Drug intelligence / Profile preview

SET 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
Intravenous, Intratumoral, Local, Ex Vivo Cellular Delivery
01

Overview

SET siRNA is a synthetic small interfering RNA designed to silence the expression of the SET gene at the mRNA level. **Small interfering RNA (siRNA)** molecules mediate gene silencing via the RNA interference (RNAi) pathway, which relies on the introduction of a double-stranded 21-23 nucleotide RNA complementary to a target mRNA[1][2][3]. Upon cellular entry, SET siRNA is incorporated into the RNA-induced silencing complex (RISC), where its guide (antisense) strand directs the complex to the complementary SET mRNA sequence; Argonaute-2, a component of RISC, then cleaves the SET mRNA, leading to its degradation and subsequent reduction of protein synthesis[3][4][5][7]. This mechanism makes SET siRNA a highly specific tool for post-transcriptional gene silencing, commonly used in research and potentially as a therapeutic for diseases where SET overexpression is linked to pathogenesis.

Brand names
SET siRNA
Other names
SET siRNA
02

Targets

TAFI (Carboxypeptidase B2)

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