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Article

The Status of RPE65 Gene Therapy Trials: Safety and Efficacy.

Cold Spring Harbor perspectives in medicine - 29 Jan 2015

Pierce Eric A, Bennett Jean

Abstract excerpt

Several groups have reported the results of clinical trials of gene augmentation therapy for Leber congenital amaurosis (LCA) because of mutations in the RPE65 gene. These studies have used subretinal injection of adeno-associated virus (AAV) vectors to deliver the human RPE65 cDNA to the retinal pigment epithelial (RPE) cells of the treated eyes. In all of the studies reported to date, this approach has been...

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Source

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Identifiers

PMID: 25635059
DOI: 10.1101/cshperspect.a017285
PMCID: PMC4561397

Genes

RPE65
RPE

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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