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Gene Therapy Shows Promise for Muscular Dystrophy
Date:5/21/2010

Small study found technique restored some muscle function for months

FRIDAY, May 21 (HealthDay News) -- Scientists have succeeded in using gene therapy to restore some muscle function in patients with a certain type of muscular dystrophy.

This is the first time such a feat has been performed in humans, state the authors, who are presenting their findings at the annual meeting of the American Society of Gene & Cell Therapy in Washington, D.C.

"This study provides additional information regarding the feasibility of gene therapy for the treatment of muscular dystrophy," said Dr. Valerie Cwik, executive vice president and research and medical director of the Muscular Dystrophy Association, which helped fund the research. "Specifically, it provides proof of principle, in people, for sustained gene expression [for at least six months] following treatment."

"This study has shown that a normal gene packed into a virus and injected directly into a muscle can actually produce the protein that is either defective or missing in this particular form of muscular dystrophy," added Dr. Rabi Tawil, a professor of neurology at the University of Rochester Medical Center. "Similar studies have been done in animal models, and this is the first to show a similar result in humans."

If replicated, the findings could provide hope for people with this and other forms of muscular dystrophy.

"Reversing or significantly blunting the severity of this weakness and wasting will give these patients major improvement in their quality of life, enhance their independence, and increase the likelihood that they can obtain employment," said Dr. Richard Moxley, director of the Neuromuscular Disease Center, also at the University of Rochester Medical Center.

The patients in this study had limb-girdle muscular dystrophy (LGMD), which is characterized by muscle weakness around the hips and shoulders. The condition resul
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