期刊
TRENDS IN GENETICS
卷 34, 期 8, 页码 600-611出版社
ELSEVIER SCIENCE LONDON
DOI: 10.1016/j.tig.2018.05.004
关键词
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资金
- Amon Carter Foundation
- Laurie Kraus Lacob Faculty Scholar Award in Pediatric Translational Research
- NIH [R01-AI097320, R01-AI120766]
- Lundbeck Foundation Fellowship, an AIAS-COFUND (Marie Curie) fellowship from Aarhus Institute of Advanced Studies (AIAS) - Aarhus University's Research Foundation
- EU's 7th Framework Program [609033]
- Novo Nordisk Foundation
- Carlsberg Foundation
- Slagtermester Max Worzner og Hustru Inger Worzners Mindelegat
- AP Moller Foundation
- Riisfort Foundation
Smithies et al. (1985) and Jasin and colleagues (1994) provided proof of concept that homologous recombination (HR) could be applied to the treatment of human disease and that its efficiency could be improved by the induction of double-strand breaks (DSBs). A key advance was the discovery of engineered nucleases, such as zinc-finger nucleases (ZFNs) and transcription activator-like (TAL) effector nucleases (TALENs), that can generate site-specific DSBs. The democratization and widespread use of genome editing was enabled by the discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 nuclease system. While genome editing using ZFNs and TALENs has already reached clinical trials, the pace at which genome editing enters human trials is bound to accelerate in the next several years with multiple promising preclinical studies heralding cures for monogenic diseases that are currently difficult to manage or even incurable. Here we review recent advances and current limitations and discuss the path forward using genome editing to understand, treat, and cure genetic diseases.
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