The use of new CRISPR tools in cardiovascular research and medicine.
The use of new CRISPR tools in cardiovascular research and medicine.
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DOI：
10.1038/s41569-021-00669-3
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发表时间：
2022-08
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在过去的几年里，许多基于CRISPR的新型基因组编辑工具已经开发出来，具有广泛的应用。最初的CRISPR/Cas9(簇状规则间隔短回文重复序列/CRISPR相关蛋白9)系统是作为一种工具以一种简单的方式改变生物体内的基因组序列。然而，新的CRISPR工具的功能并不局限于由非同源末端连接或同源定向修复介导的传统基因组编辑，而是扩展到基因表达控制、表观基因组编辑、单核苷酸编辑、RNA编辑和活细胞成像。此外，通过多重引导RNA进行遗传扰动筛查作为一种无偏见地识别致病基因和途径的方法正变得越来越受欢迎。新的CRISPR工具还可以用于体外或体内治疗性基因组编辑，以治疗高脂血症等疾病。在这篇综述中，我们首先概述了迄今为止已经开发的各种新的CRISPR工具。其次，我们总结了这些新的CRISPR工具如何被用于研究心血管研究和医学中的生物过程和疾病机制。最后，我们讨论了利用CRISPR工具进行治疗性基因组编辑以治疗遗传性心血管疾病的前景。
Many novel CRISPR-based genome editing tools, with a wide variety of applications, have been developed in the past few years. The original CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) system was developed as a tool to alter genomic sequences within living organisms in a simple way. However, the functions of new CRISPR tools are not limited to conventional genome editing mediated by nonhomologous end-joining or homology-directed repair, but expand into gene-expression control, epigenome editing, single nucleotide editing, RNA editing and live-cell imaging. Furthermore, genetic perturbation screening by multiplexing guide RNAs is gaining popularity as a method of identifying causative genes and pathways in an unbiased manner. New CRISPR tools can also be applied to ex vivo or in vivo therapeutic genome editing for the treatment of conditions such as hyperlipidaemia. In this Review, we first provide an overview of the diverse new CRISPR tools that have been developed to date. Second, we summarize how these new CRISPR tools are being used to study biological processes and disease mechanisms in cardiovascular research and medicine. Finally, we discuss the prospect of therapeutic genome editing by CRISPR tools to cure genetic cardiovascular diseases.
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DOI：
10.1126/science.aaf5573
发表时间：
2016-08-05
期刊：
Science (New York, N.Y.)
影响因子：
--
作者：
Abudayyeh OO;Gootenberg JS;Konermann S;Joung J;Slaymaker IM;Cox DB;Shmakov S;Makarova KS;Semenova E;Minakhin L;Severinov K;Regev A;Lander ES;Koonin EV;Zhang F
通讯作者：
Zhang F
使用 CRISPR/Cas9 对恒河猴的肌营养不良蛋白基因进行功能破坏。
影响因子：
3.5
作者：
Chen, Yongchang;Zheng, Yinghui;Li, Xiao-Jiang
通讯作者：
Li, Xiao-Jiang
影响因子：
82.9
作者：
Charlesworth, Carsten T.;Deshpande, Priyanka S.;Porteus, Matthew H.
通讯作者：
Porteus, Matthew H.
影响因子：
64.8
作者：
Abudayyeh OO;Gootenberg JS;Essletzbichler P;Han S;Joung J;Belanto JJ;Verdine V;Cox DBT;Kellner MJ;Regev A;Lander ES;Voytas DF;Ting AY;Zhang F
通讯作者：
Zhang F
影响因子：
64.8
作者：
Anzalone, Andrew V.;Randolph, Peyton B.;Liu, David R.
通讯作者：
Liu, David R.