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Rodent of the Week: New mouse model yields some clues about Duchenne muscular dystrophy

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Mice are used throughout medical research to provide models for human disease. The more accurate the mouse model of the disease, the better for scientists trying to understand disease processes and treatments.

Researchers this week announced they have developed what they say is a more realistic mouse model for Duchenne muscular dystrophy. In the new model, scientists can study muscle damage that is similar to what occurs in humans with the disease. The model produces human-like glycosylation, the process by which human cells coat themselves with long sugar chains that are attached to proteins. This process is vital for normal growth and function of tissues and organs.

The researchers, led by Paul Martin at the Research Institute at Nationwide Children’s Hospital in Columbus, introduced a mutation into a gene that altered glycosylation by getting rid of a sugar molecule called Neu5Gc. They found this sugar appears to stimulate negative immune responses in muscle tissue affected by Duchenne. If this finding is verified by additional research, it could mean that avoiding dietary intake of the Neu5Gc sugar molecule, which is found in red meat and dairy products, may be helpful to reduce the immune response. In any case, the new model, the authors wrote: “should facilitate the testing of therapeutics with morbidity and mortality measures more akin to the human disease.”

The study appears in the journal Science Translational Medicine. For more information on research on Duchenne muscular dystrophy and a new Duchenne clinic at UCLA, see this recent story in the Los Angeles Times.

-- Shari Roan

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