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Target intelligence / Profile preview
Galactosidase Alpha (GLA)
- Target
- GLA
- Molecular classification
- Enzyme, Glycoprotein, Hydrolase
01
Overview
The GLA gene encodes alpha-galactosidase A, a lysosomal enzyme responsible for hydrolyzing terminal alpha-galactosyl moieties from glycolipids and glycoproteins, particularly globotriaosylceramide (Gb3). Mutations in GLA lead to Fabry disease, an X-linked lysosomal storage disorder characterized by Gb3 accumulation in various tissues, causing progressive cellular damage. Enzyme replacement therapies (agalsidase alfa and beta) and pharmacological chaperones (migalastat) are used to treat Fabry disease.
- Other names
- AGAL_HUMANAgalsidase alfaAgalsidase beta
02
Mechanism of action
Enzyme replacement therapy; Pharmacological chaperone for certain amenable missense mutations by stabilizing mutant enzymes with residual function.
03
Biological functions
Sphingolipid metabolismHydrolysis of glycosphingolipids/glycoproteinsProtein homodimerizationInnate Immune System
04
Disease associations
Fabry DiseaseLysosomal Storage DisorderHypertrophic Cardiomyopathy
05
Safety considerations
Infusion reactions with enzyme replacement therapyEfficacy of Migalastat depends on mutation type/amenability criteria
06
Interacting drugs
Agalsidase alfa
2 more in the full profile.
07
Biomarkers
Globotriaosylceramide (Gb3) levelsAlpha-galactosidase A activity
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