Induced Pluripotent Stem Cells for the Treatment of Hemophilia A

Abstract

Factor VIII, one of the most complex proteins known, plays a major role in blood coagulation pathway. Defects in factor VIII protein result in hemophilia A, a severe bleeding disorder. Plasma derived factor VIII or recombinant factor VIII has been used extensively for treating hemophilia A patients. Number of attempts at gene therapy for hemophilia A has failed for various unknown/not much studied reasons including immune rejection. Here, the progress that has been made in establishing iPSC-based disease models and the potentials of iPSC technology for personalized medicine and cell therapy for hemophilia A are reviewed. The challenges of iPSC technology are also briefly discussed.