Faculty

Lindsey A. George, M.D.

faculty photo
Assistant Professor of Pediatrics (Hematology)
Department: Pediatrics
Graduate Group Affiliations

Contact information
Children's Hospital of Philadelphia
3501 Civic Center Blvd
CTRB, Room 5016
Philadelphia, PA 19104
Education:
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

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 therapy 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.

Selected Publications

Doshi BS, Markmann CA, Novak N, Rojas SJ, Davidson R, Chau JQ, Wang W, Carrig S, Rus CM, Samelson-Jones BJ, Small JC, Bhoj VG, George LA. : Use of CD19-targeted Immune Modulation to Eradicate AAV Neutralizing Antibodies. Mol Ther Mar 2025.

Sternberg AR, Martos-Rus C, Davidson RJ, Liu X, George LA.: Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice. Nat Commun 15(1): 7193, August 2024.

Lee K, Chau JQ, Suber YB, Sternberg AR, Pishko A, George LA, Bhoj V, Doshi BS, Samelson-Jones BJ. : Enhanced Procoagulant Activity of Select Hemophilia B Causing Factor IX Variants with Emicizumab. Blood 144(11): 1230-1235, Sept 2024.

Kaczmarek R, Piñeros AR, Patterson PE, Bertolini TB, Perrin GQ, Sherman A, Born J, Arisa S, Arvin MC, Kamocka MM, Martinez MM, Dunn KW, Quinn SM, Morris JJ, Wilhelm AR, Kaisho T, Munoz-Melero M, Biswas M, Kaplan MH, Linnemann AK, George LA, Camire RM, Herzog RW: Factor VIII trafficking to CD4+ T cells shapes its immunogenicity and requires several types of antigen-presenting cells. Blood 142(3): 290-305, Jul 2023.

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.

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.

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.

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.

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.

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Last updated: 03/11/2025
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