{"id":207278,"date":"2025-02-26T07:09:20","date_gmt":"2025-02-26T13:09:20","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2025\/02\/gene-therapy-can-improve-vision-in-young-children-with-aipl1-associated-retinal-dystrophy"},"modified":"2025-02-26T07:09:20","modified_gmt":"2025-02-26T13:09:20","slug":"gene-therapy-can-improve-vision-in-young-children-with-aipl1-associated-retinal-dystrophy","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2025\/02\/gene-therapy-can-improve-vision-in-young-children-with-aipl1-associated-retinal-dystrophy","title":{"rendered":"Gene therapy can improve vision in young children with AIPL1-associated retinal dystrophy"},"content":{"rendered":"<p><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/gene-therapy-can-improve-vision-in-young-children-with-aipl1-associated-retinal-dystrophy2.jpg\"><\/a><\/p>\n<p>Researchers from the NIHR Moorfields Biomedical Research Centre and University College London have found that gene therapy improved visual acuity and preserved retinal structure in young children with AIPL1-associated severe retinal dystrophy. This is the first human trial of gene supplementation therapy targeting this condition.<\/p>\n<p>Retinal dystrophy caused by biallelic variants in the AIPL1 gene leads to severe visual impairment from birth, with progressive degeneration and limited treatment options. Previous studies of early-onset rod-cone dystrophies, including AIPL1-related forms, highlighted a critical window for intervention during early childhood, when some photoreceptor structure remains intact. Prior research using Aipl1-deficient mouse models and human retinal organoids demonstrated partial restoration of photoreceptor function through <a href=\"https:\/\/medicalxpress.com\/tags\/gene+therapy\/\" rel=\"tag\" class=\"\">gene therapy<\/a>.<\/p>\n<p>In the study, \u201cGene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study,\u201d <a href=\"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0140673624028125\" target=\"_blank\">published<\/a> in <i>The Lancet<\/i>, researchers administered a single subretinal injection of a recombinant adeno-associated <a href=\"https:\/\/medicalxpress.com\/tags\/viral+vector\/\" rel=\"tag\" class=\"\">viral vector<\/a> (rAAV8.hRKp. AIPL1) carrying the AIPL1 gene to one eye of each child to assess the safety and efficacy of gene supplementation therapy in improving visual function and preserving retinal structure.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Researchers from the NIHR Moorfields Biomedical Research Centre and University College London have found that gene therapy improved visual acuity and preserved retinal structure in young children with AIPL1-associated severe retinal dystrophy. This is the first human trial of gene supplementation therapy targeting this condition. Retinal dystrophy caused by biallelic variants in the AIPL1 gene [\u2026]<\/p>\n","protected":false},"author":427,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11],"tags":[],"class_list":["post-207278","post","type-post","status-publish","format-standard","hentry","category-biotech-medical"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/207278","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\/427"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=207278"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/207278\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=207278"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=207278"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=207278"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}