Target intelligence / Profile preview

Messenger RNA (ANGPTL3) (ANGPTL3 mRNA)

Target
ANGPTL3 mRNA
Molecular classification
mRNA, Non-coding RNA, Gene transcript
01

Overview

ANGPTL3 mRNA is the messenger RNA transcript of the *ANGPTL3* gene, encoding Angiopoietin-like protein 3. It is expressed primarily in the liver. This mRNA serves as the template for synthesizing the ANGPTL3 protein, which plays a key role in lipid metabolism by inhibiting lipoprotein lipase (LPL) and endothelial lipase (EL). Consequently, ANGPTL3 mRNA is a therapeutic target for siRNA and antisense oligonucleotides aimed at reducing ANGPTL3 protein expression and lowering triglyceride and LDL-cholesterol levels to reduce the risk of atherosclerotic cardiovascular disease.

Other names
ANGPTL3 mRNAAngiopoietin-like protein 3 mRNA*ANGPTL3* transcript
02

Mechanism of action

siRNA and antisense oligonucleotides inhibit ANGPTL3 mRNA expression, leading to reduced ANGPTL3 protein levels and subsequent increase in LPL and EL activity, ultimately lowering triglycerides and LDL-C.

03

Biological functions

Template for protein synthesisRegulation of lipid metabolism (indirectly, via ANGPTL3 protein)Target for therapeutic intervention to modulate lipid levels
04

Disease associations

Cardiovascular diseaseDyslipidemiaAtherosclerosis
05

Safety considerations

Off-target effects of siRNA/ASOImmune response to siRNA/ASOLong-term effects of ANGPTL3 inhibition
06

Interacting drugs

siRNA targeting ANGPTL3

1 more in the full profile.

07

Biomarkers

ANGPTL3 mRNA levels (baseline and post-treatment)ANGPTL3 protein levels (baseline and post-treatment)Triglycerides (baseline and post-treatment)LDL-C (baseline and post-treatment)HDL-C (baseline and post-treatment)

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