New mitochondrial DNA editing technique prevents genetic diseases in mice

A new technique to edit mitochondrial DNA has been tested on mice, and if replicable in humans, could help prevent mothers living with certain incurable disease from passing them onto their children, researchers say. Mitochondria are tiny powerhouses found inside nearly all human cells, and are responsible for generating the chemical energy that allows our cells to perform many essential functions. For example, this energy enables communication between brain cells, and it allows our muscle cells to keep us moving around. However, defects in these cellular components, which have their own DNA, can lead to a range of incurable conditions, known collectively as mitochondrial diseases.

While the diseases present differently in each individual, they can generally be characterised by symptoms such as poor muscle development and weakness; hearing, vision and other neurological impairments; learning disabilities; and heart, liver, gastrointestinal and respiratory diseases. For thousands of women around the world living with one of these incurable diseases, having children is a gamble, as it’s likely their defective mitochondrial DNA will be passed on.

Now, researchers at the Salk Institute for Biological Studies in the US have developed a technique to eliminate mitochondrial mutations from eggs or early embryos, while leaving the healthy mitochondria intact. The proof-of-concept demonstration, which has been described in the journal Cell, could help prevent children from inheriting these chronic diseases. Source: New mitochondrial DNA editing technique prevents genetic diseases in mice

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