Target intelligence / Profile preview

Low-density lipoprotein receptor (LDLR)

Target
LDLR
Molecular classification
Receptor, Cell-surface glycoprotein
01

Overview

The low-density lipoprotein receptor (LDLR) is a cell-surface glycoprotein that plays a central role in cholesterol homeostasis by mediating the endocytosis of cholesterol-rich low-density lipoproteins (LDL) from the bloodstream into cells. It is encoded by the *LDLR* gene located on chromosome 19 in humans. Mutations in *LDLR* disrupt its synthesis, processing, or function—leading to familial hypercholesterolemia: an inherited disorder characterized by elevated blood cholesterol levels and increased risk for premature cardiovascular disease due to impaired clearance of LDL-cholesterol from circulation. All currently approved lipid-lowering therapies aim—directly or indirectly—to increase hepatic expression or activity of functional LDLRs as a means to lower circulating cholesterol levels and reduce cardiovascular risk.

Other names
LDL receptor
02

Mechanism of action

Increase hepatic expression or activity of functional LDLRs

03

Biological functions

Receptor-mediated endocytosisCholesterol homeostasisLipoprotein uptake
04

Disease associations

Familial hypercholesterolemiaCardiovascular diseaseMyocardial infarctionCoronary artery disease
05

Safety considerations

Side effects related to lipid-lowering therapies
06

Interacting drugs

Lipid-lowering therapies (statins, PCSK9 inhibitors, etc.)
07

Biomarkers

Plasma LDL-cholesterol levels

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