Grant: Operating Grant - Priority Announcement: Neuromuscular Research

One of the most promising therapeutic strategies for countering muscular dystrophy is to replace the missing muscle dystrophin protein that is a signature of this disease with its protein "twin sister" utrophin, which is present in small amounts in the muscles of people that have muscular dystrophy. We were the first to provide exciting new evidence showing that utrophin is under the control of another muscle protein, calciceurin, which we had previously shown to be an orchestrator of muscle growth. Indeed, we showed that when calcineurin is turned on within a muscle, utrophin is also forced to appear in abundance all over the muscle in areas usually reserved for dystrophin, and by virtue of this replacement is thus capable of rescuing fibers that are damaged by muscular dystrophy. Within the time frame of this proposal, we plan to further define the role of calcineurin in this rescuing of damaged dystrophic muscles and plan to identify novel other players in this symphony to help us better understand this disease and develop strategies and interventions to reverse its damaging effects.