jueves, 9 de julio de 2020

Gene-editing find could point to cures for mitochondrial diseases - STAT

Gene-editing find could point to cures for mitochondrial diseases - STAT

The Readout

Damian Garde & Meghana Keshavan

Genome editing finally breaks into the power source

Mitochondria, the tiny organelles that provide energy to cells, have long evaded the tools of DNA editing, much to the frustration of scientists. But a new technique, derived from a bacterial toxin, suggests cellular power plants can be tinkered with after all, lighting the way to potential treatments for rare and devastating diseases.

As STAT’s Sharon Begley reports, scientists at the Broad Institute and the University of Washington paired their expertises to edit mitochondrial DNA in lab dishes. The efficiency is only so-so, hitting roughly 20% to 40% of its genetic targets. But if the rapid advance of CRISPR-based genome editing is a guide, further study could hone the new method into a potent tool.

“With this technology, mitochondrial research will enter a golden age,” said biologist Fyodor Urnov of the University of California’s Innovative Genomics Institute.

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