The present invention relates to a method for producing a model cell based on a human cell model of GM1 gangliosidosis, induced pluripotent stem cells (iPSCs) and iPSCs-derived neural progenitor cells, IPSCs derived from a fibroblast of a GM1 patient of the present invention are useful as a neural progenitor cell (NPC) and a neural progenitor cell And the cells can differentiate into GM1 gangliosides and lysosomes, which are symptoms of GM1, as a model cell of GM1. Therefore, it is possible to identify the GM1 pathogenesis mechanism analysis study and therapeutic agent It can be useful for research for development. Also, it was confirmed that rhIL1RA or Z-YVAD-FMK, which is an inflammation-regulating complex inhibitor, could be used as a symptomatic remedy / remedy for GM1 gangliosidosis by using the GM1 cell model of the present invention.
展开▼
