Cell Biology and Toxicology ( IF 5.3 ) Pub Date : 2020-07-30 , DOI: 10.1007/s10565-020-09549-x Willian Wang 1 , Xiangdong Wang 1
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.
中文翻译:
线粒体 DNA 编辑的新潜力。
使用线粒体 DNA (mtDNA) 进行基因修饰和编辑是线粒体功能障碍常规疗法的一种有前景的选择,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 衍生的胞嘧啶碱基编辑器因其在人类 mtDNA 中的高靶标特异性和高产品纯度而成为一种很有前途的方法。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。