GSE109083
Transcriptomic analysis of microglia from 7 week Npc1 disease mouse model treated or not with 4000mg.kg-1 HPBCD
the study aims to describe the modifications occurring the microglia isolated from NPC1 disease mouse model using a transcriptomic and phenotypic approach. The analysis is also performed on animal treated with the reference drug HPBCD, currently under clinical trial in human. Overall design: FACS isolated cell from Npc1 KO and WT mice were harvested from 7-week-old animals treated with HPBCD (n=3/genotype) or vehicle (n=3/ genotype) or no treatment (n=5/ genotype). Transcriptome was analyzed for modified signaling pathway further validated by additional experiments.