Novel Therapeutics for Bleeding Disorders Publications

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Below is a list of selected publications from the Novel Therapeutics for Bleeding Disorders Frontier Program.

Xue F, et al. Total Knee Arthroplasty after Gene Therapy for Hemophilia B. N Engl J Med. 2022 Oct; 387(17):1622-1624. PMID: 36306204
Samelson-Jones BJ, George LA. Adeno-associated Virus Gene Therapy for Hemophilia. Annu Rev Med. 2022 Sep; PMID: 36103998
George LA, et al. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. N Engl J Med. 2021 Nov; 385(21):1961-1973. PMID: 34788507
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. 2021 May; 137(18):2532-2543. PMID: 33512448
Robinson MM, George LA, Carr ME, Samelson-Jones BJ, Arruda VR, Murphy JE, Rybin D, 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. 2021 May; 19(5):1212-1218. PMID: 33636038
George LA, et al. Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B. Mol Ther. 2020 Sep; 28(9):2073-2082. PMID: 32559433
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. 2020 Apr; 18(4):848-852. PMID: 31985872
George LA, et al. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. N Engl J Med. 2017 Dec; 377(23):2215-2227. PMID: 29211678