Recent Advances in Hemophilia Gene Therapy

Heterogeneous loss of function mutations at F8 and F9 genes causes X-linked recessive bleeding disorders, hemophilia A (HA) and hemophilia B (HB), respectively. HA is clinically indistinguishable from HB and accounts for more than 80% of hemophilia cases; the former affects 1/5000 and the latter 1/25000 male births worldwide. In Iran, it is estimated that around 4300 HA and 900 HB patients are under care. The standard care for hemophilia patients is lifelong on demand or prophylactic factor replacement therapy. However, the complications such as arthropathies in severe form of the disease and viral infections are not uncommon. The cost of prophylactic treatment is high and not affordable in developing countries. The annual cost of hemophilia treatment in Iran is not predictable, but it is estimated to be around 50,000-60,000 US dollars/patient...