The Take From Courtside: UC Berkeley Probably Not Going to Win CRISPR Patent Dispute
Explainer: The CRISPR/Cas9 Advance That Makes Gene Editing Significantly More Precise
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Billions at Stake: UC Berkeley's Day in Court vs. Harvard/MIT Over CRISPR
CRISPR: What You Need to Know About the Medical Science 'Breakthrough of the Year'
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Sponsored
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FM","link":"/"}},"futureofyou_441220":{"type":"posts","id":"futureofyou_441220","meta":{"index":"posts_1591205157","site":"futureofyou","id":"441220","score":null,"sort":[1525204384000]},"guestAuthors":[],"slug":"the-take-from-courtside-uc-berkeley-probably-not-going-to-win-crispr-patent-dispute","title":"The Take From Courtside: UC Berkeley Probably Not Going to Win CRISPR Patent Dispute","publishDate":1525204384,"format":"aside","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003ch3>The university clearly failed to win over at least one of the three judges and, at best, did not lose too much ground with a second.\u003c/h3>\n\u003cp class=\"danger-zone\">The University of California, which has been losing the fight over U.S. patents on the revolutionary genome-editing technology CRISPR-Cas9, did not pull any rabbits out of a hat during \u003ca href=\"https://www.statnews.com/2018/04/27/crispr-patent-fight-round-two/\" target=\"_blank\" rel=\"noopener\">oral arguments\u003c/a> Monday in its appeal of the case.\u003c/p>\n\u003cp class=\"danger-zone\">The university clearly failed to win over at least one of the three judges and, at best, did not lose too much ground with a second (while the third asked almost no questions and so did not tip his hand).\u003c/p>\n\u003cp class=\"danger-zone\">“UC came into this argument from a tough spot, and I doubt that oral arguments from either side moved the needle much,” said patent attorney Michael Stramiello of Paul Hastings, who attended the arguments at the U.S. Court of Appeals for the Federal Circuit, where attorneys for the University of California fought to get a patent win by the Broad Institute of MIT and Harvard reversed.\u003c/p>\n\u003cp class=\"danger-zone\">“It was closer than I expected,” said patent expert Jacob Sherkow of New York Law School, who also attended the argument. “But it’s still going to be tough for UC.”\u003c/p>\n\u003cp class=\"danger-zone\">That’s because in order to \u003ca href=\"https://www.statnews.com/2018/04/27/crispr-patent-fight-round-two/\" target=\"_blank\" rel=\"noopener\">reverse the Broad’s win\u003c/a>, the appeals court will have to find that the patent office committed serious legal errors, as UC’s new attorney (and former solicitor general) Donald Verrilli argued. He marshaled both legal and scientific evidence to make his case for what experts call his only path to success: persuading the judges that the patent office did not have “substantial evidence” for its decision in favor of the Broad.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>The problem for UC is that the legal definition of “substantial” differs from the plain English meaning.\u003c/p>\n\u003cfigure id=\"attachment_441233\" class=\"wp-caption alignright\" style=\"max-width: 413px\">\u003ca href=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/05/FengZhang_413x315.png\">\u003cimg src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/05/FengZhang_413x315.png\" alt=\"\" width=\"413\" height=\"315\" class=\"size-full wp-image-441233\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2018/05/FengZhang_413x315.png 413w, https://ww2.kqed.org/app/uploads/sites/13/2018/05/FengZhang_413x315-160x122.png 160w, https://ww2.kqed.org/app/uploads/sites/13/2018/05/FengZhang_413x315-240x183.png 240w, https://ww2.kqed.org/app/uploads/sites/13/2018/05/FengZhang_413x315-375x286.png 375w\" sizes=\"(max-width: 413px) 100vw, 413px\">\u003c/a>\u003cfigcaption class=\"wp-caption-text\">Biotechnologist Feng Zhang of the Broad Institute was awarded a patent for CRISPR gene-editing technology in 2014. \u003ccite>(Broad Institute)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>“It means just enough for a reasonable person to find it persuasive,” Sherkow said. In other words, when the patent office decided that the Broad gets to keep its key CRISPR patents, did it base that on a reasonable assessment of the relevant evidence about, in particular, whether the Broad’s breakthroughs were “obvious” extensions of research by UC scientists. “You can ignore some evidence that still meet the requirement of ‘substantial evidence,'” said law professor Dmitry Karshtedt, who attended the hearing.\u003c/p>\n\u003cp>Judge Kimberly Moore, in particular, seemed unconvinced by Verrilli’s arguments. “That’s how science works, Mr. Verrilli,” she said toward the end of the 35-minute hearing. Verrilli spent much of his allotted 15 minutes, and much of the written briefs, arguing that because half a dozen labs, besides that of the Broad’s \u003ca href=\"https://www.statnews.com/2015/11/06/hollywood-inspired-scientist-rewrite-code-life/\">Feng Zhang\u003c/a>, quickly got CRISPR to work in mammalian cells after UC scientists showed that it worked on DNA in a test tube, and used conventional techniques to do so, the Broad’s work was an obvious extension of UC’s and thus not deserving of patents.\u003c/p>\n\u003cp>But getting CRISPR to work in mammalian cells, and the fact that all the labs used conventional techniques of molecular biology to do it, “doesn’t mean they thought it would work,” Moore said. In pushback that could be fatal for UC, she continued, “It’s hard for me to say there wasn’t substantial evidence” to back up the patent office’s ruling against UC. When Verrilli tried to jump in, he got a sharp, “Please don’t interrupt me.”\u003c/p>\n\u003caside class=\"pullquote alignright\">'That’s how science works, Mr. Verrilli.'\u003ccite>Judge Kimberly Moore to UC Berkeley attorney Donald Verrilli\u003c/cite>\u003c/aside>\n\u003cp>The Broad issued a statement at the conclusion of the hearing: “Based on the oral arguments today, we are even more confident the Federal Circuit will affirm the PTAB’s judgment,” the Broad said. “We call on UCB and the companies that control its IP to join our ongoing efforts to simplify, share, and open the IP landscape.”\u003c/p>\n\u003cp>Not surprisingly, the University of California had a different take: “We presented compelling arguments today that the PTAB committed several legal errors, including disregarding Supreme Court and Federal Circuit precedent,” Charles F. Robinson, UC general counsel said in a statement. “Based on the questioning today, we are optimistic that the court has serious doubts about several aspects of the PTAB’s decision.”\u003c/p>\n\u003cp>To recap the plot thus far: In June 2012, UC Berkeley biochemist Jennifer Doudna and her colleagues reported that CRISPR-Cas9 could be turned into a “programmable” genome-editing tool, as they demonstrated on DNA in a test tube. They’d filed their first patent application on the technique the month before. Seven months later, the Broad Institute filed for a CRISPR patent on Zhang’s discovery of how to use the system to edit the genomes of eukaryotic cells, as he reported in January 2013.\u003c/p>\n\u003cp>Although UC filed first for a CRISPR patent, under the law at the time, patents went to the first to invent, and Zhang submitted lab notebooks showing he managed his feat before Doudna’s paper was published. In April 2014, the Broad won a far-reaching patent on the use of CRISPR-Cas9 for editing genomes in eukaryotic cells. A year later, UC asked the patent office to declare an “interference in fact”: to decide that the Broad’s CRISPR patents (which by then numbered 10) covered inventions so similar to what UC applied for (and was still waiting for) a patent on that they should be deemed invalid.\u003c/p>\n\u003cp>In February 2017, the Patent Trial and Appeal Board \u003ca href=\"https://www.statnews.com/2017/02/15/crispr-patent-ruling/\">ruled\u003c/a> that the Broad’s 2014 CRISPR patent did not “interfere” with UC’s pending ones: The Broad’s, which covers the use of CRISPR-Cas9 to edit genomes in eukaryotic cells, was different enough from UC’s and not an obvious extension of Doudna’s work to be valid.\u003c/p>\n\u003cp>UC appealed, and the oral arguments were heard on Monday.\u003c/p>\n\u003cp>The only arguments an appeals court entertains, whether about a conviction for murder or a ruling on patent interference, are those about whether the previous tribunals conducted the proceedings properly, interpreting and applying the law correctly. In this case, that means UC must persuade the three judges that PTAB did not have “substantial evidence” to support its finding of no-interference-in-fact; that PTAB made a material legal error, such as in what evidence or testimony it allowed; or that it failed to consider certain evidence.\u003c/p>\n\u003cp>Verrilli got out barely two sentences before chief judge Sharon Prost jumped in. The main ground for overturning PTAB’s decision, she pointed out, would be a finding that the board did not have substantial evidence for it. But PTAB’s long, detailed written decision suggests that it did not exactly base its decision on a whim. “How do we get around that?” Prost asked Verrilli.\u003c/p>\n\u003cp>When he tried to answer, Moore interrupted, pointing out “problems with the cases” Verrilli had cited to support his position and the low bar for evidence to be “substantial.” “Do you see the problem I’m having?” she asked him. (Shortly after, Moore had a coughing fit and excused herself from the bench, an almost unheard-of occurrence, but told the attorneys to carry on; she returned a few minutes later.)\u003c/p>\n\u003cp>Arguing for the Broad, Raymond Nimrod of Quinn Emanuel had a somewhat easier time but did not get off scot-free. Prost zeroed in on the success that those half a dozen labs had using CRISPR in mammalian cells in the months after Doudna’s success with CRISPR on test-tube DNA. UC has argued throughout the patent saga that the success by so many labs indicates that it was an obvious extension of Doudna’s work, “obviousness” that would include what Zhang achieved.\u003c/p>\n\u003cp>“What about the [patent] board’s not placing much weight on how several people came up with it in a short period of time” after Doudna’s work, Prost asked Nimrod. He replied that PTAB did not dismiss that evidence, but simply found it not to support UC’s claim.\u003c/p>\n\u003cp>The fact that PTAB considered that argument might be enough to meet the fairly low requirement that its decisions be based on “substantial evidence,” Sherkow said.\u003c/p>\n\u003cp>A ruling is expected by late summer.\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>\u003cem>This \u003ca href=\"https://www.statnews.com/2018/04/30/crispr-patent-appeal-hearing/\" 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":"In the ongoing dispute over ownership of key CRISPR patents, UC Berkeley is going to have a tough time getting a court to overturn a patent office ruling in favor of the rival Broad Institute.","status":"publish","parent":0,"modified":1535388964,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":20,"wordCount":1499},"headData":{"title":"The Take From Courtside: UC Berkeley Probably Not Going to Win CRISPR Patent Dispute | KQED","description":"In the ongoing dispute over ownership of key CRISPR patents, UC Berkeley is going to have a tough time getting a court to overturn a patent office ruling in favor of the rival Broad Institute.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"The Take From Courtside: UC Berkeley Probably Not Going to Win CRISPR Patent Dispute","datePublished":"2018-05-01T19:53:04.000Z","dateModified":"2018-08-27T16:56:04.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"441220 https://ww2.kqed.org/futureofyou/?p=441220","disqusUrl":"https://ww2.kqed.org/futureofyou/2018/05/01/the-take-from-courtside-uc-berkeley-probably-not-going-to-win-crispr-patent-dispute/","disqusTitle":"The Take From Courtside: UC Berkeley Probably Not Going to Win CRISPR Patent Dispute","source":"Your Genes","nprByline":"Sharon Begley\u003cbr />\u003ca href=\"https://www.statnews.com/\">STAT\u003c/a>","path":"/futureofyou/441220/the-take-from-courtside-uc-berkeley-probably-not-going-to-win-crispr-patent-dispute","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003ch3>The university clearly failed to win over at least one of the three judges and, at best, did not lose too much ground with a second.\u003c/h3>\n\u003cp class=\"danger-zone\">The University of California, which has been losing the fight over U.S. patents on the revolutionary genome-editing technology CRISPR-Cas9, did not pull any rabbits out of a hat during \u003ca href=\"https://www.statnews.com/2018/04/27/crispr-patent-fight-round-two/\" target=\"_blank\" rel=\"noopener\">oral arguments\u003c/a> Monday in its appeal of the case.\u003c/p>\n\u003cp class=\"danger-zone\">The university clearly failed to win over at least one of the three judges and, at best, did not lose too much ground with a second (while the third asked almost no questions and so did not tip his hand).\u003c/p>\n\u003cp class=\"danger-zone\">“UC came into this argument from a tough spot, and I doubt that oral arguments from either side moved the needle much,” said patent attorney Michael Stramiello of Paul Hastings, who attended the arguments at the U.S. Court of Appeals for the Federal Circuit, where attorneys for the University of California fought to get a patent win by the Broad Institute of MIT and Harvard reversed.\u003c/p>\n\u003cp class=\"danger-zone\">“It was closer than I expected,” said patent expert Jacob Sherkow of New York Law School, who also attended the argument. “But it’s still going to be tough for UC.”\u003c/p>\n\u003cp class=\"danger-zone\">That’s because in order to \u003ca href=\"https://www.statnews.com/2018/04/27/crispr-patent-fight-round-two/\" target=\"_blank\" rel=\"noopener\">reverse the Broad’s win\u003c/a>, the appeals court will have to find that the patent office committed serious legal errors, as UC’s new attorney (and former solicitor general) Donald Verrilli argued. He marshaled both legal and scientific evidence to make his case for what experts call his only path to success: persuading the judges that the patent office did not have “substantial evidence” for its decision in favor of the Broad.\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 problem for UC is that the legal definition of “substantial” differs from the plain English meaning.\u003c/p>\n\u003cfigure id=\"attachment_441233\" class=\"wp-caption alignright\" style=\"max-width: 413px\">\u003ca href=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/05/FengZhang_413x315.png\">\u003cimg src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2018/05/FengZhang_413x315.png\" alt=\"\" width=\"413\" height=\"315\" class=\"size-full wp-image-441233\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2018/05/FengZhang_413x315.png 413w, https://ww2.kqed.org/app/uploads/sites/13/2018/05/FengZhang_413x315-160x122.png 160w, https://ww2.kqed.org/app/uploads/sites/13/2018/05/FengZhang_413x315-240x183.png 240w, https://ww2.kqed.org/app/uploads/sites/13/2018/05/FengZhang_413x315-375x286.png 375w\" sizes=\"(max-width: 413px) 100vw, 413px\">\u003c/a>\u003cfigcaption class=\"wp-caption-text\">Biotechnologist Feng Zhang of the Broad Institute was awarded a patent for CRISPR gene-editing technology in 2014. \u003ccite>(Broad Institute)\u003c/cite>\u003c/figcaption>\u003c/figure>\n\u003cp>“It means just enough for a reasonable person to find it persuasive,” Sherkow said. In other words, when the patent office decided that the Broad gets to keep its key CRISPR patents, did it base that on a reasonable assessment of the relevant evidence about, in particular, whether the Broad’s breakthroughs were “obvious” extensions of research by UC scientists. “You can ignore some evidence that still meet the requirement of ‘substantial evidence,'” said law professor Dmitry Karshtedt, who attended the hearing.\u003c/p>\n\u003cp>Judge Kimberly Moore, in particular, seemed unconvinced by Verrilli’s arguments. “That’s how science works, Mr. Verrilli,” she said toward the end of the 35-minute hearing. Verrilli spent much of his allotted 15 minutes, and much of the written briefs, arguing that because half a dozen labs, besides that of the Broad’s \u003ca href=\"https://www.statnews.com/2015/11/06/hollywood-inspired-scientist-rewrite-code-life/\">Feng Zhang\u003c/a>, quickly got CRISPR to work in mammalian cells after UC scientists showed that it worked on DNA in a test tube, and used conventional techniques to do so, the Broad’s work was an obvious extension of UC’s and thus not deserving of patents.\u003c/p>\n\u003cp>But getting CRISPR to work in mammalian cells, and the fact that all the labs used conventional techniques of molecular biology to do it, “doesn’t mean they thought it would work,” Moore said. In pushback that could be fatal for UC, she continued, “It’s hard for me to say there wasn’t substantial evidence” to back up the patent office’s ruling against UC. When Verrilli tried to jump in, he got a sharp, “Please don’t interrupt me.”\u003c/p>\n\u003caside class=\"pullquote alignright\">'That’s how science works, Mr. Verrilli.'\u003ccite>Judge Kimberly Moore to UC Berkeley attorney Donald Verrilli\u003c/cite>\u003c/aside>\n\u003cp>The Broad issued a statement at the conclusion of the hearing: “Based on the oral arguments today, we are even more confident the Federal Circuit will affirm the PTAB’s judgment,” the Broad said. “We call on UCB and the companies that control its IP to join our ongoing efforts to simplify, share, and open the IP landscape.”\u003c/p>\n\u003cp>Not surprisingly, the University of California had a different take: “We presented compelling arguments today that the PTAB committed several legal errors, including disregarding Supreme Court and Federal Circuit precedent,” Charles F. Robinson, UC general counsel said in a statement. “Based on the questioning today, we are optimistic that the court has serious doubts about several aspects of the PTAB’s decision.”\u003c/p>\n\u003cp>To recap the plot thus far: In June 2012, UC Berkeley biochemist Jennifer Doudna and her colleagues reported that CRISPR-Cas9 could be turned into a “programmable” genome-editing tool, as they demonstrated on DNA in a test tube. They’d filed their first patent application on the technique the month before. Seven months later, the Broad Institute filed for a CRISPR patent on Zhang’s discovery of how to use the system to edit the genomes of eukaryotic cells, as he reported in January 2013.\u003c/p>\n\u003cp>Although UC filed first for a CRISPR patent, under the law at the time, patents went to the first to invent, and Zhang submitted lab notebooks showing he managed his feat before Doudna’s paper was published. In April 2014, the Broad won a far-reaching patent on the use of CRISPR-Cas9 for editing genomes in eukaryotic cells. A year later, UC asked the patent office to declare an “interference in fact”: to decide that the Broad’s CRISPR patents (which by then numbered 10) covered inventions so similar to what UC applied for (and was still waiting for) a patent on that they should be deemed invalid.\u003c/p>\n\u003cp>In February 2017, the Patent Trial and Appeal Board \u003ca href=\"https://www.statnews.com/2017/02/15/crispr-patent-ruling/\">ruled\u003c/a> that the Broad’s 2014 CRISPR patent did not “interfere” with UC’s pending ones: The Broad’s, which covers the use of CRISPR-Cas9 to edit genomes in eukaryotic cells, was different enough from UC’s and not an obvious extension of Doudna’s work to be valid.\u003c/p>\n\u003cp>UC appealed, and the oral arguments were heard on Monday.\u003c/p>\n\u003cp>The only arguments an appeals court entertains, whether about a conviction for murder or a ruling on patent interference, are those about whether the previous tribunals conducted the proceedings properly, interpreting and applying the law correctly. In this case, that means UC must persuade the three judges that PTAB did not have “substantial evidence” to support its finding of no-interference-in-fact; that PTAB made a material legal error, such as in what evidence or testimony it allowed; or that it failed to consider certain evidence.\u003c/p>\n\u003cp>Verrilli got out barely two sentences before chief judge Sharon Prost jumped in. The main ground for overturning PTAB’s decision, she pointed out, would be a finding that the board did not have substantial evidence for it. But PTAB’s long, detailed written decision suggests that it did not exactly base its decision on a whim. “How do we get around that?” Prost asked Verrilli.\u003c/p>\n\u003cp>When he tried to answer, Moore interrupted, pointing out “problems with the cases” Verrilli had cited to support his position and the low bar for evidence to be “substantial.” “Do you see the problem I’m having?” she asked him. (Shortly after, Moore had a coughing fit and excused herself from the bench, an almost unheard-of occurrence, but told the attorneys to carry on; she returned a few minutes later.)\u003c/p>\n\u003cp>Arguing for the Broad, Raymond Nimrod of Quinn Emanuel had a somewhat easier time but did not get off scot-free. Prost zeroed in on the success that those half a dozen labs had using CRISPR in mammalian cells in the months after Doudna’s success with CRISPR on test-tube DNA. UC has argued throughout the patent saga that the success by so many labs indicates that it was an obvious extension of Doudna’s work, “obviousness” that would include what Zhang achieved.\u003c/p>\n\u003cp>“What about the [patent] board’s not placing much weight on how several people came up with it in a short period of time” after Doudna’s work, Prost asked Nimrod. He replied that PTAB did not dismiss that evidence, but simply found it not to support UC’s claim.\u003c/p>\n\u003cp>The fact that PTAB considered that argument might be enough to meet the fairly low requirement that its decisions be based on “substantial evidence,” Sherkow said.\u003c/p>\n\u003cp>A ruling is expected by late summer.\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/2018/04/30/crispr-patent-appeal-hearing/\" 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/441220/the-take-from-courtside-uc-berkeley-probably-not-going-to-win-crispr-patent-dispute","authors":["byline_futureofyou_441220"],"categories":["futureofyou_1","futureofyou_73","futureofyou_1064"],"tags":["futureofyou_91","futureofyou_94","futureofyou_80","futureofyou_89"],"featImg":"futureofyou_441233","label":"source_futureofyou_441220"},"futureofyou_436872":{"type":"posts","id":"futureofyou_436872","meta":{"index":"posts_1591205157","site":"futureofyou","id":"436872","score":null,"sort":[1510768919000]},"guestAuthors":[],"slug":"explainer-the-new-gene-editing-tool-significantly-more-precise-than-crispr","title":"Explainer: The CRISPR/Cas9 Advance That Makes Gene Editing Significantly More Precise","publishDate":1510768919,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{"site":"futureofyou"},"content":"\u003cp>CRISPR/Cas9 has been justifiably ballyhooed as the best gene-editing tool scientists have ever had to correct broken genes.\u003c/p>\n\u003caside class=\"pullquote alignright\">A new tool that can home in on a problematic single letter of genetic code was substantially more accurate and left many fewer errors than classic CRISPR/Cas9, which was already the most precise gene-editing tool scientists had at their disposal.\u003c/aside>\n\u003cp>But it is by no means perfect. For instance, it only works correctly a small percentage of the time -- as little as a half-percent. And what is worse, it can add mistakes to the DNA of some cells on top of the ones it has failed to correct.\u003c/p>\n\u003cp>Recently, researchers affiliated with Harvard, MIT and the Broad Institute \u003ca href=\"https://www.nature.com/articles/nature24644\" target=\"_blank\" rel=\"noopener\">reported\u003c/a> in the journal \u003cem>Nature\u003c/em> that they had made some major improvements to CRISPR/Cas9. By homing in on single letters within the human genetic code, these tools corrected DNA significantly more often than the older version of the gene-editing tool, leaving behind a lot fewer mistakes as well. Below, you can see Broad Institute researcher David Liu describing this programmable \"machine\" he and others have created. Liu says two-thirds, or roughly 33,000, of the known genetic variants associated with a disease \"are single-letter changes that are thought to play a role in human diseases.\"\u003c/p>\n\u003cp>https://www.youtube.com/watch?v=4Rwft3tairE#action=share\u003c/p>\n\u003cp>Like the original CRISPR/Cas9, what's being called an Adenine Base Editor, or ABE, fixes the DNA of a gene. With an ABE, scientists corrected their target DNA a whopping 34-68 percent of the time, depending on the mutation that’s targeted, with not even 0.1 percent of cells showing a newly created mistake.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>That’s compared to a correction rate of only about .5 to 4 percent with CRISPR/Cas9 sent to corresponding DNA sites. The new-mistake rate for CRISPR is roughly 3 to 11 percent.\u003c/p>\n\u003cfigure id=\"attachment_437084\" class=\"wp-caption aligncenter\" style=\"max-width: 640px\">\u003ca href=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115.jpg\">\u003cimg class=\"size-large wp-image-437084\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-1020x677.jpg\" alt=\"\" width=\"640\" height=\"425\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-1020x677.jpg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-160x106.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-800x531.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-768x510.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-1180x784.jpg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-960x638.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-240x159.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-375x249.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-520x345.jpg 520w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115.jpg 1280w\" sizes=\"(max-width: 640px) 100vw, 640px\">\u003c/a>\u003cfigcaption class=\"wp-caption-text\">CRISPR/Cas9 targets specific sites in DNA, but not necessarily specific enough.\u003c/figcaption>\u003c/figure>\n\u003cp>\u003cstrong>How are ABE and CBE different from the Original CRISPR/Cas9?\u003c/strong>\u003c/p>\n\u003cp>The instructions in DNA are written using four molecules (or bases) called adenine, guanine, thymine and cytosine. These are usually abbreviated as A, G, T and C.\u003c/p>\n\u003cp>Many genetic mistakes consist of a single alteration in the combination of these letters. For example, where a G may be normally found, a patient has an A instead. In that case, the goal of editing the gene would be to change the problematic A to a G.\u003c/p>\n\u003cp>CRISPR/Cas9 does this in a roundabout way. When it is sent to that A by scientists, it makes a clean cut somewhere near in the DNA. This incision sets off alarms bells in the cell, prompting it to immediately begin closing the cut.\u003c/p>\n\u003cp>In order for this natural process to result in a corrected gene, scientists need to package CRISPR/Cas9 with a piece of DNA that contains a G in the correct position.\u003c/p>\n\u003cp>The cell uses the added DNA as a model to correct the sequence.\u003c/p>\n\u003cp>CRISPR/Cas9 then essentially\u003cem> swaps out a large part of the DNA\u003c/em> around that erroneously positioned A with the added DNA that has a correctly situated G. You’ll notice the emphasis in that last sentence, the point being: This if fairly inefficient, as a lot more than just an A is replaced.\u003c/p>\n\u003cp>But ABE neither makes an incision to the existing DNA nor adds the \"template\" DNA that CRISPR/Cas9 does. Instead, it homes in on one base, the A, then turns it into a G. A companion tool to ABE likewise transforms a C into a T.\u003c/p>\n\u003cp>In these base editing tools, the Cas9 molecule can no longer make a complete cut in the DNA. Instead, the researchers attach an enzyme, a protein from the cell, to Cas9. In the case of ABE, that enzyme converts an adenine (A) into an inosine (I). (The cell can't tell the difference between an I and a G and so treats the I as a G.)\u003c/p>\n\u003cp>\u003cstrong>Why Is This Approach Safer?\u003c/strong>\u003c/p>\n\u003cp>A big problem with CRISPR/Cas9 is that cut DNA is so harmful to the cell that it moves to repair it as fast as it can. So fast, in fact, that it often introduces a lot of mistakes. When this occurs, instead of correcting errors, CRISPR creates new ones.\u003c/p>\n\u003cp>The end result can be a hodgepodge of cells with different configurations of DNA -- some of the cells will still have the broken DNA, some will have newly damaged DNA, and a small fraction will include the repaired DNA -- that G that used to be an A, for example.\u003c/p>\n\u003cp>But since ABE does not cut the DNA, cells are not sent into a frenzy of repair. Without that incision in the DNA, the chance of introducing new errors drops by as much as a factor of 100.\u003c/p>\n\u003cp>\u003cstrong>Will These New Tools Replace CRISPR/Cas9?\u003c/strong>\u003c/p>\n\u003cp>Not completely. These new gene-editing tools appear to be very good at turning As into Gs and Cs into Ts, but they won’t work for other kinds of mutations.\u003c/p>\n\u003cp>Sometimes a genetic disease occurs when a bit of DNA is missing, called a deletion, or when there is an extra bit of DNA, called an insertion. The most common mutation in the genetic disease cystic fibrosis, for example, results from a deletion. The original CRISPR/Cas9 system can fix these kinds of mistakes, but the new editing tools can’t.\u003c/p>\n\u003cp>\u003cstrong>Another New Tool Targets RNA\u003c/strong>\u003c/p>\n\u003cp>A different tool, created by a second group of Broad Institute, Harvard and MIT researchers, changes the messenger RNA, or mRNA, in a gene, instead of changing the gene itself. This tool is called REPAIR, for RNA Editing for Programmable A to I Replacement. The best version of REPAIR corrected 13-27 percent of mRNAs.\u003c/p>\n\u003cp>In order for a gene to make changes in a cell, it must first be read by the cellular machinery. The first step, called transcription, is when the gene is copied into a short-lived molecule called mRNA. This mRNA usually goes on to be translated into a protein. It is that protein that does the work in the cell.\u003c/p>\n\u003cp>Up until now, gene editing has been done at the DNA level, where scientists are permanently changing the instructions found in the gene’s DNA. Every mRNA that emanates from the altered gene will include the change.\u003c/p>\n\u003cp>But DNA and RNA are actually very similar in that they both use bases (although RNA uses a U instead of a T). The new tool changes an A to a G in the mRNA coming from a particular gene.\u003c/p>\n\u003cp>So the DNA itself, the original instructions, are not changed. But the disease is treated because the repair has been made in the mRNA.\u003c/p>\n\u003cp>This system might be used for temporary conditions like inflammation. The technique is also ostensibly safer than CRISPR/Cas9 because any changes made are not permanent. If the base editor makes a mistake, those changes will be short-lived. In contrast, any changes made in DNA are forever.\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>\u003cem>\u003cspan style=\"font-weight: 400\">Dr. Barry Starr is a scientist in the\u003c/span>\u003ca href=\"https://med.stanford.edu/genetics.html\"> \u003cspan style=\"font-weight: 400\">Department of Genetics\u003c/span>\u003c/a>\u003cspan style=\"font-weight: 400\"> at Stanford University who runs the\u003c/span>\u003ca href=\"https://med.stanford.edu/genetics/tech.html\"> \u003cspan style=\"font-weight: 400\">Stanford at The Tech\u003c/span>\u003c/a>\u003cspan style=\"font-weight: 400\"> program and the\u003c/span>\u003ca href=\"http://genetics.thetech.org/\"> \u003cspan style=\"font-weight: 400\">Understanding Genetics\u003c/span>\u003c/a>\u003cspan style=\"font-weight: 400\"> website with\u003c/span>\u003ca href=\"https://www.thetech.org/\"> \u003cspan style=\"font-weight: 400\">The Tech Museum of Innovation\u003c/span>\u003c/a>\u003cspan style=\"font-weight: 400\"> in San Jose, California. Before running the program, he worked as a research scientist in the biotechnology field. \u003c/span>\u003c/em>\u003c/p>\n\n","blocks":[],"excerpt":"By homing in on single letters within the human genetic code, new tools corrected DNA significantly more often than CRISPR/Cas9, leaving behind a lot fewer mistakes as well. ","status":"publish","parent":0,"modified":1510783877,"stats":{"hasAudio":false,"hasVideo":true,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":31,"wordCount":1246},"headData":{"title":"Explainer: The CRISPR/Cas9 Advance That Makes Gene Editing Significantly More Precise | KQED","description":"By homing in on single letters within the human genetic code, new tools corrected DNA significantly more often than CRISPR/Cas9, leaving behind a lot fewer mistakes as well. ","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Explainer: The CRISPR/Cas9 Advance That Makes Gene Editing Significantly More Precise","datePublished":"2017-11-15T18:01:59.000Z","dateModified":"2017-11-15T22:11:17.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"436872 https://ww2.kqed.org/futureofyou/?p=436872","disqusUrl":"https://ww2.kqed.org/futureofyou/2017/11/15/explainer-the-new-gene-editing-tool-significantly-more-precise-than-crispr/","disqusTitle":"Explainer: The CRISPR/Cas9 Advance That Makes Gene Editing Significantly More Precise","nprByline":"Barry Starr\u003cbr />KQED Future of You","path":"/futureofyou/436872/explainer-the-new-gene-editing-tool-significantly-more-precise-than-crispr","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>CRISPR/Cas9 has been justifiably ballyhooed as the best gene-editing tool scientists have ever had to correct broken genes.\u003c/p>\n\u003caside class=\"pullquote alignright\">A new tool that can home in on a problematic single letter of genetic code was substantially more accurate and left many fewer errors than classic CRISPR/Cas9, which was already the most precise gene-editing tool scientists had at their disposal.\u003c/aside>\n\u003cp>But it is by no means perfect. For instance, it only works correctly a small percentage of the time -- as little as a half-percent. And what is worse, it can add mistakes to the DNA of some cells on top of the ones it has failed to correct.\u003c/p>\n\u003cp>Recently, researchers affiliated with Harvard, MIT and the Broad Institute \u003ca href=\"https://www.nature.com/articles/nature24644\" target=\"_blank\" rel=\"noopener\">reported\u003c/a> in the journal \u003cem>Nature\u003c/em> that they had made some major improvements to CRISPR/Cas9. By homing in on single letters within the human genetic code, these tools corrected DNA significantly more often than the older version of the gene-editing tool, leaving behind a lot fewer mistakes as well. Below, you can see Broad Institute researcher David Liu describing this programmable \"machine\" he and others have created. Liu says two-thirds, or roughly 33,000, of the known genetic variants associated with a disease \"are single-letter changes that are thought to play a role in human diseases.\"\u003c/p>\n\u003cp>https://www.youtube.com/watch?v=4Rwft3tairE#action=share\u003c/p>\n\u003cp>Like the original CRISPR/Cas9, what's being called an Adenine Base Editor, or ABE, fixes the DNA of a gene. With an ABE, scientists corrected their target DNA a whopping 34-68 percent of the time, depending on the mutation that’s targeted, with not even 0.1 percent of cells showing a newly created mistake.\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>That’s compared to a correction rate of only about .5 to 4 percent with CRISPR/Cas9 sent to corresponding DNA sites. The new-mistake rate for CRISPR is roughly 3 to 11 percent.\u003c/p>\n\u003cfigure id=\"attachment_437084\" class=\"wp-caption aligncenter\" style=\"max-width: 640px\">\u003ca href=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115.jpg\">\u003cimg class=\"size-large wp-image-437084\" src=\"https://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-1020x677.jpg\" alt=\"\" width=\"640\" height=\"425\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-1020x677.jpg 1020w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-160x106.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-800x531.jpg 800w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-768x510.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-1180x784.jpg 1180w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-960x638.jpg 960w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-240x159.jpg 240w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-375x249.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115-520x345.jpg 520w, https://ww2.kqed.org/app/uploads/sites/13/2017/11/DESKTOP_CRISPR_171115.jpg 1280w\" sizes=\"(max-width: 640px) 100vw, 640px\">\u003c/a>\u003cfigcaption class=\"wp-caption-text\">CRISPR/Cas9 targets specific sites in DNA, but not necessarily specific enough.\u003c/figcaption>\u003c/figure>\n\u003cp>\u003cstrong>How are ABE and CBE different from the Original CRISPR/Cas9?\u003c/strong>\u003c/p>\n\u003cp>The instructions in DNA are written using four molecules (or bases) called adenine, guanine, thymine and cytosine. These are usually abbreviated as A, G, T and C.\u003c/p>\n\u003cp>Many genetic mistakes consist of a single alteration in the combination of these letters. For example, where a G may be normally found, a patient has an A instead. In that case, the goal of editing the gene would be to change the problematic A to a G.\u003c/p>\n\u003cp>CRISPR/Cas9 does this in a roundabout way. When it is sent to that A by scientists, it makes a clean cut somewhere near in the DNA. This incision sets off alarms bells in the cell, prompting it to immediately begin closing the cut.\u003c/p>\n\u003cp>In order for this natural process to result in a corrected gene, scientists need to package CRISPR/Cas9 with a piece of DNA that contains a G in the correct position.\u003c/p>\n\u003cp>The cell uses the added DNA as a model to correct the sequence.\u003c/p>\n\u003cp>CRISPR/Cas9 then essentially\u003cem> swaps out a large part of the DNA\u003c/em> around that erroneously positioned A with the added DNA that has a correctly situated G. You’ll notice the emphasis in that last sentence, the point being: This if fairly inefficient, as a lot more than just an A is replaced.\u003c/p>\n\u003cp>But ABE neither makes an incision to the existing DNA nor adds the \"template\" DNA that CRISPR/Cas9 does. Instead, it homes in on one base, the A, then turns it into a G. A companion tool to ABE likewise transforms a C into a T.\u003c/p>\n\u003cp>In these base editing tools, the Cas9 molecule can no longer make a complete cut in the DNA. Instead, the researchers attach an enzyme, a protein from the cell, to Cas9. In the case of ABE, that enzyme converts an adenine (A) into an inosine (I). (The cell can't tell the difference between an I and a G and so treats the I as a G.)\u003c/p>\n\u003cp>\u003cstrong>Why Is This Approach Safer?\u003c/strong>\u003c/p>\n\u003cp>A big problem with CRISPR/Cas9 is that cut DNA is so harmful to the cell that it moves to repair it as fast as it can. So fast, in fact, that it often introduces a lot of mistakes. When this occurs, instead of correcting errors, CRISPR creates new ones.\u003c/p>\n\u003cp>The end result can be a hodgepodge of cells with different configurations of DNA -- some of the cells will still have the broken DNA, some will have newly damaged DNA, and a small fraction will include the repaired DNA -- that G that used to be an A, for example.\u003c/p>\n\u003cp>But since ABE does not cut the DNA, cells are not sent into a frenzy of repair. Without that incision in the DNA, the chance of introducing new errors drops by as much as a factor of 100.\u003c/p>\n\u003cp>\u003cstrong>Will These New Tools Replace CRISPR/Cas9?\u003c/strong>\u003c/p>\n\u003cp>Not completely. These new gene-editing tools appear to be very good at turning As into Gs and Cs into Ts, but they won’t work for other kinds of mutations.\u003c/p>\n\u003cp>Sometimes a genetic disease occurs when a bit of DNA is missing, called a deletion, or when there is an extra bit of DNA, called an insertion. The most common mutation in the genetic disease cystic fibrosis, for example, results from a deletion. The original CRISPR/Cas9 system can fix these kinds of mistakes, but the new editing tools can’t.\u003c/p>\n\u003cp>\u003cstrong>Another New Tool Targets RNA\u003c/strong>\u003c/p>\n\u003cp>A different tool, created by a second group of Broad Institute, Harvard and MIT researchers, changes the messenger RNA, or mRNA, in a gene, instead of changing the gene itself. This tool is called REPAIR, for RNA Editing for Programmable A to I Replacement. The best version of REPAIR corrected 13-27 percent of mRNAs.\u003c/p>\n\u003cp>In order for a gene to make changes in a cell, it must first be read by the cellular machinery. The first step, called transcription, is when the gene is copied into a short-lived molecule called mRNA. This mRNA usually goes on to be translated into a protein. It is that protein that does the work in the cell.\u003c/p>\n\u003cp>Up until now, gene editing has been done at the DNA level, where scientists are permanently changing the instructions found in the gene’s DNA. Every mRNA that emanates from the altered gene will include the change.\u003c/p>\n\u003cp>But DNA and RNA are actually very similar in that they both use bases (although RNA uses a U instead of a T). The new tool changes an A to a G in the mRNA coming from a particular gene.\u003c/p>\n\u003cp>So the DNA itself, the original instructions, are not changed. But the disease is treated because the repair has been made in the mRNA.\u003c/p>\n\u003cp>This system might be used for temporary conditions like inflammation. The technique is also ostensibly safer than CRISPR/Cas9 because any changes made are not permanent. If the base editor makes a mistake, those changes will be short-lived. In contrast, any changes made in DNA are forever.\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\">Dr. Barry Starr is a scientist in the\u003c/span>\u003ca href=\"https://med.stanford.edu/genetics.html\"> \u003cspan style=\"font-weight: 400\">Department of Genetics\u003c/span>\u003c/a>\u003cspan style=\"font-weight: 400\"> at Stanford University who runs the\u003c/span>\u003ca href=\"https://med.stanford.edu/genetics/tech.html\"> \u003cspan style=\"font-weight: 400\">Stanford at The Tech\u003c/span>\u003c/a>\u003cspan style=\"font-weight: 400\"> program and the\u003c/span>\u003ca href=\"http://genetics.thetech.org/\"> \u003cspan style=\"font-weight: 400\">Understanding Genetics\u003c/span>\u003c/a>\u003cspan style=\"font-weight: 400\"> website with\u003c/span>\u003ca href=\"https://www.thetech.org/\"> \u003cspan style=\"font-weight: 400\">The Tech Museum of Innovation\u003c/span>\u003c/a>\u003cspan style=\"font-weight: 400\"> in San Jose, California. Before running the program, he worked as a research scientist in the biotechnology field. \u003c/span>\u003c/em>\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/436872/explainer-the-new-gene-editing-tool-significantly-more-precise-than-crispr","authors":["byline_futureofyou_436872"],"categories":["futureofyou_1","futureofyou_1064"],"tags":["futureofyou_1383","futureofyou_91","futureofyou_94","futureofyou_80"],"featImg":"futureofyou_437037","label":"futureofyou"},"futureofyou_338066":{"type":"posts","id":"futureofyou_338066","meta":{"index":"posts_1591205157","site":"futureofyou","id":"338066","score":null,"sort":[1487263047000]},"guestAuthors":[],"slug":"broad-institute-wins-decision-over-uc-berkeley-in-crispr-patent-battle","title":"Broad Institute Wins Decision Over UC Berkeley in CRISPR Patent Battle","publishDate":1487263047,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{},"content":"\u003cp>The U.S. patent office has delivered a potentially lucrative victory to bioengineer \u003ca href=\"https://www.broadinstitute.org/zhang-lab\">Feng Zhang\u003c/a> of the Broad Institute in Massachusetts, regarding patents for an extraordinarily useful gene-editing tool.\u003c/p>\n\u003cp>\u003ca href=\"http://www.npr.org/tags/419142387/crispr\">CRISPR\u003c/a>, a technology that's already worth billions of dollars, is shaping up to play a big role in medicine and medical research because it can edit DNA with unprecedented accuracy. But exactly who has the right to profit from the technology has been up for debate.\u003c/p>\n\u003cp>Wednesday the U.S. Patent and Trademark Office said patents issued to the Broad Institute in 2014, and then challenged by the University of California, Berkeley, are in fact valid.\u003c/p>\n\u003cp>\"It's a pretty monumental decision here,\" said \u003ca href=\"http://www.nyls.edu/faculty/faculty-profiles/faculty_profiles/jacob-s-sherkow/\">Jacob Sherkow\u003c/a>, an associate professor at the New York Law School, who has been tracking the dispute closely.\u003c/p>\n\u003cp>\"It seems to reward the most valuable aspect of CRISPR to the Broad Institute,\" Sherkow told Shots.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>The proceedings aren't entirely settled, but as Sherkow sees the situation, the Broad Institute — a joint venture of Harvard University and MIT — will hold the patent for using CRISPR in human beings, other animals, and plants. Sherkow told Shots he believes Cal's patent, which has not yet been issued, could be limited to bacteria.\u003c/p>\n\u003cp>\"Obviously the patents covering the application of this technology in human cells ... are going to be much more financially valuable than using the same technology in bacteria,\" Sherkow says, \"because one can develop drugs and other therapies from them.\"\u003c/p>\n\u003cp>Investors Wednesday seemed to agree with this assessment. The value of companies that were spun off to license the Broad patents rose sharply, while the company based on the Berkeley patent lost value.\u003c/p>\n\u003cp>Potentially, tens of billions of dollars are at stake here, both for the companies and for the universities.\u003c/p>\n\u003cp>Biochemist \u003ca href=\"https://www.hhmi.org/scientists/jennifer-doudna\">Jennifer Doudna\u003c/a>, of U.C. Berkeley and the Howard Hughes Medical Institute, discovered the biology that underlies this technology along with a European colleague, \u003ca href=\"https://www.mpg.de/9343753/infektionsbiologie-charpentier\">Emmanuelle Charpentier\u003c/a>, who is now director of the Institute for Infection Biology at the Max Planck Institute in Berlin.\u003c/p>\n\u003cp>Doudna told Shots she isn't convinced that Berkeley is the big loser here. She said the ruling paves the way for her patent application to move forward.\u003c/p>\n\u003cp>\"We're looking forward to having our patent issued,\" she said. \"And our patent is a very broad patent that covers the composition and the use of this technology in all cell types.\"\u003c/p>\n\u003cp>If the patent office rules the way Doudna hopes it will, people wanting to use CRISPR in higher organisms will have get licenses from both Berkeley and the Broad Institute.\u003c/p>\n\u003cp>\"That's the thing that I think is a bit crazy about the way the decision comes down,\" Doudna said. \"It leaves the field — the situation — where a license would be necessary from both parties. There's not further clarity at this stage.\"\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>There's yet another possibility: Berkeley could appeal Wednesday's ruling, and once again challenge the Broad Institute's patents. Doudna said the university hasn't decided what to do just yet.\u003c/p>\n\u003cdiv class=\"fullattribution\">Copyright 2017 NPR. To see more, visit http://www.npr.org/.\u003cimg src=\"http://www.google-analytics.com/__utm.gif?utmac=UA-5828686-4&utmdt=Broad+Institute+Wins+Big+Battle+Over+CRISPR+Gene-Editing+Patent&utme=8(APIKey)9(MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004)\">\u003c/div>\n\n","blocks":[],"excerpt":"CRISPR technology is already worth billions of dollars, investors say. This ruling seems to affirm the biggest piece of the pie goes to the Broad, over patent rival University of California, Berkeley.","status":"publish","parent":0,"modified":1487263912,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":17,"wordCount":523},"headData":{"title":"Broad Institute Wins Decision Over UC Berkeley in CRISPR Patent Battle | KQED","description":"CRISPR technology is already worth billions of dollars, investors say. This ruling seems to affirm the biggest piece of the pie goes to the Broad, over patent rival University of California, Berkeley.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Broad Institute Wins Decision Over UC Berkeley in CRISPR Patent Battle","datePublished":"2017-02-16T16:37:27.000Z","dateModified":"2017-02-16T16:51:52.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"338066 https://ww2.kqed.org/futureofyou/?p=338066","disqusUrl":"https://ww2.kqed.org/futureofyou/2017/02/16/broad-institute-wins-decision-over-uc-berkeley-in-crispr-patent-battle/","disqusTitle":"Broad Institute Wins Decision Over UC Berkeley in CRISPR Patent Battle","source":"KQED Future of You","nprByline":"Richard Harris\u003cbr />NPR Shots","nprStoryId":"515459773","nprApiLink":"http://api.npr.org/query?id=515459773&apiKey=MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004","nprHtmlLink":"http://www.npr.org/sections/health-shots/2017/02/15/515459773/broad-institute-wins-big-battle-over-crispr-gene-editing-patent?ft=nprml&f=515459773","nprRetrievedStory":"1","nprPubDate":"Thu, 16 Feb 2017 09:22:00 -0500","nprStoryDate":"Wed, 15 Feb 2017 19:31:00 -0500","nprLastModifiedDate":"Thu, 16 Feb 2017 09:05:42 -0500","nprAudio":"https://ondemand.npr.org/anon.npr-mp3/npr/me/2017/02/20170216_me_broad_institute_wins_big_battle_over_crispr_gene-editing_patent.mp3?orgId=1&topicId=1128&d=190&p=3&story=515459773&t=progseg&e=515529274&seg=16&ft=nprml&f=515459773","nprAudioM3u":"http://api.npr.org/m3u/1515529417-49b234.m3u?orgId=1&topicId=1128&d=190&p=3&story=515459773&t=progseg&e=515529274&seg=16&ft=nprml&f=515459773","path":"/futureofyou/338066/broad-institute-wins-decision-over-uc-berkeley-in-crispr-patent-battle","audioUrl":"https://ondemand.npr.org/anon.npr-mp3/npr/me/2017/02/20170216_me_broad_institute_wins_big_battle_over_crispr_gene-editing_patent.mp3?orgId=1&topicId=1128&d=190&p=3&story=515459773&t=progseg&e=515529274&seg=16&ft=nprml&f=515459773","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>The U.S. patent office has delivered a potentially lucrative victory to bioengineer \u003ca href=\"https://www.broadinstitute.org/zhang-lab\">Feng Zhang\u003c/a> of the Broad Institute in Massachusetts, regarding patents for an extraordinarily useful gene-editing tool.\u003c/p>\n\u003cp>\u003ca href=\"http://www.npr.org/tags/419142387/crispr\">CRISPR\u003c/a>, a technology that's already worth billions of dollars, is shaping up to play a big role in medicine and medical research because it can edit DNA with unprecedented accuracy. But exactly who has the right to profit from the technology has been up for debate.\u003c/p>\n\u003cp>Wednesday the U.S. Patent and Trademark Office said patents issued to the Broad Institute in 2014, and then challenged by the University of California, Berkeley, are in fact valid.\u003c/p>\n\u003cp>\"It's a pretty monumental decision here,\" said \u003ca href=\"http://www.nyls.edu/faculty/faculty-profiles/faculty_profiles/jacob-s-sherkow/\">Jacob Sherkow\u003c/a>, an associate professor at the New York Law School, who has been tracking the dispute closely.\u003c/p>\n\u003cp>\"It seems to reward the most valuable aspect of CRISPR to the Broad Institute,\" Sherkow told Shots.\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 proceedings aren't entirely settled, but as Sherkow sees the situation, the Broad Institute — a joint venture of Harvard University and MIT — will hold the patent for using CRISPR in human beings, other animals, and plants. Sherkow told Shots he believes Cal's patent, which has not yet been issued, could be limited to bacteria.\u003c/p>\n\u003cp>\"Obviously the patents covering the application of this technology in human cells ... are going to be much more financially valuable than using the same technology in bacteria,\" Sherkow says, \"because one can develop drugs and other therapies from them.\"\u003c/p>\n\u003cp>Investors Wednesday seemed to agree with this assessment. The value of companies that were spun off to license the Broad patents rose sharply, while the company based on the Berkeley patent lost value.\u003c/p>\n\u003cp>Potentially, tens of billions of dollars are at stake here, both for the companies and for the universities.\u003c/p>\n\u003cp>Biochemist \u003ca href=\"https://www.hhmi.org/scientists/jennifer-doudna\">Jennifer Doudna\u003c/a>, of U.C. Berkeley and the Howard Hughes Medical Institute, discovered the biology that underlies this technology along with a European colleague, \u003ca href=\"https://www.mpg.de/9343753/infektionsbiologie-charpentier\">Emmanuelle Charpentier\u003c/a>, who is now director of the Institute for Infection Biology at the Max Planck Institute in Berlin.\u003c/p>\n\u003cp>Doudna told Shots she isn't convinced that Berkeley is the big loser here. She said the ruling paves the way for her patent application to move forward.\u003c/p>\n\u003cp>\"We're looking forward to having our patent issued,\" she said. \"And our patent is a very broad patent that covers the composition and the use of this technology in all cell types.\"\u003c/p>\n\u003cp>If the patent office rules the way Doudna hopes it will, people wanting to use CRISPR in higher organisms will have get licenses from both Berkeley and the Broad Institute.\u003c/p>\n\u003cp>\"That's the thing that I think is a bit crazy about the way the decision comes down,\" Doudna said. \"It leaves the field — the situation — where a license would be necessary from both parties. There's not further clarity at this stage.\"\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>There's yet another possibility: Berkeley could appeal Wednesday's ruling, and once again challenge the Broad Institute's patents. Doudna said the university hasn't decided what to do just yet.\u003c/p>\n\u003cdiv class=\"fullattribution\">Copyright 2017 NPR. To see more, visit http://www.npr.org/.\u003cimg src=\"http://www.google-analytics.com/__utm.gif?utmac=UA-5828686-4&utmdt=Broad+Institute+Wins+Big+Battle+Over+CRISPR+Gene-Editing+Patent&utme=8(APIKey)9(MDAxOTAwOTE4MDEyMTkxMDAzNjczZDljZA004)\">\u003c/div>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/338066/broad-institute-wins-decision-over-uc-berkeley-in-crispr-patent-battle","authors":["byline_futureofyou_338066"],"categories":["futureofyou_452","futureofyou_1062","futureofyou_1","futureofyou_73"],"tags":["futureofyou_91","futureofyou_94","futureofyou_790","futureofyou_80","futureofyou_89"],"label":"source_futureofyou_338066"},"futureofyou_295997":{"type":"posts","id":"futureofyou_295997","meta":{"index":"posts_1591205157","site":"futureofyou","id":"295997","score":null,"sort":[1481216406000]},"guestAuthors":[],"slug":"billions-at-stake-uc-berkeley-gets-day-in-court-vs-harvardmit","title":"Billions at Stake: UC Berkeley's Day in Court vs. Harvard/MIT Over CRISPR","publishDate":1481216406,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{"site":"futureofyou"},"content":"\u003cp>It's called CRISPR-Cas9 — one of the century's biggest scientific breakthroughs in genetic engineering\u003cem> — \u003c/em>and now three major universities are battling it out in court over who owns a patent on the revolutionary technology.\u003c/p>\n\u003cp>On Tuesday, UC Berkeley lawyers defended the university's claim to patent\u003cem> \u003c/em>CRISPR in a dispute that went before a panel of judges at the U.S Patent and Trademark headquarters in Alexandria, Virginia\u003cem>. \u003c/em>The Broad Institute of MIT and Harvard is also claiming it owns the rights to CRISPR.\u003c/p>\n\u003cp>CRISPR is a gene-editing tool that allows scientists to manipulate DNA by snipping out part of a mutated gene and substituting a healthy gene. It has huge implications \u003cem>—\u003c/em> from yielding new cancer therapies to correcting genetic disorders to modifying plant and animal DNA.\u003c/p>\n\u003ch3>Editing DNA Using CRISPR\u003c/h3>\n\u003cdiv class=\"row textColumnWidth\">\n\u003cdiv class=\"small-12 large-4 columns\">\u003cimg class=\"aligncenter size-full wp-image-296418\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2016/12/CRISPR01.png\" alt=\"crispr01\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR01.png 750w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR01-160x285.png 160w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR01-240x427.png 240w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR01-375x667.png 375w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR01-520x925.png 520w\" sizes=\"(max-width: 750px) 100vw, 750px\">\u003c/div>\n\u003cdiv class=\"small-12 large-4 columns\">\u003cimg class=\"aligncenter size-full wp-image-296419\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2016/12/CRISPR02.png\" alt=\"crispr02\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR02.png 750w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR02-160x285.png 160w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR02-240x427.png 240w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR02-375x667.png 375w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR02-520x925.png 520w\" sizes=\"(max-width: 750px) 100vw, 750px\">\u003c/div>\n\u003cdiv class=\"small-12 large-4 columns large-4 columns\">\u003cimg class=\"aligncenter size-full wp-image-296420\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2016/12/CRISPR_03_Bug.jpg\" alt=\"crispr_03_bug\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR_03_Bug.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR_03_Bug-160x285.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR_03_Bug-240x427.jpg 240w\" sizes=\"(max-width: 375px) 100vw, 375px\">\u003c/div>\n\u003c/div>\n\u003cp>\"If Berkeley wins it means they’re essentially going to control which commercial companies are able to develop the technology going forward and that will be a huge change in the status quo,\" says New York Law School professor says Jake Sherkow.\u003c/p>\n\u003cp>Sherkow says billions of dollars could be at stake. Companies that use the technology will likely need to pay royalties to whomever owns it -- Broad or Berkeley. Many start-ups including \u003ca href=\"https://www.addgene.org/\" target=\"_blank\">Addgene\u003c/a> -- a biotech non-profit working with Broad -- are already using CRISPR.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>Both UC Berkeley and the Broad Institute of MIT and Harvard claim to have invented the technology and filed for patents.\u003cem> \u003c/em>\u003c/p>\n\u003cp>Berkeley filed first and even though Broad filed later, Broad fast-tracked its application, which was approved while Berkeley's was still pending. This is what Berkeley is contesting -- the fact that Broad's application was approved while its own was still under review.\u003c/p>\n\u003cp>The court has already ruled that the patent will be issued to the scientists who prove the CRISPR technology works in transforming the genes of a particular kind of cell -- \u003cem>eukaryotic\u003c/em> cells, those found in plants and animals, including humans.\u003c/p>\n\u003cp>In their research, Broad Institute bioengineer Feng Zhang and his team used eukaryotic cells, whereas UC Berkeley biochemist Jennifer Doudna used bacteria cells, not eukaryotic cells.\u003c/p>\n\u003cp>However, Doudna argues that UC Berkeley has a right to the patent because any skilled scientist could apply her team's research technique to eukaryotic cells.\u003c/p>\n\u003cp>Berkeley's lawyers faced more intense questioning on Tuesday than the Broad's.\u003c/p>\n\u003cp>\"It does speak to some of the judges' skepticism of the University of California's claims,\" says Sherkow.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>The patent judges could make a decision by February but Sherkow says whichever side loses will likely appeal. So a final resolution may not come until 2018 or 2019.\u003c/p>\n\n","blocks":[],"excerpt":"Who owns the rights to CRISPR? That's up to U.S. patent judges and the decision could be worth billions. ","status":"publish","parent":0,"modified":1481222679,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":15,"wordCount":464},"headData":{"title":"Billions at Stake: UC Berkeley's Day in Court vs. Harvard/MIT Over CRISPR | KQED","description":"Who owns the rights to CRISPR? That's up to U.S. patent judges and the decision could be worth billions. ","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Billions at Stake: UC Berkeley's Day in Court vs. Harvard/MIT Over CRISPR","datePublished":"2016-12-08T17:00:06.000Z","dateModified":"2016-12-08T18:44:39.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"295997 http://ww2.kqed.org/futureofyou/?p=295997","disqusUrl":"https://ww2.kqed.org/futureofyou/2016/12/08/billions-at-stake-uc-berkeley-gets-day-in-court-vs-harvardmit/","disqusTitle":"Billions at Stake: UC Berkeley's Day in Court vs. Harvard/MIT Over CRISPR","path":"/futureofyou/295997/billions-at-stake-uc-berkeley-gets-day-in-court-vs-harvardmit","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>It's called CRISPR-Cas9 — one of the century's biggest scientific breakthroughs in genetic engineering\u003cem> — \u003c/em>and now three major universities are battling it out in court over who owns a patent on the revolutionary technology.\u003c/p>\n\u003cp>On Tuesday, UC Berkeley lawyers defended the university's claim to patent\u003cem> \u003c/em>CRISPR in a dispute that went before a panel of judges at the U.S Patent and Trademark headquarters in Alexandria, Virginia\u003cem>. \u003c/em>The Broad Institute of MIT and Harvard is also claiming it owns the rights to CRISPR.\u003c/p>\n\u003cp>CRISPR is a gene-editing tool that allows scientists to manipulate DNA by snipping out part of a mutated gene and substituting a healthy gene. It has huge implications \u003cem>—\u003c/em> from yielding new cancer therapies to correcting genetic disorders to modifying plant and animal DNA.\u003c/p>\n\u003ch3>Editing DNA Using CRISPR\u003c/h3>\n\u003cdiv class=\"row textColumnWidth\">\n\u003cdiv class=\"small-12 large-4 columns\">\u003cimg class=\"aligncenter size-full wp-image-296418\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2016/12/CRISPR01.png\" alt=\"crispr01\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR01.png 750w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR01-160x285.png 160w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR01-240x427.png 240w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR01-375x667.png 375w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR01-520x925.png 520w\" sizes=\"(max-width: 750px) 100vw, 750px\">\u003c/div>\n\u003cdiv class=\"small-12 large-4 columns\">\u003cimg class=\"aligncenter size-full wp-image-296419\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2016/12/CRISPR02.png\" alt=\"crispr02\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR02.png 750w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR02-160x285.png 160w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR02-240x427.png 240w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR02-375x667.png 375w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR02-520x925.png 520w\" sizes=\"(max-width: 750px) 100vw, 750px\">\u003c/div>\n\u003cdiv class=\"small-12 large-4 columns large-4 columns\">\u003cimg class=\"aligncenter size-full wp-image-296420\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2016/12/CRISPR_03_Bug.jpg\" alt=\"crispr_03_bug\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR_03_Bug.jpg 375w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR_03_Bug-160x285.jpg 160w, https://ww2.kqed.org/app/uploads/sites/13/2016/12/CRISPR_03_Bug-240x427.jpg 240w\" sizes=\"(max-width: 375px) 100vw, 375px\">\u003c/div>\n\u003c/div>\n\u003cp>\"If Berkeley wins it means they’re essentially going to control which commercial companies are able to develop the technology going forward and that will be a huge change in the status quo,\" says New York Law School professor says Jake Sherkow.\u003c/p>\n\u003cp>Sherkow says billions of dollars could be at stake. Companies that use the technology will likely need to pay royalties to whomever owns it -- Broad or Berkeley. Many start-ups including \u003ca href=\"https://www.addgene.org/\" target=\"_blank\">Addgene\u003c/a> -- a biotech non-profit working with Broad -- are already using CRISPR.\u003c/p>\n\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"ad","attributes":{"named":{"label":"fullwidth"},"numeric":["fullwidth"]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\n\u003cp>Both UC Berkeley and the Broad Institute of MIT and Harvard claim to have invented the technology and filed for patents.\u003cem> \u003c/em>\u003c/p>\n\u003cp>Berkeley filed first and even though Broad filed later, Broad fast-tracked its application, which was approved while Berkeley's was still pending. This is what Berkeley is contesting -- the fact that Broad's application was approved while its own was still under review.\u003c/p>\n\u003cp>The court has already ruled that the patent will be issued to the scientists who prove the CRISPR technology works in transforming the genes of a particular kind of cell -- \u003cem>eukaryotic\u003c/em> cells, those found in plants and animals, including humans.\u003c/p>\n\u003cp>In their research, Broad Institute bioengineer Feng Zhang and his team used eukaryotic cells, whereas UC Berkeley biochemist Jennifer Doudna used bacteria cells, not eukaryotic cells.\u003c/p>\n\u003cp>However, Doudna argues that UC Berkeley has a right to the patent because any skilled scientist could apply her team's research technique to eukaryotic cells.\u003c/p>\n\u003cp>Berkeley's lawyers faced more intense questioning on Tuesday than the Broad's.\u003c/p>\n\u003cp>\"It does speak to some of the judges' skepticism of the University of California's claims,\" says Sherkow.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp>The patent judges could make a decision by February but Sherkow says whichever side loses will likely appeal. So a final resolution may not come until 2018 or 2019.\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/295997/billions-at-stake-uc-berkeley-gets-day-in-court-vs-harvardmit","authors":["5432"],"categories":["futureofyou_452","futureofyou_73","futureofyou_1064"],"tags":["futureofyou_91","futureofyou_94","futureofyou_790","futureofyou_80","futureofyou_89"],"featImg":"futureofyou_297041","label":"futureofyou"},"futureofyou_370":{"type":"posts","id":"futureofyou_370","meta":{"index":"posts_1591205157","site":"futureofyou","id":"370","score":null,"sort":[1451462462000]},"guestAuthors":[],"slug":"a-crispr-solution-to-bubble-boy-disease","title":"CRISPR: What You Need to Know About the Medical Science 'Breakthrough of the Year'","publishDate":1451462462,"format":"audio","headTitle":"Future of You | KQED Future of You | KQED Science","labelTerm":{"term":54,"site":"futureofyou"},"content":"\u003cp>http://www.kqed.org/.stream/anon/radio/science/2015/03/20150313ScienceCRISPR.mp3\u003c/p>\n\u003cp>\u003cem>Editor's Note: One of our most fascinating stories of 2015 was this one, about a dramatic breakthrough that could help children suffering from the tragic illness known as \"Bubble-Boy Disease.\" This breakthrough in gene editing, using a tool called CRISPR, is \u003ca href=\"http://www.npr.org/sections/health-shots/2015/12/28/460705645/gene-editing-tool-hailed-as-a-breakthrough-and-it-really-is-one\" target=\"_blank\">revolutionizing medical research\u003c/a>. Science magazine has \u003ca href=\"http://news.sciencemag.org/scientific-community/2015/12/and-science-s-breakthrough-year\" target=\"_blank\">named CRISPR\u003c/a> the 2015 Breakthrough of the Year. We present this story again here, in case you missed it in March.\u003c/em>\u003c/p>\n\u003cp>They named him Phoenix because he was born five weeks early while his parents were on vacation, and spent his first few weeks in an incubator. Kristen and Patrick Wilkinson thought they knew exactly which ashes their son might soon rise from.\u003c/p>\n\u003cp>But when they got him home to San Francisco things just got worse, Kristen says.\u003c/p>\n\u003cp>Phoenix wasn’t gaining weight. He had a persistent skin rash. Eventually he was admitted to \u003ca href=\"https://www.ucsfbenioffchildrens.org/\">UCSF Benioff Children's Hospital\u003c/a> with a diagnosis of “failure to thrive.”\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>Phoenix had been born in Kentucky, a state where, unlike in California, infants are not routinely screened for a disease called SCID -- Severe Combined Immunodeficiency. So at first, California doctors puzzled over what might be wrong with him.\u003c/p>\n\u003cfigure id=\"attachment_384\" class=\"wp-caption alignleft\" style=\"max-width: 669px\">\u003ca href=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/ucsf-8.jpg\">\u003cimg class=\"size-medium wp-image-384\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/ucsf-8-669x600.jpg\" alt=\"\" width=\"669\" height=\"600\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2015/03/ucsf-8-669x600.jpg 669w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/ucsf-8-400x359.jpg 400w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/ucsf-8-768x689.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/ucsf-8-320x287.jpg 320w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/ucsf-8.jpg 1024w\" sizes=\"(max-width: 669px) 100vw, 669px\">\u003c/a>\u003cfigcaption class=\"wp-caption-text\">Phoenix was lucky: His doctors found a good match for a bone marrow transplant. But he still had to undergo chemotherapy. Researchers say CRISPR could lead to better treatments. (Mark Andrew Boyer/KQED)\u003c/figcaption>\u003c/figure>\n\u003cp>But Dr. Jennifer Puck, a SCID specialist at UCSF who pioneered the screening test, says Phoenix’s rash was the hallmark of an almost completely deficient immune system.\u003c/p>\n\u003cp>(The Jeffrey Modell Foundation maintains a map of states that have implemented infant SCID screening (\u003ca href=\"http://downloads.info4pi.org/pdfs/JMF_International_Map_3-9-2015.pdf\">PDF\u003c/a>), as well as a \u003ca href=\"http://www.info4pi.org/town-hall/newborn-screening\">list of states\u003c/a>.)\u003c/p>\n\u003cp>“The baby’s rogue [immune] cells were attacking his own skin,” says Puck.\u003c/p>\n\u003cp>Left unchecked, she says, SCID is fatal. “Babies who don’t have this immunity start out getting one infection after another and can’t get over any of them.”\u003c/p>\n\u003cp>\u003cstrong>From Bubbles to Transplants\u003c/strong>\u003c/p>\n\u003cp>A generation ago, SCID was commonly known as “bubble boy disease,” named after the kids (including one portrayed by John Travolta in a 1976 \u003ca href=\"http://en.wikipedia.org/wiki/The_Boy_in_the_Plastic_Bubble\">made-for-TV movie\u003c/a>) who spent their lives in plastic bubbles to shield them from germs.\u003c/p>\n\u003cp>Nowadays SCID babies receive bone marrow transplants. Healthy donors contribute bone marrow capable of producing infection-fighting white blood cells.\u003c/p>\n\u003cp>Transplanted into the blood stream of a SCID baby, the donated cells take up residence in the infant’s bone marrow and begin building a functional immune system.\u003c/p>\n\u003cp>But transplants are tough on babies and their families.\u003c/p>\n\u003cp>It can be difficult to find a good donor match. And even once one is found, most SCID babies must undergo chemotherapy to prepare their bodies for the transplanted cells.\u003c/p>\n\u003cfigure id=\"attachment_386\" class=\"wp-caption alignright\" style=\"max-width: 442px\">\u003ca href=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Phoenix-baby.jpg\">\u003cimg class=\" wp-image-386\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Phoenix-baby-600x600.jpg\" alt=\"\" width=\"442\" height=\"442\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-600x600.jpg 600w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-400x400.jpg 400w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-768x768.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-320x320.jpg 320w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-32x32.jpg 32w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-64x64.jpg 64w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-96x96.jpg 96w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-128x128.jpg 128w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-75x75.jpg 75w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby.jpg 1024w\" sizes=\"(max-width: 442px) 100vw, 442px\">\u003c/a>\u003cfigcaption class=\"wp-caption-text\">Phoenix is back at home now and doing \"great,\" says his mom, Kristen Wilkinson. (Photo courtesy Kristen Wilkinson)\u003c/figcaption>\u003c/figure>\n\u003cp>Kristen Wilkinson says the chemo was the hardest part.\u003c/p>\n\u003cp>Phoenix had painful sores on the inside of his mouth. He stopped breastfeeding, had to be fed intravenously.\u003c/p>\n\u003cp>“He was throwing up, really nauseous,” she says. “Anytime he was awake he was screaming. He never was awake and not screaming.”\u003c/p>\n\u003cp>\u003cstrong>Hope for Future Treatment\u003c/strong>\u003c/p>\n\u003cp>Today Phoenix is six months old, home with his parents and doing well. But Dr. Puck and others want better treatment for babies like him.\u003c/p>\n\u003cp>The best idea, says Puck, would be to fix the SCID mutation in Phoenix’s own cells: Take them out, reprogram them so that they can make a functional immune system, and then put them back into his body.\u003c/p>\n\u003cp>Phoenix would be his own donor. There would be no search for a donor match, no fear of rejection, no chemo.\u003c/p>\n\u003cp>“Actually just correcting the spelling mistake of the gene in the person, and not putting in any new genetic material is very attractive,” Puck says. \"That should be safer.”\u003c/p>\n\u003cp>Gene therapy, as this is called, is not a new strategy. But it’s proved to be a challenging one.\u003c/p>\n\u003cp>One way to explain why is to picture a string of Christmas tree lights containing one broken bulb.\u003c/p>\n\u003cp>\u003cstrong>A Promising Treatment Backfires\u003c/strong>\u003c/p>\n\u003cp>That string is the DNA, wound up inside the nucleus of a cell. The broken bulb is the mutation.\u003c/p>\n\u003cp>Say you want to replace the broken bulb, but the string is too small to see. How do you know you’re taking out the right bulb and putting a new one in the correct place?\u003c/p>\n\u003cp>In the 1990s, doctors in Europe tried this kind of fix on a group of babies with SCID, with mixed results.\u003c/p>\n\u003cp>“In placing the correct copy of the gene into a chromosome, we couldn’t direct where it was going to land,” says Puck. “And sometimes it landed in a dangerous spot.”\u003c/p>\n\u003cp>The “dangerous spot” in this case, was next to a growth factor gene that was inadvertently turned on by the insertion.\u003c/p>\n\u003cp>“Growth factor genes incorrectly turned on are called oncogenes,” explains Puck. “Cancer genes.”\u003c/p>\n\u003cp>Five children got Leukemia, induced by a treatment that had been intended to save their lives.\u003c/p>\n\u003cp>Four of them recovered. One baby died. Puck says it was a devastating time for the entire field.\u003c/p>\n\u003cp>“The whole world mourned the loss of that baby,” she says.\u003c/p>\n\u003cp>\u003cstrong>Enter CRISPR\u003c/strong>\u003c/p>\n\u003cp>Today, Puck is excited about a new tool for making precise changes in a baby’s genome. It’s called CRISPR, an acronym for “clustered regularly interspaced short palindromic repeats.”\u003c/p>\n\u003cp>As UC Berkeley biologist Jennifer Doudna puts it, think of CRISPR as “a molecular scalpel.”\u003c/p>\n\u003cfigure id=\"attachment_389\" class=\"wp-caption alignleft\" style=\"max-width: 427px\">\u003ca href=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Cas9_CRISPR.jpg\">\u003cimg class=\"size-full wp-image-389\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Cas9_CRISPR.jpg\" alt=\"The enzyme Cas9, shown in blue and gray, can cut DNA, shown in gold, at selected sites. The enzyme can be programmed to snip out mutated DNA and replace it with healthy DNA. This model was created from electron microscope images. (David Taylor and Jennifer Doudna/UC Berkeley)\" width=\"427\" height=\"477\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2015/03/Cas9_CRISPR.jpg 427w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Cas9_CRISPR-400x447.jpg 400w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Cas9_CRISPR-320x357.jpg 320w\" sizes=\"(max-width: 427px) 100vw, 427px\">\u003c/a>\u003cfigcaption class=\"wp-caption-text\">The enzyme Cas9, shown in blue and gray, can cut DNA, shown in gold, at selected sites. The enzyme can be programmed to snip out mutated DNA and replace it with healthy DNA. This model was created from electron microscope images. (David Taylor and Jennifer Doudna/UC Berkeley)\u003c/figcaption>\u003c/figure>\n\u003cp>Doudna isn’t a medical doctor, or even a medical researcher. She studies bacteria -- more specifically, an enzyme, called Cas9, produced by bacteria.\u003c/p>\n\u003cp>Previous scientists had noticed that these bacteria possess an unusually effective way of fighting off viruses. The bacteria were able to recognize viral DNA and use Cas9 to precisely disable the virus.\u003c/p>\n\u003cp>In 2012, Doudna and collaborators, including \u003ca href=\"http://www.helmholtz-hzi.de/en/research/research_topics/bacterial_and_viral_pathogens/regulation_in_infection_biology/e_charpentier/\">Emmanuelle Charpentier\u003c/a>, took this a step further \u003ca href=\"http://www.sciencemag.org/content/337/6096/816.abstract?sid=3750cfc2-3490-4c65-ab99-fb2a2b6099bc\">in a paper\u003c/a> in the journal Science that thrilled cell biologists around the world.\u003c/p>\n\u003cp>The paper showed that biologists could program Cas9 -- arm it, in other words -- with a customized DNA payload.\u003c/p>\n\u003cp>They could direct the enzyme at virtually any cell, programmed to make whatever genetic change they wanted.\u003c/p>\n\u003cp>In other words, says Doudna, “scientists can go into the DNA and make a precise change that corrects the mutations that give rise to disease.\"\u003c/p>\n\u003cp>\u003cstrong>A “State of Wonder”\u003c/strong>\u003c/p>\n\u003cp>Fixing the DNA in a cell: that idea had been around for a while. But CRISPR works dramatically better than the old ways. It’s more accurate, faster. A process that could take years of trial and error now took an inexpensive few weeks.\u003c/p>\n\u003cp>“Many scientists are just -- they're sort of in a state of wonder about this,” Doudna says.\u003c/p>\n\u003caside class=\"pullquote alignright\">“CRISPR works in almost every organism it's been tried in.”\u003ccite>George Church, Harvard geneticist\u003c/cite>\u003c/aside>\n\u003cp>On the day we met, Doudna was showing visitors around a brand-new research center at UC Berkeley, the \u003ca href=\"http://innovativegenomics.org/\">Innovative Genomics Initiative\u003c/a>, which is devoted to CRISPR research.\u003c/p>\n\u003cp>There are now several such centers around the world and hundreds of researchers working with CRISPR on the cells of mice, cows, monkeys, plants and humans.\u003c/p>\n\u003cp>“CRISPR works in almost every organism it’s been tried in,” says Harvard geneticist George Church. “Dozens.”\u003c/p>\n\u003cp>Church is quick to point out that there are currently no CRISPR-based treatments in clinical trials. The technology is too new.\u003c/p>\n\u003cp>He believes this could change in a few years, though for which disease, it’s hard to say.\u003c/p>\n\u003cp>Church reels off a few of what he thinks might be early contenders: “Sickle cell anemia, clotting diseases, muscular dystrophies, blindness.”\u003c/p>\n\u003cp>\u003cstrong>“Oodles and Oodles of Money”\u003c/strong>\u003c/p>\n\u003cp>If CRISPR is a big deal for people who have these diseases, it’ll also be a big financial deal for the scientists who helped develop it.\u003c/p>\n\u003cp>And by big, Jacob Sherkow, an associate professor at New York Law School and author of a recent \u003ca href=\"http://www.nature.com/nbt/journal/v33/n3/full/nbt.3160.html\">paper\u003c/a> on CRISPR in Nature Biotechnology, “big” means “oodles and oodles of money. Potentially in the $200 billion to $300 billion range.”\u003c/p>\n\u003cp>\u003ca href=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Crispr-Publications.jpg\">\u003cimg class=\"alignleft size-medium wp-image-392\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Crispr-Publications-322x600.jpg\" alt=\"Crispr-Publications\" width=\"322\" height=\"600\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2015/03/Crispr-Publications-322x600.jpg 322w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Crispr-Publications-400x745.jpg 400w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Crispr-Publications-320x596.jpg 320w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Crispr-Publications.jpg 550w\" sizes=\"(max-width: 322px) 100vw, 322px\">\u003c/a>This windfall, he says, could go to whoever holds key patents on CRISPR.\u003c/p>\n\u003cp>As has been \u003ca href=\"http://www.technologyreview.com/featuredstory/532796/who-owns-the-biggest-biotech-discovery-of-the-century/\">widely\u003c/a> \u003ca href=\"https://www.genomeweb.com/rnai/crispr-cas9-technology-sets-take-uncertainty-swirls-around-ip-landscape\">reported\u003c/a>, even though the University of California filed Doudna and Charpentier's application first, a key CRISPR patent went to another researcher -- Feng Zhang at MIT’s Broad Institute, which filed afterwards, but paid an additional fee to have its application expedited.\u003c/p>\n\u003cp>On the subject of CRISPR patents, Doudna says only that she is “very confident in the UC position.”\u003c/p>\n\u003cp>If Zhang’s patent prevails, Doudna and her collaborators could find themselves in the position of having to license a technology they are widely credited with discovering.\u003c/p>\n\u003cp>“We’re very confident that we’re going to be able to do incredible science,\" she says, \"and we’ll let the dispute play out as it will.”\u003c/p>\n\u003cp>Sherkow says there are a number of ways this dispute could play out.\u003c/p>\n\u003cp>The US Patent Office could deny UC’s application and let Zhang's patent stand. Or it could reverse its earlier decision, revoking the Broad patent and awarding it to Doudna and her collaborators instead.\u003c/p>\n\u003cp>Doudna and Zhang both point to a third possibility: that among what will likely be dozens of CRISPR patents it could be difficult, at this point, to know which one will be most lucrative or important.\u003c/p>\n\u003cp>These early CRISPR patents, says Zhang “are just the first ones. There will be many more.”\u003c/p>\n\u003cp>There’s also a fourth possibility: that CRISPR could disappoint everyone, a “tempest in the teapot,” as George Church puts it.\u003c/p>\n\u003cp>[ad floatright]\u003c/p>\n\u003cp>\"But I hope that’s not the case,” he says, laughing.\u003c/p>\n\n","blocks":[],"excerpt":"This hot new gene-editing technique may help researchers find new cures for disease. Here's your primer for 2016 and beyond. ","status":"publish","parent":0,"modified":1477272814,"stats":{"hasAudio":true,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":69,"wordCount":1763},"headData":{"title":"CRISPR: What You Need to Know About the Medical Science 'Breakthrough of the Year' | KQED","description":"This hot new gene-editing technique may help researchers find new cures for disease. Here's your primer for 2016 and beyond. ","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"CRISPR: What You Need to Know About the Medical Science 'Breakthrough of the Year'","datePublished":"2015-12-30T08:01:02.000Z","dateModified":"2016-10-24T01:33:34.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"370 http://ww2.kqed.org/futureofyou/?p=370","disqusUrl":"https://ww2.kqed.org/futureofyou/2015/12/30/a-crispr-solution-to-bubble-boy-disease/","disqusTitle":"CRISPR: What You Need to Know About the Medical Science 'Breakthrough of the Year'","customPermalink":"CRISPR%3A-What-You-Need-to-Know-About-the-Medical-Science-%27Breakthrough-of-the-Year%27","path":"/futureofyou/370/a-crispr-solution-to-bubble-boy-disease","audioUrl":"http://www.kqed.org/.stream/anon/radio/science/2015/03/20150313ScienceCRISPR.mp3","audioDuration":null,"audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003c/p>\u003c/div>","attributes":{"named":{},"numeric":[]}},{"type":"component","content":"","name":"audioLink","attributes":{"named":{"src":"http://www.kqed.org/.stream/anon/radio/science/2015/03/20150313ScienceCRISPR.mp3"},"numeric":[]}},{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp>\u003cem>Editor's Note: One of our most fascinating stories of 2015 was this one, about a dramatic breakthrough that could help children suffering from the tragic illness known as \"Bubble-Boy Disease.\" This breakthrough in gene editing, using a tool called CRISPR, is \u003ca href=\"http://www.npr.org/sections/health-shots/2015/12/28/460705645/gene-editing-tool-hailed-as-a-breakthrough-and-it-really-is-one\" target=\"_blank\">revolutionizing medical research\u003c/a>. Science magazine has \u003ca href=\"http://news.sciencemag.org/scientific-community/2015/12/and-science-s-breakthrough-year\" target=\"_blank\">named CRISPR\u003c/a> the 2015 Breakthrough of the Year. We present this story again here, in case you missed it in March.\u003c/em>\u003c/p>\n\u003cp>They named him Phoenix because he was born five weeks early while his parents were on vacation, and spent his first few weeks in an incubator. Kristen and Patrick Wilkinson thought they knew exactly which ashes their son might soon rise from.\u003c/p>\n\u003cp>But when they got him home to San Francisco things just got worse, Kristen says.\u003c/p>\n\u003cp>Phoenix wasn’t gaining weight. He had a persistent skin rash. Eventually he was admitted to \u003ca href=\"https://www.ucsfbenioffchildrens.org/\">UCSF Benioff Children's Hospital\u003c/a> with a diagnosis of “failure to thrive.”\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>Phoenix had been born in Kentucky, a state where, unlike in California, infants are not routinely screened for a disease called SCID -- Severe Combined Immunodeficiency. So at first, California doctors puzzled over what might be wrong with him.\u003c/p>\n\u003cfigure id=\"attachment_384\" class=\"wp-caption alignleft\" style=\"max-width: 669px\">\u003ca href=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/ucsf-8.jpg\">\u003cimg class=\"size-medium wp-image-384\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/ucsf-8-669x600.jpg\" alt=\"\" width=\"669\" height=\"600\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2015/03/ucsf-8-669x600.jpg 669w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/ucsf-8-400x359.jpg 400w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/ucsf-8-768x689.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/ucsf-8-320x287.jpg 320w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/ucsf-8.jpg 1024w\" sizes=\"(max-width: 669px) 100vw, 669px\">\u003c/a>\u003cfigcaption class=\"wp-caption-text\">Phoenix was lucky: His doctors found a good match for a bone marrow transplant. But he still had to undergo chemotherapy. Researchers say CRISPR could lead to better treatments. (Mark Andrew Boyer/KQED)\u003c/figcaption>\u003c/figure>\n\u003cp>But Dr. Jennifer Puck, a SCID specialist at UCSF who pioneered the screening test, says Phoenix’s rash was the hallmark of an almost completely deficient immune system.\u003c/p>\n\u003cp>(The Jeffrey Modell Foundation maintains a map of states that have implemented infant SCID screening (\u003ca href=\"http://downloads.info4pi.org/pdfs/JMF_International_Map_3-9-2015.pdf\">PDF\u003c/a>), as well as a \u003ca href=\"http://www.info4pi.org/town-hall/newborn-screening\">list of states\u003c/a>.)\u003c/p>\n\u003cp>“The baby’s rogue [immune] cells were attacking his own skin,” says Puck.\u003c/p>\n\u003cp>Left unchecked, she says, SCID is fatal. “Babies who don’t have this immunity start out getting one infection after another and can’t get over any of them.”\u003c/p>\n\u003cp>\u003cstrong>From Bubbles to Transplants\u003c/strong>\u003c/p>\n\u003cp>A generation ago, SCID was commonly known as “bubble boy disease,” named after the kids (including one portrayed by John Travolta in a 1976 \u003ca href=\"http://en.wikipedia.org/wiki/The_Boy_in_the_Plastic_Bubble\">made-for-TV movie\u003c/a>) who spent their lives in plastic bubbles to shield them from germs.\u003c/p>\n\u003cp>Nowadays SCID babies receive bone marrow transplants. Healthy donors contribute bone marrow capable of producing infection-fighting white blood cells.\u003c/p>\n\u003cp>Transplanted into the blood stream of a SCID baby, the donated cells take up residence in the infant’s bone marrow and begin building a functional immune system.\u003c/p>\n\u003cp>But transplants are tough on babies and their families.\u003c/p>\n\u003cp>It can be difficult to find a good donor match. And even once one is found, most SCID babies must undergo chemotherapy to prepare their bodies for the transplanted cells.\u003c/p>\n\u003cfigure id=\"attachment_386\" class=\"wp-caption alignright\" style=\"max-width: 442px\">\u003ca href=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Phoenix-baby.jpg\">\u003cimg class=\" wp-image-386\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Phoenix-baby-600x600.jpg\" alt=\"\" width=\"442\" height=\"442\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-600x600.jpg 600w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-400x400.jpg 400w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-768x768.jpg 768w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-320x320.jpg 320w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-32x32.jpg 32w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-64x64.jpg 64w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-96x96.jpg 96w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-128x128.jpg 128w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby-75x75.jpg 75w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Phoenix-baby.jpg 1024w\" sizes=\"(max-width: 442px) 100vw, 442px\">\u003c/a>\u003cfigcaption class=\"wp-caption-text\">Phoenix is back at home now and doing \"great,\" says his mom, Kristen Wilkinson. (Photo courtesy Kristen Wilkinson)\u003c/figcaption>\u003c/figure>\n\u003cp>Kristen Wilkinson says the chemo was the hardest part.\u003c/p>\n\u003cp>Phoenix had painful sores on the inside of his mouth. He stopped breastfeeding, had to be fed intravenously.\u003c/p>\n\u003cp>“He was throwing up, really nauseous,” she says. “Anytime he was awake he was screaming. He never was awake and not screaming.”\u003c/p>\n\u003cp>\u003cstrong>Hope for Future Treatment\u003c/strong>\u003c/p>\n\u003cp>Today Phoenix is six months old, home with his parents and doing well. But Dr. Puck and others want better treatment for babies like him.\u003c/p>\n\u003cp>The best idea, says Puck, would be to fix the SCID mutation in Phoenix’s own cells: Take them out, reprogram them so that they can make a functional immune system, and then put them back into his body.\u003c/p>\n\u003cp>Phoenix would be his own donor. There would be no search for a donor match, no fear of rejection, no chemo.\u003c/p>\n\u003cp>“Actually just correcting the spelling mistake of the gene in the person, and not putting in any new genetic material is very attractive,” Puck says. \"That should be safer.”\u003c/p>\n\u003cp>Gene therapy, as this is called, is not a new strategy. But it’s proved to be a challenging one.\u003c/p>\n\u003cp>One way to explain why is to picture a string of Christmas tree lights containing one broken bulb.\u003c/p>\n\u003cp>\u003cstrong>A Promising Treatment Backfires\u003c/strong>\u003c/p>\n\u003cp>That string is the DNA, wound up inside the nucleus of a cell. The broken bulb is the mutation.\u003c/p>\n\u003cp>Say you want to replace the broken bulb, but the string is too small to see. How do you know you’re taking out the right bulb and putting a new one in the correct place?\u003c/p>\n\u003cp>In the 1990s, doctors in Europe tried this kind of fix on a group of babies with SCID, with mixed results.\u003c/p>\n\u003cp>“In placing the correct copy of the gene into a chromosome, we couldn’t direct where it was going to land,” says Puck. “And sometimes it landed in a dangerous spot.”\u003c/p>\n\u003cp>The “dangerous spot” in this case, was next to a growth factor gene that was inadvertently turned on by the insertion.\u003c/p>\n\u003cp>“Growth factor genes incorrectly turned on are called oncogenes,” explains Puck. “Cancer genes.”\u003c/p>\n\u003cp>Five children got Leukemia, induced by a treatment that had been intended to save their lives.\u003c/p>\n\u003cp>Four of them recovered. One baby died. Puck says it was a devastating time for the entire field.\u003c/p>\n\u003cp>“The whole world mourned the loss of that baby,” she says.\u003c/p>\n\u003cp>\u003cstrong>Enter CRISPR\u003c/strong>\u003c/p>\n\u003cp>Today, Puck is excited about a new tool for making precise changes in a baby’s genome. It’s called CRISPR, an acronym for “clustered regularly interspaced short palindromic repeats.”\u003c/p>\n\u003cp>As UC Berkeley biologist Jennifer Doudna puts it, think of CRISPR as “a molecular scalpel.”\u003c/p>\n\u003cfigure id=\"attachment_389\" class=\"wp-caption alignleft\" style=\"max-width: 427px\">\u003ca href=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Cas9_CRISPR.jpg\">\u003cimg class=\"size-full wp-image-389\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Cas9_CRISPR.jpg\" alt=\"The enzyme Cas9, shown in blue and gray, can cut DNA, shown in gold, at selected sites. The enzyme can be programmed to snip out mutated DNA and replace it with healthy DNA. This model was created from electron microscope images. (David Taylor and Jennifer Doudna/UC Berkeley)\" width=\"427\" height=\"477\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2015/03/Cas9_CRISPR.jpg 427w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Cas9_CRISPR-400x447.jpg 400w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Cas9_CRISPR-320x357.jpg 320w\" sizes=\"(max-width: 427px) 100vw, 427px\">\u003c/a>\u003cfigcaption class=\"wp-caption-text\">The enzyme Cas9, shown in blue and gray, can cut DNA, shown in gold, at selected sites. The enzyme can be programmed to snip out mutated DNA and replace it with healthy DNA. This model was created from electron microscope images. (David Taylor and Jennifer Doudna/UC Berkeley)\u003c/figcaption>\u003c/figure>\n\u003cp>Doudna isn’t a medical doctor, or even a medical researcher. She studies bacteria -- more specifically, an enzyme, called Cas9, produced by bacteria.\u003c/p>\n\u003cp>Previous scientists had noticed that these bacteria possess an unusually effective way of fighting off viruses. The bacteria were able to recognize viral DNA and use Cas9 to precisely disable the virus.\u003c/p>\n\u003cp>In 2012, Doudna and collaborators, including \u003ca href=\"http://www.helmholtz-hzi.de/en/research/research_topics/bacterial_and_viral_pathogens/regulation_in_infection_biology/e_charpentier/\">Emmanuelle Charpentier\u003c/a>, took this a step further \u003ca href=\"http://www.sciencemag.org/content/337/6096/816.abstract?sid=3750cfc2-3490-4c65-ab99-fb2a2b6099bc\">in a paper\u003c/a> in the journal Science that thrilled cell biologists around the world.\u003c/p>\n\u003cp>The paper showed that biologists could program Cas9 -- arm it, in other words -- with a customized DNA payload.\u003c/p>\n\u003cp>They could direct the enzyme at virtually any cell, programmed to make whatever genetic change they wanted.\u003c/p>\n\u003cp>In other words, says Doudna, “scientists can go into the DNA and make a precise change that corrects the mutations that give rise to disease.\"\u003c/p>\n\u003cp>\u003cstrong>A “State of Wonder”\u003c/strong>\u003c/p>\n\u003cp>Fixing the DNA in a cell: that idea had been around for a while. But CRISPR works dramatically better than the old ways. It’s more accurate, faster. A process that could take years of trial and error now took an inexpensive few weeks.\u003c/p>\n\u003cp>“Many scientists are just -- they're sort of in a state of wonder about this,” Doudna says.\u003c/p>\n\u003caside class=\"pullquote alignright\">“CRISPR works in almost every organism it's been tried in.”\u003ccite>George Church, Harvard geneticist\u003c/cite>\u003c/aside>\n\u003cp>On the day we met, Doudna was showing visitors around a brand-new research center at UC Berkeley, the \u003ca href=\"http://innovativegenomics.org/\">Innovative Genomics Initiative\u003c/a>, which is devoted to CRISPR research.\u003c/p>\n\u003cp>There are now several such centers around the world and hundreds of researchers working with CRISPR on the cells of mice, cows, monkeys, plants and humans.\u003c/p>\n\u003cp>“CRISPR works in almost every organism it’s been tried in,” says Harvard geneticist George Church. “Dozens.”\u003c/p>\n\u003cp>Church is quick to point out that there are currently no CRISPR-based treatments in clinical trials. The technology is too new.\u003c/p>\n\u003cp>He believes this could change in a few years, though for which disease, it’s hard to say.\u003c/p>\n\u003cp>Church reels off a few of what he thinks might be early contenders: “Sickle cell anemia, clotting diseases, muscular dystrophies, blindness.”\u003c/p>\n\u003cp>\u003cstrong>“Oodles and Oodles of Money”\u003c/strong>\u003c/p>\n\u003cp>If CRISPR is a big deal for people who have these diseases, it’ll also be a big financial deal for the scientists who helped develop it.\u003c/p>\n\u003cp>And by big, Jacob Sherkow, an associate professor at New York Law School and author of a recent \u003ca href=\"http://www.nature.com/nbt/journal/v33/n3/full/nbt.3160.html\">paper\u003c/a> on CRISPR in Nature Biotechnology, “big” means “oodles and oodles of money. Potentially in the $200 billion to $300 billion range.”\u003c/p>\n\u003cp>\u003ca href=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Crispr-Publications.jpg\">\u003cimg class=\"alignleft size-medium wp-image-392\" src=\"http://ww2.kqed.org/futureofyou/wp-content/uploads/sites/13/2015/03/Crispr-Publications-322x600.jpg\" alt=\"Crispr-Publications\" width=\"322\" height=\"600\" srcset=\"https://ww2.kqed.org/app/uploads/sites/13/2015/03/Crispr-Publications-322x600.jpg 322w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Crispr-Publications-400x745.jpg 400w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Crispr-Publications-320x596.jpg 320w, https://ww2.kqed.org/app/uploads/sites/13/2015/03/Crispr-Publications.jpg 550w\" sizes=\"(max-width: 322px) 100vw, 322px\">\u003c/a>This windfall, he says, could go to whoever holds key patents on CRISPR.\u003c/p>\n\u003cp>As has been \u003ca href=\"http://www.technologyreview.com/featuredstory/532796/who-owns-the-biggest-biotech-discovery-of-the-century/\">widely\u003c/a> \u003ca href=\"https://www.genomeweb.com/rnai/crispr-cas9-technology-sets-take-uncertainty-swirls-around-ip-landscape\">reported\u003c/a>, even though the University of California filed Doudna and Charpentier's application first, a key CRISPR patent went to another researcher -- Feng Zhang at MIT’s Broad Institute, which filed afterwards, but paid an additional fee to have its application expedited.\u003c/p>\n\u003cp>On the subject of CRISPR patents, Doudna says only that she is “very confident in the UC position.”\u003c/p>\n\u003cp>If Zhang’s patent prevails, Doudna and her collaborators could find themselves in the position of having to license a technology they are widely credited with discovering.\u003c/p>\n\u003cp>“We’re very confident that we’re going to be able to do incredible science,\" she says, \"and we’ll let the dispute play out as it will.”\u003c/p>\n\u003cp>Sherkow says there are a number of ways this dispute could play out.\u003c/p>\n\u003cp>The US Patent Office could deny UC’s application and let Zhang's patent stand. Or it could reverse its earlier decision, revoking the Broad patent and awarding it to Doudna and her collaborators instead.\u003c/p>\n\u003cp>Doudna and Zhang both point to a third possibility: that among what will likely be dozens of CRISPR patents it could be difficult, at this point, to know which one will be most lucrative or important.\u003c/p>\n\u003cp>These early CRISPR patents, says Zhang “are just the first ones. There will be many more.”\u003c/p>\n\u003cp>There’s also a fourth possibility: that CRISPR could disappoint everyone, a “tempest in the teapot,” as George Church puts it.\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>\"But I hope that’s not the case,” he says, laughing.\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/370/a-crispr-solution-to-bubble-boy-disease","authors":["210"],"programs":["futureofyou_54"],"categories":["futureofyou_1064"],"tags":["futureofyou_91","futureofyou_896","futureofyou_93","futureofyou_94","futureofyou_90","futureofyou_88","futureofyou_80","futureofyou_89"],"featImg":"futureofyou_380","label":"futureofyou_54"},"futureofyou_6154":{"type":"posts","id":"futureofyou_6154","meta":{"index":"posts_1591205157","site":"futureofyou","id":"6154","score":null,"sort":[1435170302000]},"guestAuthors":[],"slug":"google-and-broad-institute-team-up-to-bring-genomic-analysis-to-the-cloud","title":"Google and Broad Institute Team Up to Bring Genomic Analysis to the Cloud","publishDate":1435170302,"format":"standard","headTitle":"KQED Future of You | KQED Science","labelTerm":{"site":"futureofyou"},"content":"\u003cp class=\"p1\">\u003ca href=\"google.com\">Google\u003c/a> has teamed up with one of the world's top genomics centers, the \u003ca href=\"https://www.broadinstitute.org/\">Broad Institute of MIT\u003c/a> and Harvard, to work on a series of projects it claims will propel biomedical research.\u003c/p>\n\u003cp class=\"p1\">For the first joint project, engineers from both organizations will bring \"GATK,\" the Broad Institute's widely-used genome analysis toolkit, onto Google's cloud service and into the hands of researchers.\u003c/p>\n\u003cp class=\"p1\">\"The limiting factor is no longer getting the DNA sequenced,\" said Dr. Barry Starr, a Stanford geneticist and a contributor to KQED. \"It is now interpreting all of that information in a meaningful way.\"\u003c/p>\n\u003cp>The Broad Institute alone \u003ca href=\"http://www.technologyreview.com/news/532266/google-wants-to-store-your-genome/\">analyzed a massive 200 terabytes\u003c/a> of raw data in a single month. In the past decade, the institute has genotyped more than 1.4 million biological samples.\u003c/p>\n\u003cp>Google isn't the only tech company vying to use cloud-based technology to store and analyze this massive volume of genetic information. This is a point of competition between Google, IBM, Amazon, and Microsoft.\u003c/p>\n\u003cp>[ad fullwidth]\u003c/p>\n\u003cp>But Google is now the only public cloud provider to offer the GATK toolkit as a service. By making the software available in the cloud, researchers can run it on large data-sets without access to local computing -- and that frees up both time and resources.\u003c/p>\n\u003cp class=\"p1\">\u003cspan class=\"s1\">\"GATK was already available to researchers and tens of thousands have used the software to analyze their data,\" said Starr. \"Google adds the power of being able to handle much more data at a time.\"\u003c/span>\u003c/p>\n\u003cp class=\"p1\">\u003cspan class=\"s1\">Google Genomics' product manager Jonathan Bingham told KQED two groups will benefit most from this partnership: small research groups who lack sophisticated computing, and any individual who wants to analyze large genomic data sets without needing to download them. \u003c/span>\u003c/p>\n\u003cp class=\"p1\">\u003cspan class=\"s1\">“Broad Institute has got a tremendous amount of expertise working with large numbers of biological samples and huge volumes of genomic data,\" Bingham explained. \"Meanwhile, Google has built the infrastructure and tools to process and analyze the data and keep it secure.”\u003c/span>\u003c/p>\n\u003cp class=\"p1\">The toolkit will be available for free to nonprofits and academics. Businesses will need to \u003ca href=\"https://www.broadinstitute.org/gatk/about/#licensing\">pay to license it\u003c/a> from the Broad Institute.\u003c/p>\n\u003cp class=\"p1\">Some genetics experts say this announcement is evidence that the health industry is increasingly willing to embrace cloud computing. In the past, health organizations have been hesitant due to concerns about compliance and security.\u003c/p>\n\u003cp class=\"p1\">\"This suggests that the genomics industry has moved beyond the cloud debate,\" said Jonathan Hirsch, president and co-founder of Syapse, a Silicon Valley-based company that wants to bring more genomics data into routine clinical use.\u003c/p>\n\u003cp class=\"p1\">\"It is OK for researchers and clinicians to do genomics work in the cloud, and trust that cloud provider's hardware and software.\"\u003c/p>\n\u003cp class=\"p1\">In the future, Bingham said there may be opportunities to work on projects to further our genetic understanding of cancer and diabetes.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp class=\"p1\">But for now, he said, the organizations are focused on \"general purpose\" tools that aren't specific to a disease and can be used by researchers everywhere.\u003c/p>\n\n","blocks":[],"excerpt":"Google has teamed up with one of the world's top genomics centers, the Broad Institute of Harvard and MIT, to work on a series of projects it claims will propel biomedical research.","status":"publish","parent":0,"modified":1477281302,"stats":{"hasAudio":false,"hasVideo":false,"hasChartOrMap":false,"iframeSrcs":[],"hasGoogleForm":false,"hasGallery":false,"hasHearkenModule":false,"hasPolis":false,"paragraphCount":5,"wordCount":532},"headData":{"title":"Google and Broad Institute Team Up to Bring Genomic Analysis to the Cloud | KQED","description":"Google has teamed up with one of the world's top genomics centers, the Broad Institute of Harvard and MIT, to work on a series of projects it claims will propel biomedical research.","ogTitle":"","ogDescription":"","ogImgId":"","twTitle":"","twDescription":"","twImgId":"","schema":{"@context":"http://schema.org","@type":"Article","headline":"Google and Broad Institute Team Up to Bring Genomic Analysis to the Cloud","datePublished":"2015-06-24T18:25:02.000Z","dateModified":"2016-10-24T03:55:02.000Z","image":"https://cdn.kqed.org/wp-content/uploads/2020/02/KQED-OG-Image@1x.png"}},"disqusIdentifier":"6154 http://ww2.kqed.org/futureofyou/?p=6154","disqusUrl":"https://ww2.kqed.org/futureofyou/2015/06/24/google-and-broad-institute-team-up-to-bring-genomic-analysis-to-the-cloud/","disqusTitle":"Google and Broad Institute Team Up to Bring Genomic Analysis to the Cloud","path":"/futureofyou/6154/google-and-broad-institute-team-up-to-bring-genomic-analysis-to-the-cloud","audioTrackLength":null,"parsedContent":[{"type":"contentString","content":"\u003cdiv class=\"post-body\">\u003cp>\u003cp class=\"p1\">\u003ca href=\"google.com\">Google\u003c/a> has teamed up with one of the world's top genomics centers, the \u003ca href=\"https://www.broadinstitute.org/\">Broad Institute of MIT\u003c/a> and Harvard, to work on a series of projects it claims will propel biomedical research.\u003c/p>\n\u003cp class=\"p1\">For the first joint project, engineers from both organizations will bring \"GATK,\" the Broad Institute's widely-used genome analysis toolkit, onto Google's cloud service and into the hands of researchers.\u003c/p>\n\u003cp class=\"p1\">\"The limiting factor is no longer getting the DNA sequenced,\" said Dr. Barry Starr, a Stanford geneticist and a contributor to KQED. \"It is now interpreting all of that information in a meaningful way.\"\u003c/p>\n\u003cp>The Broad Institute alone \u003ca href=\"http://www.technologyreview.com/news/532266/google-wants-to-store-your-genome/\">analyzed a massive 200 terabytes\u003c/a> of raw data in a single month. In the past decade, the institute has genotyped more than 1.4 million biological samples.\u003c/p>\n\u003cp>Google isn't the only tech company vying to use cloud-based technology to store and analyze this massive volume of genetic information. This is a point of competition between Google, IBM, Amazon, and Microsoft.\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 Google is now the only public cloud provider to offer the GATK toolkit as a service. By making the software available in the cloud, researchers can run it on large data-sets without access to local computing -- and that frees up both time and resources.\u003c/p>\n\u003cp class=\"p1\">\u003cspan class=\"s1\">\"GATK was already available to researchers and tens of thousands have used the software to analyze their data,\" said Starr. \"Google adds the power of being able to handle much more data at a time.\"\u003c/span>\u003c/p>\n\u003cp class=\"p1\">\u003cspan class=\"s1\">Google Genomics' product manager Jonathan Bingham told KQED two groups will benefit most from this partnership: small research groups who lack sophisticated computing, and any individual who wants to analyze large genomic data sets without needing to download them. \u003c/span>\u003c/p>\n\u003cp class=\"p1\">\u003cspan class=\"s1\">“Broad Institute has got a tremendous amount of expertise working with large numbers of biological samples and huge volumes of genomic data,\" Bingham explained. \"Meanwhile, Google has built the infrastructure and tools to process and analyze the data and keep it secure.”\u003c/span>\u003c/p>\n\u003cp class=\"p1\">The toolkit will be available for free to nonprofits and academics. Businesses will need to \u003ca href=\"https://www.broadinstitute.org/gatk/about/#licensing\">pay to license it\u003c/a> from the Broad Institute.\u003c/p>\n\u003cp class=\"p1\">Some genetics experts say this announcement is evidence that the health industry is increasingly willing to embrace cloud computing. In the past, health organizations have been hesitant due to concerns about compliance and security.\u003c/p>\n\u003cp class=\"p1\">\"This suggests that the genomics industry has moved beyond the cloud debate,\" said Jonathan Hirsch, president and co-founder of Syapse, a Silicon Valley-based company that wants to bring more genomics data into routine clinical use.\u003c/p>\n\u003cp class=\"p1\">\"It is OK for researchers and clinicians to do genomics work in the cloud, and trust that cloud provider's hardware and software.\"\u003c/p>\n\u003cp class=\"p1\">In the future, Bingham said there may be opportunities to work on projects to further our genetic understanding of cancer and diabetes.\u003c/p>\n\u003cp>\u003c/p>\n\u003cp class=\"p1\">But for now, he said, the organizations are focused on \"general purpose\" tools that aren't specific to a disease and can be used by researchers everywhere.\u003c/p>\n\n\u003c/div>\u003c/p>","attributes":{"named":{},"numeric":[]}}],"link":"/futureofyou/6154/google-and-broad-institute-team-up-to-bring-genomic-analysis-to-the-cloud","authors":["3252"],"categories":["futureofyou_1064"],"tags":["futureofyou_465","futureofyou_91","futureofyou_120","futureofyou_196","futureofyou_131","futureofyou_453"],"featImg":"futureofyou_6396","label":"futureofyou"}},"programsReducer":{"possible":{"id":"possible","title":"Possible","info":"Possible is hosted by entrepreneur Reid Hoffman and writer Aria Finger. Together in Possible, Hoffman and Finger lead enlightening discussions about building a brighter collective future. The show features interviews with visionary guests like Trevor Noah, Sam Altman and Janette Sadik-Khan. Possible paints an optimistic portrait of the world we can create through science, policy, business, art and our shared humanity. It asks: What if everything goes right for once? How can we get there? 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You can also visit the MindShift website for episodes and supplemental blog posts or tweet us \u003ca href=\"https://twitter.com/MindShiftKQED\">@MindShiftKQED\u003c/a> or visit us at \u003ca href=\"/mindshift\">MindShift.KQED.org\u003c/a>","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Mindshift-Podcast-Tile-703x703-1.jpg","imageAlt":"KQED MindShift: How We Will Learn","officialWebsiteLink":"/mindshift/","meta":{"site":"news","source":"kqed","order":"2"},"link":"/podcasts/mindshift","subscribe":{"apple":"https://podcasts.apple.com/us/podcast/mindshift-podcast/id1078765985","google":"https://podcasts.google.com/feed/aHR0cHM6Ly9mZWVkcy5tZWdhcGhvbmUuZm0vS1FJTkM1NzY0NjAwNDI5","npr":"https://www.npr.org/podcasts/464615685/mind-shift-podcast","stitcher":"https://www.stitcher.com/podcast/kqed/stories-teachers-share","spotify":"https://open.spotify.com/show/0MxSpNYZKNprFLCl7eEtyx"}},"morning-edition":{"id":"morning-edition","title":"Morning Edition","info":"\u003cem>Morning Edition\u003c/em> takes listeners around the country and the world with multi-faceted stories and commentaries every weekday. Hosts Steve Inskeep, David Greene and Rachel Martin bring you the latest breaking news and features to prepare you for the day.","airtime":"MON-FRI 3am-9am","imageSrc":"https://cdn.kqed.org/wp-content/uploads/2024/04/Morning-Edition-Podcast-Tile-360x360-1.jpg","officialWebsiteLink":"https://www.npr.org/programs/morning-edition/","meta":{"site":"news","source":"npr"},"link":"/radio/program/morning-edition"},"onourwatch":{"id":"onourwatch","title":"On Our Watch","tagline":"Police secrets, unsealed","info":"For decades, the process for how police police themselves has been inconsistent – if not opaque. In some states, like California, these proceedings were completely hidden. After a new police transparency law unsealed scores of internal affairs files, our reporters set out to examine these cases and the shadow world of police discipline. 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