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Clinical Implications of Basic Research
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Volume 359:1403-1405 September 25, 2008 Number 13
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Cell Therapies for Muscular Dystrophy
Helen M. Blau, Ph.D.

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Duchenne's muscular dystrophy is a devastating, progressive, X-linked muscle-wasting disease. It is the most common form of muscular dystrophy, affecting 1 in 3500 boys. With an onset in early childhood, the disease progresses to final stages that are characterized by cardiorespiratory failure and death, which usually occurs in the teenage years or early 20s. The disease affects striated muscles of the limbs, diaphragm, and heart and is associated with a progressive loss of muscle mass, leading to impaired ambulation and paralysis. Skeletal myofibers degenerate and undergo necrosis; muscles progressively accumulate calcium deposits and are replaced by connective tissue and fat.

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From Stanford University School of Medicine, Stanford, CA.




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