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

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 integration effic...

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Detalles Bibliográficos
Autores principales: Zhang, Jian-Ping, Cheng, Xin-Xin, Zhao, Mei, Li, Guo-Hua, Xu, Jing, Zhang, Feng, Yin, Meng-Di, Meng, Fei-Ying, Dai, Xin-Yue, Fu, Ya-Wen, Yang, Zhi-Xue, Arakaki, Cameron, Su, Ruijun Jeanna, Wen, Wei, Wang, Wen-Tian, Chen, Wanqiu, Choi, Hannah, Wang, Charles, Gao, Guangping, Zhang, Lei, Cheng, Tao, Zhang, Xiao-Bing
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6912951/
https://www.ncbi.nlm.nih.gov/pubmed/31843008
http://dx.doi.org/10.1186/s13059-019-1907-9

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