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, 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. 2021 Nov; 385(21):1961-1973. doi: 10.1056/NEJMoa2104205. PMID: 34788507; PMCID: PMC8672712
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. 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; In Press, 2020
George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, 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. 2017 Dec; 377(23):2215-2227. PMID: 29211678