Target intelligence / Profile preview

Beta-glucocerebrosidase (GCase)

Target
GCase
Molecular classification
Enzyme, Glycosyl hydrolase
01

Overview

Beta-glucocerebrosidase (GCase) is a lysosomal enzyme responsible for the hydrolysis of glucocerebroside into glucose and ceramide. It is essential for cellular lipid metabolism and membrane recycling. Mutations in the GBA gene, encoding GCase, cause Gaucher disease and increase the risk of Parkinson's disease. Therapeutic strategies include enzyme replacement therapy.

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

Mechanism of action

Enzyme replacement therapy

03

Biological functions

Membrane lipid recyclingSkin barrier formationHydrolysis of glucocerebroside
04

Disease associations

Gaucher DiseaseParkinson’s Disease
05

Safety considerations

ImmunogenicityInfusion reactionsBlood-brain barrier penetration (for neurological symptoms)
06

Interacting drugs

Imiglucerase
07

Biomarkers

Glucocerebroside levelsBeta-glucocerebrosidase activity

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