Lindsey A. George, M.D.

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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.
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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

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.

Pierce GF, Coffin D; Members of the WFH Gene Therapy Round Table Program Committee and Organizing Committee.: The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world. Haemophilia 25(2): 189-194, Mar 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.

George LA: Gene Therapy for Hemophilia Comes of Age. Hematology Am Soc Hematol Educ Program 1(1): 587-594, Dec 2017 Notes: Selected as top 3 ASH Education Program.

Doshi B, Arruda VA, Lambert M, Raffini LJ, George LA: Anti-CD20 and Sirolimus for Immune Tolerance in Congenital Hemophilia A with Inhibitors. Res Pract Thromb Haemost 1(Suppl 1): 665, Jul 2017.

George LA, Ragni MV, Samuelson-Jones BJ, Cuker C, Rinoski AR, Cole G, Wright JF, Chen Y, Hui DJ, Wachtel K, Takefman D, Couto LB, Reape KZ, Carr ME, Anguela XM, High KA: SPK-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia A. Blood 130(1): 604, Dec 2017 Notes: podium presentation.

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.

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Last updated: 03/08/2021
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