, Berkeley have filed an appeal to overturn \u003ca href=\"https://ww2.kqed.org/futureofyou/2017/02/16/broad-institute-wins-decision-over-uc-berkeley-in-crispr-patent-battle/\" target=\"_blank\">a decision\u003c/a> by the \u003ca href=\"https://www.broadinstitute.org/\" target=\"_blank\">Patent Trial and Appeal Board\u003c/a> filed in mid-February. The board granted a patent to UC for CRISPR-Cas9 gene-editing in bacterial cells, but awarded a separate a patent to the East-Coast based \u003ca href=\"https://www.broadinstitute.org/\" target=\"_blank\">Broad Institute\u003c/a>, jointly owned by Harvard and MIT, for CRISPR's use in plant and animal cells.\u003c/p>\n\u003cp>UC believes they were the first inventors and should have the patent for use in all applications.\u003c/p>\n\u003cp>\"We expect to establish definitively that the team led by [UC's] Jennifer Doudna and Emmanuelle Charpentier was the first to engineer CRISPR-Cas9 for use in all types of environments, including in non-cellular settings and within plant, animal and even human cells,” said Edward Penhoet, associate dean of biology at UC Berkeley \u003ca href=\"http://news.berkeley.edu/2017/04/13/uc-appeals-u-s-patent-board-decision-on-crispr-cas9/\" target=\"_blank\">in a statement\u003c/a>.\u003c/p>\n\u003cp>In \u003ca href=\"https://www.broadinstitute.org/crispr/journalists-statement-and-background-crispr-patent-process\" target=\"_blank\">a separate statement\u003c/a>, the Broad Institute says they expect the outcome of the appeal to remain the same.\u003c/p>\n\u003cp>Billions are at stake. The patent for CRISPR gene editing will make whoever holds it a powerful player in the biomed industry, university research and the future of medicine.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>For background on the patent dispute, and the new era of gene editing, catch our previous reporting.\u003c/p>\n\u003cp>\u003ca href=\"https://ww2.kqed.org/futureofyou/2016/01/15/crispr-patent-war-billions-at-stake-for-uc-berkeley/\" target=\"_blank\">CRISPR Patent War: Billions at Stake for UC Berkeley\u003c/a> (Jan. 15, 2016 )\u003c/p>\n\u003cp>\u003ca href=\"https://ww2.kqed.org/futureofyou/2017/02/23/crispr-patent-ruling-3-different-takes/\" target=\"_blank\">CRISPR Patent Ruling: 3 Different Takes\u003c/a> (Feb. 23, 2017)\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>\u003ca href=\"https://ww2.kqed.org/futureofyou/2016/04/29/how-a-new-crisprcas9-technique-may-get-us-closer-to-curing-genetic-diseases/\" target=\"_blank\">How New CRISPR/Cas9 Technique Could Be a Game Changer in Curing Genetic Diseases\u003c/a> (Apr. 29, 2016)\u003c/p>\n\n","blocks":[],"excerpt":null,"status":"publish","parent":0,"modified":1492117567,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":11,"wordCount":255},"headData":{"title":"UC Berkeley Renews Legal Fight Over CRISPR-Cas9 Patent Rights | KQED","description":"Scientists at the University of California, Berkeley have filed an appeal to overturn a decision by the Patent Trial and Appeal Board filed in mid-February. The board granted a patent to UC for CRISPR-Cas9 gene-editing in bacterial cells, but awarded a separate a patent to the East-Coast based Broad Institute, jointly owned by Harvard and","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"UC Berkeley Renews Legal Fight Over CRISPR-Cas9 Patent Rights","datePublished":"2017-04-13T20:01:09.000Z","dateModified":"2017-04-13T21:06:07.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"372137 https://ww2.kqed.org/futureofyou/?p=372137","disqusUrl":"https://ww2.kqed.org/futureofyou/2017/04/13/uc-berkeley-renews-legal-fight-over-crispr-cas9-patent-rights/","disqusTitle":"UC Berkeley Renews Legal Fight Over CRISPR-Cas9 Patent Rights","source":" KQED Future of You","path":"/futureofyou/372137/uc-berkeley-renews-legal-fight-over-crispr-cas9-patent-rights","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>Scientists at the University of California, Berkeley have filed an appeal to overturn \u003ca href=\"https://ww2.kqed.org/futureofyou/2017/02/16/broad-institute-wins-decision-over-uc-berkeley-in-crispr-patent-battle/\" target=\"_blank\">a decision\u003c/a> by the \u003ca href=\"https://www.broadinstitute.org/\" target=\"_blank\">Patent Trial and Appeal Board\u003c/a> filed in mid-February. The board granted a patent to UC for CRISPR-Cas9 gene-editing in bacterial cells, but awarded a separate a patent to the East-Coast based \u003ca href=\"https://www.broadinstitute.org/\" target=\"_blank\">Broad Institute\u003c/a>, jointly owned by Harvard and MIT, for CRISPR's use in plant and animal cells.\u003c/p>\n\u003cp>UC believes they were the first inventors and should have the patent for use in all applications.\u003c/p>\n\u003cp>\"We expect to establish definitively that the team led by [UC's] Jennifer Doudna and Emmanuelle Charpentier was the first to engineer CRISPR-Cas9 for use in all types of environments, including in non-cellular settings and within plant, animal and even human cells,” said Edward Penhoet, associate dean of biology at UC Berkeley \u003ca href=\"http://news.berkeley.edu/2017/04/13/uc-appeals-u-s-patent-board-decision-on-crispr-cas9/\" target=\"_blank\">in a statement\u003c/a>.\u003c/p>\n\u003cp>In \u003ca href=\"https://www.broadinstitute.org/crispr/journalists-statement-and-background-crispr-patent-process\" target=\"_blank\">a separate statement\u003c/a>, the Broad Institute says they expect the outcome of the appeal to remain the same.\u003c/p>\n\u003cp>Billions are at stake. The patent for CRISPR gene editing will make whoever holds it a powerful player in the biomed industry, university research and the future of medicine.\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>For background on the patent dispute, and the new era of gene editing, catch our previous reporting.\u003c/p>\n\u003cp>\u003ca href=\"https://ww2.kqed.org/futureofyou/2016/01/15/crispr-patent-war-billions-at-stake-for-uc-berkeley/\" target=\"_blank\">CRISPR Patent War: Billions at Stake for UC Berkeley\u003c/a> (Jan. 15, 2016 )\u003c/p>\n\u003cp>\u003ca href=\"https://ww2.kqed.org/futureofyou/2017/02/23/crispr-patent-ruling-3-different-takes/\" target=\"_blank\">CRISPR Patent Ruling: 3 Different Takes\u003c/a> (Feb. 23, 2017)\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>\u003ca href=\"https://ww2.kqed.org/futureofyou/2016/04/29/how-a-new-crisprcas9-technique-may-get-us-closer-to-curing-genetic-diseases/\" target=\"_blank\">How New CRISPR/Cas9 Technique Could Be a Game Changer in Curing Genetic Diseases\u003c/a> (Apr. 29, 2016)\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/372137/uc-berkeley-renews-legal-fight-over-crispr-cas9-patent-rights","authors":["11088"],"categories":["futureofyou_452","futureofyou_1","futureofyou_73"],"tags":["futureofyou_1250","futureofyou_94","futureofyou_295"],"label":"source_futureofyou_372137"}},"programsReducer":{"possible":{"id":"possible","title":"Possible","info":"Possible is hosted by entrepreneur Reid Hoffman and writer Aria Finger. 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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. 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On Our Watch brings listeners into the rooms where officers are questioned and witnesses are interrogated to find out who this system is really protecting. 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For one hour a week, the show tries to lift the veil from the process of \"making media,\" especially news media, because it's through that lens that we see the world and the world sees us","airtime":"SUN 2pm-3pm, MON 12am-1am","imageSrc":"https://ww2.kqed.org/radio/wp-content/uploads/sites/50/2018/04/onTheMedia.png","officialWebsiteLink":"https://www.wnycstudios.org/shows/otm","meta":{"site":"news","source":"wnyc"},"link":"/radio/program/on-the-media","subscribe":{"apple":"https://itunes.apple.com/us/podcast/on-the-media/id73330715?mt=2","tuneIn":"https://tunein.com/radio/On-the-Media-p69/","rss":"http://feeds.wnyc.org/onthemedia"}},"our-body-politic":{"id":"our-body-politic","title":"Our Body Politic","info":"Presented by KQED, KCRW and KPCC, and created and hosted by award-winning journalist Farai Chideya, Our Body Politic is unapologetically centered on reporting on not just how women of color experience the major political events of today, but how they’re impacting those very issues.","airtime":"SAT 6pm-7pm, SUN 1am-2am","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Our-Body-Politic-Podcast-Tile-360x360-1.jpg","officialWebsiteLink":"https://our-body-politic.simplecast.com/","meta":{"site":"news","source":"kcrw"},"link":"/radio/program/our-body-politic","subscribe":{"apple":"https://podcasts.apple.com/us/podcast/our-body-politic/id1533069868","google":"https://podcasts.google.com/feed/aHR0cHM6Ly9mZWVkcy5zaW1wbGVjYXN0LmNvbS9feGFQaHMxcw","spotify":"https://open.spotify.com/show/4ApAiLT1kV153TttWAmqmc","rss":"https://feeds.simplecast.com/_xaPhs1s","tuneIn":"https://tunein.com/podcasts/News--Politics-Podcasts/Our-Body-Politic-p1369211/"}},"pbs-newshour":{"id":"pbs-newshour","title":"PBS NewsHour","info":"Analysis, background reports and updates from the PBS NewsHour putting today's news in context.","airtime":"MON-FRI 3pm-4pm","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/PBS-News-Hour-Podcast-Tile-360x360-1.jpg","officialWebsiteLink":"https://www.pbs.org/newshour/","meta":{"site":"news","source":"pbs"},"link":"/radio/program/pbs-newshour","subscribe":{"apple":"https://itunes.apple.com/us/podcast/pbs-newshour-full-show/id394432287?mt=2","tuneIn":"https://tunein.com/radio/PBS-NewsHour---Full-Show-p425698/","rss":"https://www.pbs.org/newshour/feeds/rss/podcasts/show"}},"perspectives":{"id":"perspectives","title":"Perspectives","tagline":"KQED's series of of daily listener commentaries since 1991","info":"KQED's series of of daily listener commentaries since 1991.","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Perspectives-Podcast-Tile-703x703-1.jpg","officialWebsiteLink":"/perspectives/","meta":{"site":"radio","source":"kqed","order":"15"},"link":"/perspectives","subscribe":{"apple":"https://podcasts.apple.com/us/podcast/id73801135","npr":"https://www.npr.org/podcasts/432309616/perspectives","rss":"https://ww2.kqed.org/perspectives/category/perspectives/feed/","google":"https://podcasts.google.com/feed/aHR0cHM6Ly93dzIua3FlZC5vcmcvcGVyc3BlY3RpdmVzL2NhdGVnb3J5L3BlcnNwZWN0aXZlcy9mZWVkLw"}},"planet-money":{"id":"planet-money","title":"Planet Money","info":"The economy explained. 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The result is stories that inform and inspire, arming our listeners with information to right injustices, hold the powerful accountable and improve lives.Reveal is hosted by Al Letson and showcases the award-winning work of CIR and newsrooms large and small across the nation. In a radio and podcast market crowded with choices, Reveal focuses on important and often surprising stories that illuminate the world for our listeners.","airtime":"SAT 4pm-5pm","imageSrc":"https://ww2.kqed.org/radio/wp-content/uploads/sites/50/2018/04/reveal300px.png","officialWebsiteLink":"https://www.revealnews.org/episodes/","meta":{"site":"news","source":"npr"},"link":"/radio/program/reveal","subscribe":{"apple":"https://itunes.apple.com/us/podcast/reveal/id886009669","tuneIn":"https://tunein.com/radio/Reveal-p679597/","rss":"http://feeds.revealradio.org/revealpodcast"}},"says-you":{"id":"says-you","title":"Says You!","info":"Public radio's game show of bluff and bluster, words and whimsy. 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