Biomedical Graduate Studies

Description of Clinical Expertise

Coagulation disorders and gene therapy for monogenic disorders

Description of Research Expertise

Dr. Lindsey George is an Assistant Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania and Director of Clinical In Vivo Gene Therapy and Attending Hematologist at the Children’s Hospital of Philadelphia. Her laboratory studies the molecular basis of coagulation with a focus on mechanisms that regulate factor VIII/VIIIa cofactor function to improve understanding of associated disease states of deficiency (hemophilia A) or excess function (thrombosis). Ongoing studies in her group aim to exploit their biochemical insights of FVIII function to develop novel gene-based therapies for hemophilia A. Dr. George has led early-phase, adeno-associated virus (AAV) mediated gene transfer trials for hemophilia A and B. Her Clinical In Vivo Gene Therapy group conducts and supports translational and clinical research in in vivo gene addition and editing as well as aims to implement licensed AAV vectors safely and efficiently across the institution. Dr. George is a member of the Board of Directors of the American Society of Gene and Cell Therapy and participates in multiple national and international professional societies for hemostasis and gene therapy research.