Potential Therapy For Congenital Muscular Dystrophy

Main Category: Muscular Dystrophy / ALS
Article Date: 02 Jan 2009 - 3:00 PDT

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Current research suggests laminin, a protein that helps cells stick together, may lead to enhanced muscle repair in muscular dystrophy. The related report by Rooney et al, "Laminin-111 restores regenerative capacity in a mouse model for alpha 7 integrin congenital myopathy," appears in the January 2009 issue of The American Journal of Pathology.

Muscular dystrophy is a group of inherited genetic diseases that cause progressive muscle weakness. In one type of muscular dystrophy, patients with mutations in the adhesion molecule alpha 7 integrin experience delayed developmental milestones and impaired mobility. There is currently no treatment or cure for alpha 7 integrin congenital myopathy.

Interactions of alpha 7 integrin with laminin, an extracellular protein found surrounding muscle fibers, promote muscle cell health and survival. Alpha 7 integrin has also been implicated in muscle repair. To determine if alpha 7 integrin is critical for muscle repair, researchers led by Dr. Dean Burkin at The University of Nevada School of Medicine examined the response to muscle damage in alpha 7 integrin-deficient mice. They found that alpha 7 integrin-deficient muscle exhibited defective muscular regeneration. Injection of laminin-111, however, restored muscle repair and regeneration.

The data from Rooney et al "indicate a critical role for the alpha7beta1 integrin and laminin in muscle repair and suggest direct muscle injections of laminin may serve as an exciting novel therapy for patients with alpha 7 integrin congenital myopathy and other muscle diseases." Dr. Burkin's group is "currently investigating the potential of this technology to treat Duchenne and other forms of muscular dystrophy. This work opens a whole new modality in therapeutics, of injecting extracellular matrix proteins to treat genetic diseases."

This work was supported by grants from the National Institutes of Health.

Rooney JE, Gurpur PB, Yablonka-Reuveni Z, and Burkin DJ: Laminin-111 restores regenerative capacity in a mouse model for alpha 7 integrin congenital myopathy. Am J Pathol 2009 174: 256-264

The American Journal of Pathology, official journal of the American Society for Investigative Pathology, seeks to publish high-quality, original papers on the cellular and molecular biology of disease. The editors accept manuscripts that advance basic and translational knowledge of the pathogenesis, classification, diagnosis, and mechanisms of disease, without preference for a specific analytic method. High priority is given to studies on human disease and relevant experimental models using cellular, molecular, animal, biological, chemical, and immunological approaches in conjunction with morphology.

American Journal of Pathology