Drug intelligence / Profile preview

serping1 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
Subcutaneous, Topical, Intratumoral, Intravenous
01

Overview

serping1 siRNA is an experimental RNA interference (RNAi) therapeutic designed to silence the expression of the *SERPING1* gene, which encodes the Plasma protease C1 inhibitor (C1-INH) protein. While C1-INH is traditionally associated with the complement system and hereditary angioedema, research in Parkinson's disease (PD) models suggests that elevated Serping1 levels contribute to neuroinflammation and dopaminergic cell death in the substantia nigra. By knocking down Serping1, this siRNA reduces the accumulation of phosphorylated α-synuclein (pSer129) and decreases the expression of pro-inflammatory markers such as cyclooxygenase-2 (COX-2) and inducible nitric oxide synthase (iNOS). Preclinical studies in MPTP-induced chronic PD mouse models have demonstrated that treatment with serping1 siRNA can normalize motor ability and maintain tyrosine hydroxylase (TH) levels, suggesting a potential neuroprotective therapeutic approach for Parkinson's disease.

Other names
SERPING1 siRNASERPING-1 siRNASERPING 1 siRNA
02

Targets

SERPING1 (Serpin family G member 1)

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