Functionalized Cyclophellitols Are Selective Glucocerebrosidase Inhibitors and Induce a Bona Fide Neuropathic Gaucher Model in Zebrafish.
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ABSTRACT:
SUBMITTER: Artola M
PROVIDER: S-EPMC6418866 | biostudies-literature | 2019 Mar
REPOSITORIES: biostudies-literature
Publications
Functionalized Cyclophellitols Are Selective Glucocerebrosidase Inhibitors and Induce a Bona Fide Neuropathic Gaucher Model in Zebrafish.
Artola Marta M Kuo Chi-Lin CL Lelieveld Lindsey T LT Rowland Rhianna J RJ van der Marel Gijsbert A GA Codée Jeroen D C JDC Boot Rolf G RG Davies Gideon J GJ Aerts Johannes M F G JMFG Overkleeft Herman S HS
Journal of the American Chemical Society 20190304 10
Gaucher disease is caused by inherited deficiency in glucocerebrosidase (GBA, a retaining β-glucosidase), and deficiency in GBA constitutes the largest known genetic risk factor for Parkinson's disease. In the past, animal models of Gaucher disease have been generated by treatment with the mechanism-based GBA inhibitors, conduritol B epoxide (CBE), and cyclophellitol. Both compounds, however, also target other retaining glycosidases, rendering generation and interpretation of such chemical knock ...[more]
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