Gene therapy may be the key to defeating hemophilia, an inherited disease in which the blood clotting process is disrupted, resulting in bleeding into the joints, muscles and internal organs.. Scientists at Queen Mary University have been testing a new approach to treating this disease, and it looks very, very promising..

A single use of the drug was enough to maintain normal blood clotting in patients with hemophilia type A caused by congenital deficiency of blood coagulation factor VIII for a year.. This is the most common type of hemophilia.

Until now, the only treatment for hemophilia has been weekly injections of the missing clotting factors to control and prevent bleeding..

During the trial, volunteers from the UK were injected with a copy of the blood clotting factor VIII gene.. As a result, the levels of this protein increased in the participants..

In 85% of volunteers, clotting factor VIII levels remained normal or nearly normal even many months after therapy.. All 13 patients were able to refuse treatment they previously required.. Experts say that the result of testing a new approach exceeded all their expectations.

aspect. net.