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...

Full description

Bibliographic Details
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:English
Published: Nature Publishing Group 2017-06-01
Series:Scientific Reports
Online Access:https://doi.org/10.1038/s41598-017-04625-5

Internet

https://doi.org/10.1038/s41598-017-04625-5

Similar Items