Hemophilia Gene Therapy

AAV Vector Successfully Overcomes Inhibitors to Express Human Factor IX

Published in Blood Weekly, April 13th, 1998

The use of recombinant adeno-associated virus (rAAV) vectors for the correction of serum protein deficiencies was discussed at a recent conference.

H. Nakai and colleagues from Avigen Inc. and the University of Southern California, demonstrated that persistent expression of a transgene product transferred via rAAV vectors can be obtained in the presence of inhibitors following intramuscular injection in immunocompetent mice (abstract "Persistent Expression of Circulating Human Coagulation Factor IX in Mice Carrying Inhibitors Following Intramuscular Administration of an AAV Vector"). The research was presented at the Keystone Symposium, Molecular and Cellular Biology...

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Source: Blood Weekly (1998-04-13)

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