GENE AND NEURAL STEM CELL THERAPY FOR LYSOSOMAL STORAGE DISEASES
 

The overarching goal of the lab is to study and treat rare genetic diseases affecting the CNS, with a special focus on Lysosomal Storage Disorders (LSDs), including leukodystrophies (Alexander Disease, Metachromatic leukodystrophy, Globoid-cell Leukodystrophy) and GM2 gangliosidosis (Sandhoff Disease and Tay-Sachs Disease).
Our projects span from basic to translational research, using murine and human models, and testing different therapeutic platforms (i.e. in vivo GT, ex vivo GT; cell therapy, gene addition, gene editing).