WORLDSymposium™ 2023 poster presentation: In vitro modeling of GM1 Gangliosidosis using iPSC-derived cellular and organoid CNS models
GM1 Gangliosidosis (GM1) is a rare, inherited, neurodegenerative, lysosomal storage disorder, caused by absent or impaired β-galactosidase activity and resulting in the cellular accumulation of GM1 gangliosides and associated metabolites. There are currently no disease-modifying treatments available. Developing robust in vitro paradigms using patient cells that recapitulate the phenotype of GM1 is important for validating emerging treatment strategies. The objective of the work presented in this poster was to develop a suite of cell, spheroid, and organoid-based central nervous system (CNS) models for use in drug development, including enzyme replacement and gene therapy modalities.