Article
Structure-activity relationship studies of Niemann-Pick type C1-like 1 (NPC1L1) ligands identified by screening assay monitoring pharmacological chaperone effect.
Bioorganic & medicinal chemistry - 1 Sept 2013
Karaki Fumika, Ohgane Kenji, Dodo Kosuke, Hashimoto Yuichi
Abstract excerpt
A number of hereditary diseases are caused by defective protein trafficking due to a folding defect resulting from point mutations in proteins. Ligands that bind to the folding intermediates of such mutant proteins and rescue their trafficking defects, known as pharmacological chaperones, have promise for the treatment of certain genetic diseases, including Fabry disease, cystic fibrosis, and Niemann-Pick disease...
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