Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient- and cell type-specific disease phenotypes
Elisabeth Mangiameli, Anna Cecchele, Francesco Morena, Francesca Sanvito, Vittoria Matafora, Angela Cattaneo, Lucrezia della Volpe, Daniela Gnani, Marianna Paulis, Lucia Susani, Sabata Martino, Raffaella Di Micco, Angela Bachi, Angela Gritti
This study uses patient-specific iPSCs to model GLD, revealing cell type-specific disease phenotypes and the importance of GALC restoration for neural differentiation.
Stem Cell Reports · May 13, 2021Peer-reviewedGenetics