This New Gene Editing Tool Targets the Powerhouse of the Cell

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Gene editing tools like CRISPR have the potential to let us change discrete segments of DNA to treat a host of diseases directly at the root. But there is one genetic frontier scientists have yet to break through: the mitochondria. Passed down from mother to child, mitochondria possess 37 genes in which mutations can give rise to around 300 different metabolic illnesses, like Leigh syndrome (which affects the brain’s role in motor movements) or Pearson syndrome (which causes anemia and other blood issues). And unfortunately, we’ve had limited success in using conventional gene-editing methods to study or correct these disastrous mistakes.

But scientists are steps closer to lifting away the mysterious mitochondrial veil. In a new study published April 25 in the journal Cell, South Korean researchers have devised a new gene-editing tool that can precisely swap out the nucleotide adenine for another nucleotide guanine within the mitochondrial genome.

“This is a creative study that has the potential to greatly expand the range of mitochondrial mutations—and thus diseases—that are accessible to genome editing-based therapeutic approaches,” Joseph Mougous, a microbiologist and Howard Hughes Medical Institute investigator at the University of Washington, who was not involved in the study, told The Daily Beast in an email.

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