Drug intelligence / Profile preview

CBS 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
Parenteral, Intravenous, Intratumoral
01

Overview

CBS siRNA is a synthetic small interfering RNA specifically designed to silence the expression of the cystathionine β-synthase (CBS) gene through the RNA interference (RNAi) pathway. The mechanism involves the double-stranded siRNA being incorporated into the RNA-induced silencing complex (RISC), where the guide (antisense) strand directs the complex to complementary CBS mRNA molecules in the cytoplasm. The Argonaute-2 (AGO2) protein in RISC cleaves the target mRNA, leading to its degradation and inhibition of CBS protein synthesis[1][3][4]. CBS siRNA is being explored as a research tool and a therapeutic candidate for diseases associated with CBS overexpression or dysregulation, such as certain metabolic disorders and cancers. Information on a clinically approved or late-stage development CBS siRNA is not available; it is most commonly referenced in preclinical and basic research contexts.

02

Targets

CBS (Cystathionine beta-synthase)

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