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 Genetic Adjustment. Story by Alan Bavley.

 

Whether you've viewed the famous telethon or not, you're likely aware that muscular dystrophy has been cutting short youthful lives and challenging the talents of medical researchers for decades.

But hope is on the horizon. Thanks in part to gene therapies being developed by MU microbiologist Dongsheng Duan, doctors and patient advocates are optimistic that an effective treatment for the most common form of the illness, Duchenne muscular dystrophy, will be forthcoming. "It's amazing how fast we're moving," Duan says. For kids with the disease and their parents, obviously, such a therapy could not come soon enough.

Children born with Duchenne muscular dystrophy, almost all of them boys, experience a progressive degeneration of muscles that should be growing stronger. As babies, they may find it difficult to advance from crawling to walking. As adolescents, they're often forced into wheelchairs. Eventually their heart and diaphragm muscles stop working. About 45,000 infants, about one in every 3,500 male births in the United States, are born with the illness every year. Few survive to their 30th birthdays.

In his lab at MU's Medical Science Building, Duan maintains hundreds of cages of mice bred with a genetic defect that mimics the disease. Many spend their days on miniature treadmills, connected to tiny heart monitors. As his research projects proliferate, his laboratories crowd into whatever extra space he can find.

"If we get funded for another grant," he quips, "we'll have space problems again."

Duan's cramped quarters in no way reflect his growing prominence. The 41-year-old assistant professor of molecular microbiology and immunology often publishes in important scientific journals, and his work has garnered national attention.

"He's considered one of the up-and-coming stars in gene therapy. He's taken things other people have worked on and taken them another step farther," says Bill Moore, director of research program development at the Muscular Dystrophy Association.

Gene therapy uses engineered viruses as vectors to deliver normal versions of genes to replace genes that are missing or defective. The technique holds enormous promise for treating a variety of genetic diseases, from cystic fibrosis to hemophilia. The Muscular Dystrophy Association, which helps fund Duan's work, has a large gene therapy program. Duchenne muscular dystrophy is the organization's first target.

"If we try to tackle the absolutely worst kind of muscular dystrophy, it will lay the groundwork for all the others," Moore says.

       
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Published by the Office of Research.

©2006 Curators of the University of Missouri. Click here to contact the editor.