%0 Journal Article
%T Iminosugar-Dihydroazulenes as Mutant L444P Glucocerebrosidase Enhancers.
%A Davighi MG
%A Clemente F
%A Andreasen ES
%A Nielsen MB
%A Matassini C
%A Goti A
%A Morrone A
%A Paoli P
%A Cardona F
%A Cacciarini M
%J Chem Biodivers
%V 0
%N 0
%D 2024 Jun 7
%M 38847390
%F 2.745
%R 10.1002/cbdv.202401104
%X A remarkable enhancer of human glucocerebrosidase enzyme (GCase) was identified among a set of dihydroazulene-tagged iminosugars. An unprecedented 3.9-fold increase in GCase activity was detected on fibroblasts bearing the homozygous L444P mutation, which is frequently associated with neuronopathic Gaucher forms, and which commonly results refractory to chaperone-induced refolding.