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Chem Biodivers
; 21(8): e202401104, 2024 Aug.
Article
in English
| MEDLINE
| ID: mdl-38847390
ABSTRACT
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.