Cortical organoids from congenital DM1 PSCs reveal MBNL-dependent corticogenesis defects and enable preclinical testing of therapeutic compounds
Azania Abatan, Jérôme Polentes, Margot Bouquier…·Cécile Martinat
This study uses cortical organoids from congenital DM1 patient cells to show MBNL-dependent neurodevelopmental defects and test therapeutic compounds.