Cortical organoids from congenital DM1 PSCs reveal MBNL-dependent corticogenesis defects and enable preclinical testing of therapeutic compounds
Azania Abatan, Jérôme Polentes, Margot Bouquier, Adeline Beuriot, Olivier Chose, Hamel Mahiou, Lina El Kassar, Karine Giraud-Triboult, Laure Chatrousse, Alexandra Benchoua, Stéphanie Tomé, Geneviève Gourdon, Mário Gomes-Pereira, Sandrine Baghdoyan, Cécile Martinat
This study uses cortical organoids from congenital DM1 patient cells to show MBNL-dependent neurodevelopmental defects and test therapeutic compounds.
bioRxiv · Jul 27, 2026 · PreprintPathology