A recent study showed that giving hemophilia patients blood clotting genes can partially make up for their deficiencies and reduce bleeding. Hemophilia B, also known as the 'Christmas Disease', is a heredity disorder caused by a defective gene for the blood clotting protein called factor IX, which causes patients to have excessive hemorrhage (internal bleeding) specially on joints or muscles. While previous gene therapy attempts have been short-lived, British scientists successfully treated 6 people with hemophilia by giving them a gene with a functional form of factor IX.
Researchers used a modified viral vector that targeted the DNA specifically to the liver where factor IX is normally produced. This strategy was used based on previous literature that had shown the vector could be expressed in the livers of large animals for at least 10 years. It worked, at least a year out after treatment. The gene raised factor IX levels in the participants between 2 and 12 %. Severe hemophiliacs have to receive injections of the factor IX protein 2 to 3 times a week to avoid severe bleeding. 4 of the participants were able to stop treatment injections altogether and the other two were able to extend time between injections. There is hope for these patients again.
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Dr. Amit Nathwani is a faculty of medical sciences in the Research Department of Haematology and Cancer Institute in London. The main thrust of his research is the development of novel treatment avenues for haematological disorders and cancer using the gene transfer technology.
Gene Therapy: The Cure for Hemophilia Patients?