NeurosciencebioRxivPreprint
Generation and characterization of human iPSC-derived NPC1I1061T/I10161T i3Neurons as a model for NPC1 disease
Shikha Salhotra, Niamh X. Cawley, Christian White…·Forbes D. Porter
The paper develops a human iPSC-derived neuronal model for NPC1 disease, showing it recapitulates disease features and responds to a proteostasis modulator.
DOI 10.64898/2026.02.11.705111