Precision BioSciences Announces New Study Published in Nature Communications Using Engineered ARCUS Nuclease to Target Mutant Mitochondrial DNA In Vivo
Additionally, both mutant and wild-type mtDNA can co-exist within the mitochondria of a cell, a phenomenon called mtDNA heteroplasmy.
- Additionally, both mutant and wild-type mtDNA can co-exist within the mitochondria of a cell, a phenomenon called mtDNA heteroplasmy.
- When specific threshold levels of mutant mtDNA are reached, cell function can be compromised, and disease can manifest1.
- In this study, a mitochondrial-targeted ARCUS nuclease (mitoARCUS) used to edit mutant mtDNA was particularly effective, in part because of the nucleases small size and single protein nature.
- Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its wholly proprietary ARCUS genome editing platform.