Lindsey A. George, MD

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Lindsey A. George, MD, is the Director of Clinical In Vivo Gene Therapy and an attending physician in the Division of Hematology at Children's Hospital of Philadelphia.

Areas of Expertise: Hemostasis and thrombosis disorders, Hemophilia gene therapy, Hemophilia

Appointments and Referrals: 1-800-TRY-CHOP (1-800-879-2467)


Dr. George is a physician-scientist whose clinical expertise is in disorders of hemostasis and thrombosis with a particular interest in hemophilia and hemophilia gene therapy. Her basic science laboratory studies the molecular basis of coagulation that in diminished or excess functional states leads to disorders of hemostasis and thrombosis, respectively. The current focus of the George lab is to merge mechanistic studies aimed at understanding the regulation of factor VIII cofactor function with translational efforts in hemophilia A gene therapy. Her group is additionally interested in understanding the mechanistic basis of questions that have emerged from current hemophilia gene therapy clinical trials as well as general studies of adeno-associated viral vectors (AAV). Dr. George was previously the lead clinical principal investigator of multiple early phase hemophilia A and B adeno-associated virus-mediated gene addition trials. In addition to her clinical practice and laboratory, she directs 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.

Education and Training

Medical School

MD - State University of New York School of Medicine at Buffalo, Buffalo, NY


Pediatrics - Weill Cornell Medical College New York, NY (Chief Resident)


Pediatric Hematology/Oncology - The Children’s Hospital of Philadelphia, Philadelphia, PA

Board Certification

Pediatrics – American Board of Pediatrics

Graduate Degree

MS in Translational Research - The Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA

Titles and Academic Titles

Director, Clinical In Vivo Gene Therapy

Attending Physician

Principal Investigators

Tenure Assistant Professor of Pediatrics, The Perelman School of Medicine at the University of Pennsylvania

Conditions Treated

Departments and Services




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.

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: Hemophilia Gene Therapy: Ushering in a New Treatment Paradigm? American Society of Hematology Education Program Dec 2021.

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.

Konkle BA, Coffin D, Pierce GF, Clark C, George LA, Iorio A, Mahlangu J, Naccache M, O'Mahony B, Peyvandi F, Pipe S, Quartel A, Sawyer EK, Skinner MW, Tortella B, Watson C, Winburn I, Members of the WFH Gene Therapy Registry Steering Committee. : World Federation of Hemophilia Gene Therapy Registry. Haemophilia 26: 563-564, May 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.

Konkle BA, Coffin D, Pierce GF, Clark C, George L, Iorio A, Mahlangu J, Naccache M, O'Mahony B, Peyvandi F, Pipe S, Quartel A, Sawyer EK, Skinner MW, Tortella B, Watson C, Winburn I: World Federation of Hemophilia Gene Therapy Registry. Haemophilia 26(4): 563-564, Jul 2020.

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.


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.

Ragni MV, George LA: Scientific Priorities and Innnovative Implementation Strategies for FVIII Inhibitor Clinical Trials: Report from Working Group 1 of the NHLBI State of the Science Worskhop on FVIII Inhibitors. Haemophilia 25(4): 581-589, July 2019.


Bergmeier W, Antoniak S, Conway EM, Denis CV, George LA, Isermann B, Key NS, Krishnaswamy S, Lam WA, Lillicrap D, Liu J, Looney MR, L pez JA, Maas C, Peyvandi F, Ruf W, Sood AK, Versteeg HH, Wolberg AS, Wong PC, Wood JP, Weiler H.: Advances in Clinical and Basic Science of Coagulation: Illustrated abstracts of the 9th Chapel Hill Symposium on Hemostasis. Res Pract Thromb Haemost 2(3): 407-428, Apr 2018.


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. PMCID: 29211678


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, Law C, Jung M, Walton S, Ignjatovic V, Monagle P, Raffini LJ: Lack of anti-factor Xa assay standardization results in significant low molecular weight heparin (enoxaparin) dose variation in neonates and children. J Thromb Haemost 12(9): 1554-7, Sep 2014.

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.


Greene LA, Goldenberg NA, Simpson ML, Villalobos-Menuey E, Bombardier C, Acharya SS, Santiago-Borrero PJ, Cambara A, DiMichele DM: Use of global assays to understand clinical phenotype in congenital factor VII deficiency. Haemophilia 19(5): 765-72, Sep 2013.


Cordeiro JM, Greene L, Heilmann C, Antzelevitch D, Antzelevitch C: Transmural heterogeneity of calcium activity and mechanical function in the canine left ventricle. Am J Physiol Heart Circ Physiol 286(4): H1471-9, Apr 2004.


George LA, Lambert MP: Coagulation and fibrinolysis pathways-tests and interpretation. Nonmalignant Hematology. Abutalib SA, Connors JM, Ragni MV (eds.). Springer International Publishing, 2016.

Posters and Presentations


George LA, Sullivan S, Luk A, Urich T, Teitel J, Cuker A, Wright, F, Chen Y, Hui D, Wachtel K, Galvao A, McMillan LE, Takefman D, Carr ME, Cuoto L, Anguela X, High KA: SPK-9001 adeno-associated virus mediated gene therapy for hemophilia B: sustained factor IX expression at therapeutic levels without immunosuppression. Haemophilia 22: 151, Jul 2016 Notes: plenary presentation.

Invited Lectures


George LA. "Factor VIII Biochemistry and Therapeutic Application", University of Toronto, City-Wide Grand Rounds, Toronto, Canada. May, 2018.

George LA. "Gene Therapy for Hemophilia: State of the Art", World Federation of Hemophilia, Quebec, Canada. May, 2018.

George LA. "Licensing AAV Gene Therapy for Hemophilia: Clinical Endpoints and Long-Term Safety Monitoring", U.S. Food and Drug Administration, Silver Springs, Maryland. May, 2018.

George LA. "Novel Treatment Approaches for Hemophilia", 9th Symposium on Hemostasis and Thrombosis, Advances in the Clinical and Basic Sciences of Coagulation, University of North Carolina, Chapel Hill, North Carolina. Apr, 2018.

George LA. "Hemophilia Gene Therapy in 2018 and Beyond", Education Program, Thrombosis and Hemostasis Societies of North America, San Diego, California. Mar, 2018.

George LA. "Updates in Hemophilia A and B Gene Therapy", National Hemophilia Foundation Gene Therapy Workshop, Washington D.C. Feb, 2018.


George LA. "Gene Therapy for Hemophilia Comes of Age", American Society of Hematology Education Program, American Society of Hematology Annual Meeting, Atlanta, Georgia. Dec, 2017.


George LA. "SPK-9001 adeno-associated virus mediated gene transfer for hemophilia B achieves sustained mean factor IX activity levels of >30% without immunosuppression", Scientific Plenary Session, American Society of Hematology Annual Meeting, San Diego, California. Dec, 2016.

George LA. "SPK-9001 adeno-associated virus mediated gene therapy for hemophilia B: sustained factor IX expression without immunosuppression confers disease amelioration", Spotlight on Gene Therapy, World Federation of Hemophilia, Orlando, Florida. Jun, 2016.

Awards and Honors

2022, Outstanding New Investigator Award, American Society of Gene and Cell Therapy
2022, American Society of Clinical Investigation, Young Physician-Scientist Award
2022, Martin Villar Basic Science Award, Milan, Italy
2019, Scholar Award of the National Blood Foundation, Washington, D.C.
2019, National Top 10 Clinical/Translational Research Award, Clinical Research Forum, Washington, D.C.
2018, Professor Heimburger Award in Hemostasis, Marburg, Germany
2017, Eberhard Mammen Young Investigator Award for Excellence in Thrombosis and Hemostasis
2017, Next Gen Innovator, HemOnc Today
2015, Young Investigator Award, International Society of Thrombosis and Hemostasis, Toronto, CA
2015, Best Oral Abstract Presentation, Philadelphia Workshop in Thrombosis and Hemostasis, Philadelphia, PA
2015, Young Investigator Award, International Society of Thrombosis and Hemostasis, Toronto, CA
2014, Outstanding Abstract, Thrombosis and Hemostasis Society of North America, Chicago, IL
2014, Future Leaders in Hematology, Celgene Biopharmaceuticals, Anaheim, CA
2013, Abstract Achievement Award, American Society of Hematology, New Orleans, LA
2012, Abstract Travel Award, European Hematology Association, Amsterdam, NE
2011, Distinguished Clinician in Training, New York-Presbyterian Hospital, New York, NY

Editorial and Academic Positions

Editorial Positions 

Ad hoc reviewer

2021-present, The New England Journal of Medicine
2019-present, Science Translational Medicine
2019-present, Journal of Thrombosis and Hemostasis
2019-present, Molecular Therapy
2019-present, Hematology (American Society of Hematology Education Program)
2019-present, Blood
2018-present, Human Gene Therapy
2018-present, Nature Reviews Drug Discovery
2018-present, Human Gene Therapy
2017-present, British Journal of Haematology
2016-present, Pediatric Blood and Cancer
2015-present, Haematologica
2015-present, Thrombosis Research
2015-present, Haemophilia

Leadership and Memberships

Memberships in Professional Organizations

2013-present, International Society of Thrombosis and Haemostasis


2022-present, American Society of Clinical Investigation

2015-present, American Society of Gene and Cell Therapy

2010-present, American Society of Hematology