Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.

Abstract

In a phase I study, administration of an AAV2-FIX vector into the skeletal muscle of eight hemophilia B subjects proved safe and achieved local gene transfer and FIX expression for at least 10 months after vector injection, the last time point assessed by muscle biopsy. In hemophilia B dogs we have demonstrated FIX in both muscle biopsies and circulation >4… (More)

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@article{Jiang2006EvidenceOM, title={Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.}, author={Haiyan Jiang and Glenn F. Pierce and Margareth Castro Ozelo and E V De Paula and Joseph A Vargas and Peter Smith and J{\"u}rg M Sommer and Alvin Luk and Catherine S. Manno and Katherine A. High and Valder Roberval Arruda}, journal={Molecular therapy : the journal of the American Society of Gene Therapy}, year={2006}, volume={14 3}, pages={452-5} }