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Keywords:

  • chaperone proteins;
  • enzymes;
  • Gaucher disease;
  • inhibitors;
  • isofagomines
Thumbnail image of graphical abstract

Heat protection: A series of C-alkylated isofagomines were synthesised and tested as inhibitors of the human glucocerebrosidase. Nanomolar and subnanomolar competitive inhibition was seen in each case, and all served to stabilise the enzyme against thermal denaturation (see figure). Pharmacologically useful levels of chaperoning of enzyme activity were seen in all cases, particularly for the propyl and nonyl derivatives.