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 GSK3β-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. Comparative expression of WT and HSA mice treated with either vehicle or GSK3 inhibitor tideglusib.