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Article

Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis.

Human gene therapy - 1 Aug 2006

Jacobson Samuel G, Boye Sanford L, Aleman Tomas S, Conlon Thomas J, Zeiss Caroline J, Roman Alejandro J, Cideciyan Artur V, Schwartz Sharon B, Komaromy Andras M, Doobrajh Michelle, Cheung Andy Y, Sumaroka Alexander, Pearce-Kelling Susan E, Aguirre Gustavo D, Kaushal Shalesh, Maguire Albert M, Flotte Terence R, Hauswirth William W

Abstract excerpt

Leber congenital amaurosis (LCA) is a molecularly heterogeneous disease group that leads to blindness. LCA caused by RPE65 mutations has been studied in animal models and vision has been restored by subretinal delivery of AAV-RPE65 vector. Human ocular gene transfer trials are being considered. Our safety studies of subretinal AAV-2/2.RPE65 in RPE65-mutant dogs showed evidence of modest photoreceptor loss in the...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 16942444
DOI: 10.1089/hum.2006.17.845

Genes

RPE65

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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