1.
Chem Biodivers
; 21(8): e202401104, 2024 Aug.
Artículo
en Inglés
| MEDLINE
| ID: mdl-38847390
RESUMEN
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.