Safety and Efficacy of Subretinal Readministration of a Viral Vector in Large Animals to Treat Congenital Blindness

Leber’s congenital amaurosis (LCA) is a group of severe inherited retinal degenerations that are symptomatic in infancy and lead to total blindness in adulthood. Recent clinical trials using recombinant adeno-associated virus serotype 2 (rAAV2) successfully reversed blindness in patients with LCA ca...

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Autors principals: Amado, Defne, Mingozzi, Federico, Hui, Daniel, Bennicelli, Jeannette L., Wei, Zhangyong, Chen, Yifeng, Bote, Erin, Grant, Rebecca L., Golden, Jeffrey A., Narfstrom, Kristina, Syed, Nasreen A., Orlin, Stephen E., High, Katherine A., Maguire, Albert M., Bennett, Jean
Format: Artigo
Idioma:Inglês
Publicat: 2010
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4169124/
https://ncbi.nlm.nih.gov/pubmed/20374996
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.3000659
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