Target intelligence / Profile preview

ATP-binding cassette sub-family G member 4 (ABCG4)

Target
ABCG4
Molecular classification
Transporter, ATP-binding cassette (ABC) transporter, White subfamily of ABC transporters
01

Overview

ATP-binding cassette sub-family G member 4 (ABCG4) is a member of the ATP-binding cassette transporter superfamily, specifically the White subfamily. It functions as an ATP-dependent transmembrane transporter involved primarily in the efflux of sterols (particularly cholesterol and desmosterol) and amyloid-β peptide. ABCG4 is highly expressed in the brain and liver, plays a role in cellular cholesterol homeostasis, and likely contributes to the regulation of amyloid-β levels at the blood-brain barrier. The gene is associated with certain cancers and lipid metabolism disorders and is under investigation as a potential target in neurodegenerative diseases like Alzheimer’s due to its role in amyloid-β clearance.

Other names
ABCG4WHITE2ATP binding cassette subfamily G member 4ATP-binding cassette, sub-family G, member 4Putative ABC transporter, sub-family G, member 4
02

Mechanism of action

Inhibition of transporter activity (e.g., probucol and L-thyroxine bind and block export of sterols and Aβ). Efflux modulation: promotes export of substrates (sterols, Aβ) from cells, including across the blood-brain barrier; inhibition leads to increased substrate retention.

03

Biological functions

Cholesterol homeostasisEfflux (export) of sterols, including cholesterol and desmosterolEfflux of amyloid-β peptide (Aβ)May form homodimers or heterodimers (e.g., with ABCG1) to function
04

Disease associations

Neurodegenerative disease (e.g., Alzheimer’s disease, due to involvement in amyloid-β efflux)Prostate cancerCongenital bile acid synthesis defectOther potential roles in lipid metabolism disorders
05

Safety considerations

Potential disruption of cholesterol and lipid homeostasis if targeted or inhibitedPossible impact on neurodegenerative disease processes due to altered amyloid-β transport
06

Interacting drugs

Probucol

2 more in the full profile.

07

Biomarkers

Amyloid-β peptide efflux (efflux capacity may be a functional biomarker for research)No standard clinical biomarkers specific for ABCG4 are widely established at this time

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