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A **c-myc antisense oligonucleotide** is a synthetic strand of nucleic acid designed to specifically bind to the mRNA of the c-myc proto-oncogene, inhibiting its translation and thereby reducing c-myc protein expression. c-myc is a well-established oncogene whose overexpression drives tumor growth, proliferation, and survival in many cancers. Mechanistically, these antisense oligonucleotides (ASOs) work by hybridizing to target c-myc mRNA, leading to its degradation or blocking its translation, thus downregulating oncogenic Myc protein levels. Early clinical candidates included phosphorothioate oligodeoxynucleotides (e.g., INX-3280) and phosphorodiamidate morpholino oligomers (e.g., AVI-4126/Resten-RG), as well as more recent locked nucleic acid-based gapmers. Development has focused on cancer (lymphoma, solid tumors, leukemia, melanoma, hepatocellular carcinoma) and vascular restenosis, though no candidate has reached market approval. Delivery approaches have included intravenous, local/intracoronary, and microencapsulated sustained-release formulations[1][2][3][4][5].
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