{"id":189585,"date":"2024-05-17T01:22:23","date_gmt":"2024-05-17T06:22:23","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2024\/05\/gene-editing-breakthrough-crispr-improves-vision-in-clinical-trial"},"modified":"2024-05-17T01:22:23","modified_gmt":"2024-05-17T06:22:23","slug":"gene-editing-breakthrough-crispr-improves-vision-in-clinical-trial","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2024\/05\/gene-editing-breakthrough-crispr-improves-vision-in-clinical-trial","title":{"rendered":"Gene Editing Breakthrough: CRISPR Improves Vision in Clinical Trial"},"content":{"rendered":"<p><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/gene-editing-breakthrough-crispr-improves-vision-in-clinical-trial2.jpg\"><\/a><\/p>\n<p>Jason Comander, MD, PhD, performs the procedure to deliver the CRISPR-based medicine as part of the BRILLIANCE trial in September 2020 at Mass Eye and Ear. Credit: Mass Eye and Ear.<\/p>\n<p>All 14 trial participants, including 12 adults (ages 17 to 63) and two children (ages 10 and 14), were born with a form of Leber Congenital Amaurosis (LCA) caused by mutations in the centrosomal protein 290 (<em>CEP290<\/em>) gene. They underwent a single injection of a CRISPR\/Cas9 genome editing medicine, EDIT-101 in one eye via a specialized surgical procedure. This trial, which included the first patient to ever receive a CRISPR-based investigational medicine directly inside the body, focused primarily on safety with a secondary analysis for efficacy.<\/p>\n<p>No serious treatment or procedure-related adverse events were reported, nor were there any dose-limiting toxicities. For efficacy, the researchers looked at four measures: best-corrected visual acuity (BCVA); dark-adapted full-field stimulus testing (FST), visual function navigation (VNC, as measured by a maze participants completed), and vision-related quality of life.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Jason Comander, MD, PhD, performs the procedure to deliver the CRISPR-based medicine as part of the BRILLIANCE trial in September 2020 at Mass Eye and Ear. Credit: Mass Eye and Ear. All 14 trial participants, including 12 adults (ages 17 to 63) and two children (ages 10 and 14), were born with a form of [\u2026]<\/p>\n","protected":false},"author":511,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1902,11,412],"tags":[],"class_list":["post-189585","post","type-post","status-publish","format-standard","hentry","category-bioengineering","category-biotech-medical","category-genetics"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/189585","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/511"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=189585"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/189585\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=189585"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=189585"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=189585"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}