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Protein / target

Superoxide dismutase [Cu-Zn]

Encoded bySOD1P00441Homo sapiensSwiss-Prot
UniProt: P00441Ensembl: ENSG00000142168HGNC: HGNC:11179NCBI Gene: 6647
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

Protein homodimerization

Strongest disease association

Amyotrophic Lateral Sclerosis

Via encoding gene SOD1 · Genetic evidence · score 0.94

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

Destroys radicals which are normally produced within the cells and which are toxic to biological systems.

View complete UniProt function annotation

Destroys radicals which are normally produced within the cells and which are toxic to biological systems (PubMed:18948262, PubMed:24140062). Catalyzes the oxidation of hydrogen sulfide (H2S) to sulfate, playing an important role in detoxifying H2S and limiting the accumulation of reactive sulfur species (RSS) such as persulfides and polysulfides (PubMed:36630448)

Subcellular location

CytoplasmNucleus
Domains and Gene Ontology detail (77)

Gene Ontology

  • Caxon cytoplasm
  • Ccytoplasm
  • Ccytoplasmic vesicle
  • Ccytosol
  • Cdendrite cytoplasm
  • Cdense core granule
  • Cextracellular exosome
  • Cextracellular region
  • Cextracellular space
  • Clysosome
  • Cmitochondrial intermembrane space
  • Cmitochondrial matrix

154 aa · 16 kDa

Biological roles

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

Immune signallingGOMetabolic enzyme activityGOApoptosis & cell deathGO
View supporting evidence

Immune signalling

  • ·negative regulation of inflammatory response
  • ·positive regulation of cytokine production
  • ·regulation of T cell differentiation in thymus

Metabolic enzyme activity

  • ·glutathione metabolic process
  • ·reactive oxygen species metabolic process
  • ·superoxide metabolic process

Apoptosis & cell death

  • ·negative regulation of neuron apoptotic process
  • ·positive regulation of apoptotic 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 SOD1

Gene-level evidence surfaced through the gene SOD1 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.

Amyotrophic Lateral Sclerosis
0.99Well supported

Genetic evidence dominant · Open Targets 0.88

Motor Neuron Disease
0.73Moderately supported

Genetic evidence dominant · Open Targets 0.59

Neurodegenerative Diseases
0.38Preliminary

Pathway evidence dominant · Open Targets 0.55 · no direct causal or clinical evidence

View evidence synthesis (3)
Amyotrophic Lateral SclerosisWell supported
0.99
agreement 0.89–1.00
Genetic47%Clinical35%Animal model11%Literature8%Genetic literaturedup

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

Motor Neuron DiseaseModerately supported
0.73
agreement 0.59–0.87
Genetic94%Literature7%

Open Targets aggregate 0.59 · 2 independent evidence families

Neurodegenerative DiseasesPreliminary
0.38
agreement 0.20–0.56
Pathway92%Literature8%

Open Targets aggregate 0.55 · 2 independent evidence families · no direct causal or clinical evidence

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
Amyotrophic Lateral Sclerosis0.88
Motor Neuron Disease0.59
Neurodegenerative Diseases0.55

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)
TOFERSENApproval

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 and high-quality pocket) — no clinical-stage drug of this modality recorded.

Antibodies — Emerging

Feasibility evidence (go cc high conf) — no clinical-stage drug of this modality recorded.

Protein degraders — Emerging

Feasibility evidence (uniprot ubiquitination and database ubiquitination) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (7)
SM · Structure with LigandSM · High-Quality PocketAB · GO CC high confPR · UniProt UbiquitinationPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

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

Copper-zinc superoxide dismutase (SOD1) is released by microglial cells and confers neuroprotection against 6-OHDA neurotoxicity.21 cites

Polazzi E · Neuro-Signals · 2013

Recent

Copper-zinc superoxide dismutase (SOD1) is released by microglial cells and confers neuroprotection against 6-OHDA neurotoxicity.

Polazzi E · Neuro-Signals · 2013

Europe PMC papers linked directly to this protein.