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Article

Gene therapy outcomes in young patients with RPE65-retinal degeneration.

Canadian journal of ophthalmology. Journal canadien d'ophtalmologie - 1 Jun 2026

Stephenson Kirk A J, Alsalamah Abrar K, Tumber Anupreet, Mercer Gareth D, Ballios Brian G, Muni Rajeev H, Tavares Erika, Kertes Peter J, Vincent Ajoy, Héon Elise

Abstract excerpt

OBJECTIVES: RPE65-associated Leber's congenital amaurosis (RPE65-LCA) is now treatable with a subretinal injection of voretigene neparvovec-rzyl (VN). Outcomes specific to young patients are not often reported. We present outcomes of young patients ≤21 years old with RPE65-LCA who have received VN. DESIGN: A single-centre, retrospective review of 18 patients ≤21 years old (mean age: 14.6 ± 3.4 years, 10 females)...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 41679721
DOI: 10.1016/j.jcjo.2026.01.016

Genes

RPE65
FST

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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