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  • A Fluorescence Polarization...
    Lahav, Daniël; Liu, Bing; van den Berg, Richard J. B. H. N; van den Nieuwendijk, Adrianus M. C. H; Wennekes, Tom; Ghisaidoobe, Amar T; Breen, Imogen; Ferraz, Maria J; Kuo, Chi-Lin; Wu, Liang; Geurink, Paul P; Ovaa, Huib; van der Marel, Gijsbert A; van der Stelt, Mario; Boot, Rolf G; Davies, Gideon J; Aerts, Johannes M. F. G; Overkleeft, Herman S

    Journal of the American Chemical Society, 10/2017, Volume: 139, Issue: 40
    Journal Article

    Human nonlysosomal glucosylceramidase (GBA2) is one of several enzymes that controls levels of glycolipids and whose activity is linked to several human disease states. There is a major need to design or discover selective GBA2 inhibitors both as chemical tools and as potential therapeutic agents. Here, we describe the development of a fluorescence polarization activity-based protein profiling (FluoPol-ABPP) assay for the rapid identification, from a 350+ library of iminosugars, of GBA2 inhibitors. A focused library is generated based on leads from the FluoPol-ABPP screen and assessed on GBA2 selectivity offset against the other glucosylceramide metabolizing enzymes, glucosylceramide synthase (GCS), lysosomal glucosylceramidase (GBA), and the cytosolic retaining β-glucosidase, GBA3. Our work, yielding potent and selective GBA2 inhibitors, also provides a roadmap for the development of high-throughput assays for identifying retaining glycosidase inhibitors by FluoPol-ABPP on cell extracts containing recombinant, overexpressed glycosidase as the easily accessible enzyme source.