Lindsey A. George, M.D.

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Assistant Professor of Pediatrics
Member, Perelman Center for Cellular and Molecular Therapeutics, University of Pennsylvania School of Medicine
Department: Pediatrics
Graduate Group Affiliations

Contact information
Children's Hospital of Philadelphia
3501 Civic Center Blvd
CTRB, Room 5016
Philadelphia, PA 19104
B.S. (Biology)
Cornell University, 2004.
M.D. (Medicine)
State University at Buffalo, School of Medicine and Biological Sciences, 2008.
MSTR (Translational Research)
University of Pennsylvania, School of Medicine, 2021.
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Description of Clinical Expertise

Disorders of hemostasis and thrombosis with a particular interest in hemophilia and hemophilia gene therapy.

Description of Research Expertise

My laboratory studies the molecular basis of coagulation that in diminished or excess functional states leads to disorders of hemostasis and thrombosis, respectively. We are particularly interested in the intrinsic tenase enzyme complex that, in deficiency states, results in hemophilia A or B. The current primary focus of our work is the regulation of factor VIII cofactor function with the goal of exploiting our biochemical understanding to develop gene based therapies for hemophilia A. We are additionally interested in understanding the mechanistic basis of translational questions that have emerged from hemophilia gene therapy clinical trials. I was previously the lead clinical principal investigator of early phase hemophilia A and B adeno-associated virus-mediated gene addition trials. Lastly, I am the director of 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.

Selected Publications

George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA: Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med 385(21): 1961-1973, Nov 2021 Notes: Corresponding author.

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 137(18): doi: 10.1182/blood.2020007562, May 2021.

George LA: No CpGs for AAV? Blood 137(6): 721-723, Feb 2021 Notes: Invited Commentary.

George LA: Hemophilia Gene Therapy: Ushering in a New Treatment Paradigm? American Society of Hematology Education Program 2021(1): 226-233, Dec 2021.

Robinson MM, George LA, Carr ME, Samelson-Jones BJ, Arruda VR, Murphy JE, Rybin D, Jeremy Rupon J, High KA, Tiefenbacher S: Factor IX Assay Discrepancies in the Setting of Liver Gene Therapy Using a Hyperfunctional Variant Factor IX-Padua. J Thromb Haemost 19(5): 1212-1218, May 2021.

George LA, Ragni MV, Rasko JEJ, Raffini LJ, Samelson-Jones BS, Hazbon M, Runowski AR, Wellman JA, Wachtel K, Chen Y, Anguela XM, Kuranda K, Mingozzi F, High KA : Long-Term Follow-Up of the First In Human Intravascular Delivery of AAV For Gene Transfer: AAV2 hFIX16 For Severe Hemophilia B. Mol Ther 28(9): 2073-2082, June 2020.

Doshi BS, Raffini LJ, George LA: Combined anti‐CD20 and mTOR inhibition with factor VIII for immune tolerance induction in hemophilia A patients with refractory inhibitors. J Thromb Haemost 18(4): 848-852, Mar 2020.

Samelson-Jones BJ, Finn JD, George LA, Camire RM, Arruda VR1: Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity. JCI Insight 4(14), June 2019.

George LA, Sullivan SK, Giermasz G, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majuddar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hu Di, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA : Hemophilia B Gene Therapy with a High Specific Activity Factor IX Variant N Engl J Med 377(2): 2215-2227, Dec 2017 Notes: Corresponding author, published with accompanying editorial.

Nguyen GN, George LA, Siner JI, Davidson RJ, Zander CB, Zheng XL, Arruda VR, Camire RM, Sabatino DE: Novel human factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia. J Thromb Haemost 14(10): 1-12, Oct 2016.

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Last updated: 01/03/2023
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