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CRISPR-Cas9:从细菌免疫系统到临床试验中的基因组编辑人类细胞。

CRISPR-Cas9: From a bacterial immune system to genome-edited human cells in clinical trials.

机构信息

a Center for Integrated Protein Science at the Department of Chemistry , Technische Universität München , Garching , Germany.

出版信息

Bioengineered. 2017 May 4;8(3):280-286. doi: 10.1080/21655979.2017.1299834. Epub 2017 Mar 13.

DOI:10.1080/21655979.2017.1299834
PMID:28287876
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5470512/
Abstract

The adaptive bacterial immune system CRISPR-Cas is revolutionizing all fields of life science and has opened up new frontiers toward personalised medicine. Since the elucidation of the molecular mechanism of Cas9 from Streptococcus pyogenes in 2012 and its development as a genomic engineering tool, genetic modifications in more than 40 species have been performed, over 290 patents have been filed worldwide and the first clinical trials using CRISPR-Cas-modified T-cells have recently been started in China and in the US. In this review we summarise current design developments, novel Cas systems and their antagonists, present and potential future applications as well as the ongoing debate on ethical issues, which has arisen through the CRISPR-Cas technology.

摘要

适应性细菌免疫系统 CRISPR-Cas 正在彻底改变生命科学的各个领域,并为个性化医疗开辟了新的前沿。自 2012 年阐明来自酿脓链球菌的 Cas9 的分子机制及其作为基因组工程工具的发展以来,已经对 40 多种物种进行了遗传修饰,全世界已经提交了超过 290 项专利,并且最近在中国和美国开始了使用 CRISPR-Cas 修饰的 T 细胞的首次临床试验。在这篇综述中,我们总结了当前的设计进展、新型 Cas 系统及其拮抗剂、当前和潜在的未来应用以及通过 CRISPR-Cas 技术产生的关于伦理问题的持续争论。

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本文引用的文献

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Naturally Occurring Off-Switches for CRISPR-Cas9.CRISPR-Cas9的天然存在的开关
Cell. 2016 Dec 15;167(7):1829-1838.e9. doi: 10.1016/j.cell.2016.11.017. Epub 2016 Dec 8.
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CRISPR/Cas9 mediated genome editing in ES cells and its application for chimeric analysis in mice.CRISPR/Cas9 介导的胚胎干细胞基因组编辑及其在小鼠嵌合分析中的应用。
Sci Rep. 2016 Aug 17;6:31666. doi: 10.1038/srep31666.
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