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adenoviral Notch3 short hairpin RNA
- Development stage
- Preclinical
- Lead developer
- Wenzhou Medical University
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intratracheal, Intravenous
01
Overview
Adenoviral Notch3 short hairpin RNA is an experimental gene therapy and RNA interference (RNAi) agent designed to silence the expression of the Notch3 receptor. It utilizes an adenoviral vector to deliver a specific short hairpin RNA (shRNA) sequence that targets Notch3 messenger RNA (mRNA) for degradation. Notch3 is a transmembrane receptor critical for cell fate determination, and its overactivation is linked to the excessive proliferation and apoptosis resistance of pulmonary artery smooth muscle cells (PASMCs). By knocking down Notch3 expression, this agent inhibits pathological vascular remodeling and ventricular hypertrophy, particularly in models of persistent pulmonary hypertension of the newborn (PPHN) and pulmonary arterial hypertension (PAH). It is primarily used as a research tool to validate Notch3 as a therapeutic target and as a potential candidate for gene-based intervention in vascular diseases.
- Other names
- adenovirus-mediated Notch3 shRNANotch3 shRNA adenovirusNotch-3 shRNA adenovirusNotch 3 shRNA adenovirusadenoviral-delivered Notch3 short hairpin RNA
02
Targets
NOTCH (Neurogenic locus notch homolog protein 3)
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