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Article

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

2 Apr 2003

Abstract excerpt

Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated viral (rAAV) vector expressing F.IX. In this study we investigated the safety of this approach in patients with...

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Identifiers

PMID: 12515715
DOI: 10.1182/blood-2002-10-3296

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