Title : Unesterified cholesterol accumulation in late endosomes/lysosomes causes neurodegeneration and is prevented by driving cholesterol export from this compartment.

Pub. Date : 2011 Jun 22

PMID : 21697390






1 Functional Relationships(s)
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1 When injected into the CNS of the npc1(-/-) mouse, 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD), a compound known to prevent this C accumulation, diffused throughout the brain and was excreted with a t(½) of 6.5 h. This agent caused suppression of C synthesis, elevation of C esters, suppression of sterol regulatory-binding protein 2 (SREBP2) target genes, and activation of liver X receptor-controlled genes. 2-Hydroxypropyl-beta-cyclodextrin sterol regulatory element binding factor 2 Mus musculus