Hemophilia Therapy
Adeno-associated virus delivering factor VIII provides multiyear benefit in hemophilia A
Published in Hematology Week, August 21st, 2006
According to a report recently published in the journal Blood, "Hemophilia A, a deficiency of functional coagulation factor VIII (FVIII), is treated via protein replacement therapy. Restoring 1% to 5% of normal blood FVIII activity prevents spontaneous bleeding, making the disease an attractive gene therapy target."
"Previously, we have demonstrated short-term activity of a liver-specific AAV2 vector expressing canine B-domain-deleted FVIII (cFVIII) in a hemophilia canine model. Here, we report the...
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Source: Hematology Week (2006-08-21)
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