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JAK1 siRNA
- Development stage
- Preclinical
- Lead developer
- Commissariat à l’Énergie Atomique et aux Énergies Alternatives
- 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
- Oral, Rectal, Topical
01
Overview
JAK1 siRNA refers to small interfering RNA molecules designed to specifically target and silence the expression of Janus kinase 1 (JAK1) mRNA. By utilizing RNA interference (RNAi), JAK1 siRNA prevents the translation of JAK1 protein, thereby inhibiting downstream JAK/STAT signaling pathways. This approach is being investigated as a highly selective, local therapeutic alternative to systemic small-molecule JAK inhibitors (Jakinibs) for the treatment of inflammatory bowel diseases (IBD), such as Crohn's disease and ulcerative colitis. Researchers from institutions including the University of Grenoble Alpes, CEA, INSERM, and IDIBAPS have explored delivering JAK1 siRNA locally to the intestinal mucosa using lipid nanoparticle (LNP) technology to minimize systemic side effects.
- Other names
- JAK1-targeting siRNAJAK-1-targeting siRNAJAK 1-targeting siRNAJanus kinase 1 siRNAsiJAK1siJAK-1siJAK 1
02
Targets
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