Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system

Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system

Abstract Background Replacement therapy for hemophilia remains a lifelong treatment. Only gene therapy can cure hemophilia at a fundamental level. The clustered regularly interspaced short palindromic repeats–CRISPR associated nuclease 9 (CRISPR-Cas9) system is a versatile and convenient genome edit...

Full description

Bibliographic Details
Main Authors: Cuicui Lyu, Jun Shen, Rui Wang, Haihui Gu, Jianping Zhang, Feng Xue, Xiaofan Liu, Wei Liu, Rongfeng Fu, Liyan Zhang, Huiyuan Li, Xiaobing Zhang, Tao Cheng, Renchi Yang, Lei Zhang
Format: Article
Language:English
Published: BMC 2018-04-01
Series:Stem Cell Research & Therapy
Subjects:
CRISPR-Cas systems
Induced pluripotent stem cells
Hemophilia B
Genetic therapy
Cellular therapy
Hepatocyte differentiation
Online Access:http://link.springer.com/article/10.1186/s13287-018-0839-8

Internet

http://link.springer.com/article/10.1186/s13287-018-0839-8

Similar Items