Article
Chemical screen to reduce sterol accumulation in Niemann-Pick C disease cells identifies novel lysosomal acid lipase inhibitors.
Biochimica et biophysica acta - 1 Dec 2009
Rosenbaum Anton I, Rujoi Madalina, Huang Amy Y, Du Hong, Grabowski Gregory A, Maxfield Frederick R
Abstract excerpt
Niemann-Pick C disease (NPC) is a lysosomal storage disorder causing abnormal accumulation of unesterified free cholesterol in lysosomal storage organelles. High content phenotypic microscopy chemical screens in both human and hamster NPC-deficient cells have identified several compounds that par...
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