Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse

Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse

Abstract Background Hemophilia A, a bleeding disorder resulting from F8 mutations, can only be cured by gene therapy. A promising strategy is CRISPR-Cas9-mediated precise insertion of F8 in hepatocytes at highly expressed gene loci, such as albumin (Alb). Unfortunately, the precise in vivo integrati...

Full description

Bibliographic Details
Main Authors: Jian-Ping Zhang, Xin-Xin Cheng, Mei Zhao, Guo-Hua Li, Jing Xu, Feng Zhang, Meng-Di Yin, Fei-Ying Meng, Xin-Yue Dai, Ya-Wen Fu, Zhi-Xue Yang, Cameron Arakaki, Ruijun Jeanna Su, Wei Wen, Wen-Tian Wang, Wanqiu Chen, Hannah Choi, Charles Wang, Guangping Gao, Lei Zhang, Tao Cheng, Xiao-Bing Zhang
Format: Article
Language:English
Published: BMC 2019-12-01
Series:Genome Biology
Subjects:
Hemophilia A
CRISPR-Cas9
Genome editing
Knock-in
NHEJ
Online Access:https://doi.org/10.1186/s13059-019-1907-9

Internet

https://doi.org/10.1186/s13059-019-1907-9

Similar Items