{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,3]],"date-time":"2026-11-03T09:26:51Z","timestamp":1793698011457,"version":"4.5.1"},"reference-count":15,"publisher":"Elsevier BV","issue":"11","license":[{"start":{"date-parts":[[2026,11,1]],"date-time":"2026-11-01T00:00:00Z","timestamp":1793491200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,11,1]],"date-time":"2026-11-01T00:00:00Z","timestamp":1793491200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Ophthalmology Retina"],"published-print":{"date-parts":[[2026,11]]},"DOI":"10.1016\/j.oret.2026.06.012","type":"journal-article","created":{"date-parts":[[2026,6,22]],"date-time":"2026-06-22T16:01:42Z","timestamp":1782144102000},"page":"1118-1126","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Clinical Experience with Gene Therapy for Autosomal Recessive RPE65-Related Retinal Dystrophy in the United Arab Emirates"],"prefix":"10.1016","volume":"10","author":[{"given":"Zabila","family":"Khan","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9255-8025","authenticated-orcid":false,"given":"Nicola G.","family":"Ghazi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven","family":"Hay","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Arif O.","family":"Khan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.oret.2026.06.012_bib1","doi-asserted-by":"crossref","first-page":"261","DOI":"10.4103\/sjopt.sjopt_295_23","article-title":"Inherited retinal diseases and gene therapy update","volume":"37","author":"Khan","year":"2023","journal-title":"Saudi J Ophthalmol"},{"key":"10.1016\/j.oret.2026.06.012_bib2","doi-asserted-by":"crossref","first-page":"1115","DOI":"10.1001\/jamaophthalmol.2019.2512","article-title":"Cost-effectiveness of voretigene neparvovec-rzyl vs standard care for RPE65-mediated inherited retinal disease","volume":"137","author":"Johnson","year":"2019","journal-title":"JAMA Ophthalmol"},{"key":"10.1016\/j.oret.2026.06.012_bib3","doi-asserted-by":"crossref","first-page":"1273","DOI":"10.1016\/j.ophtha.2019.06.017","article-title":"Efficacy, safety, and durability of voretigene neparvovec-rzyl in RPE65 mutation-associated inherited retinal dystrophy: results of phase 1 and 3 trials","volume":"126","author":"Maguire","year":"2019","journal-title":"Ophthalmology"},{"key":"10.1016\/j.oret.2026.06.012_bib4","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1016\/j.ymthe.2020.11.029","article-title":"Clinical perspective: treating RPE65-associated retinal dystrophy","volume":"29","author":"Maguire","year":"2021","journal-title":"Mol Ther"},{"key":"10.1016\/j.oret.2026.06.012_bib5","doi-asserted-by":"crossref","first-page":"58","DOI":"10.1016\/j.ajo.2018.09.024","article-title":"The natural history of inherited retinal dystrophy due to biallelic mutations in the RPE65 gene","volume":"199","author":"Chung","year":"2019","journal-title":"Am J Ophthalmol"},{"key":"10.1016\/j.oret.2026.06.012_bib6","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1016\/S0140-6736(17)31868-8","article-title":"Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial","volume":"390","author":"Russell","year":"2017","journal-title":"Lancet"},{"key":"10.1016\/j.oret.2026.06.012_bib7","doi-asserted-by":"crossref","first-page":"122","DOI":"10.3390\/biom14010122","article-title":"Real-world safety and effectiveness of voretigene neparvovec: results up to 2 years from the prospective, registry-based PERCEIVE study","volume":"14","author":"Fischer","year":"2024","journal-title":"Biomolecules"},{"key":"10.1016\/j.oret.2026.06.012_bib8","doi-asserted-by":"crossref","first-page":"764","DOI":"10.1016\/j.ophtha.2023.02.015","article-title":"Full-field scotopic threshold improvement after voretigene neparvovec-rzyl treatment correlates with chorioretinal atrophy","volume":"130","author":"Stingl","year":"2023","journal-title":"Ophthalmology"},{"key":"10.1016\/j.oret.2026.06.012_bib9","doi-asserted-by":"crossref","DOI":"10.1016\/j.xops.2026.101090","article-title":"Expert consensus on characteristics, etiology, and management of chorioretinal atrophy in patients treated with voretigene neparvovec","volume":"6","author":"Fischer","year":"2026","journal-title":"Ophthalmol Sci"},{"key":"10.1016\/j.oret.2026.06.012_bib10","doi-asserted-by":"crossref","first-page":"361","DOI":"10.1080\/15569527.2025.2573463","article-title":"Clinical and pharmacovigilance safety evaluation of LUXTURNA\u00ae (voretigene neparvovec-rzyl)","volume":"44","author":"Simoens","year":"2025","journal-title":"Cutan Ocul Toxicol"},{"key":"10.1016\/j.oret.2026.06.012_bib11","doi-asserted-by":"crossref","first-page":"156","DOI":"10.1001\/jamaophthalmol.2025.5505","article-title":"Vision outcomes in children treated with voretigene neparvovec for RPE65-associated retinopathy","volume":"144","author":"Thompson","year":"2026","journal-title":"JAMA Ophthalmol"},{"key":"10.1016\/j.oret.2026.06.012_bib12","doi-asserted-by":"crossref","first-page":"42","DOI":"10.1016\/j.oret.2023.08.017","article-title":"Classification and growth rate of chorioretinal atrophy after voretigene neparvovec-rzyl for RPE65-mediated retinal degeneration","volume":"8","author":"Bommakanti","year":"2024","journal-title":"Ophthalmol Retina"},{"key":"10.1016\/j.oret.2026.06.012_bib13","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1136\/bjo-2022-321488","article-title":"Subretinal deposits in young patients treated with voretigene neparvovec-rzyl for RPE65-mediated retinal dystrophy","volume":"107","author":"Lopez","year":"2023","journal-title":"Br J Ophthalmol"},{"key":"10.1016\/j.oret.2026.06.012_bib14","doi-asserted-by":"crossref","first-page":"e602","DOI":"10.1016\/j.jcjo.2024.12.013","article-title":"\u201cBlindness\u201d is not a contraindication for voretigene neparvovec-rzyl treatment: a review of 9 cases","volume":"60","author":"Parameswarappa","year":"2025","journal-title":"Can J Ophthalmol"},{"key":"10.1016\/j.oret.2026.06.012_bib15","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1136\/bjo-2024-326221","article-title":"12-month outcomes after voretigene neparvovec gene therapy in paediatric patients with RPE65-mediated inherited retinal dystrophy","volume":"109","author":"Daruich","year":"2025","journal-title":"Br J Ophthalmol"}],"container-title":["Ophthalmology Retina"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2468653026003040?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2468653026003040?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,11,3]],"date-time":"2026-11-03T08:37:36Z","timestamp":1793695056000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2468653026003040"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,11]]},"references-count":15,"journal-issue":{"issue":"11","published-print":{"date-parts":[[2026,11]]}},"alternative-id":["S2468653026003040"],"URL":"https:\/\/doi.org\/10.1016\/j.oret.2026.06.012","relation":{},"ISSN":["2468-6530"],"issn-type":[{"value":"2468-6530","type":"print"}],"subject":[],"published":{"date-parts":[[2026,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Clinical Experience with Gene Therapy for Autosomal Recessive RPE65-Related Retinal Dystrophy in the United Arab Emirates","name":"articletitle","label":"Article Title"},{"value":"Ophthalmology Retina","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.oret.2026.06.012","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 American Academy of Ophthalmology, Inc. Published by Elsevier Inc. All rights are reserved, including those for text and data mining, AI training, and similar technologies.","name":"copyright","label":"Copyright"}]}}