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Protein / target

RNA-binding protein FUS

Encoded byFUSP35637Homo sapiensSwiss-Prot
UniProt: P35637Ensembl: ENSG00000089280HGNC: HGNC:4010NCBI Gene: 2521
Small-molecule tractable
Druggability
Structure with Ligand
1
Research papers

Protein at a glance

Biological role

Molecular condensate scaffold

Strongest disease association

Amyotrophic Lateral Sclerosis

Via encoding gene FUS · Genetic evidence · score 0.87

Research activity

Emerging research

1 papers · latest 2019

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

DNA/RNA-binding protein that plays a role in various cellular processes such as transcription regulation, RNA splicing, RNA transport, DNA repair and damage response.

View complete UniProt function annotation

DNA/RNA-binding protein that plays a role in various cellular processes such as transcription regulation, RNA splicing, RNA transport, DNA repair and damage response (PubMed:27731383). Binds to ssRNA containing the consensus sequence 5'-AGGUAA-3' (PubMed:21256132). Binds to nascent pre-mRNAs and acts as a molecular mediator between RNA polymerase II and U1 small nuclear ribonucleoprotein thereby coupling transcription and splicing (PubMed:26124092). Also binds its own pre-mRNA and autoregulates its expression; this autoregulation mechanism is mediated by non-sense-mediated decay (PubMed:24204307). Plays a role in DNA repair mechanisms by promoting D-loop formation and homologous recombination during DNA double-strand break repair (PubMed:10567410). In neuronal cells, plays crucial roles in dendritic spine formation and stability, RNA transport, mRNA stability and synaptic homeostasis (By similarity)

Subcellular location

Nucleus
Domains and Gene Ontology detail (25)

Domains & features

RRM

Gene Ontology

  • CGABA-ergic synapse
  • Cglutamatergic synapse
  • Cnucleoplasm
  • Cnucleus
  • Cpostsynaptic cytosol
  • Cpresynaptic cytosol
  • Fchromatin binding
  • FDNA binding
  • Fidentical protein binding
  • Fmolecular condensate scaffold activity
  • FmRNA 3'-UTR binding
  • FRNA binding

526 aa · 53 kDa · 2 isoforms

Biological roles

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

Transcriptional regulationUniProt · GO
View supporting evidence

Transcriptional regulation

  • ·DNA/RNA-binding protein that plays a role in various cellular processes such as transcri…
  • ·transcription coactivator activity
  • ·transcription coregulator activity
  • ·regulation of DNA-templated transcription

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 FUS

Gene-level evidence surfaced through the gene FUS 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
0.89Well supported

Genetic evidence dominant · Open Targets 0.71

Essential Tremor
0.77Well supported

Genetic evidence dominant · Open Targets 0.56

Genetic Diseases, Inborn
0.68Moderately supported

Genetic evidence dominant · Open Targets 0.42

Liposarcoma
0.45Limited support

Somatic mutation evidence dominant · Open Targets 0.39

View evidence synthesis (4)
Amyotrophic Lateral SclerosisWell supported
0.89
agreement 0.75–1.00
Genetic85%Literature15%Genetic literaturedup

Open Targets aggregate 0.71 · 2 independent evidence families · 1 not counted as duplicate

Essential TremorWell supported
0.77
agreement 0.63–0.91
Genetic97%Literature3%Genetic literaturedup

Open Targets aggregate 0.56 · 2 independent evidence families · 1 not counted as duplicate

Genetic Diseases, InbornModerately supported
0.68
agreement 0.55–0.82
Genetic99%Literature2%

Open Targets aggregate 0.42 · 2 independent evidence families

LiposarcomaLimited support
0.45
agreement 0.28–0.61
Somatic mutation83%Literature18%

Open Targets aggregate 0.39 · 2 independent evidence families

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 Sclerosis0.71
Essential Tremor0.56
Genetic Diseases, Inborn0.42
Liposarcoma0.39

Tractability

Small molecules — Emerging

Feasibility evidence (structure with ligand) — 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 (4)
SM · Structure with LigandPR · UniProt UbiquitinationPR · Database UbiquitinationPR · Half-life Data

Raw Open Targets tractability assessment buckets, by modality.

Research activity

1 papers · to 2019

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

Most cited

Circular RNA CircFndc3b modulates cardiac repair after myocardial infarction via FUS/VEGF-A axis.354 cites

Garikipati VNS · Nature communications · 2019

Recent

Circular RNA CircFndc3b modulates cardiac repair after myocardial infarction via FUS/VEGF-A axis.

Garikipati VNS · Nature communications · 2019

Europe PMC papers linked directly to this protein.