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miR-1291 is a bioengineered, fully-humanized microRNA (miRNA) agent designed as a tumor suppressor therapy. It specifically targets the miR-1291-5p strand to modulate the transcriptome of cancer cells, particularly in pancreatic cancer. Its mechanism involves the downregulation of key metabolic proteins, including pyridoxine 5'-phosphate oxidase (PNPO), which is involved in vitamin B6 synthesis, as well as the glucose transporter GLUT1 (SLC2A1) and the amino acid transporter LAT1 (SLC7A5). By disrupting nutrient transport and metabolism, miR-1291 suppresses glycolysis, mitochondrial function, and increases oxidative stress in tumor cells. It has shown synergistic effects when combined with 5-fluorouracil (5-FU) in preclinical models.
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