Therapeutic targeting of the GSK3b-CUGBP1 pathway in Myotonic Dystrophy

ABSTRACT: 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.

ORGANISM(S): Mus musculus

PROVIDER: GSE230821 | GEO | 2025/07/22

REPOSITORIES: GEO