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Lalistat 1 is an inhibitor of lysosomal acid lipase (LAL; IC50 = 68 nM using purified human LAL).1 It is selective for LAL over human pancreatic and bovine milk lipoprotein lipases up to a concentration of 10 µM.2 Lalistat 1 (0.01-10 µM) reduces cholesterol accumulation in lysosome-like storage organelles in GM03123 human fibroblast cells deficient in NPC1, a membrane protein found in late endosomes, in a concentration-dependent manner but has little effect on cholesterol accumulation in NPC1-deficient CHO cells.
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1. Chemical screen to reduce sterol accumulation in Niemann-
2. Thiadiazole carbamates: Potent inhibitors of lysosomal acid lipase and potential Niemann-