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PTTG1 siRNA
- Development stage
- Preclinical
- Lead developer
- Hospital Clínic of Barcelona
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
PTTG1 siRNA is a small interfering RNA (siRNA) therapeutic designed to silence the expression of pituitary tumor-transforming gene 1 (PTTG1). PTTG1 is a multifunctional protein and securin that is typically upregulated in liver cirrhosis and hepatocellular carcinoma (HCC), where it acts as a key regulator of cell proliferation, extracellular matrix remodeling, and angiogenesis. By targeting and silencing PTTG1, this siRNA therapy downregulates key profibrogenic genes such as COL1alpha1 and alpha-SMA, thereby mitigating the progression of liver fibrosis and portal hypertension. It has been evaluated in preclinical models, including human precision-cut liver slices (hPCLS) and hepatic stellate cell lines, using lipid nanoparticle or poly(beta-amino ester) (pBAE) delivery systems.
- Other names
- PTTG1-targeted siRNAPTTG-1-targeted siRNAPTTG 1-targeted siRNAPttg1 siRNAPttg-1 siRNAPttg 1 siRNAsmall interfering RNA targeting PTTG1
02
Targets
PTTG1 (Pituitary tumor-transforming gene 1 protein)
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