Focusing on fibrosis: halofuginone-induced functional improvement in the mdx mouse model of Duchenne muscular dystrophy

摘要

Duchenne muscular dystrophy (DMD) is a devastating X-linked disease caused by mutations in the gene encoding for dystrophin and characterized by widespread muscle degeneration that leads to death (20). The major focus of research has been directed toward alleviating the primary genetic deficit, using gene therapy and myoblast transfer approaches to promote dystrophin expression in muscle fibers. Unfortunately for patients with this ultimately fatal disease, there is currently no cure. Thus the development of complementary and supportive therapies that slow the progression of the disease and allow patients to have an improved quality of life is critically important.