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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Opis bibliograficzny
Główni autorzy: 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
Format: Artigo
Język:Inglês
Wydane: Nature Publishing Group 2009
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Original Articles
Dostęp online: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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