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Hemophilia A

Short-Term Correction of Canine Hemophilia A by In Vivo Gene Therapy

Published in Gene Therapy Weekly, December 9th, 1996

Adenovirus-mediated in vivo gene therapy temporarily corrects a canine model of hemophilia A.

Hemophilia A is an X-linked bleeding disorder caused by a deficiency of blood coagulation factor VIII (FVIII). The most widely accepted therapy during a crisis is infusion of plasma-derived or recombinant FVIII. Although prophylactic treatment reduces the frequency and severity of bleeding episodes, the treatment is limited by the availability and high cost of purified FVIII, the short half-life of FVIII in vivo, and difficulties associated with repeated intravenous administration.

A gene therapy protocol for hemophilia A which could provide constant blood...

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