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NPC2 siRNA
- Development stage
- Preclinical
- Lead developer
- CHA University
- 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
- Intrathecal
01
Overview
NPC2 siRNA is a small interfering RNA (siRNA) designed to silence the expression of the Niemann-Pick type C2 (NPC2) protein. NPC2 is a lysosomal protein involved in cholesterol transport, but recent research has identified it as a key driver of angiogenesis in the mesenchymal subtype of glioblastoma (GBM). In this context, NPC2 is highly expressed in tumor-associated endothelial cells, and its expression levels correlate with poor overall and disease-free survival. By targeting and degrading NPC2 mRNA, NPC2 siRNA inhibits endothelial cell proliferation, migration, and invasion, effectively suppressing tumor-associated angiogenesis. This approach is being explored as a potential therapeutic strategy to treat aggressive, highly vascularized glioblastomas by modulating the tumor microenvironment.
- Other names
- siRNA targeting NPC2NPC2-targeting siRNANPC-2-targeting siRNANPC 2-targeting siRNA
02
Targets
NPC2 (NPC intracellular cholesterol transporter 2 (NPC2) mRNA)
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