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Dystrophin Substitute Shows Promise in Mouse Model of Muscular Dystrophy

Researchers have shown that use of a dystrophin-like molecule with a cell-penetrating tag can effectively repair weakened muscle in a mouse model of Duchenne muscular dystrophy (DMD). Muscular dystrophy causes the muscles in the body to progressively weaken. DMD is the most common and most severe form of childhood muscular dystrophy. About one of 3,500 boys is born with the crippling disease. Symptoms usually begin in children who are 2 to 3 years-old, most are in a wheelchair by age 12, and many who have the disease pass away by their late teens to early 20s. Current treatment, limited to corticosteroids, is minimally effective and can cause serious side effects. DMD is caused by mutation in the gene for the dystrophin protein, which is an important structural component within muscle tissue. In this work, the researchers used the dystrophin-like molecule utrophin, attached to a cell-penetrating tag called TAT. "This unique approach can replace the missing protein without the complexities of gene replacement or stem cell approaches," said Dr. James Ervasti, principal investigator of the study. This new method would not be a cure for muscular dystrophy. Rather, it would be a therapy most likely administered on a regular basis. Dr. Ervasti is hopeful that the therapy can move into human clinical trials within three years. This work was published in the May 26 edition of PLoS Medicine. [Press release] [PLoS Medicine article]