Key points from article :
Precision BioSciences has published a peer-reviewed article in the journal Nature Metabolism demonstrating the ability of its ARCUS gene editing technology to eliminate mutant mitochondrial DNA (mtDNA) in cells.
The study used a mitochondrial-targeted ARCUS nuclease, mitoARCUS, to specifically target and cleave the pathogenic m.3243G point mutation in mtDNA.
MitoARCUS was able to eliminate all of the mitochondrial DNA containing the mutation, leaving the wild-type mtDNA untouched.
This wild-type mtDNA was then able to be rapidly replicated by the cell in order to maintain a steady mitochondrial DNA copy number.
The elimination of mutant mtDNA and the repopulation of wild-type mtDNA is known as shifting heteroplasmy, and it is believed to be a potential therapeutic approach for mitochondrial diseases.
The study provides further evidence that ARCUS is a promising gene editing technology for the treatment of mitochondrial diseases and other genetic diseases.
