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Gemcitabine-modified microRNA-15a (Gem-miR-15a) is a novel nucleic acid-based therapeutic developed by researchers at Stony Brook University and Curamir Therapeutics. It is an engineered microRNA (miRNA) mimic where the endogenous cytidine bases in the guide strand of miR-15a are replaced with the nucleoside analog gemcitabine. This chemical modification significantly enhances the stability and potency of the miRNA. Gem-miR-15a functions as a multi-targeted agent, downregulating several key oncogenic proteins including WEE1, CHK1, BMI1, and YAP1. In preclinical studies for pancreatic ductal adenocarcinoma (PDAC) and colorectal cancer (CRC), the drug has demonstrated the ability to induce S-phase cell cycle arrest and apoptosis, showing high cytotoxicity in both parental and chemoresistant cell lines and effectively inhibiting tumor growth in vivo without significant toxicity.
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