Gene therapy has proven successful in the treatment of hemophilia


German scientists first used gene therapy against hemophilia B in humans.

In the body of patients with hemophilia does not produce important clotting factors, causing a minor injury becomes a cause of spontaneous bleeding, including internal. Treatment by injection of these factors is often accompanied by a defensive reaction of the organism, which blocks the action of foreign proteins. The experience of a group of scientists with participation of specialists from Germany has shown that effective alternative is gene therapy of hemophilia.

The experiment involved 10 male patients with hemophilia B who have virtually or completely absent production of blood clotting factor IX. With the help of the virus in the liver cells of subjects introduced genetic information, including gene formulation of factor IX , which was 10 times more active than normal. Participants were surveyed after six months and a year and a half. It turned out that the gene was successfully delivered into the cells of the liver, which result in synthesized need protein. Patients stopped spontaneous bleeding, as well as improved overall health.

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