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Article

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

Scientific reports - 23 Jun 2017

Ohmori Tsukasa, Nagao Yasumitsu, Mizukami Hiroaki, Sakata Asuka, Muramatsu Shin-Ichi, Ozawa Keiya, Tominaga Shin-Ichi, Hanazono Yutaka, Nishimura Satoshi, Nureki Osamu, Sakata Yoichi

Abstract excerpt

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 (CRISPR)/Cas9 system. Administration of adeno-associated virus (AAV) 8 vector harbouring Staphylococcus aureus Cas9...

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Identifiers

PMID: 28646206
DOI: 10.1038/s41598-017-04625-5
PMCID: PMC5482879

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