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

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

Abstract 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 palindrom...

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Main Authors: Tsukasa Ohmori, Yasumitsu Nagao, Hiroaki Mizukami, Asuka Sakata, Shin-ichi Muramatsu, Keiya Ozawa, Shin-ichi Tominaga, Yutaka Hanazono, Satoshi Nishimura, Osamu Nureki, Yoichi Sakata
Format: article
Language:EN
Published: Nature Portfolio 2017
Subjects:
Medicine
R
Science
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Online Access:https://doaj.org/article/b79acbb0c9004973b1526e004ab7e566
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https://doaj.org/article/b79acbb0c9004973b1526e004ab7e566

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