Current Clinical Applications of In Vivo Gene Therapy with AAVs

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2020

2019

Nathwani, 2011, Adenovirus-associated virus vector-mediated gene transfer in hemophilia B, N. Engl. J. Med., 365, 2357, 10.1056/NEJMoa1108046

Nathwani, 2014, Long-term safety and efficacy of factor IX gene therapy in hemophilia B, N. Engl. J. Med., 371, 1994, 10.1056/NEJMoa1407309

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Chapin, 2017, SHP648: an analysis of bleeding rates and Factor IX Consumption in the Phase I/II BAX 335 Gene Therapy Trial in Subjects with Hemophilia B, Res. Pract. Thromb. Haemost., 2017, 144

Miesbach, 2018, Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B, Blood, 131, 1022, 10.1182/blood-2017-09-804419

Miesbach, 2019, Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B, Blood, 134, 2059, 10.1182/blood-2019-122535

2018

George, 2017, Hemophilia gene therapy comes of age, Hematology (Am. Soc. Hematol. Educ. Program), 2017, 587, 10.1182/asheducation-2017.1.587

Chowdary, 2020, A novel adeno associated virus (AAV) gene therapy (FLT180a) achieves normal FIX activity levels in severe Hemophilia B (HB) patients (B-AMAZE study), Res. Pract. Thromb. Haemost., 4

George, 2017, Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant, N. Engl. J. Med., 377, 2215, 10.1056/NEJMoa1708538

George, 2019, Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year, Blood, 134, 3347, 10.1182/blood-2019-124091

Majowicz, 2020, Therapeutic human Factor IX (hFIX) activity after single treatment with AMT-060 (AAV5-hFIX) or AMT-061 (AAV5-Padua hFIX) in Hemophilia B patients with pre-existing anti-AAV5 humoral immunity, Haemophilia, 26, 20

Rangarajan, 2017, AAV5-Factor VIII Gene Transfer in Severe Hemophilia A, N. Engl. J. Med., 377, 2519, 10.1056/NEJMoa1708483

Pasi, 2020, Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A, N. Engl. J. Med., 382, 29, 10.1056/NEJMoa1908490

2020

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