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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Autores principales: 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
Lenguaje:Inglês
Publicado: Nature Publishing Group 2009
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Original Articles
Acceso en línea: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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