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CRISPR/Cas 系统对线粒体 DNA 的修饰:挑战与未来方向。

Mitochondrial DNA modification by CRISPR/Cas system: Challenges and future direction.

机构信息

Department of Protein Chemistry and Technology, CSIR-Central Food Technological Research Institute, Mysuru, India; Academy of Scientific and Innovative Research (AcSIR), CSIR-Central Food Technological Research Institute (CSIR-CFTRI) Campus, Mysuru, India.

Department of Protein Chemistry and Technology, CSIR-Central Food Technological Research Institute, Mysuru, India; Academy of Scientific and Innovative Research (AcSIR), CSIR-Central Food Technological Research Institute (CSIR-CFTRI) Campus, Mysuru, India.

出版信息

Prog Mol Biol Transl Sci. 2021;178:193-211. doi: 10.1016/bs.pmbts.2020.12.009. Epub 2021 Jan 27.

Abstract

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas (CRISPR associated endonuclease), a hotshot genome editing tool which is originally known to be the form of prokaryotic adaptive immune system against viral infections has gained all the attention of scientific community as a promising genome editing platform. This review encompasses a brief description of mitochondrial disease conditions associated with the alteration in mitochondrial genome (mtDNA) and highlights the key role of the CRISPR/Cas system pertaining to its working mechanism and its involvement in gene-based therapeutics in treating the foresaid mitochondrial diseases. Here, we also extend the perception related to the detailed mechanism of CRISPR/Cas system in mtDNA modification.

摘要

CRISPR(成簇规律间隔短回文重复)/Cas(CRISPR 相关内切酶)是一种热门的基因组编辑工具,最初被认为是原核生物对抗病毒感染的适应性免疫系统的形式,现已成为科学界关注的焦点,成为一种有前途的基因组编辑平台。本综述简要描述了与线粒体基因组(mtDNA)改变相关的线粒体疾病状况,并强调了 CRISPR/Cas 系统在其工作机制及其在治疗上述线粒体疾病的基因治疗中的关键作用。在这里,我们还扩展了对 CRISPR/Cas 系统在 mtDNA 修饰中的详细机制的相关认识。

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