Gene Therapy for Hemophilia

Abstract

Adeno-associated viral vectors have been developed for the treatment of hemophilia A and B. Derivation of vector particles is achieved after multiplasmid transfection of cells that package the vector genome to yield vector particles. To date, three clinical trials have been performed for hemophilia B. The results of these trials are described. The trial that we conducted with our collaborators has yielded evidence of clinical efficacy for hemophilia B. A vector for treating hemophilia A has been developed and a clinical trial is planned.