Treatment (Hemophilia B)

Gene Therapy Approach Shows Promise with Single Intramuscular Administration

Published in Gene Therapy Weekly, June 8th, 1998

Scientists at Avigen, Inc., Seattle, Washington, and The Children's Hospital of Philadelphia, Pennsylvania, have developed a gene therapy approach for the treatment of hemophilia B using a single intramuscular administration of an adeno-associated virus (AAV) vector containing the gene for coagulation factor IX.

Katherine A. High, M.D., The Children's Hospital of Philadelphia and Avigen scientific advisor, presented research at the inaugural meeting of the American Society of Gene Therapy that shows the ability of AAV-mediated gene therapy to achieve sustained production of factor IX resulting in improved blood clotting times in a large animal model of hemophilia...

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Source: Gene Therapy Weekly (1998-06-08)

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