Duchenne Muscular Dystrophy

Department of Pharmacology

Integrin therapeutics for the treatment of DMD

The α7β1 integrin is the predominant laminin-binding integrin in cardiac and skeletal muscle. Several studies have demonstrated the α7β1 integrin is a major modifier of disease progression in DMD.

• Transgenic over-expression of the α7 integrin in the skeletal muscle of mouse models of DMD improves muscle pathology and increases life expectancy.
• Loss of α7 integrin in the dystrophin deficient mdx mouse model of DMD accelerates muscle disease progression.
• AAV-mediated gene transfer of α7 or β1D integrin alleviates muscle disease in mouse models of DMD.

Together these results support the idea the α7β1 integrin is target for drug-based therapies for the treatment of DMD.