Safety and efficacy of gene transfer for Leber's congenital amaurosis.

doi:10.1056/NEJMoa0802315

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Article

Safety and efficacy of gene transfer for Leber's congenital amaurosis.

The New England journal of medicine - 22 May 2008

Maguire Albert M, Simonelli Francesca, Pierce Eric A, Pugh Edward N, Mingozzi Federico, Bennicelli Jeannette, Banfi Sandro, Marshall Kathleen A, Testa Francesco, Surace Enrico M, Rossi Settimio, Lyubarsky Arkady, Arruda Valder R, Konkle Barbara, Stone Edwin, Sun Junwei, Jacobs Jonathan, Dell'Osso Lou, Hertle Richard, Ma Jian-xing, Redmond T Michael, Zhu Xiaosong, Hauck Bernd, Zelenaia Olga, Shindler Kenneth S, Maguire Maureen G, Wright J Fraser, Volpe Nicholas J, McDonnell Jennifer Wellman, Auricchio Alberto, High Katherine A, Bennett Jean

Abstract excerpt

Leber's congenital amaurosis (LCA) is a group of inherited blinding diseases with onset during childhood. One form of the disease, LCA2, is caused by mutations in the retinal pigment epithelium-specific 65-kDa protein gene (RPE65). We investigated the safety of subretinal delivery of a recombinant adeno-associated virus (AAV) carrying RPE65 complementary DNA (cDNA) (ClinicalTrials.gov number, NCT00516477...

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Identifiers

PMID: 18441370
DOI: 10.1056/NEJMoa0802315
PMCID: PMC2829748

Genes

RPE65

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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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