Encouraging results for targeted gene therapy and a step closer to a possible cure for haemophilia

An amazing breakthrough in disease-targeted therapy has been slowly unfolding using viral vectors to improve the treatment of haemophilia! Current therapy for haemophilia relies on giving either purified plasma-derived or recombinant clotting factors. This treatment has been shown to be beneficial but not a cure and has its limitations. Haemophilia is a genetic disease that is biologically well understood, having clear endpoint markers such as clotting factors and bleed times and has valid animal models making it an ideal disease to research.

Building upon the research using adeno-associated viral vector (AAV) serotype 2 that included a factor IX (FIX) gene that was introduced into the muscle and liver resulted in responses that lasted about 1 month. The current theory on the limited effect was that AAV2 had been eliminated by T cells due to previous exposure to serotype 2. Because of this, researchers changed the AAV to serotype 8 and created a self-complementary (sc) configuration of …