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MicroRNA 124-3 is a non-coding RNA (miRNA) gene expressed in humans, located at chromosome 20q13.33, and one of three loci producing mature miR-124 isoforms[2][8]. Like other miRNAs, miR-124-3 regulates gene expression post-transcriptionally, affecting both mRNA stability and translation through imperfect base pairing, typically resulting in translational repression or degradation of target mRNAs[1][5]. It is highly expressed in neuronal tissues, where it contributes to regulation of neuronal differentiation and function[3][8]. miR-124-3 acts as a tumor suppressor in multiple cancers by targeting oncogenes and pathways associated with cell cycle, migration, and resistance to chemotherapy (e.g., STAT3, CDK4, BIRC3)[7][8]. Hypermethylation and reduced expression of MIR124-3 have been linked to cancers (e.g., ovarian, breast, NSCLC), poor outcomes, and psychiatric disorders[2][5][6]. While it is not a druggable receptor or enzyme in the classic sense, it is considered a therapeutic target in RNA-based and epigenetic therapies for oncology and neurological diseases[8].
Drugs or mimics restore or modulate miR-124-3 expression to inhibit tumor cell proliferation, migration, and invasion by targeting specific oncogenes and key signaling pathways (e.g., STAT3, CDK4, BIRC3, SphK1, Wnt/β-catenin pathway)[7][8].
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