{"id":26015,"date":"2025-05-16T00:12:14","date_gmt":"2025-05-16T00:12:14","guid":{"rendered":"https:\/\/site.itshrt.com\/worldnews\/infant-becomes-worlds-first-patient-to-undergo-personalized-gene-editing-treatment\/"},"modified":"2025-05-16T00:12:14","modified_gmt":"2025-05-16T00:12:14","slug":"infant-becomes-worlds-first-patient-to-undergo-personalized-gene-editing-treatment","status":"publish","type":"post","link":"https:\/\/site.itshrt.com\/worldnews\/infant-becomes-worlds-first-patient-to-undergo-personalized-gene-editing-treatment\/","title":{"rendered":"Infant becomes world&#8217;s first patient to undergo personalized gene-editing treatment"},"content":{"rendered":"<p> <script async src=\"https:\/\/pagead2.googlesyndication.com\/pagead\/js\/adsbygoogle.js?client=ca-pub-6606220950177433\"\r\n     crossorigin=\"anonymous\"><\/script>\r\n<!-- ItShrt World News -->\r\n<ins class=\"adsbygoogle\"\r\n     style=\"display:block\"\r\n     data-ad-client=\"ca-pub-6606220950177433\"\r\n     data-ad-slot=\"1882483372\"\r\n     data-ad-format=\"auto\"\r\n     data-full-width-responsive=\"true\"><\/ins>\r\n<script>\r\n     (adsbygoogle = window.adsbygoogle || []).push({});\r\n<\/script>\r\n<br \/>\n<\/p>\n<div id=\"\">\n<p><em>Philadelphia<\/em> \u2014 Soon after KJ Muldoon was born in the summer of 2024, he was diagnosed with a rare genetic disorder that is fatal for about half the infants who are born with it.\u00a0<\/p>\n<p>Until now, the only effective long-term treatment for the rare metabolic disease known as severe Carbamoyl Phosphate Synthetase 1 deficiency, or CPS1, had been a liver transplant. <\/p>\n<p>Instead, doctors at the Children&#8217;s Hospital of Philadelphia told KJ&#8217;s family they could try something never done before. They would use a technology known as CRISPR, a personalized gene-editing therapy, to find the one uniquely mutated gene out of 20,000 in his little body, and fix it. <\/p>\n<p>KJ subsequently received three infusions of the experimental therapy to his liver earlier this year under treatment by a medical team at the hospital and Penn Medicine.\u00a0<\/p>\n<p>He became the first known patient in the world to be treated using CRISPR personalized just for him, according to a news release from Penn Medicine. <a href=\"https:\/\/www.nejm.org\/doi\/full\/10.1056\/NEJMoa2504747\" target=\"_blank\" rel=\"nofollow noopener\">His case<\/a> was published Thursday in the New England Journal of Medicine.<strong>\u00a0<\/strong><\/p>\n<p>He has so far shown no serious side effects to the treatment, Penn Medicine said. He is still in the hospital, gaining weight and thriving. He will turn 1 in August.\u00a0<\/p>\n<p>&#8220;While KJ will need to be monitored carefully for the rest of his life, our initial findings are quite promising,&#8221; Dr. Rebecca Ahrens-Nicklas, director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program at Children&#8217;s Hospital of Philadelphia, said in a statement.<\/p>\n<p>&#8220;Now, when I get to hold him, and he&#8217;s laughing and jumping around, that is a very heartwarming, because I didn&#8217;t know if that was going to happen, at one point,&#8221; his mother, Nicole Muldoon, told CBS News in an interview over Zoom Thursday.\u00a0<\/p>\n<p>According to Penn Medicine, CRISPR \u2014 which stands for clustered regularly interspaced short palindromic repeats \u2014 can &#8220;precisely correct disease-causing variants in the human genome.&#8221; The researchers cautioned that although personalized CRISPR treatment is in its early days, the technology could potentially one day serve to address other rare genetic disorders. \u00a0  <\/p>\n<p>&#8220;This is mind-blowing and we should all be very, very excited,&#8221; said Dr. Brian Brown, a bioengineer and immunologist who serves as director of the Icahn Genomics Institute at the Icahn School of Medicine at Mount Sinai in New York City.\u00a0<\/p>\n<p>&#8220;We are at day one of, you know, the future of how we are going to treat different diseases,&#8221; Brown said. &#8220;&#8230;We may have, potentially, treatments for horrible diseases children may be born with.&#8221; <\/p>\n<p>    <!-- data-recirc-source=\"queryly\" --><\/p>\n<aside class=\"component list recirculation component--type-recirculation \">\n        <\/aside>\n<p>    <!-- tags --><\/p>\n<div class=\"content-author\">\n      <a href=\"https:\/\/www.cbsnews.com\/team\/dr-jonathan-lapook\/\" class=\"content-author__name\" data-invalid-url-rewritten-http=\"\">Jon  LaPook<\/a><\/p>\n<div class=\"content-author__bio\">\n<div class=\"content-author__full-information\">\n<p class=\"content-author__text\">Jon LaPook, M.D. is the award-winning chief medical correspondent for CBS News. Since joining CBS News in 2006, LaPook has delivered more than 1,200 reports on a wide variety of breaking news and trending stories in the health and medical fields, as well as feature stories on music, lifestyle and profiles of entertainment stars.<\/p>\n<\/p><\/div>\n<\/p><\/div>\n<\/p><\/div>\n<\/p><\/div>\n<p><script async src=\"https:\/\/pagead2.googlesyndication.com\/pagead\/js\/adsbygoogle.js?client=ca-pub-6606220950177433\"\r\n     crossorigin=\"anonymous\"><\/script>\r\n<!-- ItShrt World News -->\r\n<ins class=\"adsbygoogle\"\r\n     style=\"display:block\"\r\n     data-ad-client=\"ca-pub-6606220950177433\"\r\n     data-ad-slot=\"1882483372\"\r\n     data-ad-format=\"auto\"\r\n     data-full-width-responsive=\"true\"><\/ins>\r\n<script>\r\n     (adsbygoogle = window.adsbygoogle || []).push({});\r\n<\/script>\r\n<br \/>\n<br \/><a href=\"https:\/\/www.cbsnews.com\/news\/infant-worlds-first-patient-personalized-gene-editing-treatment-crispr-liver-disorder\/\">Source link <\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Philadelphia \u2014 Soon after KJ Muldoon was born in the summer of 2024, he was diagnosed with a rare genetic<\/p>\n","protected":false},"author":1,"featured_media":26016,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[33],"tags":[310,5221,817],"class_list":["post-26015","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-us","tag-health","tag-new-england-journal-of-medicine","tag-philadelphia"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v24.2 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Infant becomes world&#039;s first patient to undergo personalized gene-editing treatment - World News<\/title>\n<meta name=\"description\" content=\"KJ Muldoon became the first patient to undergo personalized CRISPR treatment, a therapy that found the one uniquely mutated gene out of 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