Implantable Microcapsules for Gene Therapy for Hemophilia

Hemophilia is an X-linked recessive disorder caused by the deficiency of blood clotting factors VIII (hemophilia A) or IX (hemophilia B), which affects about 1 in 5000 live male births (Furie and Furie 1988, Hedner and Davie, 1989). Patients with severe hemophilia suffer from lifelong episodes of spontaneous bleeding. Common presentations include hematomas, bleeding into the joints, and intracranial hemorrhage, with the latter being a common cause of death. Long-term complications include chronic hemophilie arthropathy and progressive degeneration of the joints, leading to severe crippling deformity. Typically, one or more joints are affected in patients before the age of 12. Hemophilia is a debilitating disease imposing a heavy burden on both patients and families.