Hemophilia (Animal Studies)

Possible Gene Therapy for Hemophilia A

Published in Gene Therapy Weekly, June 28th, 1999

Results from gene therapy studies conducted in mice at the University of North Carolina at Chapel Hill (UNC-CH) may lead to an effective long-term treatment, if not a cure, for hemophilia A.

Studies in mice involved attaching a cloned gene responsible for human clotting factor VIII - a protein severely deficient in people with hemophilia A - to a genetically-engineered virus called AAV. A single injection of the virus led to a gradual increase in blood levels of factor VIII, which have remained stable for 11 months.

"This is the first result in animals demonstrating the efficacy of this approach to hemophilia A," said Dr. Christopher E. Walsh,...

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Source: Gene Therapy Weekly (1999-06-28)

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