Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy

Pasi, KJ; Rangarajan, S; Georgiev, P; Mant, T; Creagh, MD; Lissitchkov, T; Bevan, D; Austin, S; Hay, CR; Hegemann, I; Kazmi, R; Chowdary, P; Gercheva-Kyuchukova, L; Mamonov, V; Timofeeva, M; Soh, CH; Garg, P; Vaishnaw, A; Akinc, A; Sorensen, B; Ragni

Pasi, KJ (reprint author), Royal London Hosp, Haemophilia Ctr, London E1 1BB, England.

NEW ENGLAND JOURNAL OF MEDICINE, 2017; 377 (9): 819

Abstract

BACKGROUND Current hemophilia treatment involves frequent intravenous infusions of clotting factors, which is associated with variable hemostatic prot......

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