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GCNT3 siRNA
- Development stage
- Preclinical
- Lead developer
- University of Oklahoma Health Sciences Center
- 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
- Intraperitoneal
01
Overview
GCNT3 siRNA is a small interfering RNA (siRNA) designed to silence the expression of the GCNT3 gene, which encodes the enzyme glucosaminyl (N-acetyl) transferase 3. This enzyme plays a critical role in the biosynthesis of core 2 branched O-glycans on mucins, such as MUC1 and MUC4, which are frequently overexpressed in pancreatic cancer and contribute to a dense extracellular matrix that limits the penetration of chemotherapeutic agents. By degrading GCNT3 mRNA, the siRNA reduces mucin production, thereby potentially increasing the intratumoral uptake and efficacy of drugs like gemcitabine. Preclinical studies in xenograft models have demonstrated that GCNT3 siRNA can significantly inhibit tumor growth and volume.
- Other names
- GCNT3 SiRNAGCNT-3 SiRNAGCNT 3 SiRNA
02
Targets
Glucosaminyl (N-acetyl) transferase 3, mucin type mRNAIFIH1 (MDA5)EIF2AK2 (Interferon-induced protein kinase R)DDX58 (Retinoic acid-inducible gene I protein)TLR3 (Toll-like receptor 3)TLR7/8 (Toll-like receptor 7 and Toll-like receptor 8)C2GnT (Core 2 beta-1,6-N-acetylglucosaminyltransferase 1)
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