Optical Control of CRISPR/Cas9 Gene Editing.

Optical Control of CRISPR/Cas9 Gene Editing.
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DOI:
10.1021/ja512664v
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发表时间:
2015-05-06
影响因子:
15
通讯作者:
Deiters A
Deiters A
中科院分区:
化学1区
文献类型:
--
作者:
Hemphill J;Borchardt EK;Brown K;Asokan A;Deiters A

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CRISPR/Cas9系统已成为生物医学研究的重要工具,具有广泛的应用,在基因组工程和基因治疗方面具有巨大的潜力。为了实现 CRISPR/Cas9 系统的条件控制,通过在位点特异性安装笼状赖氨酸氨基酸,设计了基因编码的光激活 Cas9。几个潜在的赖氨酸残基被确定为可行的笼养位点,可以对其进行修饰以光学控制 Cas9 功能,正如通过外源和内源基因功能的光学激活和失活所证明的那样。
The CRISPR/Cas9 system has emerged as an important tool in biomedical research for a wide range of applications, with significant potential for genome engineering and gene therapy. In order to achieve conditional control of the CRISPR/Cas9 system, a genetically encoded light-activated Cas9 was engineered through the site-specific installation of a caged lysine amino acid. Several potential lysine residues were identified as viable caging sites that can be modified to optically control Cas9 function, as demonstrated through optical activation and deactivation of both exogenous and endogenous gene function.