NPC1-deficient neurons are selectively vulnerable for statin treatment
Sign Up to like & getrecommendations! Published in 2019 at "Neuropharmacology"
DOI: 10.1016/j.neuropharm.2019.04.012
Abstract: ABSTRACT Niemann Pick C (NPC) is a fatal hereditary neurovisceral disorder associated with a progressive loss of neurons of unknown mechanism. The disease is caused by mutation in either of two genes, termed npc1 and… read more here.
Keywords: npc1 deficient; neurons selectively; deficient neurons; statin ... See more keywords
Structure-function relationships of cholesterol mobilization from the endo-lysosome compartment of NPC1-deficient human cells by β-CD polyrotaxanes
Sign Up to like & getrecommendations! Published in 2022 at "PLOS ONE"
DOI: 10.1371/journal.pone.0268613
Abstract: Niemann-Pick Type C is a rare metabolic disorder characterized by the cellular accumulation of cholesterol within endosomal and lysosomal compartments. 2-Hydroxypropyl-β-cyclodextrin (HP-β-CD) containing polyrotaxanes represent an attractive approach for treating this disease due to their… read more here.
Keywords: npc1 deficient; mobilization endo; cells polyrotaxanes; cholesterol mobilization ... See more keywords