Grant: Operating Grant

Duchenne muscular dystrophy is the most frequent X-linked lethal disorder in man, affecting approximately 1 in 3500 males. Patients most often die of respiratory failure caused by involvement of the diaphragm and other respiratory muscles by the pathology. The disease is characterized by progressive muscle fiber destruction and a failure of adequate compensation by muscle repair mechanisms. Therefore, a better understanding of the factors which regulate muscle damage and repair in this disease could offer new therapeutic avenues. In this proposal, we will explore the role of the host immune system in this process. We believe that activation of innate immunity through chemokine and Toll-like receptors plays an important role in regulating the balance between muscle damage and repair in the dystrophic diaphragm. We will use genetically modified dystrophic mice and test a novel therapeutic agent to determine whether manipulation of innate immunity could be a new and promising therapeutic strategy for Duchenne muscular dystrophy.