An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia.

doi:10.1038/nm.3847

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Article

An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia.

Nature medicine - 1 May 2015

Sehgal Alfica, Barros Scott, Ivanciu Lacramioara, Cooley Brian, Qin June, Racie Tim, Hettinger Julia, Carioto Mary, Jiang Yongfeng, Brodsky Josh, Prabhala Harsha, Zhang Xuemei, Attarwala Husain, Hutabarat Renta, Foster Don, Milstein Stuart, Charisse Klaus, Kuchimanchi Satya, Maier Martin A, Nechev Lubo, Kandasamy Pachamuthu, Kel'in Alexander V, Nair Jayaprakash K, Rajeev Kallanthottathil G, Manoharan Muthiah, Meyers Rachel, Sorensen Benny, Simon Amy R, Dargaud Yesim, Negrier Claude, Camire Rodney M, Akinc Akin

Abstract excerpt

Hemophilia A and B are inherited bleeding disorders characterized by deficiencies in procoagulant factor VIII (FVIII) or factor IX (FIX), respectively. There remains a substantial unmet medical need in hemophilia, especially in patients with inhibitory antibodies against replacement factor therapy, for novel and improved therapeutic agents that can be used prophylactically to provide effective hemostasis. Guided...

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PMID: 25849132
DOI: 10.1038/nm.3847

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