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microRNA-216b (miR-216b) is a naturally occurring microRNA that acts as a tumor suppressor and is frequently downregulated in various cancers, including pancreatic ductal adenocarcinoma (PDAC) and ovarian cancer. As a therapeutic candidate, synthetic miR-216b mimics (often modified with unlocked nucleic acids or delivered via viral vectors) are being investigated for their ability to silence oncogenic drivers. In PDAC, miR-216b targets the KRAS oncogene, particularly the G12D mutant, to inhibit cell proliferation. In ovarian cancer, it has been shown to target PARP1, potentially modulating cisplatin resistance. More recently, artificial miR-216b sequences delivered via adeno-associated virus (AAV) vectors have been explored by companies like Uni-Pioneers BioMed and AskBio for gene silencing in trinucleotide repeat expansion disorders such as Huntington's disease and Myotonic Dystrophy Type 1, where they target the junction-to-junction regions of expanded transcripts.
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