Article
Glucosylceramide mimics: highly potent GCase inhibitors and selective pharmacological chaperones for mutations associated with types 1 and 2 Gaucher disease.
ChemMedChem - 1 Nov 2013
Schönemann Wojciech, Gallienne Estelle, Ikeda-Obatake Kyoko, Asano Naoki, Nakagawa Shinpei, Kato Atsushi, Adachi Isao, Górecki Marcin, Frelek Jadwiga, Martin Olivier R
Abstract excerpt
A series of iminoxylitol derivatives carrying a C-linked di-O-acyl or di-O-alkyl glyceryl substituent were prepared and characterized. All of these compounds, which were designed as glucosylceramide (GlcCer) mimics, were nanomolar inhibitors of lysosomal β-glucosidase (glucocerebrosidase, GCase). Two of these pseudoglycolipids were further evaluated for their ability to enhance the activity of mutant GCase in...
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