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CRISPR-Cas:从细菌适应性免疫系统到基因组工程的多功能工具。

CRISPR-Cas: From the Bacterial Adaptive Immune System to a Versatile Tool for Genome Engineering.

机构信息

Center for Integrated Protein Science, Department of Chemistry, Technische Universität München, Lichtenbergstrasse 4, 85748 Garching (Germany).

出版信息

Angew Chem Int Ed Engl. 2015 Nov 9;54(46):13508-14. doi: 10.1002/anie.201504741. Epub 2015 Sep 18.

Abstract

The field of biology has been revolutionized by the recent advancement of an adaptive bacterial immune system as a universal genome engineering tool. Bacteria and archaea use repetitive genomic elements termed clustered regularly interspaced short palindromic repeats (CRISPR) in combination with an RNA-guided nuclease (CRISPR-associated nuclease: Cas) to target and destroy invading DNA. By choosing the appropriate sequence of the guide RNA, this two-component system can be used to efficiently modify, target, and edit genomic loci of interest in plants, insects, fungi, mammalian cells, and whole organisms. This has opened up new frontiers in genome engineering, including the potential to treat or cure human genetic disorders. Now the potential risks as well as the ethical, social, and legal implications of this powerful new technique move into the limelight.

摘要

生物学领域最近出现了一种适应性细菌免疫系统,它作为一种通用的基因组工程工具,引发了革命性的变化。细菌和古菌利用重复的基因组元件,称为成簇规律间隔短回文重复序列(CRISPR),结合 RNA 指导的核酸酶(CRISPR 相关核酸酶:Cas)来靶向并破坏入侵的 DNA。通过选择适当的向导 RNA 序列,这个双组分系统可以有效地修饰、靶向和编辑植物、昆虫、真菌、哺乳动物细胞和整个生物体中感兴趣的基因组位点。这为基因组工程开辟了新的前沿,包括治疗或治愈人类遗传疾病的潜力。现在,这项强大新技术的潜在风险以及伦理、社会和法律影响成为了焦点。

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