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Target intelligence / Profile preview
Lysosomal acid glucosylceramidase (GCase)
- Target
- GCase
- Molecular classification
- Enzyme, Glycoside hydrolase
01
Overview
Lysosomal acid glucosylceramidase (GCase), encoded by the GBA1 gene, is a lysosomal enzyme responsible for the hydrolysis of glucocerebroside into glucose and ceramide. It plays a crucial role in sphingolipid metabolism and cellular membrane turnover. Mutations in GBA1 lead to Gaucher disease, a lysosomal storage disorder. GBA1 variants are also associated with an increased risk of Parkinson's disease.
- Other names
- Glucosylceramidase beta 1β-glucocerebrosidaseAcid β-glucosidaseD-glucosyl-N-acylsphingosine glucohydrolaseGBA1
02
Mechanism of action
Enzyme replacement therapy (for Gaucher Disease); Chaperone therapy (for Gaucher Disease)
03
Biological functions
Hydrolysis of glucocerebrosideSphingolipid metabolismLysosome functionCell membrane turnover
04
Disease associations
Gaucher diseaseParkinson's diseaseParkinsonism
05
Safety considerations
Potential for immune reactions to enzyme replacement therapyNeurological complications in neuronopathic Gaucher diseaseIncreased risk of Parkinson's disease with certain variants
06
Biomarkers
Glucocerebroside levelsGBA1 enzyme activity
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