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Target intelligence / Profile preview
Galactosylceramidase
- Molecular classification
- Enzyme, Hydrolase, Lysosomal enzyme
01
Overview
Galactosylceramidase (GALC) is a lysosomal hydrolase that catalyzes the breakdown of galactosylceramide and psychosine, essential for myelin turnover. Deficiency due to GALC mutations causes Krabbe disease, a severe demyelinating disorder. It is considered a therapeutic target, and potential therapeutic strategies include enzyme replacement therapy, gene therapy, and small molecule inhibitors.
- Other names
- GalcGalCERaseGalcosidic ceramidase beta-galctocerebrosidaseGALC_HUMANGalactocerebrosidase
02
Mechanism of action
Enzyme inhibition (potential therapeutic strategy)
03
Biological functions
Hydrolysis of galactosylceramideHydrolysis of psychosineMyelin turnoverSphingolipid catabolism
04
Disease associations
Krabbe diseaseGloboid cell leukodystrophyNeurodegenerative diseaseDemyelination
05
Safety considerations
Off-target effects of inhibitorsBlood-brain barrier penetration for CNS diseasesEnzyme replacement therapy challenges (delivery, immune response)Gene therapy risks (immune response, insertional mutagenesis)
06
Biomarkers
Galactosylceramide levelsPsychosine levelsGALC enzyme activityGALC gene mutations
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