New Discovery May Help Develop Therapies For Childhood Muscular Dystrophy

Scientists claim to have identified a new function for the protein missing in people with the most common and ultimately lethal form of childhood muscular dystrophy - a finding that will lead to new therapies to combat the disease.

Patients with Duchenne muscular dystrophy lack the protein dystrophin, which causes their muscles to become weak and eventually die.

Studies have shown that dystrophin protects muscle cells by directly connecting to two of the three filament types that give cells their shape and durability.

In the new study, the researchers from University of Minnesota and National Institutes of Health showed that dystrophin also directly links to the third structural filament type named microtubules.

Microtubules form a highly ordered lattice in muscle. They found that microtubules become disorganized when dystrophin is missing.

"It's remarkable that scientists have been intensively studying dystrophin for more than 20 years, yet we continue to identify new features that better define its important contribution to healthy muscle." said James Ervasti, Ph.D., a professor in the Department of Biochemistry, Molecular Biology and Biophysics, who directed the investigation.

The new findings suggest that loss of microtubule organization might contribute to the devastating symptoms of Duchenne muscular dystrophy, information that will hopefully lead to the development of therapies to combat the disease.

The study appears online in The Journal of Cell Biology.

Source: ANI
SRM

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