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Target intelligence / Profile preview
Non-lysosomal Neutral Glucosylceramidase (GBA2)
- Target
- GBA2
- Molecular classification
- Enzyme, Glycosidase, Glucosylceramidase
01
Overview
Non-lysosomal neutral glucosylceramidase (GBA2) is an enzyme that catalyzes the hydrolysis of glucosylceramide into glucose and ceramide outside of lysosomes. It is a microsomal beta-glucosidase primarily located in the endoplasmic reticulum and Golgi apparatus. GBA2 plays a role in lipid metabolism, bile acid metabolism, and cholesterol modification. Mutations in GBA2 are associated with hereditary spastic paraplegia, cerebellar ataxia, and male infertility.
- Other names
- β-glucosidase 2
02
Mechanism of action
Inhibition of glucosylceramidase activity
03
Biological functions
Glucosylceramide hydrolysisLipid metabolismBile acid metabolismCholesterol modification
04
Disease associations
Hereditary spastic paraplegiaCerebellar ataxiaMale infertilitySphingolipid storage disorders
05
Safety considerations
Inhibition impairs spermatogenesis
06
Interacting drugs
Miglustat
1 more in the full profile.
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