Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

doi:10.1016/S0140-6736(16)30371-3

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Article

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Lancet (London, England) - 13 Aug 2016

Bennett Jean, Wellman Jennifer, Marshall Kathleen A, McCague Sarah, Ashtari Manzar, DiStefano-Pappas Julie, Elci Okan U, Chung Daniel C, Sun Junwei, Wright J Fraser, Cross Dominique R, Aravand Puya, Cyckowski Laura L, Bennicelli Jeannette L, Mingozzi Federico, Auricchio Alberto, Pierce Eric A, Ruggiero Jason, Leroy Bart P, Simonelli Francesca, High Katherine A, Maguire Albert M

Abstract excerpt

BACKGROUND: Safety and efficacy have been shown in a phase 1 dose-escalation study involving a unilateral subretinal injection of a recombinant adeno-associated virus (AAV) vector containing the RPE65 gene (AAV2-hRPE65v2) in individuals with inherited retinal dystrophy caused by RPE65 mutations. This finding, along with the bilateral nature of the disease and intended use in treatment, prompted us to determine...

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Identifiers

PMID: 27375040
DOI: 10.1016/S0140-6736(16)30371-3
PMCID: PMC5351775

Genes

RPE65

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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