Myotonic Dystrophy type 1 (DM1) is a neuromuscular disease associated with toxic RNA containing expanded CUG repeats. Treating mice with the GSK3 inhibitor tideglusib corrected expression of 17% of DM1-related genes. correction of the GSK3β-CUGBP1 pathway in mice expressing long CUG repeats (DMSXL m...
ORGANISM(S): Mus musculus