Glucocerebrosidase-IN-2 is a potent inhibitor of glucocerebrosidase, a critical enzyme in lipid metabolism linked to Gaucher disease. This quinazoline derivative enhances glucocerebrosidase translocation to lysosomes in patient-derived cells, particularly those with the N370S mutation. It effectively inhibits the hydrolysis of substrates such as 4-methylumbelliferone β-D-glucopyranoside and fluorescent glycosylceramide in N370S mutant tissues, exhibiting an AC50 of 25.29 μM. Glucocerebrosidase-IN-2 is valuable for research focused on therapeutic approaches for Gaucher disease and related lysosomal storage disorders.
Glucocerebrosidase-IN-2 is a potent inhibitor of glucocerebrosidase, a critical enzyme in lipid metabolism linked to Gaucher disease. This quinazoline derivative enhances glucocerebrosidase translocation to lysosomes in patient-derived cells, particularly those with the N370S mutation. It effectively inhibits the hydrolysis of substrates such as 4-methylumbelliferone β-D-glucopyranoside and fluorescent glycosylceramide in N370S mutant tissues, exhibiting an AC50 of 25.29 μM. Glucocerebrosidase-IN-2 is valuable for research focused on therapeutic approaches for Gaucher disease and related lysosomal storage disorders.
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