Investigation of N-Aryl-3-alkylidenepyrrolinones as Potential Niemann−Pick Type C Disease Therapeutics
Journal of Medicinal Chemistry2009Vol. 52(20), pp. 6494–6498
Citations Over TimeTop 17% of 2009 papers
Casey C. Cosner, John T. Markiewicz, Pauline Bourbon, Christopher J. Mariani, Olaf Wiest, Madalina Rujoi, Anton I. Rosenbaum, Amy Y. Huang, Frederick R. Maxfield, Paul Helquist
Abstract
A five-step synthesis of an array of N-aryl-3-alkylidenepyrrolinones, which are potential Niemann-Pick type C (NPC) disease therapeutics, is described. The synthetic route allows for the production of analogues, including photoaffinity and biotinylated derivatives. Compound 1a increased esterification by acyl-coenzyme A:cholesteryl acyltransferase in NPC1 mutant cells. It also decreased LDL uptake and increased cholesterol efflux in both NPC1-deficient and normal cells.
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