Target intelligence / Profile preview

Glucocerebrosidase (GCase)

Target
GCase
Molecular classification
Enzyme, Glycoside hydrolase
01

Overview

Glucocerebrosidase (GCase) is a lysosomal enzyme that catalyzes the hydrolysis of glucosylceramide into ceramide and glucose. It is encoded by the GBA1 gene. Deficiency in GCase activity leads to Gaucher disease and is also a risk factor for Parkinson's disease. Therapeutic strategies include enzyme replacement therapy (ERT) and substrate reduction therapy (SRT).

Other names
β-glucocerebrosidaseAcid β-glucosidaseD-glucosyl-N-acylsphingosine glucohydrolase
02

Mechanism of action

Hydrolyzes glucosylceramide into ceramide and glucose.

03

Biological functions

Glycolipid catabolismHydrolysis of glucosylceramideHydrolysis of glucosylsphingosineSkin barrier formation
04

Disease associations

Gaucher diseaseParkinson's disease
05

Safety considerations

Infusion reactions (ERT)Neurological side effects (substrate reduction therapy)
06

Interacting drugs

Imiglucerase

4 more in the full profile.

07

Biomarkers

Glucosylceramide levelsGlucosylsphingosine levelsGCase enzyme activity

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