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Antithrombin mRNA (null)

Target: null
Molecular classification: mRNA, Nucleic acid, Gene transcript

Overview

Antithrombin mRNA is the transcribed messenger RNA genome product of the SERPINC1 gene, encoding antithrombin, a serine protease inhibitor critical for inactivating key procoagulant enzymes (thrombin, factor Xa, factor IXa). In the context of hemophilia, the antithrombin mRNA in hepatocytes is targeted by small interfering RNA (siRNA) drugs, notably fitusiran, to inhibit its translation, decrease circulating antithrombin protein, and thereby enhance thrombin production and reduce bleeding risk in patients lacking normal clotting factors.

Other names: AT mRNASERPINC1 mRNA

Mechanism of action

RNA interference (RNAi) leading to degradation of antithrombin mRNA, reducing antithrombin protein synthesis and thereby increasing thrombin generation, restoring hemostasis in hemophilia

Biological functions

Template for antithrombin protein synthesisPost-transcriptional regulation point for coagulation control

Disease associations

Cardiovascular disease (specifically hemophilia and thrombosis via modulation of coagulation)

Safety considerations

Increased risk of thrombosis due to over-inhibition of antithrombinPotential imbalance in coagulation homeostasis, possible liver effects due to hepatocyte targeting

Interacting drugs

Fitusiran

Biomarkers

Plasma antithrombin protein levels (for efficacy/safety monitoring in patients receiving siRNA)

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