Abstract：
The manipulation of animal mitochondrial genomes has long been a challenge due to the lack of an effective transformation method. With the discovery of specific gene editing enzymes, designed to target pathogenic mitochondrial DNA mutations (often heteroplasmic), the selective removal or modification of mutant variants has become a reality. Because mitochondria cannot efficiently import RNAs, CRISPR has not been the first choice for editing mitochondrial genes. However, the last few years witnessed an explosion in novel and optimized non-CRISPR approaches to promote double-strand breaks or base-edit of mtDNA in vivo. Engineered forms of specific nucleases and cytidine/adenine deaminases form the basis for these techniques. I will review the newest developments that constitute the current toolbox for animal mtDNA gene editing in vivo, bringing these approaches not only to the exploration of mitochondrial function, but also closer to clinical use.
摘要：
由于缺乏有效的转化方法，动物线粒体基因组的操作长期以来一直是一个挑战。随着特定基因编辑酶的发现,旨在针对致病性线粒体DNA突变（通常是异质），突变变体的选择性去除或修饰已成为现实。因为线粒体不能有效地导入RNA,CRISPR并不是编辑线粒体基因的首选。然而,在过去的几年中，新型优化的非CRISPR方法在体内促进mtDNA的双链断裂或碱基编辑方面出现了爆炸式增长。特定核酸酶和胞苷/腺嘌呤脱氨酶的工程化形式形成这些技术的基础。我将回顾构成当前体内动物mtDNA基因编辑工具箱的最新进展，这些方法不仅用于探索线粒体功能，而且更接近临床使用。
