Bicyclic derivatives of L-idonojirimycin as pharmacological chaperones for neuronopathic forms of Gaucher disease

Chembiochem. 2013 May 27;14(8):943-9. doi: 10.1002/cbic.201200708. Epub 2013 Apr 18.

Abstract

New human β-glucocerebrosidase (GCase) ligands with rigid 1,6-anhydro-β-L-idonojirimycin cores have been designed with the aid of molecular modeling. Efficient pharmacological chaperones for the L444P (trafficking-incompetent) mutant GCase enzyme associated with type 2 and 3 Gaucher disease (GD) were identified.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • COS Cells
  • Cell Line
  • Chlorocebus aethiops
  • Gaucher Disease / enzymology*
  • Gaucher Disease / genetics
  • Glucosylceramidase / genetics
  • Glucosylceramidase / metabolism*
  • Humans
  • Imino Pyranoses / chemistry*
  • Imino Pyranoses / pharmacology*
  • Ligands
  • Molecular Docking Simulation
  • Mutation

Substances

  • Imino Pyranoses
  • Ligands
  • Glucosylceramidase