Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities.
Bibekananda KarSantiago R CastilloAnkit SabharwalKarl J ClarkStephen C EkkerPublished in: International journal of molecular sciences (2023)
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.
Keyphrases
- mitochondrial dna
- copy number
- induced apoptosis
- crispr cas
- genome wide
- cell cycle arrest
- cell death
- dna methylation
- oxidative stress
- endothelial cells
- reactive oxygen species
- endoplasmic reticulum stress
- gene expression
- single cell
- stem cells
- signaling pathway
- induced pluripotent stem cells
- pi k akt
- endoplasmic reticulum
- transcription factor
- bone marrow