{"page":"\u003clink rel=\"stylesheet\" href=\"https://lessonplanet.com/assets/packs/css/resources-c03aa079.css\" /\u003e\n\u003clink rel=\"stylesheet\" href=\"https://lessonplanet.com/assets/packs/css/lp_boclips_stylesheets-517835be.css\" media=\"all\" /\u003e\n\u003cdiv data-title='2nd man has gene editing' data-url='/boclips/videos/5c54d71ed8eafeecae20926e' data-video-url='/boclips/videos/5c54d71ed8eafeecae20926e' id='bo_player_modal'\u003e\n\u003cdiv class='boclips-resource-page modal-dialog panel-container'\u003e\n\u003cdiv class='react-notifications-root'\u003e\u003c/div\u003e\n\u003cdiv class='rp-header'\u003e\n\u003cdiv class='rp-type'\u003e\n\u003ci aria-hidden='true' class='fai fa-regular fa-circle-play'\u003e\u003c/i\u003e\nVideo\n\u003c/div\u003e\n\u003ch1 class='rp-title' id='video-title'\u003e\n2nd man has gene editing\n\u003c/h1\u003e\n\u003cdiv class='rp-actions'\u003e\n\u003cdiv class='mr-1'\u003e\n\u003ca class=\"btn btn-success\" data-posthog-event=\"Signup: LP Signup Activity\" data-posthog-location=\"body_link_boclips\" data-remote=\"true\" href=\"/subscription/new\"\u003e\u003cspan\u003e\u003cspan\u003eGet Free Access\u003c/span\u003e\u003cspan class=\"\"\u003e for 10 Days\u003c/span\u003e\u003cspan\u003e!\u003c/span\u003e\u003c/span\u003e\u003c/a\u003e\n\u003c/div\u003e\n\u003c/div\u003e\n\u003c/div\u003e\n\u003cdiv class='rp-body'\u003e\n\u003cdiv class='rp-info'\u003e\n\u003cdiv aria-label='Hide resource details' class='rp-hide-info' role='button' tabindex='0'\u003e\u0026times;\u003c/div\u003e\n\u003ci aria-label='Expand resource details' class='rp-expand-info fai fa-solid fa-up-right-and-down-left-from-center' role='button' tabindex='0'\u003e\u003c/i\u003e\n\u003ci aria-label='Compress resource details' class='rp-compress-info fai fa-solid fa-down-left-and-up-right-to-center' role='button' tabindex='0'\u003e\u003c/i\u003e\n\u003cdiv class='rp-rating'\u003e\n\u003cspan class='resource-pool'\u003e\n\u003cspan class='pool-label'\u003ePublisher:\u003c/span\u003e\n\u003cspan class='pool-name'\u003e\n\u003cspan class='text'\u003e\u003ca data-publisher-id=\"30356011\" href=\"/search?publisher_ids%5B%5D=30356011\"\u003eCurated Video\u003c/a\u003e\u003c/span\u003e\n\u003c/span\u003e\n\u003c/span\u003e\n\u003c/div\u003e\n\u003cdiv class='rp-description'\u003e\n\u003cspan class='short-description'\u003eLEAD IN:               A second patient has been treated in an historic gene editing study in California.              And no major side effects or safety issues have emerged from the first man's treatment nearly three months ago,...\u003c/span\u003e\n\u003cspan class='full-description hide'\u003eLEAD IN: \u003cbr/\u003e              A second patient has been treated in an historic gene editing study in California.\u003cbr/\u003e              And no major side effects or safety issues have emerged from the first man's treatment nearly three months ago, doctors revealed Tuesday.\u003cbr/\u003e              Gene editing is a more precise way to do gene therapy, and aims to permanently change someone's DNA to try to cure a disease.\u003cbr/\u003e              \u003cbr/\u003e              STORY-LINE: \u003cbr/\u003e              In November, 44-year-old Brian Madeux became the first person to have gene editing inside the body for a metabolic disease called Hunter syndrome that's caused by a bad gene. Through an IV, he received many copies of a corrective gene and a genetic tool to put it in a precise spot in his DNA.\u003cbr/\u003e              Now, a second man has been treated in the same way for the same disease and according to Dr. Paul Harmatz of UCSF Benioff Children's Hospital Oakland, who treated both men, he is doing well. \u003cbr/\u003e              At a medical conference in San Diego, Harmatz reported safety results for the first six weeks after Madeux's treatment. Sangamo Therapeutics, the company that makes the gene editing tool called zinc finger nucleases, is testing them for two metabolic diseases and haemophilia, a bleeding disorder.\u003cbr/\u003e              The company said more safety information and initial results on effectiveness should come by mid-year. \u003cbr/\u003e              Gene editing aims to permanently change a person's native DNA to treat or cure a disease.  The therapy uses proteins called 'zinc fingers' to install a corrective gene.\u003cbr/\u003e              The therapy has three parts: The new gene and two zinc fingers. DNA instructions for each part are put inside a modified virus to ferry them into cells. Many copies of these are injected into the patient's bloodstream. They travel through the bloodstream to the liver.\u003cbr/\u003e              Liver cells use the DNA instructions to make the zinc fingers and prepare the corrective gene.The zinc fingers attach to a specific piece of DNA in liver cells and cut it at that spot.\u003cbr/\u003e              This allows the new gene to slip in and become a permanent part of the patient's DNA. The new gene then directs the cell to make the enzyme the patient previously lacked.\u003cbr/\u003e              Madeux had dizziness, cold sweats and weakness four days after the treatment but they went away on their own in a day, Harmatz said. Madeux also had a severe cough and a partially collapsed lung, but these were deemed unrelated to the gene therapy because he had had similar problems previously.\u003cbr/\u003e              Importantly, there were no signs of harm to his liver.\u003cbr/\u003e              The liver results were welcome news after some other recent reports caused alarm. A prominent gene therapy scientist, Dr. James Wilson of the University of Pennsylvania, published two studies reporting liver and other serious problems in monkeys and piglets that were given experimental gene therapies. Several had to be euthanized.\u003cbr/\u003e              The animal studies tested very high intravenous doses of a therapy that used a certain virus to carry the gene into cells. Relatives of this virus are widely used in human gene therapies, but Wilson said he does not believe that the results in animals have any bearing on use of lower doses, different types of the virus, or therapies given in different ways such as a shot.\u003cbr/\u003e              The results might mean it will be harder to develop gene therapies for some neuro-muscular disorders - higher doses in the animal studies were thought necessary to get the therapy into the brain and throughout muscles.\u003cbr/\u003e              The Sangamo study that Madeux is in used much lower doses of a different type of the virus.\u003cbr/\u003e              Wilson said it was important to the field that any safety concerns be published quickly. He helped lead a very early gene therapy experiment that killed a teen in 1999, putting some other studies on hold for years.\u003cbr/\u003e              An editorial in the journal Human Gene Therapy, which published one of Wilson's animal studies, said gene therapy experiments should not stop because that might deprive patients of potentially life-saving treatments.\u003cbr/\u003e              In the last year, the first gene therapies were approved in the United States to treat cancer and an inherited form of blindness.\u003cbr/\u003e            \u003cbr/\u003e\u003cbr/\u003e              ++MUTED++\u003cbr/\u003e              1.  Animation demonstrating gene editing technique called Zinc Fingers\u003c/span\u003e\n\u003c/div\u003e\n\u003cdiv class='action-container flex justify-between'\u003e\n\u003cbutton aria-expanded='false' aria-label='Read more description' class='rp-full-description' type='button'\u003e\n\u003ci class='fai fa-solid fa-align-left'\u003e\u003c/i\u003e\n\u003cspan 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