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Arylsulfatase A (ARSA)
- Target
- ARSA
- Molecular classification
- Enzyme, Sulfatase, Lysosomal enzyme
01
Overview
Arylsulfatase A (ARSA) is a lysosomal enzyme responsible for the degradation of sulfatides, particularly cerebroside 3-sulfate. It is essential for maintaining myelin integrity in the nervous system. Deficiency in ARSA leads to the accumulation of sulfatides, causing metachromatic leukodystrophy (MLD). ARSA activity is assayed to diagnose MLD, and therapies are being developed to restore ARSA function.
- Other names
- ASACerebroside-sulfataseMLD
02
Mechanism of action
Hydrolysis of cerebroside 3-sulfate into cerebroside and sulfate. Phosphate can act as an inhibitor.
03
Biological functions
Sulfatide degradationMyelin maintenanceSphingolipid metabolism
04
Disease associations
Metachromatic leukodystrophyMultiple sulfatase deficiencyEnd-stage kidney disease (potential)Type 2 diabetes mellitus (potential)Parkinson's syndrome (potential)Prostate cancer (potential)HIV infection (potential)
05
Safety considerations
Enzyme replacement therapy has shown promise but faces challenges in delivery and immunogenicityGene therapy approaches are under investigation but require careful consideration of vector safety and off-target effects
06
Biomarkers
Arylsulfatase A activity levels in leukocytes or fibroblasts for MLD diagnosisSulfatide levels in urine or tissues for MLD diagnosis
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