Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

The safety and efficacy of gene therapy for inherited retinal diseases is being tested in humans affected with Leber's congenital amaurosis (LCA), an autosomal recessive blinding disease. Three independent studies have provided evidence that the subretinal administration of adeno-associated vir...

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Detalhes bibliográficos
Main Authors: Simonelli, Francesca, Maguire, Albert M, Testa, Francesco, Pierce, Eric A, Mingozzi, Federico, Bennicelli, Jeannette L, Rossi, Settimio, Marshall, Kathleen, Banfi, Sandro, Surace, Enrico M, Sun, Junwei, Redmond, T Michael, Zhu, Xiaosong, Shindler, Kenneth S, Ying, Gui-Shuang, Ziviello, Carmela, Acerra, Carmela, Wright, J Fraser, McDonnell, Jennifer Wellman, High, Katherine A, Bennett, Jean, Auricchio, Alberto
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2009
Assuntos:
Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2839440/
https://ncbi.nlm.nih.gov/pubmed/19953081
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.277
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