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,...

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Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2009
Matèries:
Brief Reports
Accés en línia: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
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