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3p-siRNA targeting bcl-2
- Development stage
- Unknown
- Lead developer
- University of Bonn
- 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
01
Overview
3p-siRNA targeting bcl-2 is a bifunctional modified small interfering RNA (siRNA) designed to simultaneously silence the anti-apoptotic B-cell lymphoma 2 (Bcl-2) gene and activate the innate immune system. The molecule incorporates a 5'-triphosphate (3p) modification, which acts as a ligand for the cytosolic RNA sensor retinoic acid-inducible gene I (RIG-I). This dual mechanism of action involves the RNA interference (RNAi) pathway to degrade Bcl-2 mRNA, thereby sensitizing tumor cells to apoptosis, while the RIG-I activation triggers the production of type I interferons and pro-inflammatory cytokines. Primarily studied in preclinical models of melanoma, this compound represents a strategy to combine targeted gene silencing with immune stimulation to overcome tumor-mediated immunosuppression.
- Other names
- 5'-triphosphate-siRNA targeting Bcl-2bifunctional 3p-siRNA targeting Bcl-2bifunctional3p-siRNA targeting Bcl-2bifunctional-3p-siRNA targeting Bcl-23p-siRNA-Bcl2
02
Targets
BCL-2 (BCL-2 family)
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