Paired CRISPR/Cas9 Nickases Mediate Efficient Site-Specific Integration of F9 into rDNA Locus of Mouse ESCs

Paired CRISPR/Cas9 Nickases Mediate Efficient Site-Specific Integration of F9 into rDNA Locus of Mouse ESCs

Hemophilia B (HB) is an X-linked recessive bleeding disorder, caused by F9 gene deficiency. Gene therapy combined with the CRISPR/Cas9 technology offers a potential cure for hemophilia B. Now the Cas9 nickase (Cas9n) shows a great advantage in reducing off-target effect compared with wild-type Cas9....

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Bibliographic Details
Main Authors: Yanchi Wang, Junya Zhao, Nannan Duan, Wei Liu, Yuxuan Zhang, Miaojin Zhou, Zhiqing Hu, Mai Feng, Xionghao Liu, Lingqian Wu, Zhuo Li, Desheng Liang
Format: Article
Language:English
Published: MDPI AG 2018-10-01
Series:International Journal of Molecular Sciences
Subjects:
CRISPR/Cas9 nickase
gene targeting
Hemophilia B
ribosomal DNA
gene therapy
mESCs
hepatic progenitor like cells
intrasplenic transplantation
Online Access:http://www.mdpi.com/1422-0067/19/10/3035

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http://www.mdpi.com/1422-0067/19/10/3035

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