{"id":105828,"date":"2025-05-16T08:46:10","date_gmt":"2025-05-16T08:46:10","guid":{"rendered":"https:\/\/www.europesays.com\/uk\/105828\/"},"modified":"2025-05-16T08:46:10","modified_gmt":"2025-05-16T08:46:10","slug":"for-the-first-time-a-crispr-drug-treats-a-childs-unique-mutation","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/uk\/105828\/","title":{"rendered":"For the first time, a CRISPR drug treats a child\u2019s unique mutation"},"content":{"rendered":"<p data-component=\"paragraph\" class=\"css-1l5amll e1y9q0ei0\">WITHIN DAYS after KJ was born in Philadelphia in August 2024 it was clear that something was wrong. He was not eating and slept too much. Blood tests revealed sky-high levels of ammonia, a toxic substance the body usually expels. Genome sequencing confirmed that he had a rare genetic disease called carbamoyl-phosphate synthetase 1 (CPS1) deficiency, which often kills in infancy, and for which no good neonatal treatment exists. Then one of his doctors suggested something radical: a gene-editing drug designed specifically for him.<\/p>\n","protected":false},"excerpt":{"rendered":"WITHIN DAYS after KJ was born in Philadelphia in August 2024 it was clear that something was wrong.&hellip;\n","protected":false},"author":2,"featured_media":105829,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[4316],"tags":[105,3941,4348,70,229,16,15],"class_list":{"0":"post-105828","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-healthcare","8":"tag-health","9":"tag-health-care","10":"tag-healthcare","11":"tag-science","12":"tag-science-technology","13":"tag-uk","14":"tag-united-kingdom"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@uk\/114516642579629051","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/105828","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/comments?post=105828"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/105828\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/105829"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=105828"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=105828"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=105828"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}