Protein / target

Phospholipid-transporting ATPase ABCA1

Encoded byABCA1O95477Homo sapiensSwiss-Prot

UniProt: O95477 Ensembl: ENSG00000165029 HGNC: HGNC:29 NCBI Gene: 19

Clinically validated

Therapeutic maturity

Approved medicines

Open Targets target-level

Small-molecule tractable

Druggability

Approved Drug

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.