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Article

Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Dec 2005

Acland Gregory M, Aguirre Gustavo D, Bennett Jean, Aleman Tomas S, Cideciyan Artur V, Bennicelli Jeannette, Dejneka Nadine S, Pearce-Kelling Susan E, Maguire Albert M, Palczewski Krzysztof, Hauswirth William W, Jacobson Samuel G

Abstract excerpt

The short- and long-term effects of gene therapy using AAV-mediated RPE65 transfer to canine retinal pigment epithelium were investigated in dogs affected with disease caused by RPE65 deficiency. Results with AAV 2/2, 2/1, and 2/5 vector pseudotypes, human or canine RPE65 cDNA, and constitutive or tissue-specific promoters were similar. Subretinally administered vectors restored retinal function in 23 of 26 eyes,...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 16226919
DOI: 10.1016/j.ymthe.2005.08.008
PMCID: PMC3647373

Genes

RPE65

Provenance

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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