Glucosylceramidase/GBA Small Molecules and Peptides

Glucosylceramidase is a lysosomal enzyme that cleaves the beta-glucosidic linkage of glucosylceramide, an intermediate in glycolipid metabolism. The mature enzyme has 497 amino acids with a molecular weight of 62 kDa. Glycosylation occurs at four of five N-glycosylation sites and is essential for the trafficking and activity of the enzyme. The enzyme is activated in lysosomes by saposin C, although the mechanism of activation is not well understood. Defects in Glucosylceramidase are the cause ...