Studies have indicated that myotonic dystrophy in adults, which is the most common type of myotonic dystrophy could be due to a toxic RNA. //
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Doctors at the University of Virginia Health System have shown for the first time that by getting rid of poisonous RNA (ribonucleic acid) in muscle cells could result in the reversing myotonic dystrophy.
Statistics show that around 40,000 people in the United States have myotonic muscular dystrophy (MMD). The disease is known to cause a slow, progressive wasting of the muscles, irregular heartbeat, cataracts and insulin resistance. Many of the people with the condition are usually oblivious of it until their teens.
To prove the theory that toxic RNA is involved in myotonic muscular dystrophy, a research team led by Dr. Mani Mahadevan, a UVa pathologist, duplicated the disease in mice. "We showed in our mouse model that when you make this poisonous RNA the mice get various aspects of myotonic dystrophy," Mahadevan said. "Then, when you take away the toxic RNA, the mice get back to normal."
Mahadevan hopes the research might lead to new therapies for MMD in the next few years. "If we develop a therapy to silence the expression of the toxic RNA molecule, that would be a viable approach to treat people with myotonic muscular dystrophy," he said. Mahadevan's research in published in the September 2006 issue of Nature Genetics and can be found online at: http://www.nature.com/ng/index.html.
Making RNA is the second step in the conversion of DNA into proteins that determine the function of the body's cells. Myotonic muscular dystrophy is the first example of a disease caused by toxic RNA.
In 1992, Mahadevan discovered the gene mutation that causes myotonic muscular dystrophy (type 1) as part of a research group in Canada. The mutation is an increased number of CTG repeats in a gene called DMPK. Everyone with myotonic muscular dystrophy has that mutation on chromoso
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