MECP2-Related Pathways Are Dysregulated in a Cortical Organoid Model of Myotonic Dystrophy

0
102
The authors showed that CUG foci found early in the maturation of 3D cortical organoids from DM1 patient–derived iPSCs caused hyperphosphorylation of CUGBP Elav-like family member 2 protein.
[Science Translational Medicine]
Abstract