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Protein / target

Choline O-acetyltransferase

Encoded byCHATP28329Homo sapiensSwiss-Prot
UniProt: P28329Ensembl: ENSG00000070748HGNC: HGNC:1912NCBI Gene: 1103
Small-molecule tractable
Druggability
Structure with Ligand
1
Research papers

Protein at a glance

Biological role

Choline O-acetyltransferase

Strongest disease association

Congenital myasthenic syndromes

Via encoding gene CHAT · Genetic evidence · score 0.94

Research activity

Emerging research

1 papers · latest 1978

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

Catalyzes the reversible synthesis of acetylcholine (ACh) from acetyl CoA and choline at cholinergic synapses

Domains and Gene Ontology detail (10)

Gene Ontology

  • Ccytoplasm
  • Ccytosol
  • Cneuron projection
  • Cnucleus
  • Csynapse
  • Fcholine O-acetyltransferase activity
  • Pacetylcholine biosynthetic process
  • Pneuromuscular synaptic transmission
  • Pneurotransmitter transport
  • Pphosphatidylcholine biosynthetic process

748 aa · 83 kDa · 3 isoforms

Biological roles

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

Synaptic signallingUniProt · GO
View supporting evidence

Synaptic signalling

  • ·Catalyzes the reversible synthesis of acetylcholine (ACh) from acetyl CoA and choline at…
  • ·synapse
  • ·neuromuscular synaptic transmission

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 CHAT

Gene-level evidence surfaced through the gene CHATthat 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.

Congenital myasthenic syndromes
0.96Well supported

Genetic evidence dominant · Open Targets 0.80

Gastroesophageal Reflux
0.70Moderately supported

Genetic evidence dominant · Open Targets 0.43

View evidence synthesis (2)
Congenital myasthenic syndromesWell supported
0.96
agreement 0.84–1.00
Genetic76%Animal model21%Literature3%Genetic literaturedup

Open Targets aggregate 0.80 · 3 independent evidence families · 1 not counted as duplicate

Gastroesophageal RefluxModerately supported
0.70
agreement 0.56–0.84
Genetic99%Literature2%

Open Targets aggregate 0.43 · 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
Congenital myasthenic syndromes0.80
Gastroesophageal Reflux0.43

Tractability

Small molecules — Emerging

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

Protein degraders — Emerging

Feasibility evidence (small molecule binder) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (4)
SM · Structure with LigandSM · Med-Quality PocketSM · Druggable FamilyPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

Research activity

1 papers · to 1978

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

Most cited

Correlation of cholinergic abnormalities with senile plaques and mental test scores in senile dementia.1,224 cites

Perry EK · British medical journal · 1978

Recent

Correlation of cholinergic abnormalities with senile plaques and mental test scores in senile dementia.

Perry EK · British medical journal · 1978

Europe PMC papers linked directly to this protein.