Drug intelligence / Profile preview

small interfering RNA targeting NOS3

Development stage
Preclinical
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
Intracochlear, Middle Ear
01

Overview

Small interfering RNA targeting NOS3 is an experimental RNA interference (RNAi) therapeutic designed to specifically silence the expression of the Nitric Oxide Synthase 3 (NOS3) gene. NOS3, also known as endothelial nitric oxide synthase (eNOS), is a key enzyme in arginine metabolism that has been identified as a potential driver of malignant progression in hematological cancers such as diffuse large B-cell lymphoma (DLBCL). By utilizing the RNAi pathway, this agent facilitates the sequence-specific degradation of NOS3 mRNA, thereby reducing the levels of the NOS3 protein. Research indicates that silencing NOS3 disrupts the IGF2BP2-GLS/NOS3 metabolic axis, leading to inhibited cell proliferation, delayed cell cycle progression, and the induction of apoptosis in malignant cells. This mechanism suggests that targeting NOS3 via siRNA could serve as a novel therapeutic strategy for patients with DLBCL and other cancers where NOS3 is overexpressed.

Other names
NOS3 siRNANOS-3 siRNANOS 3 siRNAsi-NOS3si-NOS-3si-NOS 3eNOS siRNANitric oxide synthase 3 siRNA
02

Targets

NOS3 (eNOS)

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