Denise E. Sabatino, Ph.D., is a Research Associate Professor of Pediatrics at the Perelman School of Medicine, University of Pennsylvania. Her research focuses on hemophilia, an inherited disorder characterized by the inability to properly form blood clots. Dr. Sabatino's laboratory investigates various variants of coagulation factor VIII to gain insights into their biochemical properties and to identify variants that enhance therapeutic efficacy. She is dedicated to developing gene-based therapies aimed at providing sustained levels of factor VIII expression to improve treatment outcomes for hemophilia A patients. Current therapeutic methods, which often involve frequent protein infusions, are complicated by the development of immune responses in some patients. Dr. Sabatino's pioneering studies utilizing adeno-associated viral vectors have shown promising results in sustaining therapeutic levels of factor VIII expression and in promoting immune tolerance in animal models. Her work is pivotal for advancing gene therapy approaches that could revolutionize treatment options for hemophilia sufferers.