Scientists Reverse Key Muscular Dystrophy Symptom in Mice
November 19th, 2007 Posted by: Dr. Cox
(Ivanhoe Newswire) — A new way to get rid of a critical symptom of muscular dystrophy is a success in mice.
Researchers from the University of Rochester Medical Center found a compound that eliminates myotonia — prolonged muscle tensing that is a symptom of muscular dystrophy — in the lab animals. The scientists designed a synthetic RNA-based molecule that restores a pathway that controls electrical activity in muscles. They found once the pathway was restored, normal muscle function returned.
âThe significance of this work is the proof of concept that a fundamental aspect of this genetic disease can be reversed even after it is very well established,â senior author Charles Thornton, M.D., University of Rochester Medical Center, was quoted as saying. âIt encourages us to believe that other parts of the disease could be reversible as well.â
Myotonic dystrophy is the most common form of muscular dystrophy in adults. Patients with the disease have progressive muscle wasting and weakness, especially in the lower legs, hands, neck, and face. They also have prolonged muscle tensing (myotonia) and canât relax certain muscles after they use them.
The study showed when the synthetic compound — called a morpholino — was injected into the muscle cells of mice with myotonic dystrophy, the myotonia all but disappeared and did not come back for several weeks.
Researchers say they are encouraged by what they found and believe it could ultimately be a step toward a breakthrough treatment for the disease. But they caution more work needs to be done before they can test this compound in people.
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SOURCE: Journal of Clinical Investigation published online November 15, 2007