Gene therapy methods that specifically target muscle may reverse the symptoms of a rare form of muscular dystrophy, according to new research in mice conducted by medical geneticists at Duke University Medical Center. Infants born with the inherited muscular disorder called Pompe disease usually die before they reach the age of two. The researchers also said their approach of targeting corrective genes to muscles may have application in treating other muscular dystrophies.
Patients with Pompe disease have a defect in a key enzyme that converts glycogen, a stored form of sugar, into glucose, the bodys primary energy source. As a result, glycogen builds up in muscles throughout the body, including the heart, causing muscles to degenerate.
Using genetically altered mice in which the gene for the enzyme had been rendered nonfunctional, the researchers demonstrated they could introduce the functioning gene and correct glycogen buildup in heart and skeletal muscle. The findings suggest that such an approach should be considered as a potential gene therapy strategy for Pompe disease patients, the researchers report in a forthcoming issue of Molecular Therapy (now available online).
Kendall Morgan | EurekAlert!
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