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Protein / target

Actin nucleation-promoting factor WAS

Encoded byWASP42768Homo sapiensSwiss-Prot
UniProt: P42768Ensembl: ENSG00000015285HGNC: HGNC:12731NCBI Gene: 7454
Small-molecule tractable
Druggability
Structure with Ligand
1
Research papers

Protein at a glance

Biological role

GTPase regulator

Strongest disease association

Wiskott-Aldrich Syndrome

Via encoding gene WAS · Genetic evidence · score 0.93

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

Effector protein for Rho-type GTPases that regulates actin filament reorganization via its interaction with the Arp2/3 complex.

View complete UniProt function annotation

Effector protein for Rho-type GTPases that regulates actin filament reorganization via its interaction with the Arp2/3 complex (PubMed:12235133, PubMed:12769847, PubMed:16275905). Important for efficient actin polymerization (PubMed:12235133, PubMed:16275905, PubMed:8625410). Possible regulator of lymphocyte and platelet function (PubMed:9405671). Mediates actin filament reorganization and the formation of actin pedestals upon infection by pathogenic bacteria (PubMed:18650809). In addition to its role in the cytoplasmic cytoskeleton, also promotes actin polymerization in the nucleus, thereby regulating gene transcription and repair of damaged DNA (PubMed:20574068). Promotes homologous recombination (HR) repair in response to DNA damage by promoting nuclear actin polymerization, leading to drive motility of double-strand breaks (DSBs) (PubMed:29925947)

Subcellular location

Cytoplasm, cytoskeletonNucleus
Domains and Gene Ontology detail (35)

Domains & features

WH1CRIBWH2

Gene Ontology

  • Cactin cytoskeleton
  • Cactin filament
  • Ccell-cell junction
  • Ccytosol
  • Cextracellular exosome
  • Cnucleus
  • Cphagocytic vesicle
  • Csite of double-strand break
  • Cvesicle membrane
  • Factin binding
  • FGTPase regulator activity
  • Fidentical protein binding

502 aa · 53 kDa

Biological roles

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

Cell migrationGOTranscriptional regulationUniProt · GOHaemostasisUniProt · GOImmune signallingGO
View supporting evidence

Cell migration

  • ·negative regulation of cell motility

Transcriptional regulation

  • ·Effector protein for Rho-type GTPases that regulates actin filament reorganization via i…
  • ·positive regulation of transcription by RNA polymerase II

Haemostasis

  • ·Effector protein for Rho-type GTPases that regulates actin filament reorganization via i…
  • ·blood coagulation

Immune signalling

  • ·immune response
  • ·regulation of T cell antigen processing and presentation

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 WAS

Gene-level evidence surfaced through the gene WASthat 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.

Wiskott-Aldrich Syndrome
0.97Well supported

Genetic evidence dominant · Open Targets 0.85

Genetic Diseases, Inborn
0.68Moderately supported

Genetic evidence dominant · Open Targets 0.41

Lymphoma
0.63Moderately supported

Genetic literature evidence dominant · Open Targets 0.47

Severe congenital neutropenia
0.62Moderately supported

Genetic literature evidence dominant · Open Targets 0.47

Melanoma
0.41Limited support

Somatic mutation evidence dominant · Open Targets 0.38

View evidence synthesis (5)
Wiskott-Aldrich SyndromeWell supported
0.97
agreement 0.87–1.00
Genetic57%Somatic mutation22%Animal model16%Literature6%Genetic literaturedup

Open Targets aggregate 0.85 · 4 independent evidence families · 1 not counted as duplicate

Genetic Diseases, InbornModerately supported
0.68
agreement 0.54–0.82
Genetic99%Literature1%

Open Targets aggregate 0.41 · 2 independent evidence families

LymphomaModerately supported
0.63
agreement 0.48–0.78
Genetic literature91%Literature9%

Open Targets aggregate 0.47 · 2 independent evidence families

Severe congenital neutropeniaModerately supported
0.62
agreement 0.46–0.77
Genetic literature97%Literature3%

Open Targets aggregate 0.47 · 2 independent evidence families

MelanomaLimited support
0.41
agreement 0.25–0.57
Somatic mutation93%Literature7%

Open Targets aggregate 0.38 · 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
Wiskott-Aldrich Syndrome0.85
Lymphoma0.47
Severe congenital neutropenia0.47
Genetic Diseases, Inborn0.41
Melanoma0.38

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 human protein atlas loc) — no clinical-stage drug of this modality recorded.

Protein degraders — Emerging

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

View underlying tractability evidence (5)
SM · Structure with LigandAB · GO CC high confAB · Human Protein Atlas locPR · Database UbiquitinationPR · 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 in patients with Wiskott-Aldrich syndrome.799 cites

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

Recent

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

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

Europe PMC papers linked directly to this protein.