title: Unraveling the sterol-trafficking defect in Niemann-Pick C disease.
authors.affiliation: Department of Pediatrics and Institute of Human Nutrition, Columbia University Medical Center, 630 West 168th Street, New York, NY 10032, USA.
authors.forename: Stephen L
authors.initials: SL
authors.lastname: Sturley
authors.forename: Marc C
authors.initials: MC
authors.lastname: Patterson
authors.forename: Peter
authors.initials: P
authors.lastname: Pentchev
mesh_terms: D000073738:2-Hydroxypropyl-beta-cyclodextrin; D000818:Animals; D001692:Biological Transport; D002784:Cholesterol / Q000378:metabolism; D005079:Excipients / Q000494:pharmacology; D006801:Humans; D047908:Intracellular Signaling Peptides and Proteins; D008055:Lipids / Q000737:chemistry / Q000502:physiology*; D008247:Lysosomes / Q000378:metabolism; D051379:Mice; D008822:Mice, Transgenic; D008954:Models, Biological; D009154:Mutation; D000077296:Niemann-Pick C1 Protein; D009542:Niemann-Pick Diseases / Q000378:metabolism*; D011506:Proteins / Q000378:metabolism; D013261:Sterols / Q000378:metabolism*; D047392:beta-Cyclodextrins / Q000494:pharmacology
chemical_list: D005079:Excipients; D047908:Intracellular Signaling Peptides and Proteins; D008055:Lipids; D000077296:Niemann-Pick C1 Protein; C106831:Npc1 protein, mouse; D011506:Proteins; D013261:Sterols; D047392:beta-Cyclodextrins; D000073738:2-Hydroxypropyl-beta-cyclodextrin; D002784:Cholesterol