Tag Archives: duchenne muscular dystrophy

Another Muscular Dystrophy Mystery Solved; MU Scientists Inch Closer to a Therapy for Patients

COLUMBIA, Mo. — Approximately 250,000 people in the United States suffer from muscular dystrophy, which occurs when damaged muscle tissue is replaced with fibrous, bony or fatty tissue and loses function. Three years ago, University of Missouri scientists found a molecular compound that is vital to curing the disease, but they didn’t know how to make the compound bind to the muscle cells. In a new study, published in the Proceedings of the National Academies of Science, MU School of Medicine scientists Yi Lai and Dongsheng Duan have discovered the missing pieces to this puzzle that could ultimately lead to a therapy and, potentially, a longer lifespan for patients suffering from 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, a protein essential for muscle cell survival and function. Absence of dystrophin starts a chain reaction that eventually leads to muscle cell degeneration and death. 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 that can keep muscle cells healthy after exercise. (more…)

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MU Researchers Find New Insight into Fatal Spinal Disease

*Discovery could lead to treatments for muscular dystrophy and ALS*

COLUMBIA, Mo. – Researchers at the University of Missouri have identified a communication breakdown between nerves and muscles in mice that may provide new insight into the debilitating and fatal human disease known as spinal muscular atrophy (SMA).

“Critical communication occurs at the point where nerves and muscles ‘talk’ to each other. When this communication between nerves and muscles is disrupted, muscles do not work properly,” said Michael Garcia, associate professor of biological sciences in the College of Arts and Science and the Christopher S. Bond Life Sciences Center.  “In this study, we found that delivery of ‘the words’ a nerve uses to communicate with muscles was disrupted before they arrived at the nerve ending.” (more…)

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