The goals of the Program in Human Gene Therapy are to establish the scientific principles and develop the technologies needed for achieving persistent and therapeutic levels of gene expression in vivo. While our ultimate aim is to use gene transfer to treat human disease, we plan to address basic biological questions that will be important for rational design of vectors for gene therapy applications. Towards this goal, we are working on developing new non-viral and viral vectors for gene transfer and establishing the cellular and molecular mechanisms involved in gene transduction in animals.

Our vector development includes: new episomal DNA vectors, DNA transposons, viral gene deleted adenoviruses, and adeno-associated virus. Hemophilia, human hepatitis virus infection, and diabetes are the three clinical disorders on which we have concentrated our efforts, but the results obtained will be useful for treating a wide variety of diseases. In the case of hemophilia, we have used our preclinical studies to help support clinical trials using recombinant AAV vectors to deliver a normal copy of the factor IX gene into factor IX-deficient individuals. We are currently conducting a Phase I/II trial, "Liver-directed AAV-hFIX administration into severe hemophilia B patients." Recently, we have developed RNAi approaches for treating human Hepatitis B and C infection in animal models, and are pursuing this approach for developing a therapy suitable for humans.