Hemophilia A: current treatment and future gene therapy

Summary The last two decades has seen significant progress in the treatment of hemophilia A. The development of highly purified and recombinant FVIII pharmaceutical products has dramatically increased the life expectancy and quality of life for many hemophiliacs. However, the high cost and short supply of these replacement products has resulted in their availability limited to less than 10% of the world’s hemophiliac population. Gene therapy for hemophilia A would provide prophylactic expression of FVIII and correction of the coagulation defect. A gene therapy protocol allowing simple, infrequent vector administration may extend hemophilia treatment to remote locations worldwide that currently lack access to FVIII replacement therapy. While progress has been made with each of the gene therapy vector systems described below, each still faces obstacles to its clinically utility. However, with the efforts that are currently directed toward overcoming these limitations, gene therapy for hemophilia A will ultimately become a reality.