Lindsey A. George, M.D.

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
3501 Civic Center Blvd
CTRB, Room 5016
Philadelphia, PA 19104
Publications
Links
Search PubMed for articles
Perelman School of Medicine at the University of Pennsylvania website
Raymond Perelman Center for Cellular and Molecular Therapeutics website
The Children's Hospital of Philadelphia
Search PubMed for articles
Perelman School of Medicine at the University of Pennsylvania website
Raymond Perelman Center for Cellular and Molecular Therapeutics website
The Children's Hospital of Philadelphia
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.
Permanent linkB.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.
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
Byrne BJ, Flanigan KM, Matesanz SE, Finkel RS, Waldrop MA, D'Ambrosio ES, Johnson NE, Smith BK, Bönnemann C, Carrig S, Rossano JW, Greenberg B, Lalaguna L, Lara-Pezzi E, Subramony S, Corti M, Mercado-Rodriguez C, Leon-Astudillo C, Ahrens-Nicklas R, Bharucha-Goebel D, Gao G, Gessler DJ, Hwu WL, Chien YH, Lee NC, Boye SL, Boye SE, George LA: Current clinical applications of AAV-mediated gene therapy. Mol Ther 33(6): 2479-2516, Jun 2025.Musunuru K, Grandinette SA, Wang X, Hudson TR, Briseno K, Berry AM, Hacker JL, Hsu A, Silverstein RA, Hille LT, Ogul AN, Robinson-Garvin NA, Small JC, McCague S, Burke SM, Wright CM, Bick S, Indurthi V, Sharma S, Jepperson M, Vakulskas CA, Collingwood M, Keogh K, Jacobi A, Sturgeon M, Brommel C, Schmaljohn E, Kurgan G, Osborne T, Zhang H, Kinney K, Rettig G, Barbosa CJ, Semple SC, Tam YK, Lutz C, George LA, Kleinstiver BP, Liu DR, Ng K, Kassim SH, Giannikopoulos P, Alameh MG, Urnov FD, Ahrens-Nicklas RC: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. N Engl J Med 392(22): 2235-2243, Jun 2025.
Rasko JEH, Samelson-Jones BJ, George LA, Giermasz A, Ducore JM, Teitel JM, McGuinn CE, High KA, de Jong YP, Chhabra A, O’Brien A, Smith LM, Winburn I, Rupon J : Fidanacogene Elaparvovec for Hemophilia B: A Multi-Year Follow-Up Study. N Engl J Med 392(15): (accepted), Apr 2025.
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 8: 177-7, Mar 2025.
Morris JJ, Parsons NA, Wilhelm AR, Davidson RJ, Olenick LK, Watson CT, Vanden Heuvel A, George LA: Factor IXa and Factor X Influence Factor VIIIa Stability and Inactivation Mechanisms In Vitro and In Vivo. Blood in press, 2025.
Samelson-Jones BJ, Doshi BS, George LA: Coagulation Factor VIII: Biological Basis of Emerging Hemophilia A Therapies Blood 144(21): 2185-2197, Nov 2024.
Samelson-Jones BJ, Small JC, George LA. : Roctavian Gene Therapy for Hemophilia A. Blood Adv 8(19): 5179-5189, Oct 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.
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.
Cao W, Trask AR, Bignotti AI, George LA, Doshi BS, Sabatino DE, Yada N, Zheng L, Camire RM, Zheng XL. : Coagulation factor VIII regulates von Willebrand factor homeostasis in vivo. J Thromb Haemost 21((12)), Dec 2023.