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NPC1L1: evolution from pharmacological target to physiological sterol transporter
Ist Teil von
Arteriosclerosis, thrombosis, and vascular biology, 2006-11, Vol.26 (11), p.2433
Ort / Verlag
United States
Erscheinungsjahr
2006
Quelle
MEDLINE
Beschreibungen/Notizen
Niemann-Pick C1-like 1 protein (NPC1L1) was recently shown to be the molecular target of the cholesterol absorption inhibitor class of drugs, of which ezetimibe is the first widely used member. Since its discovery, NPC1L1 has also been shown to play a focal physiological role in intestinal absorption of sterols, including plant sterols and cholesterol. Evidence in support of this new metabolic pathway has been garnered not only through human, animal, and cell studies of function but also through the use of human genetics as an approach to study the association of NPC1L1 sequence variation with metabolic and drug-response phenotypes. The example of NPC1L1 shows how the elucidation of a pharmacological target can serve as a means to gain understanding of a key physiological pathway.