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Evidence for a Cholesterol Transport Pathway from Lysosomes to Endoplasmic Reticulum That Is Independent of the Plasma Membrane*
TLDR
Evidence is presented that supports a vesicular model of cholesterol transport from lysosomes to the endoplasmic reticulum that is independent of the plasma membrane and is normal in a Chinese hamster ovary mutant with defective plasma membrane-to-ACAT movement.
Identification of a pharmaceutical compound that partially corrects the Niemann-Pick C phenotype in cultured cells DOI 10.1194/jlr.M200179-JLR200
TLDR
An amphotericin B-mediated cytotoxicity assay is developed and implemented to screen for potential therapeutic drugs that induce cholesterol movement in cultured NPC cells and one compound, NP-27, corrected the NPC phenotype by four different measures of cholesterol homeostasis.