Scientists have for the first time been able to switch on and off the effects of a genetic disease, myotonic dystrophy, in human muscle tissue.
Although the technique has so far only been attempted in cells, the group are optimistic about its potential to help provide a better understanding of the disease. “In complex diseases, there are always unanticipated mechanisms,” said Professor Matthew Disney, who led the study at the Scripps Research Institute in Florida. “Now that we can reverse the disease at will, we can study those aspects of it.”
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