MicroRNA 124-1 (miR-124-1)

MicroRNA 124-1 is a small, non-coding RNA gene (located at human chromosome 8p23.1) that produces miR-124, one of the most abundant brain-specific microRNAs[1][2][4][5]. The mature miR-124-1 is produced through a complex biochemical process involving transcription by RNA polymerase II and sequential cleavage by Drosha and Dicer enzymes, ultimately leading to its incorporation into the RNA-induced silencing complex (RISC)[1][2]. miR-124-1 acts as a critical post-transcriptional regulator of gene expression, with widespread functions in neuronal differentiation, brain development, cell cycle control, autophagy, immune cell differentiation, and inflammation[1][3][4][5]. It is well characterized as a tumor suppressor in multiple cancer types, where decreased expression is linked to enhanced tumor proliferation, invasion, migration, and angiogenesis[1][2][3]. There are three miR-124 variants (miR-124-1, -2, -3), but all give rise to the same mature sequence and share overlapping functional roles[1]. miR-124-1 is being investigated as both a diagnostic biomarker and a potential therapeutic agent—primarily as a mimic to restore its tumor-suppressive functions in cancer or as a modulator in neurodegenerative and immune diseases. However, clinical translation is limited by delivery and specificity challenges. miR-124-1 is not itself a conventional "therapeutic target" such as a receptor or enzyme, but rather a regulatory RNA, with indirect but significant therapeutic implications[1][2][3][4][5].

Other names

MIR124AMIR124A1MIRN124-1MIRN124A1mir-124-1hsa-mir-124-1hsa-mir-124a-1microRNA 124a-1

Regulation of target mRNA by translational inhibition or degradation via the RNA-induced silencing complex (RISC); Suppression of oncogene expression (e.g., CDK6, STAT3, EGFR, etc.); Modulation of signaling pathways (e.g., Wnt/β-catenin, JAK/STAT, PI3K/AKT, etc.)