Precision BioSciences Announces Publication in Nature Metabolism Supporting ARCUS® In Vivo Gene Editing as a Potentially Curative Treatment for Mitochondrial Diseases
For mitochondrial diseases, what makes ARCUS such an elegant and simple tool is that it is a single protein that both recognizes and eliminates the mutant mitochondrial DNA,” said Jeff Smith, Ph.D., Chief Research Officer of Precision BioSciences.
- For mitochondrial diseases, what makes ARCUS such an elegant and simple tool is that it is a single protein that both recognizes and eliminates the mutant mitochondrial DNA,” said Jeff Smith, Ph.D., Chief Research Officer of Precision BioSciences.
- “Today’s publication further validates the ability of ARCUS to overcome the limitations of CRISPR-based gene editing technologies for treating mitochondrial disease and eliminating mutated mitochondrial DNA with high specificity to improve overall mitochondrial function.
- By eliminating the mutant mitochondrial DNA and allowing the wild-type mitochondrial DNA to repopulate, mitoARCUS drives a shift toward healthy mitochondrial DNA, a process known as shifting heteroplasmy.
- “To date, there are no curative treatments for mitochondrial diseases, so a gene editing approach is a novel way to offer hope to the patients suffering from mitochondrial myopathy.