Potential Therapy For Congenital Muscular Dystrophy

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 the new study by 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.