Lindsey A. George, M.D.

faculty photo
Assistant Professor of Pediatrics
Department: Pediatrics
Graduate Group Affiliations

Contact information
The 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.
MTR (Masters of 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 clinical and research interests are in the development of novel therapeutics for hemophilia. My laboratory research relates to coagulation factor VIII (FVIII). Studies encompass basic biochemical studies of FVIII as well as translational work as it relates to exploiting this knowledge to develop therapeutics for hemophilia A. I am also the clinical principal investigator of ongoing early phase hemophilia A and B gene therapy trials as well as long term follow up of hemophilia gene therapy patients.

Selected Publications

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

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 Page: doi: 10.1182/blood.2020007562, Jan 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: 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.

George LA, Thalji NK, Raffini LJ, Gimotty PA, Camire RM: A new bypass strategy: zymogen-like Factor XaI16L corrects Hemophilia A whole blood thromboelastometry findings. J Thromb Haemost 13(9): 1694-8, Sep 2015.

Greene LA, Chen S, Seery C, Imahiyerobo AM, Bussel JB: Beyond the platelet count: immature platelet fraction and thromboelastometry correlate with bleeding in patients with immune thrombocytopenia. Br J Haematol 166(4): 592-600, Aug 2014.

Konkle B, Pierce G, Coffin D, Naccache M, Clark RC, George L, Iorio A, O'Mahony B, Pipe S, Skinner M, Watson C, Peyvandi F, Mahlangu J: Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH. J Thromb Haemost 18(11): 3074-3077, Nov 2020.

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Last updated: 09/09/2021
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