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Lindsey A. George, M.D.
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Assistant Professor of Pediatrics (Hematology)
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Department: Pediatrics
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Graduate Group Affiliations
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Contact information
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Children's Hospital of Philadelphia
39 3501 Civic Center Blvd
Philadelphia, PA 19104
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39 3501 Civic Center Blvd
Philadelphia, PA 19104
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Publications
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Links
b6 Search PubMed for articles
af Perelman School of Medicine at the University of Pennsylvania website
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b6 Search PubMed for articles
af Perelman School of Medicine at the University of Pennsylvania website
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Education:
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2b Cornell University, 2004.
21 7 MD c
5f State University at Buffalo School of Medicine and Biological Sciences, 2008.
21 9 MSTR 23 (Translational Research) c
46 University of Pennsylvania School of Medicine, 2021.
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Permanent link21 7 BS 14 (Biology) c
2b Cornell University, 2004.
21 7 MD c
5f State University at Buffalo School of Medicine and Biological Sciences, 2008.
21 9 MSTR 23 (Translational Research) c
46 University of Pennsylvania School of Medicine, 2021.
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Description of Clinical Expertise
4e Coagulation disorders and gene therapy for monogenic disorders71
Description of Research Expertise
50b Dr. Lindsey George is an Assistant Professor of Pediatrics at the University of Pennsylvania and Director of Clinical In Vivo Gene Therapy 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 biochemical insights of FVIII function to develop novel gene-based therapies for hemophilia A, which have translated to an ongoing hemophilia A gene therapy trial. Dr. George has additionally led early-phase, adeno-associated virus (AAV) mediated gene transfer trials for hemophilia A and B and conducted a series of translational studies to mechanistically interrogate clinical observations from AAV clinical trials. Her Clinical In Vivo Gene Therapy group conducts and supports translational and clinical research of in vivo gene addition and editing 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.e 29
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1e5 Keselman DJ, Small JC, Seneviratne T, McCague S, Kaschak TL, Yum SW, O'Brien A, Brandsema JF, Diamond T, Loomes KM, Lin KY, Wittlieb-Weber CA, Mayer OH, Daniel SG, Waldman AT, Samelson-Jones BJ, Matesanz SE, George LA: Real-World Outcomes of Delandistrogene Moxeparvovec Gene Therapy: Motor Outcomes and Emerging Safety Concerns. Mol Ther 34(1): 123-130, Jan 2026 Notes: Published with an accompanying editorial.
200 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.
2b8 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.
173 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(1): 177-7, Mar 2025 Notes: Published with an accompanying editorial.
11e 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, Aug 2024.
e2 Samelson-Jones BJ, Doshi BS, George LA: Coagulation Factor VIII: Biological Basis of Emerging Hemophilia A Therapies. Blood 144(21): 2185-2197, Nov 2024.
1b2 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.
107 Wilhelm AR, Parsons 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): 2532-2543, May 2021.
163 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 54 : Hemophilia B Gene Therapy with a High Specific Activity Factor IX Variant 85 N Engl J Med 377(2): 2215-2227, Dec 2017 Notes: Corresponding author, published with accompanying editorial.
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Selected Publications
16a 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 146(18): 2259-2271, Sep 2025 Notes: Published with an accompanying editorial.1e5 Keselman DJ, Small JC, Seneviratne T, McCague S, Kaschak TL, Yum SW, O'Brien A, Brandsema JF, Diamond T, Loomes KM, Lin KY, Wittlieb-Weber CA, Mayer OH, Daniel SG, Waldman AT, Samelson-Jones BJ, Matesanz SE, George LA: Real-World Outcomes of Delandistrogene Moxeparvovec Gene Therapy: Motor Outcomes and Emerging Safety Concerns. Mol Ther 34(1): 123-130, Jan 2026 Notes: Published with an accompanying editorial.
200 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.
2b8 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.
173 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(1): 177-7, Mar 2025 Notes: Published with an accompanying editorial.
11e 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, Aug 2024.
e2 Samelson-Jones BJ, Doshi BS, George LA: Coagulation Factor VIII: Biological Basis of Emerging Hemophilia A Therapies. Blood 144(21): 2185-2197, Nov 2024.
1b2 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.
107 Wilhelm AR, Parsons 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): 2532-2543, May 2021.
163 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 54 : Hemophilia B Gene Therapy with a High Specific Activity Factor IX Variant 85 N Engl J Med 377(2): 2215-2227, Dec 2017 Notes: Corresponding author, published with accompanying editorial.
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