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...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Main 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, Willam J., Iannaccone, Alessandro, Fishman, Gerald A., Stone, Edwin M., Byrne, Barry J., Hauswirth, William W.
Formato: Artigo
Idioma:Inglês
Publicado em: 2011
Assuntos:
Article
Acesso em linha: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
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados