Lindsey A. George, MD

Papers

2017

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

2016

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.

2015

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.

2014

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.

2013

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.

2004

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.

Abstracts

2017

Parsons NA, Ho MD, Davidson RJ, Thalji NK, Samelson-Jones BJ, Camire RM, George LA: Regulation of Factor VIII By Activated Protein C: Assessment in Vitro and In Vivo. Blood Dec 2017.

Hui DJ, Liu Y, Chen Y, Patel R, George LA, Sullivan SK, Giermasz A, Rasko J, Samelson-Jones BJ, Ducore JM, Dasen S, Carr ME, Anguela XM and High KA: SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response. Blood Dec 2017.

George LA, Sullivan SK, Giermasz A, Samuelson-Jones BJ, Ducore JM, Teitel JM,Cuker A, Von Mackensen S, Majumdar S, McGuinn CW, Runoski AR, Wright JF, Dasen S, Barber K, Chen Y, Hui DJ, Patel R, Liu Y, Wachtel K, Takefman D, Couto L, Reape K, Carr ME, Anguela X, High KA: SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence. Blood Dec 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 Dec 2017.

von Mackensen S, George LA, Carr M, Galvao AM, Luk AY, High KA: Preliminary Results of SPK-9001 Gene Transfer Demonstrate Statistical Improvements on the Health-Related Quality-of-Life in Adults with Hemophilia B. Res Pract Thromb Haemost 1(Suppl 1), Jul 2017.

George LA, Sullivan SK, Giermasz A, Ducore JM, Teitel JM, Samelson-Jones BJ , Cuker A, Sullivan LM, Majumdar S, McGuinn CE, Galvao AM, Luk AY, Wright JF, Chen Y, Hui DJ, Wachtel K, Galas T, Takefman D, Couto LB, Carr ME, Anguela XM, High KA.: SPK-9001: Adeno-associated Virus Mediated Gene Transfer for Haemophilia B Achieved Durable Endogenous Prophylaxis at Levels of Activity Sufficient to Achieve Significant Mean Reduction in Annual Bleeding and Infusions Rates in Preliminary Data from an On-going Phase 1/2a Trial. Res Pract Thromb Haemost 1(Suppl 1): 143, Jul 2017.

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, Sullivan SK, Giermasz A, Ducore J, Teitel J, Cuker A, Sullivan LM, Majumdar S, McGuinn CE, Luk AY, Galvao AM, Urich T, Wright JF, Chen Y, Hui D, Wachtel K, Takefman D, Carr ME, Couto L, Anguela X, High KA: Adeno-associated virus mediated gene transfer for hemophilia B at the low vector dose achieved sustained, continuous factor IX activity levels adequate for endogenous prophylaxis preventing bleeding episodes and reducing the risk of immune response. Mol Ther 25(5 Suppl 1): 331, Mar 2017.

Books

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

2016

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

2018

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.

2017

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.

2016

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.