Drug intelligence / Profile preview

shRNA against uPAR and cathepsin B

Development stage
Preclinical
Lead developer
University of Illinois College of Medicine
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral
01

Overview

shRNA against uPAR and cathepsin B is a dual-targeting RNA interference therapeutic designed to simultaneously silence the expression of the urokinase-type plasminogen activator receptor (uPAR) and the lysosomal protease cathepsin B. Developed by researchers at the University of Illinois College of Medicine, this construct (often referred to as pUC) is primarily investigated for the treatment of glioblastoma multiforme (GBM). By downregulating these two proteins, the therapy disrupts critical signaling pathways—including PKC, integrin β1, and FAK—that drive tumor cell invasion, migration, and resistance to radiation. Preclinical studies have demonstrated its efficacy in inhibiting the growth and spread of glioma-initiating cells (GICs) in both in vitro and in vivo mouse models.

Other names
shRNA-mediated knockdown of uPAR and cathepsin BuPAR and cathepsin B knockdown
02

Targets

PLAUR (Urokinase plasminogen activator receptor)CTSB (Cathepsin B)

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