Gene Therapy for Leber Congenital Amaurosis caused by RPE65 mutations: Safety and Efficacy in Fifteen Children and Adults Followed up to Three Years

OBJECTIVE: To determine safety and efficacy of subretinal gene therapy in the RPE65 form of Leber congenital amaurosis using recombinant adeno-associated virus 2 (rAAV2) carrying human RPE65 gene. DESIGN: Open-label, dose-escalation Phase I study of 15 patients (11-30 years) evaluated after subretin...

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Autors principals: 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, Willam J., Iannaccone, Alessandro, Fishman, Gerald A., Stone, Edwin M., Byrne, Barry J., Hauswirth, William W.
Format: Artigo
Idioma:Inglês
Publicat: 2011
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3600816/
https://ncbi.nlm.nih.gov/pubmed/21911650
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/archophthalmol.2011.298
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