A novel approach targeting inflammation and muscle stem cells simultaneously to mitigate Duchenne Muscular Dystrophy

Description

Abstract

Duchenne muscular dystrophy (DMD) is a severe childhood muscle disease characterized by the absence of dystrophin, a structural protein critical for muscle fiber stability. Different factors contribute to the progression of the disease such as myofiber fragility, chronic inflammation, fibroadipose tissue deposition, and muscle stem cell dysfunction. So far, glucocorticoids remain the only drugs that are able to delay the progression of the disease; however, they also directly stimulate protein catabolism and long-term muscle wasting. Therefore, the therapeutic potential of glucocorticoids is mitigated by their harmful side effects. Our research project investigates the therapeutic potential of a novel class of bioactive lipids that have potent anti-inflammatory capacities and can simultaneously target muscle stem cells. Our findings indicate that these novel mediators improve myogenesis, muscle growth and function in dystrophic mice to a higher level than glucocorticoids. Thus, our findings suggest that this new therapeutic approach is a significant improvement compared to the standard-of-care treatment for DMD.