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BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice.
Seth Chhabra E, Liu T, Kulman J, Patarroyo-White S, Yang B, Lu Q, Drager D, Moore N, Liu J, Holthaus AM, Sommer JM, Ismail A, Rabinovich D, Liu Z, van der Flier A, Goodman A, Furcht C, Tie M, Carlage T, Mauldin R, Dobrowsky TM, Liu Z, Mercury O, Zhu L, Mei B, Schellenberger V, Jiang H, Pierce GF, Salas J, Peters R. Seth Chhabra E, et al. Among authors: zhu l. Blood. 2020 Apr 23;135(17):1484-1496. doi: 10.1182/blood.2019001292. Blood. 2020. PMID: 32078672 Free PMC article.
Single synonymous mutation in factor IX alters protein properties and underlies haemophilia B.
Simhadri VL, Hamasaki-Katagiri N, Lin BC, Hunt R, Jha S, Tseng SC, Wu A, Bentley AA, Zichel R, Lu Q, Zhu L, Freedberg DI, Monroe DM, Sauna ZE, Peters R, Komar AA, Kimchi-Sarfaty C. Simhadri VL, et al. Among authors: zhu l. J Med Genet. 2017 May;54(5):338-345. doi: 10.1136/jmedgenet-2016-104072. Epub 2016 Dec 22. J Med Genet. 2017. PMID: 28007939 Free PMC article.
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