Professor of Pediatrics
Pediatrics, Herman B Wells Center for Pediatric Research
Gene Therapy for Hemophilia
My research led to the first clinical trials on AAV gene therapy for hemophilia and to immune modulatory protocols used in patients with genetic disease receiving AAV gene therapy. My laboratory's research continue to provide guidance for others conducting gene therapy trials. We are developing novel gene and immunotherapies for hemophilia and other diseases using murine, canine, non-human primate, and primate human cell-based models.