期刊
STEM CELL REPORTS
卷 3, 期 6, 页码 940-947出版社
CELL PRESS
DOI: 10.1016/j.stemcr.2014.09.013
关键词
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资金
- NIH [EB015300, AI096305, UH2TR000505, DP2OD008586, R01DA036865]
- National Science Foundation [CBET-1151035]
- American Heart Association [10SDG3060033]
- Muscular Dystrophy Association [MDA277360]
- Flight Attendant Medical Research Institute
- Directorate For Engineering
- Div Of Chem, Bioeng, Env, & Transp Sys [1151035] Funding Source: National Science Foundation
Gene activation by the CRISPR/Cas9 system has the potential to enable new approaches to science and medicine, but the technology must be enhanced to robustly control cell behavior. We show that the fusion of two transactivation domains to Cas9 dramatically enhances gene activation to a level that is necessary to reprogram cell phenotype. Targeted activation of the endogenous Myod1 gene locus with this system led to stable and sustained reprogramming of mouse embryonic fibroblasts into skeletal myocytes. The levels of myogenic marker expression obtained by the activation of endogenous Myod1 gene were comparable to that achieved by overexpression of lentivirally delivered MYOD1 transcription factor.
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