4.6 Review Book Chapter

Molecular Mechanisms and Treatment Options for Muscle Wasting Diseases

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DOI: 10.1146/annurev-pharmtox-010510-100537

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atrophy; muscular dystrophy; proteasome; dystrophin glycoprotein complex; exon skipping; protein synthesis; insulin-like growth factor; nonsense suppression

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Loss of muscle mass can be the consequence of pathological changes, as observed in muscular dystrophies; or it can be secondary to cachexia-inducing diseases that cause muscle atrophy, such as cancer, heart disease, or chronic obstructive pulmonary disease; or it can be a consequence of aging or simple disuse. Although muscular dystrophies are rare, muscle loss affects millions of people worldwide. We discuss the molecular mechanisms involved in muscular dystrophy and in muscle atrophy and present current strategies aimed at ameliorating these diseases. Finally, we discuss whether lessons learned from studying muscular dystrophies will also be helpful for halting muscle loss secondary to nondystrophic diseases and whether strategies to halt muscle atrophy have potential for the treatment of muscular dystrophies.

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