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Duke scientists reverse a rare form of muscular dystrophy in mice using gene therapy

DURHAM, N.C. -- Using a modified virus to deliver a therapeutic gene, scientists at Duke University Medical Center have shown that, in mice, they can reverse the damage caused by an inherited muscle-wasting disease with a single injected dose.

The study findings, which appear in the Aug. 3 issue of the Proceedings of the National Academy of Science, show for the first time that it appears possible to deliver a therapeutic gene product throughout all of the muscles of the body to reverse muscle wasting, a result that has implications for treating dozens of forms of muscular dystrophy.

The researchers note, however, that to date they have only demonstrated a short-term reversal of symptoms in laboratory mice, and further experiments are needed to determine if the approach could become practical for use in people.

The study is part of a large, collaborative effort at Duke University to find an effective treatment for Pompe disease, a rare inherited disorder in which the body can not process glycogen, a stored form of sugar the body needs for energy. People born with Pompe disease have a defect in an enzyme called acid alpha-glucosidase (GAA), which normally processes glycogen and converts it to sugar. The glycogen builds up in muscle tissues throughout the body, including the heart, causing the various muscles to degenerate.

Several forms of Pompe disease affect more than 5,000 people in the United States. If symptoms appear during infancy, the disease is usually fatal. It is usually less severe when symptoms first appear late in childhood, but life expectancy is greatly deceased. Although Pompe disease is a relatively rare disease, it is but one of a group of lysosomal storage diseases, which in total occur in about one in 5,000 births in the United States.

Duke pediatric geneticist Y. T. Chen has been simultaneously pursuing two avenues to treating the disease: replacing the missing GAA en
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Contact: Karyn Hede
Hede0001@mc.duke.edu
919-684-4148
Duke University Medical Center
2-Aug-1999


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