Effect of gene therapy on visual function in Leber's congenital amaurosis.

doi:10.1056/NEJMoa0802268

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Article

Effect of gene therapy on visual function in Leber's congenital amaurosis.

The New England journal of medicine - 22 May 2008

Bainbridge James W B, Smith Alexander J, Barker Susie S, Robbie Scott, Henderson Robert, Balaggan Kamaljit, Viswanathan Ananth, Holder Graham E, Stockman Andrew, Tyler Nick, Petersen-Jones Simon, Bhattacharya Shomi S, Thrasher Adrian J, Fitzke Fred W, Carter Barrie J, Rubin Gary S, Moore Anthony T, Ali Robin R

Abstract excerpt

Early-onset, severe retinal dystrophy caused by mutations in the gene encoding retinal pigment epithelium-specific 65-kD protein (RPE65) is associated with poor vision at birth and complete loss of vision in early adulthood. We administered to three young adult patients subretinal injections of recombinant adeno-associated virus vector 2/2 expressing RPE65 complementary DNA (cDNA) under the control of a human...

Topics

Adolescent
Adult
Blindness
Carrier Proteins
DNA, Complementary
Dependovirus
Eye Proteins
Gene Transfer Techniques
Genetic Therapy

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Genetic Vectors

Humans

Injections

Mutation

Retina

Retinal Degeneration

Visual Acuity

cis-trans-Isomerases

Source

Open in PubMed

Identifiers

PMID: 18441371
DOI: 10.1056/NEJMoa0802268

Genes

RPE65

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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