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pituitary tumor-transforming gene 1 siRNA + nitric oxide donor
- 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, Small Molecules
- Administration
- Intravenous
01
Overview
pituitary tumor-transforming gene 1 siRNA + nitric oxide donor is a preclinical combination therapy consisting of a small interfering RNA (siRNA) targeting pituitary tumor-transforming gene 1 (Pttg1) and a nitric oxide (NO) donor [FRI-414]. Delivered via liver-targeted, retinol-functionalized poly(β-amino ester) nanoparticles (ret-pBAE NPs), this dual-action therapeutic is designed to treat advanced liver fibrosis and portal hypertension [FRI-414]. The Pttg1 siRNA component silences Pttg1 expression to inhibit profibrogenic pathways and extracellular matrix accumulation, while the NO donor restores intrahepatic vascular tone and improves hemodynamics [FRI-414]. In preclinical models of advanced liver disease, the combination therapy has demonstrated robust antifibrotic, hepatoprotective, and hemodynamic-improving effects, significantly reducing portal pressure, hepatic collagen content, and steatosis [FRI-414].
- Other names
- Pttg1 siRNA + NO donorPttg1 siRNA + nitric oxide donorPttg1 siRNA + nitric oxide donor-loaded ret-pBAE NPspituitary tumor-transforming gene 1 siRNA + NO donor
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