Development and applications of CRISPR-Cas9 for genome engineering.

Development and applications of CRISPR-Cas9 for genome engineering.
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DOI:
10.1016/j.cell.2014.05.010
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发表时间:
2014-06-05
期刊:
影响因子:
64.5
通讯作者:
Zhang F
Zhang F
中科院分区:
生物学1区
文献类型:
--
作者:
Hsu PD;Lander ES;Zhang F

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基于CRISPR相关的RNA引导的核酸内切酶CAS9的基因组工程技术的最新进展正在实现哺乳动物基因组功能的系统询问。类似于现代文字处理器中的搜索函数,CAS9可以通过短的RNA搜索字符串引导到复杂基因组中的特定位置。使用此系统,现在几乎可以在任何选择的生物体中对内源基因组中的DNA序列及其功能输出进行编辑或调节。 CAS9介导的遗传扰动是简单且可扩展的,赋予了研究人员的能力,以阐明基因组在系统级别的功能组织,并在遗传变异和生物表型之间建立因果关系。在这篇综述中,我们描述了CAS9在各种研究或翻译应用中的开发和应用,同时强调挑战以及未来的方向。 Cas9源自显着的微生物防御系统,将创新的应用从基本生物学推向生物技术和医学。
Recent advances in genome engineering technologies based on the CRISPR-associated RNA-guided endonuclease Cas9 are enabling the systematic interrogation of mammalian genome function. Analogous to the search function in modern word processors, Cas9 can be guided to specific locations within complex genomes by a short RNA search string. Using this system, DNA sequences within the endogenous genome and their functional outputs are now easily edited or modulated in virtually any organism of choice. Cas9-mediated genetic perturbation is simple and scalable, empowering researchers to elucidate the functional organization of the genome at the systems level and establish causal linkages between genetic variations and biological phenotypes. In this Review, we describe the development and applications of Cas9 for a variety of research or translational applications while highlighting challenges as well as future directions. Derived from a remarkable microbial defense system, Cas9 is driving innovative applications from basic biology to biotechnology and medicine.
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