Fluorescence polarisation activity-based protein profiling for the identification of deoxynojirimycin-type inhibitors selective for lysosomal retaining alpha- and beta-glucosidases
Daniël van der Gracht, R.J. Rowland, V. Roig-Zamboni, Maria J. Ferraz, Max Louwerse, Paul P. Geurink, Johannes M. F. G. Aerts, Gerlind Sulzenbacher, G.J. Davies, Herman S. Overkleeft, Marta Artola
Abstract
-alkyldeoxynojirimycins that inhibit GAA, but not GBA1, and that may form the starting point for the development of pharmacological chaperone therapeutics for the lysosomal glycogen storage disease that results from genetic deficiency in GAA: Pompe disease.
Topics & Concepts
IminosugarSubstrate reduction therapyLysosomal storage disordersChemistryLysosomal storage diseaseBiochemistryGlycoside hydrolaseGlucosidasesEnzymeLysosomeComputational biologyBiologyEnzyme replacement therapyDiseaseMedicinePathologyLysosomal Storage Disorders ResearchCarbohydrate Chemistry and SynthesisTrypanosoma species research and implications