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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Bibliografische gegevens
Hoofdauteurs: 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
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Nature Publishing Group 2009
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Original Articles
Online toegang: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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