Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

Human gene therapy with rAAV2-vector was performed for the RPE65 form of childhood blindness called Leber congenital amaurosis. In three contemporaneous studies by independent groups, the procedure was deemed safe and there was evidence of visual gain in the short term. At 12 months after treatment,...

Descrizione completa

Salvato in:
Dettagli Bibliografici
Autori principali: Cideciyan, Artur V., Hauswirth, William W., Aleman, Tomas S., Kaushal, Shalesh, Schwartz, Sharon B., Boye, Sanford L., Windsor, Elizabeth A.M., Conlon, Thomas J., Sumaroka, Alexander, Pang, Ji-jing, Roman, Alejandro J., Byrne, Barry J., Jacobson, Samuel G.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc. 2009
Soggetti:
Brief Reports
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2829287/
https://ncbi.nlm.nih.gov/pubmed/19583479
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2009.086
Tags: Aggiungi Tag
Nessun Tag, puoi essere il primo ad aggiungerne! !

Documenti analoghi