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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主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: Nature Publishing Group 2009
主題:
Original Articles
オンライン･アクセス: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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