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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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Detalles Bibliográficos
Autores principales:	Wang, Yanchi, Zhao, Junya, Duan, Nannan, Liu, Wei, Zhang, Yuxuan, Zhou, Miaojin, Hu, Zhiqing, Feng, Mai, Liu, Xionghao, Wu, Lingqian, Li, Zhuo, Liang, Desheng
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6213315/ https://www.ncbi.nlm.nih.gov/pubmed/30301136 http://dx.doi.org/10.3390/ijms19103035

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