Transporters as Drug Targets: Discovery and Development of NPC1L1 Inhibitors
- 11 November 2009
- journal article
- research article
- Published by Wiley in Clinical Pharmacology & Therapeutics
- Vol. 87 (1) , 117-121
- https://doi.org/10.1038/clpt.2009.209
Abstract
The potent cholesterol absorption inhibitor ezetimibe was developed as a first-in-class drug for treating hypercholesterolemia even before its molecular target, Niemann-Pick C1-like 1 (NPC1L1), had been identified. The NPC1L1 protein mediates sterol transport across the enterocyte brush border membrane and is essential for intestinal cholesterol absorption, a major pathway controlling whole-body cholesterol homeostasis. An elucidation of the mechanism underlying NPC1L1-dependent cholesterol absorption would greatly facilitate the discovery and development of new cholesterol-lowering agents for treating hypercholesterolemia and other cholesterol-related metabolic disorders.This publication has 32 references indexed in Scilit:
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