Target intelligence / Profile preview

ATP-binding cassette sub-family A member 2 (ABCA2)

Target
ABCA2
Molecular classification
Transporter, ATP-binding cassette transporter, ABC transporter
01

Overview

ATP-binding cassette sub-family A member 2 (ABCA2) is a membrane-bound transporter protein from the ABC superfamily, most highly expressed in neural tissues and macrophages. It is localized primarily to late endosomal/lysosomal membranes where it participates in the intracellular transport and homeostasis of cholesterol and sphingolipids—molecules critical for proper myelin formation and compaction in the nervous system. Expression of ABCA2 is associated with resistance to cancer drugs (especially those with steroid backbones) in several tumor models, and its genetic variants and regulation are implicated in pathologies such as Alzheimer’s disease, atherosclerosis, and cardiovascular disease. ABCA2 uses ATP hydrolysis to actively transport small hydrophobic molecules, contributing to homeostasis and potentially disease processes through effects on lipid metabolism and drug disposition.

Other names
ABC2KIAA1062ATP-binding cassette transporter 2ATP-binding cassette, sub-family A (ABC1), member 2ATP-binding cassette subfamily A member 2
02

Mechanism of action

Drug efflux by ATP-dependent active transport; Sequestration of cholesterol in late endosomal/lysosomal compartments; Modulation of cellular lipid environment affecting drug retention and resistance

03

Biological functions

Intracellular lipid transportCholesterol homeostasisSphingolipid metabolismMyelin formation and compactionMacrophage lipid metabolismDrug resistance mediation
04

Disease associations

Cancer (drug resistance)Neurodegenerative disease (Alzheimer’s disease)Cardiovascular disease (cholestrol-related)Atherosclerosis
05

Safety considerations

Overexpression may confer resistance to multiple chemotherapeutic agentsPotential adverse effects on neuronal lipid homeostasis (from knock-out studies: abnormal myelination, neurological symptoms)Possible involvement in progression/worsening of neurodegenerative and cardiovascular diseases due to lipid imbalances
06

Interacting drugs

Estramustine

3 more in the full profile.

07

Biomarkers

SNP in exon 14 linked to early-onset Alzheimer’s diseaseABCA2 expression in cancer cells correlates with drug resistance phenotype

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