Therapeutic targeting of the GSK3b-CUGBP1 pathway in Myotonic Dystrophy

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 GSK3ß-CUGBP1 pathway in mice expressing long CUG repeats (DMSXL model) is important not only at prenatal and postnatal stage, but also beneficial in adult mice. Overall design: Comparative expression of WT and HSA mice treated with either vehicle or GSK3 inhibitor tideglusib.