Muscular dystrophy is a disease that, even when it’s not fatal, still makes life difficult at best for the thousands of sufferers. But we might just be on the verge of stopping it altogether.
A University of Texas research team has been working on removing the mutation using CRISPR/Cas9-mediated genome editing and found some surprising results. According to Gizmag, trimming out the mutated gene that caused muscular dystrophy in the mice caused it to be replaced with a normal gene. The mice didn’t suffer from the disease.
This doesn’t mean, however, that we’re on the verge of a cure just yet. First, they have to ensure that the mutation doesn’t creep back in over time, and then they have to work out how to scale it up and turn it into a treatment fit for humans. Tweaking a mouse’s genetics and tweaking a human’s are two entirely different things, after all, and it’s not clear that, even if this works, that we can ever use it on humans.
But still, this is promising news. The mortality rate for MD sufferers increases dramatically over time; according to the CDC, almost half of sufferers won’t make it to their early 20s. But there’s hope, at least, and that’s something anybody with a disease can use.