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Scientific reports, v.7, 2017년, pp.4159 -
Ohmori, Tsukasa (Department of Biochemistry, Jichi Medical University School of Medicine, Tochigi, 329-0498 Japan) , Nagao, Yasumitsu (Center for Experimental Medicine, Jichi Medical University, Tochigi, 329-0498 Japan) , Mizukami, Hiroaki (Division of Genetic Therapeutics, Center for Molecular Medicine, Jichi Medical University, Tochigi, 329-0498 Japan) , Sakata, Asuka (Division of Cell and Molecular Medicine, Center for Molecular Medicine, Jichi Medical University, Tochigi, 329-0498 Japan) , Muramatsu, Shin-ichi (Department of Neurology, Jichi Medical University School of Medicine, Tochigi, 329-0498 Japan) , Ozawa, Keiya (The Institute of Medical Science, The University of Tokyo, Tokyo, 108-0071 Japan) , Tominaga, Shin-ichi (Department of Biochemistry, Jichi Medical University School of Medicine, Tochigi, 329-0498 Japan) , Hanazono, Yutaka (Division of Regenerative Medicine, Center for Molecular Medicine, Jichi Medical University, Tochigi, 329-0498 Japan) , Nishimura, Satoshi (Division of Cell and Molecular Medicine, Center for Molecular Medicine, Jichi Medical University, Tochigi, 329-0498 Japan) , Nureki, Osamu (Department of Biological Sciences, Graduate School of) , Sakata, Yoichi
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Haemophilia B, a congenital haemorrhagic disease caused by mutations in coagulation factor IX gene (F9), is considered an appropriate target for genome editing technology. Here, we describe treatment strategies for haemophilia B mice using the clustered regularly interspaced short palindromic repeat...
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