Mitochondrial diseases and mtDNA editing

Genes Dis. 2023 Aug 9;11(3):101057. doi: 10.1016/j.gendis.2023.06.026. eCollection 2024 May.

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.

Keywords: Gene editing; Mitochondrial DNA mutation; Mitochondrialdisease; Transcription activator-like effector nucleases; Zinc finger nucleases.

Publication types

  • Review