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...

Volledige beschrijving

Bewaard in:
Bibliografische gegevens
Hoofdauteurs: 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.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2011
Onderwerpen:
Article
Online toegang: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: Voeg label toe
Geen labels, Wees de eerste die dit record labelt!

Gelijkaardige items