Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

Transduction with recombinant adeno-associated virus (AAV) vectors is limited by the need to convert its single-stranded (ss) genome to transcriptionally active double-stranded (ds) forms. For AAV-mediated hemophilia B (HB) gene therapy, we have overcome this obstacle by constructing a liver-restric...

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Main Authors: Nathwani, Amit C., Gray, John T., Ng, Catherine Y. C., Zhou, Junfang, Spence, Yunyu, Waddington, Simon N., Tuddenham, Edward G. D., Kemball-Cook, Geoffrey, McIntosh, Jenny, Boon-Spijker, Mariette, Mertens, Koen, Davidoff, Andrew M.
Formáid: Artigo
Teanga:Inglês
Foilsithe: The American Society of Hematology 2006
Ábhair:
Gene Therapy
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC1895379/
https://ncbi.nlm.nih.gov/pubmed/16322469
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2005-10-4035
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