Publication | Closed Access
Bicyclic Derivatives of <scp>L</scp>‐Idonojirimycin as Pharmacological Chaperones for Neuronopathic Forms of Gaucher Disease
33
Citations
68
References
2013
Year
Pharmaceutical ScienceBicyclic DerivativesGlycobiologyMolecular BiologyPharmaceutical ChemistryMutant Gcase EnzymeNew Human β-GlucocerebrosidaseNeurologyGlycosylationGaucher DiseaseBiochemistryType 2Pharmacological AgentNeuroprotectionDrug DevelopmentPharmacological ChaperonesPharmacologyMolecular ModelingNeurodegenerative DiseasesNatural SciencesMedicineCarbohydrate-protein InteractionDrug Discovery
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.
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