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Article

Retinal gene therapy in RPE-65 gene mediated inherited retinal dystrophy.

Eye (London, England) - 1 Jun 2023

Jalil Assad, Ivanova Tsveta, Moussa George, Parry Neil R A, Black Graeme C M

Abstract excerpt

BACKGROUND: Voretigene neparvovec (VN) is a gene therapeutic agent for treatment of retinal dystrophies caused by bi-allelic RPE65 mutations. We illustrate, both the benefits and pitfalls associated with ocular gene therapy in the same patient. METHODS: Two eyes of one patient with bi-allelic RPE65 mutations have been treated with VN. The clinical examinations included visual acuity (VA, in normal and low...

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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: 36163489
DOI: 10.1038/s41433-022-02262-5
PMCID: PMC10275908

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026.

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