Recent Advances in Gene Therapy for Hemophilia

Course overview

Hemophilia is a hemorrhagic disease characterized by a deficiency in blood clotting. It results from gene mutations in the genes that encode clotting factor proteins that form blood clots. The disease manifests itself in excessive and prolonged periods of external and internal bleeding. The conventional hemophilia treatment replaces missing blood clotting factors through intravenous infusion of plasma and cryoprecipitates (plasma-derived) and recombinant clotting factor concentrates. While these agents and treatments are effective, the current gene therapy strategies may offer new alternatives to the replacement protocols. Recent gene therapy directions, including viral, non-viral, and gene-editing technologies, are often more effective and provide new therapeutic avenues for routine hemophilia treatment.