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Functionalized cyclophellitols are selective glucocerebrosidase inhibitors and induce a bona fide neuropathic Gaucher model in zebrafish.

著者 Artola M , Kuo CL , Lelieveld LT , Rowland RJ , van der Marel GA , Codée JDC , Boot RG , Davies GJ , Aerts JMFG , Overkleeft HS
J Am Chem Soc.2019 Feb 27 ; ():.
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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 genera-tion and interpretation of such chemical knockout models complicated. Here we demonstrate that cyclophellitol derivatives carrying a bulky hydrophobic substituent at C8 are potent and selective GBA inhibitors and that an unambiguous Gaucher animal model can be readily generated by treatment of zebrafish with these.
PMID: 30811188 [PubMed - as supplied by publisher]
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