Dysregulated MEG3 in Myotonic Dystrophy 1: nuclear retention, pathological role, and therapeutic correction by antisense conjugates.
David Seoane-Miraz, Jessica Stoodley, Natalia Galindo Riera, Yahya E. Jad, Arnaud F. Klein, Lara Nikel, Yulia N. Lomonosova, Jesús Reiné, Susana Camara, Ruben D. Artero, Denis Furling, Matthew J. A. Wood, Miguel A. Varela
This study shows MEG3 is upregulated in DM1 and exhibits nuclear retention, with antisense conjugates correcting MEG3 levels in mice.
bioRxiv · Jun 20, 2025 · PreprintMolecular Biology