Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.

doi:10.1001/archophthalmol.2011.298

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Article

Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.

Archives of ophthalmology (Chicago, Ill. : 1960) - 1 Jan 2012

Jacobson Samuel G, Cideciyan Artur V, Ratnakaram Ramakrishna, Heon Elise, Schwartz Sharon B, Roman Alejandro J, Peden Marc C, Aleman Tomas S, Boye Sanford L, Sumaroka Alexander, Conlon Thomas J, Calcedo Roberto, Pang Ji-Jing, Erger Kirsten E, Olivares Melani B, Mullins Cristina L, Swider Malgorzata, Kaushal Shalesh, Feuer William J, Iannaccone Alessandro, Fishman Gerald A, Stone Edwin M, Byrne Barry J, Hauswirth William W

Abstract excerpt

OBJECTIVE: To determine the safety and efficacy of subretinal gene therapy in the RPE65 form of Leber congenital amaurosis using recombinant adeno-associated virus 2 (rAAV2) carrying the RPE65 gene. DESIGN: Open-label, dose-escalation phase I study of 15 patients (range, 11-30 years of age) evaluated after subretinal injection of the rAAV2- RPE65 vector into the worse-functioning eye. Five cohorts represented 4...

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PMID: 21911650
DOI: 10.1001/archophthalmol.2011.298
PMCID: PMC3600816

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RPE65

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