Target intelligence / Profile preview

Phospholipid-transporting ATPase ABCA1 (ABCA1)

Target
ABCA1
Molecular classification
ATP-binding cassette transporter, Transporter, ATPase
01

Overview

Phospholipid-transporting ATPase ABCA1 is a membrane-associated protein that plays a critical role in lipid homeostasis, particularly in the efflux of cholesterol and phospholipids from cells to apolipoproteins, which leads to the formation of high-density lipoprotein (HDL). This process is essential for reverse cholesterol transport, where excess cellular cholesterol is transported to the liver for excretion or redistribution. Mutations or deficiencies in ABCA1 cause severe reductions in HDL levels due to impaired efflux of cellular lipids, underlying rare genetic disorders like Tangier disease and familial HDL deficiency, both associated with increased risk of cardiovascular disease.

02

Mechanism of action

ATP-dependent transport of phospholipids and cholesterol

03

Biological functions

Reverse cholesterol transportLipid effluxNascent HDL biogenesisPhospholipid transportCholesterol transport
04

Disease associations

Tangier diseaseFamilial HDL deficiencyAtherosclerosisCardiovascular disease
05

Safety considerations

Potential for off-target effects due to broad expressionChallenges in achieving tissue-specific modulation
06

Biomarkers

HDL levels

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