Protocol for screening of small molecules in a CLN3 disease patient-specific iPSC-derived neuronal progenitor cell model.

Although rare, CLN3 disease is the most common neurodegenerative disorder of childhood. Here, we present a protocol for screening small molecules in a CLN3 disease patient-specific induced pluripotent stem cell (iPSC)-derived neuronal progenitor cell (NPC) model. We describe steps for converting human iPSCs (hiPSCs) to neural stem cells (NSCs) and developing NPCs using feeder-free medium and cytokine-based differentiation. We then detail procedures for achieving access to mature neurons. For complete details on the use and execution of this protocol, please refer to Simeon et al.1.