A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1.

Anika V PrabhuInsung KangRaffaella De PaceChristopher A WassifHideji FujiwaraPamela KellXuntian JiangDaniel S OryJuan S BonifacinoMichael E WardForbes D Porter

Published in: BMC biology (2021)

Our data demonstrate the utility of this new cell line in high-throughput drug/chemical screens to identify potential therapeutic agents. The NPC1-/- i3Neuron line will also be a valuable tool for the NPC1 research community to explore the pathological mechanisms contributing to neuronal degeneration.

Keyphrases

high throughput
induced pluripotent stem cells
endothelial cells
cerebral ischemia
mental health
single cell
healthcare
electronic health record
pluripotent stem cells
genome wide
big data
emergency department
dna methylation
machine learning
data analysis
drug induced