Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

Adeno-associated virus vectors (AAV) show promise for liver-targeted gene therapy. In this study, we examined the long-term consequences of a single intravenous administration of a self-complementary AAV vector (scAAV2/ 8-LP1-hFIXco) encoding a codon optimized human factor IX (hFIX) gene in 24 nonhu...

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Detalhes bibliográficos
Main Authors: Nathwani, Amit C, Rosales, Cecilia, McIntosh, Jenny, Rastegarlari, Ghasem, Nathwani, Devhrut, Raj, Deepak, Nawathe, Sushmita, Waddington, Simon N, Bronson, Roderick, Jackson, Scott, Donahue, Robert E, High, Katherine A, Mingozzi, Federico, Ng, Catherine YC, Zhou, Junfang, Spence, Yunyu, McCarville, M Beth, Valentine, Marc, Allay, James, Coleman, John, Sleep, Susan, Gray, John T, Nienhuis, Arthur W, Davidoff, Andrew M
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2011
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3098629/
https://ncbi.nlm.nih.gov/pubmed/21245849
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.274
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