1. Department of Cardiovascular Surgery of the First Affiliated Hospital & Institute for Cardiovascular Science, Collaborative Innovation Center of Hematology, State Key Laboratory of Radiation Medicine and Protection, Suzhou Medical College, Soochow University,Jiangsu,Suzhou,China,215000
Min Song, Lingqun Ye, Yongjin Yan, et al. Mitochondrial diseases and mtDNA editing. [J]. Genes & Diseases 11(3).(2024)
DOI:
Min Song, Lingqun Ye, Yongjin Yan, et al. Mitochondrial diseases and mtDNA editing. [J]. Genes & Diseases 11(3).(2024) DOI: 10.1016/j.gendis.2023.06.026.
Mitochondrial diseases and mtDNA editing
摘要
Abstract
Mitochondrial diseases are a heterogeneous group of inherited disorders characterized by mitochondrial dysfunction
and these diseases are often severe or even fatal. Mitochondrial diseases are often caused by mitochondrial DNA mutations. Currently
there is no curative treatment for patients with pathogenic mitochondrial DNA mutations. With the rapid development of traditional gene editing technologies
such as zinc finger nucleases and transcription activator-like effector nucleases methods
there has been a search for a mitochondrial gene editing technology that can edit mutated mitochondrial DNA; however
there are still some problems hindering the application of these methods. The discovery of the DddA-derived cytosine base editor has provided hope for mitochondrial gene editing. In this paper
we will review the progress in the research on several mitochondrial gene editing technologies with the hope that this review will be useful for further research on mitochondrial gene editing technologies to optimize the treatment of mitochondrial diseases in the future.
关键词
Keywords
Gene editingMitochondrialdiseaseMitochondrial DNA mutationTranscription activator-like effector nucleasesZinc finger nucleases