Drug intelligence / Profile preview

nNOS siRNA

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
Intrathecal, Intratumoral, Intravenous, Intraperitoneal
01

Overview

nNOS siRNA is a small interfering RNA (siRNA) construct designed to silence the expression of the neuronal nitric oxide synthase (nNOS) gene, also known as NOS1. nNOS is a key enzyme in the central nervous system that catalyzes the production of nitric oxide (NO), a signaling molecule involved in neurotransmission, but also implicated in neurotoxicity and tumor progression when overproduced. By utilizing the RNA interference (RNAi) pathway, nNOS siRNA targets and facilitates the degradation of nNOS mRNA, thereby reducing protein levels and subsequent NO production. This experimental biologic is primarily used as a research tool in preclinical studies to investigate the role of nNOS in various pathologies, including neurodegenerative diseases like Parkinson's disease, central nervous system injuries such as traumatic brain injury and spinal cord injury, and certain cancers like melanoma and neuroblastoma. It is typically delivered using specialized systems such as liposomes, viral vectors (e.g., AAV), or nanoparticles to ensure cellular uptake and stability.

Other names
neuronal nitric oxide synthase siRNANOS1 siRNANOS-1 siRNANOS 1 siRNAnNOS-siRNA
02

Targets

NOS1 (nNOS)

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