Researchers have proven that gene therapy can reverse the pathological features of muscular dystrophy in an animal model. Before, gene therapy had only been able to prevent further muscle-wasting in mice. "We expect to build on these results in the continuing search for a way to treat a horrible disease. Our results indicate that gene therapy could be used not only to halt or prevent this disease, but also to restore normal muscle function in older patients," says Dr. Jeffrey S. Chamberlain, professor of neurology at the University of Washington School of Medicine in Seattle.

Chamberlain is the senior author of the paper describing the results, which will be published in the Proceedings of the National Academy of Sciences online Early Edition the week of Sept. 16 to 20.

Duchenne muscular dystrophy is an X-linked genetic disorder that strikes one of every 3,500 newborn boys. The genetic disorder means the body does not produce the dystrophin protein, which is necessary for the structural support of muscle. Without this protein, muscles weaken to the point where the victim cannot survive. There is no specific treatment against any form of muscular dystrophy, except for supportive measures, such as physical therapy, assistive technology and corrective surgery. Boys and men with the disorder usually die from respiratory failure before they can turn 25.

Researchers have been looking for many years for ways to introduce the dystrophin gene into the body of patients to replace the missing gene. In doing so, researchers have developed a strain of mice who lack the dystrophin gene. In the past, researchers have been able to insert the gene into newborn mice via adenoviral vectors. But those vectors have many viral properties, and results have been limited because adult mice and mice that have begun to develop symptoms of the disease developed a sharp host immune response that eliminated the therapeutic gene.

The results have also been limited
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Contact: Walter Neary
wneary@u.washington.edu
206-685-3841
University of Washington
16-Sep-2002

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