Wang Willian, Wang Xiangdong
Zhongshan Hospital Institute of Clinical Science, Fudan University Shanghai Medical College, Shanghai, China.
Cell Biol Toxicol. 2020 Oct;36(5):391-393. doi: 10.1007/s10565-020-09549-x. Epub 2020 Jul 30.
Gene modification and editing using mitochondrial DNA (mtDNA) is a promising option to conventional therapy for mitochondrial dysfunction which can result in a wide range of diseases and failure of organ and tissue functions. RNA-free DddA-derived cytosine base editors have been a promising method due to its high target specificity and high product purity in human mtDNA. However, mtDNA editing is still only a promising option and further studies and investigations are required prior to implementing it in different cell types, organelles, and various diseases as a new clinical therapy.
使用线粒体DNA（mtDNA）进行基因修饰和编辑是传统线粒体功能障碍治疗方法的一个有前景的选择，线粒体功能障碍可导致多种疾病以及器官和组织功能衰竭。无RNA的DddA衍生胞嘧啶碱基编辑器因其在人类mtDNA中的高靶向特异性和高产物纯度而成为一种有前景的方法。然而，mtDNA编辑仍然只是一个有前景的选择，在将其作为一种新的临床疗法应用于不同细胞类型、细胞器和各种疾病之前，还需要进一步的研究和调查。
