Alongside scientists, mice have played a huge role in health-related breakthroughs. Since previously advancing diabetes research, mice have now led us to a potential cure for paralysis. In particular, gene editing has eliminated muscular dystrophy in mice.
The mice in the study have a rare and severe form of congenital muscular dystrophy known as MDC1A.
The illness is caused by a splice site mutation: a genetic error makes cellular messengers misread a critical section of DNA, like the scratch that makes a record skip.
Researchers in Cohn’s lab used CRISPR to cut out the scratch. Natural cell repair mechanisms stitched the remaining strands of DNA back together, allowing the whole genetic sequence to be read normally.
Follow ups proved that the participating mice were healed completely. The method is also simple, unlike other processes that require the engineering of an entirely new genome. However, as with all animal testing, researchers must carefully consider human trials.
For the first time it’s possible to think about — and this is still at the thinking stage, let’s be clear — the possibilities of gene correction in humans with these diseases,”
Considering that patients with muscular dystrophy rarely make it through their twenties, this could be the change they need. It may be a while, but gene editing holds much promise for the future.