Researchers find on-off "switch" for muscular dystrophy

Researchers at the University of Virginia say that they've been able to reverse muscular dystrophy in mice using a therapy that could eventually be used in humans. The key was silencing toxic messenger RNA molecules--which are created by faulty DNA code--that create abnormalities which in turn causes a progressive weakening of muscles, wasting and heart ailments. Those are all common symptoms of myotonic dystrophy, the most common form of MD. The researchers first developed mice with faulty DNA that could be turned "on" or "off" by adding or eliminating an antibiotic in their water supply. In the "off" phase normal muscle activity returned in many of the mice. The team of researchers was led by Dr. Mani Mahadevan.

"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," wrote the researchers. "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."

For more detail on this therapy:
- read this article from the BBC