Biomedical Graduate Studies

Description of Clinical Expertise

hemostasis, hemophilia, gene therapy, gene editing

Description of Research Expertise

Dr. George is an Assistant Professor of Pediatrics at University of Pennsylvania and Director of Clinical In Vivo Gene Therapy at Children’s Hospital of Philadelphia. Her laboratory focuses on the development of gene-based therapies for coagulation disorders and on elucidating the molecular mechanisms underlying blood coagulation, with a particular emphasis on factor VIII biology and hemophilia A. Her studies on the regulation of factor VIII have led to the development of a next-generation gene therapy approach for hemophilia A that is currently being evaluated in a phase 2b clinical trial. Her laboratory also investigates the immunologic and molecular basis of unexplained clinical observations that have emerged from adeno-associated virus (AAV) gene therapy trials, with the goal of improving their safety and efficacy. In parallel with her laboratory research, Dr. George founded and directs the Clinical In Vivo Gene Therapy group at the Children’s Hospital of Philadelphia, which provides regulatory support for investigator-initiated studies, operational efforts for a large portfolio of interventional in vivo gene addition and editing trials and clinical infrastructure to safely and efficiently implement commercial in vivo gene therapies into clinical practice. She is a member of the National Academy of Medicine Emerging Leaders in Health and Medicine and past member of the Board of Directors of the American Society of Gene and Cell Therapy.