Target intelligence / Profile preview

Apolipoprotein C-III mRNA

Molecular classification
Other (mRNA transcript; not a protein, enzyme, or receptor)
01

Overview

Apolipoprotein C-III mRNA is the messenger RNA transcript encoded by the APOC3 gene, which is translated into Apolipoprotein C-III, a key regulator of plasma triglyceride metabolism. The mRNA serves as a pharmacological target for antisense oligonucleotide therapies aiming to lower Apolipoprotein C-III protein levels to reduce hypertriglyceridemia and cardiovascular risk. In clinical development, antisense drugs bind to APOC3 mRNA to induce its degradation, thereby decreasing protein production and lowering plasma triglyceride concentrations.

Other names
APOC3 mRNAApolipoprotein C3 mRNAApoC-III transcript
02

Mechanism of action

Antisense-mediated reduction of APOC3 mRNA, leading to decreased Apolipoprotein C-III protein production, reduction of plasma triglycerides, and lower cardiovascular risk.

03

Biological functions

Template for translation of Apolipoprotein C-III proteinIndirectly involved in triglyceride metabolism regulation (via its protein product)
04

Disease associations

Cardiovascular disease (indirect, via effect of protein product; mRNA suppression is therapeutic)Metabolic syndrome (indirect)Hypertriglyceridemia
05

Safety considerations

Thrombocytopenia (notable with antisense oligonucleotides like volanesorsen)Injection site reactionsPossible liver enzyme elevations
06

Interacting drugs

Volanesorsen

1 more in the full profile.

07

Biomarkers

Plasma levels of Apolipoprotein C-III proteinPlasma triglyceride concentration

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