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
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.
Permanent link
> Perelman School of Medicine   > Faculty   > Details

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 and international professional societies for hemostasis and gene therapy research.

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.

back to top
Last updated: 11/28/2023
The Trustees of the University of Pennsylvania