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The uPA RNAi plasmid is an experimental, research-stage gene therapy construct designed to silence the expression of urokinase-type plasminogen activator (uPA) and its receptor (uPAR) through plasmid-based RNA interference (RNAi). The construct typically consists of a plasmid vector, often driven by a CMV promoter, that expresses short hairpin RNA (shRNA) or hairpin RNA (hpRNA) sequences complementary to uPA and uPAR mRNA. By downregulating these targets, the therapy aims to disrupt the uPA/uPAR signaling system, which is critically involved in tumor cell invasion, extracellular matrix degradation, angiogenesis, and metastasis. Preclinical studies in models of pancreatic cancer, glioma, and prostate cancer have demonstrated that intratumoral or intraperitoneal administration of the plasmid can inhibit tumor growth and induce apoptosis. This research has been primarily spearheaded by investigators at the University of Illinois College of Medicine.
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