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nucleostemin shRNA
- Development stage
- Preclinical
- Lead developer
- Universidad Autónoma de Madrid
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intratumoral
01
Overview
Nucleostemin shRNA is a gene-silencing therapeutic candidate designed to target and knockdown the expression of nucleostemin (also known as GNL3), a GTP-binding protein primarily localized in the nucleolus. Nucleostemin is highly expressed in stem cells and various cancer cells, including glioblastoma-derived cancer stem cells (GBM-CSCs), where it plays a critical role in maintaining self-renewal, proliferation, and survival. By utilizing short hairpin RNA (shRNA) delivered via a viral vector (such as lentivirus), this approach aims to trigger the RNA interference (RNAi) pathway to degrade nucleostemin mRNA. Preclinical studies have demonstrated that nucleostemin knockdown leads to the inhibition of neurosphere formation, induction of apoptosis, and suppression of tumor growth in xenograft models, suggesting its potential as a targeted therapy for glioblastoma and other aggressive malignancies.
- Other names
- GNL3 shRNAGNL-3 shRNAGNL 3 shRNAanti-nucleostemin shRNA
02
Targets
Innate immune RNA sensorsGNL3 (Guanine nucleotide-binding protein-like 3)
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