Target intelligence / Profile preview

MicroRNA 501 (miR-501)

Target
miR-501
Molecular classification
MicroRNA, Non-coding RNA, miRNA family
01

Overview

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.

Other names
hsa-mir-501MIRN501mir-501MIR501microRNA-501-3p
02

Mechanism of action

Gene silencing through sequence-specific binding to mRNAs (via RISC complex), resulting in translational inhibition or mRNA degradation[5]

03

Biological functions

Post-transcriptional regulation of gene expressionMuscle regenerationRegulation of myosin heavy chain isoform transitionRegulation of synaptic structure and functionModulation of cell proliferation, migration, and invasion
04

Disease associations

Cancer (including hemangioma, hepatocellular carcinoma, other malignancies)Neurological/psychiatric disorders (schizophrenia-related synaptic dysfunction)Muscle disorders (muscle regeneration, muscle diseases such as polymyositis and dermatomyositis)
05

Safety considerations

Potential off-target effects if targeted therapeutically (as with other miRNAs)Broad impact on transcriptional/post-transcriptional regulation may lead to unintended cellular effects
06

Biomarkers

Serum biomarker for muscle regeneration and adult muscle stem cell activation[1]Potential tissue biomarker for synaptic or muscle diseases

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