Stimulating The Production Of Utrophin Protects Muscular Dystrophy Mice From Muscle Wasting

Researchers at the University of Pennsylvania School of Medicine report a novel strategy for stimulating the production of utrophin - an important muscle protein in young mice - for muscular dystrophy therapy. The investigators gave mdx mice (the mouse model for Duchenne's muscular dystrophy) heregulin, a small molecule to turn on the production of utrophin in their muscles. Utrophin improved muscle function in the mdx mice. “Our strategy boosts the levels of an existing gene using pre-existing cellular machinery rather than having to deliver a gene via gene therapy,” says lead author Tejvir S. Khurana, MD, PhD, Assistant Professor of Physiology & Member of the Pennsylvania Muscle Institute.