Hemophilia

New gene therapy tool successfully treats mice with hemophilia A

Published in Gene Therapy Weekly, July 4th, 2002

Scientists at the University of North Carolina have successfully treated mice with hemophilia A using a new approach to gene therapy - RNA trans-splicing. The experimental procedure repairs a mutated section of the gene responsible for hemophilia A.

Dr. Hengjun Chao, a research assistant professor at the UNC School of Medicine, Gene Therapy Center presented the new research June 8, 2002, in Boston at the Presidential Symposium of the American Society of Gene Therapy Annual Meeting.

Hemophilia A is a sex-linked congenital disease, occurring in 1 out of 5000-10,000 males in all populations and is caused by a defect in coagulation factor VIII. The...

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Source: Gene Therapy Weekly (2002-07-04)

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