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Gene therapy achieves early success against hereditary bleeding disorder
Date:12/10/2011

(MEMPHIS, Tenn. December 10, 2011) Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K.

The findings of the six-person study mark the first proof that gene therapy can reduce disabling, painful bleeding episodes in patients with the inherited blood disorder. Results of the Phase I study appear in today's online edition of the New England Journal of Medicine. The research is also scheduled to be presented December 11 at the 53rd annual meeting of the American Society of Hematology in San Diego.

Four study participants stopped receiving protein injections to prevent bleeding episodes after undergoing the therapy and have not suffered spontaneous bleeding. Several have also participated in marathons and other activities that would have been difficult prior to gene therapy. The study volunteers were all treated at the Royal Free Hospital in London under the care of Edward G.D. Tuddenham, M.D., Ph.D., a pioneer in the field of blood coagulation and a study co-author.

"This is a potentially life-changing treatment for patients with this disease and an important milestone for the field of gene therapy. It could have ramifications for the treatment of hemophilia A, other protein and liver disorders and chronic diseases such as cystic fibrosis," said first author Amit Nathwani, M.D., Ph.D., a faculty member at the UCL Cancer Institute, Royal Free Hospital and NHS Blood and Transplant (NHSBT).

Hemophilia B is caused by an inherited mistake in the gene for making a protein called Factor IX, which is essential for normal blood clotting. The gene is carried on the X chromosome. As a result, hemophilia B is almost exclusively a disease of men. About 1 in 30,000 individua
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Contact: Carrie Strehlau
media@stjude.org
901-595-2295
St. Jude Children's Research Hospital
Source:Eurekalert

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