In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs

摘要: The liver represents a model organ for gene therapy. A method has been developed hepatic transfer in vivo by the direct infusion of recombinant retroviral vectors into portal vasculature, which results persistent expression exogenous genes. To determine if these technologies are applicable treatment hemophilia B patients, preclinical efficacy studies were done dog model. When canine factor IX complementary DNA was transduced directly hepatocytes affected dogs vivo, animals constitutively expressed low levels more than 5 months. Persistent clotting resulted reductions whole blood and partial thromboplastin times treated animals. Thus, long-term patients may be feasible therapy vivo.