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

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  • Marta Artola
  • Chi-Lin Kuo
  • Lindsey T. Lelieveld
  • Rhianna J. Rowland
  • Gijsbert A. van der Marel
  • Jeroen D. C. Codée
  • Rolf G. Boot
  • Gideon J. Davies
  • Johannes M. F. G. Aerts
  • Herman S. Overkleeft


Publication details

JournalJournal of the American Chemical Society
DateAccepted/In press - 27 Feb 2019
DateE-pub ahead of print - 27 Feb 2019
DatePublished (current) - 13 Mar 2019
Issue number10
Number of pages5
Pages (from-to) 4214–4218
Early online date27/02/19
Original languageEnglish


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 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.

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© 2019 American Chemical Society. This is an author-produced version of the published paper. Uploaded in accordance with the publisher’s self-archiving policy. Further copying may not be permitted; contact the publisher for details.


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