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Another muscular dystrophy mystery solved; MU scientists inch closer to a therapy for patients
Date:12/7/2012

hat two particular sections of the dystrophin gene must be present for nNOS to bind to the muscle cells. The sections of the gene, known as "repeaters 16 & 17," contain a "claw" that can grab nNOS and bring it to the muscle cells so that it will bind and repair any damage from regular use. Without this "claw," nNOS doesn't bind to the cells and the damage is not repaired, leading to further problems associated with muscular dystrophy.

The other key to this puzzle is dystrophin. If the protein is not present in the body, no "claw" exists and nNOS would never make it to the muscle cells. For years, scientists have been attempting to find ways to make the body manufacture more dystrophin, and thus get more nNOS to the muscle cells. Duan and Lai said the answer might lie elsewhere.

"Everybody, including those individuals with muscular dystrophy, has another protein known as 'utrophin,'" said Duan, a professor of molecular microbiology and immunology. "Utrophin is nearly identical to dystrophin except that it is missing repeaters 16 & 17, so it cannot attract nNOS to the muscle cells. In our study, we were able to modify utrophin so that it had the repeaters, and thus, the ability to grab nNOS and bring it to the muscle cells for repair. Our study was completed in mice; if we can do the same thing in larger animals, we could eventually have a significant therapy for humans with this devastating disease."


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

Page: 1 2

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