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

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

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 integration efficiency of a l...

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Príomhchruthaitheoirí: Jianping Zhang, Xinxin Cheng, Mei Zhao, Guohua Li, Jing Xu, Feng Zhang, Mengdi Yin, Feiying Meng, Xinyue Dai, Ya-Wen Fu, Zhi-Xue Yang, Cameron Arakaki, Ruijun Su, Wei Wen, Wentian Wang, Wanqiu Chen, Hannah Choi, Charles Wang, Guangping Gao, Lei Zhang, Tao Cheng, Xiao‐Bing Zhang
Formáid: Artigo
Teanga:Béarla
Foilsithe / Cruthaithe: 2019
Rochtain ar líne:https://doi.org/10.1186/s13059-019-1907-9
https://genomebiology.biomedcentral.com/track/pdf/10.1186/s13059-019-1907-9
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https://doi.org/10.1186/s13059-019-1907-9
https://genomebiology.biomedcentral.com/track/pdf/10.1186/s13059-019-1907-9

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