Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities

Int J Mol Sci. 2023 Mar 18;24(6):5798. doi: 10.3390/ijms24065798.


Mitochondria are critical organelles that form networks within our cells, generate energy dynamically, contribute to diverse cell and organ function, and produce a variety of critical signaling molecules, such as cortisol. This intracellular microbiome can differ between cells, tissues, and organs. Mitochondria can change with disease, age, and in response to the environment. Single nucleotide variants in the circular genomes of human mitochondrial DNA are associated with many different life-threatening diseases. Mitochondrial DNA base editing tools have established novel disease models and represent a new possibility toward personalized gene therapies for the treatment of mtDNA-based disorders.

Keywords: DdCBE; TALED; base editing; heteroplasmy; mitochondria; mitochondrial DNA.

Publication types

  • Review

MeSH terms

  • DNA, Mitochondrial / genetics
  • Gene Editing
  • Genetic Therapy
  • Humans
  • Mitochondria / genetics
  • Mitochondrial Diseases* / genetics
  • Mitochondrial Diseases* / therapy


  • DNA, Mitochondrial