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MicroRNA 501 (miR-501) is a short, non-coding RNA molecule of the microRNA (miRNA) family that regulates gene expression at the post-transcriptional level. It is expressed as part of a primary transcript, processed through Drosha and Dicer enzymes, and finally incorporated into the RNA-induced silencing complex (RISC), where it binds partially complementary sequences in target mRNAs to inhibit translation or promote degradation[5]. miR-501 is specifically induced during adult muscle regeneration, where it regulates the switch between embryonic and adult myosin heavy chain isoforms and may serve as a serum biomarker for muscle stem cell activation[1]. In cancer settings, it acts predominantly as an oncogenic miRNA, promoting proliferation, migration, and invasion by targeting tumor suppressor genes such as HOXD10[2]. In the central nervous system, miR-501 is enriched in specific brain regions, where it is critical for synaptic structure and glutamatergic transmission, and its deficiency leads to behavioral and synaptic abnormalities linked to psychiatric disease models[3]. No approved drugs are known to target miR-501 directly, but its role in disease makes it a candidate for therapeutic intervention or as a disease biomarker.
Gene silencing through sequence-specific binding to mRNAs (via RISC complex), resulting in translational inhibition or mRNA degradation[5]
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