DNA Editing in Mouse Embryos Prevents Disease

Mothers may one day be able to prevent their children from inheriting mitochondrial defects. Therapies that modify diseased eggs are inching closer to the clinic, but researchers are still hotly debating the safety and ethics of the most promising techniques. These involve combining the nucleus of the mother’s egg with mitochondria from a healthy woman to create a ‘three-parent embryo’.

In the 23 April issue of Cell1, one team proposes an alternative: neutralizing the faulty mitochondria. Some researchers say that the approach could help enable the ethically questionable practice of engineering human embryos to have modifications that would be passed on to future generations. (The first instance of such engineering was published in Protein & Cell2 and reported by Nature's news team on 22 April.)

About 1 in 5,000 people worldwide has a disorder caused by faulty mitochondria, the organelles that supply the cell with energy. In others, the faulty organelles worsen diseases that arise by other means, such as some cancers. Roughly 60–95% of the mitochondria in a cell must be faulty for disease...