Ontology highlight
ABSTRACT:
SUBMITTER: Dai S
PROVIDER: S-EPMC5584846 | biostudies-literature | 2017 Aug
REPOSITORIES: biostudies-literature
Dai Sheng S Dulcey Andrés E AE Hu Xin X Wassif Christopher A CA Porter Forbes D FD Austin Christopher P CP Ory Daniel S DS Marugan Juan J Zheng Wei W
Autophagy 20170614 8
The drug 2-hydroxypropyl-β-cyclodextrin (HPβCD) reduces lysosomal cholesterol accumulation in Niemann-Pick disease, type C (NPC) and has been advanced to human clinical trials. However, its mechanism of action for reducing cholesterol accumulation in NPC cells is uncertain and its molecular target is unknown. We found that methyl-β-cyclodextrin (MβCD), a potent analog of HPβCD, restored impaired macroautophagy/autophagy flux in Niemann-Pick disease, type C1 (NPC1) cells. This effect was mediated ...[more]