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Muscular dystrophy mystery solved; Mizzou scientist moves closer to MD solution
Date:2/26/2009

COLUMBIA, Mo. Muscular dystrophy, which affects approximately 250,000 people in the United States, occurs when damaged muscle tissue is replaced with fibrous, bony or fatty tissue and loses function. While scientists have identified one protein, dystrophin, as an important piece to curing the disease, another part of the mystery has eluded scientists for the past 14 years. Now, one University of Missouri scientist and his team have identified the location of the genetic material responsible for a molecular compound that is vital to curing the disease.

Duchenne muscular dystrophy (DMD), predominantly affecting males, is the most common type of muscular dystrophy. Patients with Duchenne muscular dystrophy have a gene mutation that disrupts the production of dystrophin. Absence of dystrophin starts a chain reaction that eventually leads to muscle cell degeneration and death. A previous study by Dongsheng Duan, associate professor of molecular microbiology and immunology, discovered a potential delivery method to replace the mutated genes with healthy genes. Following the replacement of these genes, Duan observed that dystrophin production was restarted in animals with muscular dystrophy.

However, while dystrophin is vital for muscle development, the protein also needs several "helpers" to maintain the muscle tissue. One of these "helper" molecular compounds is nNOS, which produces nitric oxide. This is important for muscles that are in use during high intensity movements, such as exercise.

"When you exercise, not only does the muscle contract, but the blood vessels are constricted," Duan said. "nNOS is important because it produces nitric oxide that relaxes the blood vessels, helping to maintain the muscle with a healthy blood supply. If no blood reaches the muscle cells, they will eventually die. In DMD patients, this means the disease will progress as the muscle cells are replaced by the fibrous, bony or fatty tissue."

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Contact: Christian Basi
BasiC@missouri.edu
573-882-4430
University of Missouri-Columbia
Source:Eurekalert

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