A new study appearing in Nature Genetics says that researchers have found a way to reverse muscular dystrophy (MD) in laboratory mice. This discovery kindles hope that a cure for the human form of the disease may be found.
The University of Virginia researchers said that the therapy used by them could fully restore heart and skeletal muscle function. "The results represent the first in vivo proof of principle for a therapeutic strategy for treatment of myotonic dystrophy by ablating or silencing expression of the toxic RNA molecules,' the authors said.
In MD there is a faulty DNA, which is responsible for almost all of the abnormalities. This therapy targeted a specific molecule and silenced it during the course of the disease.
Lead researcher Dr Mani Mahadevan and his team found that toxic mRNA causes the disease and they also found a way to turn it off in mice. "The fact that the course of the disease can be reversed both overtly and at the molecular level suggests that the toxic RNA functions as a reversible metabolic toxin," the researchers said.