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

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 editing tool...

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Detalles Bibliográficos
Autores principales: Lyu, Cuicui, Shen, Jun, Wang, Rui, Gu, Haihui, Zhang, Jianping, Xue, Feng, Liu, Xiaofan, Liu, Wei, Fu, Rongfeng, Zhang, Liyan, Li, Huiyuan, Zhang, Xiaobing, Cheng, Tao, Yang, Renchi, Zhang, Lei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5889534/
https://www.ncbi.nlm.nih.gov/pubmed/29625575
http://dx.doi.org/10.1186/s13287-018-0839-8

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