Yang Xingbo, Jiang Jiacheng, Li Zongyu, Liang Jiayi, Xiang Yaozu
School of Life Sciences and Technology, Tongji University, Shanghai 200092, China.
School of Mathematics and Statistics, University of Glasgow, Glasgow G12 8QQ, United Kingdom.
Comput Struct Biotechnol J. 2021 Jun 4;19:3319-3329. doi: 10.1016/j.csbj.2021.06.003. eCollection 2021.
Mitochondria, as the energy factory of cells, participate in metabolism processes and play a critical role in the maintenance of human life activities. Mitochondria belong to semi-automatic organelles, which have their own genome different from nuclear genome. Mitochondrial DNA (mtDNA) mutations can cause a series of diseases and threaten human health. However, an effective approach to edit mitochondrial DNA, though long-desired, is lacking. In recent years, gene editing technologies, represented by restriction endonucleases (RE) technology, zinc finger nuclease (ZFN) technology, transcription activator-like effector nuclease (TALEN) technology, CRISPR system and pAgo-based system have been comprehensively explored, but the application of these technologies in mitochondrial gene editing is still to be explored and optimized. The present study highlights the progress and limitations of current mitochondrial gene editing technologies and approaches, and provides insights for development of novel strategies for future attempts.
线粒体作为细胞的能量工厂,参与新陈代谢过程,对维持人类生命活动起着关键作用。线粒体属于半自主细胞器,拥有与核基因组不同的自身基因组。线粒体DNA(mtDNA)突变可引发一系列疾病,威胁人类健康。然而,尽管长期以来人们一直渴望有一种有效的方法来编辑线粒体DNA,但目前仍缺乏这样的方法。近年来,以限制性内切酶(RE)技术、锌指核酸酶(ZFN)技术、转录激活样效应因子核酸酶(TALEN)技术、CRISPR系统和基于pAgo的系统为代表的基因编辑技术得到了全面探索,但这些技术在线粒体基因编辑中的应用仍有待探索和优化。本研究重点介绍了当前线粒体基因编辑技术和方法的进展与局限性,并为未来尝试开发新策略提供了思路。