Towards the treatment of a severe form of myopathy

Centro-nuclear myopathies are severe neuromuscular diseases of genetic origin. A French team1CNRS, Université de Strasbourg, INSERM has been able to demonstrate the efficacy of a novel therapeutic approach in an animal model. This consists in lowering the expression level of dynamin 2, a protein responsible for the organization of muscle cells. Created for transposing this promising treatment to humans, the Dynacure start-up has developed human antisense DNAs. As they bind to the gene coding for dynamin 2, the aim of such DNA fragments is to limit dynamin 2 production in muscle cells, thereby improving their function.