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- Title
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.
- Authors
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
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.
- Publication
Archives of ophthalmology (Chicago, Ill. : 1960), 2012, Vol 130, Issue 1, p9
- ISSN
1538-3601
- Publication type
Journal Article
- DOI
10.1001/archophthalmol.2011.298