Neural stem cells for disease modeling of Wolman disease and evaluation of therapeutics.

Francis AguisandaCharles D YehCatherine Z ChenRong LiJeanette BeersJizhong ZouNatasha ThorneWei Zheng

Published in: Orphanet journal of rare diseases (2017)

The results demonstrate that these WD NSCs are valid cell-based disease models with characteristic disease phenotypes that can be used to evaluate drug efficacy and screen compounds. DT and HPBCD both reduce LysoTracker dye staining in WD cells. The cells may be used to further dissect the pathology of WD, evaluate compound efficacy, and serve as a platform for high-throughput drug screening to identify new compounds for therapeutic development.

Keyphrases

high throughput
induced apoptosis
single cell
cell cycle arrest
neural stem cells
emergency department
endoplasmic reticulum stress
cell death
oxidative stress
cell therapy
cell proliferation
adverse drug
highly efficient
pi k akt