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, a hereditary bleeding disorder.
Dr. Hengjun Chao, a research assistant professor at the UNC School of Medicine, Gene Therapy Center will present the new research Saturday June 8 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 one out of 5,000 to 10,000 live males in all populations and is caused by a defect in coagulation factor VIII. The mutation renders the factor VIII gene non-functional resulting in recurrent, non-predictable, spontaneous bleeding into major joints and soft tissues. Currently, the disorder is treated with injections of factor VIII protein in response to bleeding incidents. Conventional approaches to gene therapy have not proven successful against hemophilia A, partially due to difficulties involved in packaging and delivering the large factor VIII gene.
Leslie H. Lang | EurekAlert
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