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New NIH grant helps MU scientist get to the heart of muscular dystrophy
Date:12/6/2010

developing this gene based on what we already know about skeletal muscle, so it will not only be perfectly functional for skeletal muscle but also therapeutic for heart muscle," said Duan, a Margaret Proctor Mulligan Distinguished Professor in Medical Research in MU's Department of Molecular Microbiology and Immunology.

Duan plans to evaluate the newly developed gene in dystrophic mouse and dog models. With funding from additional NIH grants, Duan's team is developing viral vectors for carrying the most important parts of the therapeutic genes to diseased muscle cells. The therapy-carrying viruses will be injected directly into the blood system or the muscles. Following therapy, researchers will perform a series of tests to evaluate the strength of the heart muscle and the effect of the treatment.

"Researchers are more advanced in their understanding of Duchenne muscular dystrophy when it comes to skeletal muscle, but cardiac and skeletal muscle need to be simultaneously treated in patients," Duan said. "This is just the beginning of research on the heart muscle."

While still being tested in labs, Duan's proposed therapy gives hope to parents like Robert McDonald, MD, of Jefferson City, Mo. His son Mark was diagnosed with DMD when the boy was 2 years old. Dr. McDonald now serves on the board of directors and the therapeutics committee for Parent Project Muscular Dystrophy, the largest nonprofit organization focused on DMD. Parent Project has also awarded funds for Duan's research.

"He is one of the most valuable people trying to find a solution for DMD," McDonald said. "Parents like me are looking to scientists like him to someday help our children grow up to be men, and hopefully old men."


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Contact: Natalie Fieleke
fieleken@health.missouri.edu
573-884-7541
University of Missouri School of Medicine
Source:Eurekalert

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