title: Niemann-Pick NPC1: sterols to the rescue and beyond.
abstract: In Niemann-Pick type C disease, the most prevalent I1061T mutation inhibits folding and trafficking of the NPC1 protein to the endosomes/lysosomes. In this issue of Chemistry & Biology, Ohgane and colleagues used pharmacological chaperones to repair the defect and identify a second sterol-binding site.
authors.affiliation: Department of Medicine and Department of Biochemistry, University of Toronto, Toronto, ON M5S 1A8, Canada.
authors.forename: Tip W
authors.initials: TW
authors.lastname: Loo
authors.forename: David M
authors.initials: DM
authors.lastname: Clarke