Viral Vector-Mediated Gene Therapy for Hemophilia B

Over the past five years, significant advances have been made in the development of novel viral vector systems for the treatment of hemophilia B by somatic gene therapy. At present, both a sustained but partial or a complete but transient correction of the hemophilia B phenotype have been observed in a clinically relevant animal model. Present efforts are being directed toward the development of safe, effective and persistent methods of virally-mediated gene transfer to achieve the complete restoration of normal hemostasis in individuals with hemophilia B.