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Protein / target

Phospholipid-transporting ATPase ABCA1

Encoded byABCA1O95477Homo sapiensSwiss-Prot
UniProt: O95477Ensembl: ENSG00000165029HGNC: HGNC:29NCBI Gene: 19
Clinically validated
Therapeutic maturity
1
Approved medicines
Open Targets target-level
Small-molecule tractable
Druggability
Approved Drug
2
Research papers

Protein at a glance

Biological role

ATPase-coupled transmembrane transporter

Strongest disease association

Metabolic Syndrome

Via encoding gene ABCA1 · Genetic evidence · score 0.90

Therapeutic position

Established drug target

Small molecules

Research activity

Emerging research

2 papers · latest 2024

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 translocation of specific phospholipids from the cytoplasmic to the extracellular/lumenal leaflet of membrane coupled to the hydrolysis of ATP.

View complete UniProt function annotation

Catalyzes the translocation of specific phospholipids from the cytoplasmic to the extracellular/lumenal leaflet of membrane coupled to the hydrolysis of ATP (PubMed:24097981, PubMed:35974019). Thereby, participates in phospholipid transfer to apolipoproteins to form nascent high density lipoproteins/HDLs (PubMed:14754908). Transports preferentially phosphatidylcholine over phosphatidylserine (PubMed:24097981). May play a similar role in the efflux of intracellular cholesterol to apolipoproteins and the formation of nascent high density lipoproteins/HDLs (PubMed:10533863, PubMed:14754908, PubMed:24097981, PubMed:35974019). Translocates phospholipids from the outer face of the plasma membrane and forces it through its gateway and annulus into an elongated hydrophobic tunnel in its extracellular domain (PubMed:35974019)

Subcellular location

Cell membraneEndosome
Domains and Gene Ontology detail (67)

Domains & features

ABC transporter 1ABC transporter 2

Gene Ontology

  • Cbasolateral plasma membrane
  • Cendocytic vesicle
  • Cendoplasmic reticulum membrane
  • Cendosome
  • Cexternal side of plasma membrane
  • CGolgi apparatus
  • Cintracellular vesicle
  • Cmembrane raft
  • Cperinuclear region of cytoplasm
  • Cphagocytic vesicle
  • Cplasma membrane
  • FABC-type transporter activity

2261 aa · 254 kDa

Biological roles

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

Lipid & lipoprotein metabolismUniProt · GOG protein-coupled signallingGO
View supporting evidence

Lipid & lipoprotein metabolism

  • ·Catalyzes the translocation of specific phospholipids from the cytoplasmic to the extrac…
  • ·apolipoprotein A-I binding
  • ·apolipoprotein A-I receptor activity
  • ·apolipoprotein binding

G protein-coupled signalling

  • ·adenylate cyclase-activating G protein-coupled receptor signaling pathway
  • ·G protein-coupled receptor signaling pathway

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 ABCA1

Gene-level evidence surfaced through the gene ABCA1that 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.

Metabolic Syndrome
0.93Well supported

Genetic evidence dominant · Open Targets 0.57

Metabolic Diseases
0.91Well supported

Genetic evidence dominant · Open Targets 0.55

Glaucoma, Open-Angle
0.90Well supported

Genetic evidence dominant · Open Targets 0.55

Glaucoma
0.89Well supported

Genetic evidence dominant · Open Targets 0.55

Coronary Artery Disease
0.87Well supported

Genetic evidence dominant · Open Targets 0.53

View evidence synthesis (5)
Metabolic SyndromeWell supported
0.93
agreement 0.81–1.00
Genetic78%Animal model14%Literature8%

Open Targets aggregate 0.57 · 3 independent evidence families

Metabolic DiseasesWell supported
0.91
agreement 0.77–1.00
Genetic97%Literature3%

Open Targets aggregate 0.55 · 2 independent evidence families

Glaucoma, Open-AngleWell supported
0.90
agreement 0.78–1.00
Genetic77%Animal model16%Literature7%

Open Targets aggregate 0.55 · 3 independent evidence families

GlaucomaWell supported
0.89
agreement 0.75–1.00
Genetic92%Literature8%

Open Targets aggregate 0.55 · 2 independent evidence families

Coronary Artery DiseaseWell supported
0.87
agreement 0.78–0.97
Genetic49%Clinical34%Literature10%Animal model7%

Open Targets aggregate 0.53 · 4 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
Metabolic Syndrome0.57
Metabolic Diseases0.55
Glaucoma, Open-Angle0.55
Glaucoma0.55
Coronary Artery Disease0.53
Alcohol drinking0.51

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)
PROBUCOLApproval

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 — Strong

Approved Drug and Structure with Ligand support this modality.

Antibodies — Emerging

Feasibility evidence (uniprot loc high conf and go cc high conf) — 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 (8)
SM · Approved DrugSM · Structure with LigandSM · Druggable FamilyAB · UniProt loc high confAB · GO CC high confAB · UniProt SigP or TMHMMAB · Human Protein Atlas locPR · Half-life Data

Raw Open Targets tractability assessment buckets, by modality.

Research activity

2 papers · to 2024

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

Most cited

Genomic and drug target evaluation of 90 cardiovascular proteins in 30,931 individuals.569 cites

Folkersen L · Nature metabolism · 2020

Macrophage-mediated myelin recycling fuels brain cancer malignancy.137 cites

Kloosterman DJ · Cell · 2024

Recent

Macrophage-mediated myelin recycling fuels brain cancer malignancy.

Kloosterman DJ · Cell · 2024

Genomic and drug target evaluation of 90 cardiovascular proteins in 30,931 individuals.

Folkersen L · Nature metabolism · 2020

Europe PMC papers linked directly to this protein.