platelet-targeted lentivirus-mediated gene therapy

Platelet-targeted lentivirus-mediated gene therapy is an ex vivo gene therapy platform designed for the treatment of hemophilia A and B. The approach involves the transduction of hematopoietic stem cells (HSCs) with lentiviral vectors that express coagulation factors, such as Factor VIII (FVIII) or Factor IX (FIX), under the control of a platelet-specific promoter (e.g., GPIIb). This strategy sequesters the therapeutic proteins within the alpha-granules of platelets, protecting them from circulating inhibitors (neutralizing antibodies) and allowing for targeted delivery and release at sites of vascular injury. Developed by researchers at the Medical College of Wisconsin and the Versiti Blood Research Institute, this platform has demonstrated a favorable safety profile and stable clonal reconstitution in long-term murine models and human xenotransplantation studies.

Other names

platelet-specific FVIII gene therapyplatelet-specific FIX gene therapyplatelet-targeted gene therapy