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Protein / target

Arylsulfatase A

Encoded byARSAP15289Homo sapiensSwiss-Prot
UniProt: P15289Ensembl: ENSG00000100299HGNC: HGNC:713NCBI Gene: 410
Clinically validated
Therapeutic maturity
1
Approved medicines
Open Targets target-level
Small-molecule tractable
Druggability
Structure with Ligand
1
Research papers

Protein at a glance

Biological role

Sulfuric ester hydrolase

Strongest disease association

Leukodystrophy, Metachromatic

Via encoding gene ARSA · Genetic evidence · score 0.99

Therapeutic position

Established drug target

Research activity

Emerging research

1 papers · latest 2013

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function overview

Lysosomal enzyme that catalyzes the hydrolysis of cerebroside-3-sulfate (sulfatide) into cerebroside and sulfate, a reaction that requires the activator protein saposin B

Subcellular location

Endoplasmic reticulumLysosome
Domains and Gene Ontology detail (11)

Gene Ontology

  • Cazurophil granule lumen
  • Cendoplasmic reticulum lumen
  • Cextracellular exosome
  • Cextracellular region
  • Clysosomal lumen
  • Clysosome
  • Farylsulfatase activity
  • Fcalcium ion binding
  • Fcerebroside-sulfatase activity
  • Fsulfuric ester hydrolase activity
  • Plipid metabolic process

507 aa · 54 kDa · 2 isoforms

Biological roles

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Lipid & lipoprotein metabolismGO
View supporting evidence

Lipid & lipoprotein metabolism

  • ·lipid metabolic process

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene ARSA

Gene-level evidence surfaced through the gene ARSA that encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Leukodystrophy, Metachromatic
0.99Well supported

Genetic evidence dominant · Open Targets 0.88

Genetic Diseases, Inborn
0.86Well supported

Genetic evidence dominant · Open Targets 0.52

Neurodevelopmental Disorders
0.67Moderately supported

Genetic evidence dominant · Open Targets 0.41

View evidence synthesis (3)
Leukodystrophy, MetachromaticWell supported
0.99
agreement 0.90–1.00
Genetic55%Clinical35%Animal model6%Literature5%Genetic literaturedup

Open Targets aggregate 0.88 · 4 independent evidence families · 1 not counted as duplicate

Genetic Diseases, InbornWell supported
0.86
agreement 0.72–1.00
Genetic99%Literature1%

Open Targets aggregate 0.52 · 2 independent evidence families

Neurodevelopmental DisordersModerately supported
0.67
agreement 0.55–0.79
Genetic100%

Open Targets aggregate 0.41 · 1 independent evidence family

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the per-type scores above show the calculation.

Show all associations
Leukodystrophy, Metachromatic0.88
Genetic Diseases, Inborn0.52
Neurodevelopmental Disorders0.41

Drug development

1 compounds recorded · 1 approved

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines.

View all recorded compounds (1)
ATIDARSAGENE AUTOTEMCELApproval

Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.

Tractability

Small molecules — Emerging

Feasibility evidence (structure with ligand) — no clinical-stage drug of this modality recorded.

Antibodies — Emerging

Feasibility evidence (go cc high conf and uniprot sigp or tmhmm) — no clinical-stage drug of this modality recorded.

Protein degraders — Emerging

Feasibility evidence (half-life data) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (4)
SM · Structure with LigandAB · GO CC high confAB · UniProt SigP or TMHMMPR · Half-life Data

Raw Open Targets tractability assessment buckets, by modality.

Research activity

1 papers · to 2013

Papers linked directly to this protein. This is the protein's own literature — descriptor-derived papers are kept separate below.

Most cited

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.892 cites

Biffi A · Science (New York, N.Y.) · 2013

Recent

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.

Biffi A · Science (New York, N.Y.) · 2013

Europe PMC papers linked directly to this protein.