Musculoskeletal

Mouse studies suggest antisense oligonucleotides (ASOs) against DNM2 could help treat X-linked centronuclear myopathies. In a myotubularin 1 (MTM1)-knockout mouse model of X-linked centronuclear myopathy, intramuscular injection of an ASO targeting mouse DNM2 before symptom onset increased muscle size and force compared with a control oligo. Also in the model, systemic delivery of the DNM2-targeting ASO before symptom onset increased body weight, respiratory function and lifespan and decreased muscle pathology, and in mice with established disease, the ASO decreased muscle pathology. Ongoing studies by Dynacure S.A.S. include testing DYN101, a version of the ASO targeting human DMN2, in non-human primates and mice expressing that gene...