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New gene therapy tool successfully treats mice with hemophilia A

(Embargoed) CHAPEL HILL -- 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.

This new study in mice with hemophilia A was conducted in collaboration with scientists from Intronn Inc., Rockville, Maryland, where RNA trans-splicing was pioneered. A "pre-trans-splicing molecule" (PTM) was injected into some of the mice. The molecule, or "cassette," is designed to produce RNA that binds and splices onto the existing faulty RNA, correcting it. The corrected RNA then encodes for the normal factor VIII protein.

"Preliminary data using the hemophilia A mice is very encouraging," said Dr. Hengjun Chao, "After injecting hemophilia A mice with the PTM cassette, factor VIII levels in the blood rose from lower than 1% to a maximum of 20 % of normal factor VIII activity. These levels of activity corrected the bleeding tendency of the hemophilia A mice, thus protect
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Contact: Leslie H. Lang
LLANG@MED.UNC.EDU
919-843-9687
University of North Carolina School of Medicine
8-Jun-2002


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