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Treatment in mice proves effective against Duchenne muscular dystrophy

obility and lived to an average age of 38 weeks.

Duchenne muscular dystrophy, caused by a recessive genetic defect, affects one in 3,300 males. The disease usually begins in early childhood and often is fatal by age 30. It is the most prevalent of the muscular dystrophy family of neuromuscular diseases. Patients with Becker, limb girdle and other muscular dystrophies also might benefit from the approach used in the study, Kaufman said.

"The potential exists to enhance the expression of the endogenous normal alpha 7 integrin gene, or extend the lifetime of the alpha 7 protein," he said. "This would even avoid the need for what we think of as classical gene therapy."


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Contact: Jim Barlow
b-james3@uiuc.edu
217-333-5802
University of Illinois at Urbana-Champaign
18-Mar-2001


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