Target intelligence / Profile preview

Apolipoprotein L1 (APOL1)

Target
APOL1
Molecular classification
Apolipoprotein, Lipid Transport Protein, Pore-forming protein
01

Overview

Apolipoprotein L1 (APOL1) is a secreted protein and a minor component of high-density lipoprotein (HDL) particles. It plays a role in lipid transport and innate immunity, particularly against Trypanosoma brucei parasites. Genetic variants (G1 and G2) provide protection against African trypanosomiasis but significantly increase the risk of developing non-diabetic chronic kidney disease, especially focal segmental glomerulosclerosis (FSGS) and hypertensive nephrosclerosis, particularly among individuals of African ancestry. The pathogenic mechanisms may involve toxic gain-of-function effects, altered ion fluxes due to disrupted channel function, or dysfunctional HDL formation.

02

Mechanism of action

N/A

03

Biological functions

Lipid transportInnate immunityAutophagy regulationRegulation of intracellular vesicle traffickingCation channel activity
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Disease associations

Chronic Kidney DiseaseFocal Segmental Glomerulosclerosis (FSGS)Hypertensive NephrosclerosisAfrican Trypanosomiasis (resistance/susceptibility)
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Safety considerations

Increased risk of non-diabetic chronic kidney disease (CKD) in individuals with two APOL1 risk alleles (G1/G1, G1/G2, G2/G2)Potential for off-target effects related to autophagy or cell death pathways
06

Biomarkers

APOL1 risk alleles (G1, G2) for CKD susceptibility

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