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A gene editing tool to treat mitochondrial mutations


Key points from article :

Scientists have developed a gene editing tool capable of making targeted, single base pair changes in the DNA of mitochondria.

The new editor, derived from a bacterial toxin, could allow researchers to better study mitochondrial diseases.

Mitochondria are inaccessible to bulky CRISPR molecules & have 37 genes.

'We’ve been able to make point mutations in human nuclear DNA for 15 years, but mitochondria have resisted that furiously... with this technology, mitochondrial research will enter a golden age.' - Fyodor Urnov, Innovative Genomics Institute researcher.

Even a single base pair change in many of these genes can lead to devastating diseases.

Gene editing tools called base editors, capable of swapping one pair of nucleotides for another.

The new method can make single nucleotide changes directly on double-stranded DNA.

In studies using human cells, the tool effectively repaired between 20% to 40% of its targets after three to six days.

Research by Broad Institute published in Nature.

Derived from bacterial toxin - can make single base pair changes

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Scientific director for technology and translation, IGI, UC Berkeley