Angela Gritti

This study uses human oligodendrocyte spheroids to model leukodystrophies and identifies disease-specific transcriptional changes and lineage-specific vulnerabilities.

This study characterizes the transcriptional and epigenetic profiles of hiPSC-NSCs and evaluates their safety in vivo, showing they resemble fetal NSCs and are safe for transplantation.

This study shows that combining gene therapy and bone marrow transplantation in a mouse model of GM2 gangliosidosis improves lifespan and reduces disease symptoms more effectively than either treatment alone.

This study uses patient-specific iPSCs to model GLD, revealing cell type-specific disease phenotypes and the importance of GALC restoration for neural differentiation.