Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

The safety and efficacy of peripheral venous administration of a self-complementary adeno-associated viral vector encoding the human FIX gene (scAAV-LP1-hFIXco) was evaluated in nonhuman primates for gene therapy of hemophilia B. Peripheral vein infusion of 1 × 10(12) vg/kg scAAV-LP1-hFIXco pseudoty...

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Detalhes bibliográficos
Main Authors: Nathwani, Amit C., Gray, John T., McIntosh, Jenny, Ng, Catherine Y. C., Zhou, Junfang, Spence, Yunyu, Cochrane, Melanie, Gray, Elaine, Tuddenham, Edward G. D., Davidoff, Andrew M.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2007
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC1794053/
https://ncbi.nlm.nih.gov/pubmed/17090654
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2006-03-010181
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