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UBTF shRNA is a short hairpin RNA-based therapeutic candidate designed to target and repress the expression of Upstream Binding Transcription Factor (UBTF). UBTF is a member of the HMG-box DNA-binding protein family and a key component of the RNA Polymerase I transcription machinery. In pancreatic cancer, UBTF is often upregulated to meet the high demand for protein synthesis by driving ribosome biogenesis. By knocking down UBTF, this agent reduces the occupancy of the RNA Polymerase I complex (including subunits like RPA194 and RPA135) on rDNA, thereby inhibiting the synthesis of pre-rRNA (5' ETS and 18S rRNA). Preclinical studies in pancreatic cancer cell lines and xenograft mouse models have demonstrated that UBTF repression leads to decreased cell proliferation, colony formation, migration, and invasion, as well as significant inhibition of tumor growth.
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