Lindsey George, MD

At-Large Director (2022-2025)


Dr. George is a physician-scientist whose clinical expertise is in disorders of hemostasis and thrombosis with a particular interest in hemophilia and hemophilia gene therapy. Her basic science laboratory studies the molecular basis of coagulation that in diminished or excess functional states leads to disorders of hemostasis and thrombosis, respectively. The current focus of the George lab is to merge mechanistic studies aimed at understanding the regulation of factor VIII cofactor function with translational efforts in hemophilia A gene therapy. Her group is additionally interested in understanding the mechanistic basis of questions that have emerged from current hemophilia gene therapy clinical trials as well as general studies of adeno-associated viral vectors (AAV). Dr. George was previously the lead clinical principal investigator of multiple early phase hemophilia A and B adeno-associated virus-mediated gene addition trials. In addition to her clinical practice and laboratory, she directs the clinical in vivo gene therapy at the Children's Hospital of Philadelphia that long-term aims to safely and efficiently advance translational and clinical research for in vivo gene therapy for children with genetic disorders.

2024

Breakthroughs in Muscular Dystrophy

November 19-20, 2024 | Chicago, IL

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