Advances in research against muscular dystrophy

Advances in research against muscular dystrophy

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American scientists discover a way to reverse muscular dystrophy
According to an article published on July 30, 2006 by BBC News, a group of American scientists from Nature Genetics has discovered a way to reverse muscular dystrophy in mice, raising hopes of finding a cure for humans.

The study animals had myotonic dystrophy, the most common in adults. The mRNA or messenger RNA is a copy of the information that a gene carries in the DNA, so if the DNA is defective, its mRNA will be too. These abnormalities are what lead to the progressive loss and weakness of the musculature and to the heart problems seen in myotonic dystrophy.

Dr. Mani Mahadevan and his team thought that removing toxic mRNA molecules could help reverse the disease process. To do this, they made the mice have defective DNA that could be activated or deactivated by adding or removing an antibiotic from the water they drink. In the DNA-activated phase, the mice exhibited all the fundamental characteristics of myotonic dystrophy; on the contrary, when the DNA was inactivated, the majority of the mice (although not all) recovered their normal functions of skeletal and cardiac muscles.

Although the treatment was not 100% effective, the researchers believe that their results constitute the proof that all scientists were waiting to show that it is possible to reverse muscular dystrophy. Their work also suggests that it is the toxic mRNA that causes the pathology.

Dr Marita Pohlschmidt from the Muscular Dystrophy Campaign in the UK says that "the research results are very encouraging in finding a treatment for myotonic dystrophy." And he adds: “Perhaps the symptoms of the disease can be reversed by neutralizing the toxic substance that produces it; however, there is still a lot of work to do before we can be sure that it will work. "

Source: BBC

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