Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities

Bibekananda Kar, Santiago R. Castillo, Ankit Sabharwal, Karl J. Clark, Stephen C. Ekker

Research output: Contribution to journalReview articlepeer-review


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.

Original languageEnglish (US)
Article number5798
JournalInternational journal of molecular sciences
Issue number6
StatePublished - Mar 2023


  • DdCBE
  • base editing
  • heteroplasmy
  • mitochondria
  • mitochondrial DNA

ASJC Scopus subject areas

  • Catalysis
  • Molecular Biology
  • Spectroscopy
  • Computer Science Applications
  • Physical and Theoretical Chemistry
  • Organic Chemistry
  • Inorganic Chemistry


Dive into the research topics of 'Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities'. Together they form a unique fingerprint.

Cite this