ROLE OF SPHINGOMYELIN METABOLISM IN SKELETAL MUSCLE REGENERATION AND REPAIR

Muscular dystrophies are high-incidence genetic diseases characterised by progressive skeletal muscle weakness and degeneration for which there is presently no therapy. Moreover, skeletal myopathies occur also in other genetic diseases, such as Marfan syndrome. The understanding of the molecular mechanisms responsible for the correct process of muscle repair, which is impaired in these muscular diseases, is critical for the identification of new molecular targets which could open new avenues for the pharmacological treatment of these diseases. This proposal is focused on the study of normal products of the metabolism belonging to the "fat" family and named sphingolipids. We have already shown that these compounds are required for fundamental processes such as those that allow muscle precursor cells to give rise to muscle fibers. This project will define the role of sphingomyelin (SM) metabolism in the skeletal muscle regeneration in vitro and in vivo. Since innovative therapeutics in various different medicine branches are presently based on the interference with sphingolipid metabolism, the identification of these molecules as critical players in skeletal muscle regeneration will individuate innovative approaches for the treatment of skeletal myopathies based on the enhancement of the regenerative capacity of skeletal muscle.