CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice

Ohmori, T; Nagao, Y; Mizukami, H; Sakata, A; Muramatsu, S; Ozawa, K; Tominaga, S; Hanazono, Y; Nishimura, S; Nureki, O; Sakata, Y

Ohmori, T (reprint author), Jichi Med Univ, Sch Med, Dept Biochem, Shimotsuke, Tochigi 3290498, Japan.

SCIENTIFIC REPORTS, 2017; 7 ( ):

Abstract

Haemophilia B, a congenital haemorrhagic disease caused by mutations in coagulation factor IX gene (F9), is considered an appropriate target for genom......

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