GeneticsStem Cell ReportsPeer-reviewed
Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient- and cell type-specific disease phenotypes
Elisabeth Mangiameli, Anna Cecchele, Francesco Morena…·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.
27 citations