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pCU shRNA
- Development stage
- Preclinical
- Lead developer
- University of Illinois College of Medicine Peoria
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous, Intravenous, Intramuscular, Inhalation, Intranasal, Intradermal, Intrathecal, Intratumoral, Intraperitoneal, Parenteral, Implant
01
Overview
**pCU shRNA** is an experimental plasmid-encoded bicistronic short hairpin RNA construct designed to simultaneously silence expression of urokinase-type plasminogen activator receptor and cathepsin B. It was developed in academic glioma research as a potential antineoplastic gene-silencing approach, including as an adjunct to ionizing radiation. In glioma cell lines, glioma-initiating cells, and mouse xenograft models, pCU-mediated knockdown reduced the target proteins, disrupted pro-invasive and survival signaling, and increased apoptosis and radiation-associated DNA damage. It has not been established as a clinically developed or marketed therapeutic product. ([ncbi.nlm.nih.gov](https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3367854/))
- Other names
- pCUpUCuPAR and cathepsin B shRNA
02
Targets
CTSB (Cathepsin B)PLAUR (Urokinase plasminogen activator receptor)
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