Ontology highlight
ABSTRACT:
SUBMITTER: Ohmori T
PROVIDER: S-EPMC5482879 | biostudies-other | 2017 Jun
REPOSITORIES: biostudies-other
Ohmori Tsukasa T Nagao Yasumitsu Y Mizukami Hiroaki H Sakata Asuka A Muramatsu Shin-Ichi SI Ozawa Keiya K Tominaga Shin-Ichi SI Hanazono Yutaka Y Nishimura Satoshi S Nureki Osamu O Sakata Yoichi Y
Scientific reports 20170623 1
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 (SaCas9) and single guide RNA (sgRNA) to wild-type adult mice induced a double-stra ...[more]