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Lindsey A. George, MD
Lindsey A. George
Tenure Assistant Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania

Dr. George's basic and clinical research studies include defining the molecular basis of coagulation and developing gene-based therapies for hemophilia.



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Dr. George's 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 research group conducts and supports translational and clinical research in in vivo gene therapy and also 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 and international professional societies for hemostasis and gene therapy research.

Education and Training

BS, Cornell University

MD, University at Buffalo School of Medicine

Pediatric Resident, Weill Cornell Medical College

Pediatric Chief Resident, Weill Cornell Medical College

Pediatric Hematology/Oncology fellowship, Children's Hospital of Philadelphia

MS, University of Pennsylvania (Translational Research)

Titles and Academic Titles

Tenure Assistant Professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania

Director, Clinical In Vivo Gene Therapy Center, Children's Hospital of Philadelphia

Attending Hematologist, Children's Hospital of Philadelphia

Professional Memberships

International Society of Thrombosis and Haemostasis World Federation of Hemophilia, 2013-

World Federation of Hemophilia, 2017-

American Society of Hematology, 2010-

American Society of Gene and Cell Therapy, 2015-

Hemostasis and Thrombosis Research Society, 2015-

Professional Awards

2017 Eberhard Mammen Young Investigator Award for Excellence in Thrombosis and Hemostasis, San Diego, CA

2018 Professor Heimburger Award in Hemostasis, Marburg, Germany

2019 National Top 10 Clinical/Translational Research Award, Clinical Research Forum, Washington, D.C.

2019 Scholar Award of the National Blood Foundation, Washington, D.C.

2020 Martin Villar Basic Science Award

2022 Young Physician-Scientist Award, American Society of Clinical Investigation

2022 Outstanding New Investigator, American Society of Gene and Cell Therapy

Publication Highlights

George LA. Hemophilia A Gene Therapy - Some Answers, More Questions. N Engl J Med. 2023 Feb; 388(8):761-763. PMID: 36812440
George LA, et al. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med. 2021 Nov; 385(21):1961-1973. PMID: 34788507
Wilhelm AR, Parson NA, Samelson-Jones BJ, Davidson RJ, Esmon CT, Camire RM, George LA. Activated protein C has a regulatory role in factor VIII function. Blood. 2021 May; 137(18):2532-2543. PMID: 33512448
George LA, et al. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. N Engl J Med. 2017 Dec; 377(23):2215-2227. PMID: 29211678