MicroRNA 124-3p (miR-124-3p) (miR-124-3p)

MicroRNA 124-3p (miR-124-3p) is a brain-enriched, non-coding RNA that serves as a master regulator of neuronal differentiation and central nervous system (CNS) homeostasis [Sun et al., 2015, Journal of Biomedical Science]. It is primarily expressed in neurons and is crucial for the transition from neural stem cells to mature neurons by post-transcriptionally repressing non-neuronal transcripts [Yoo et al., 2011, Nature]. Beyond development, miR-124-3p modulates neuroinflammation by maintaining microglia in a quiescent state and regulates synaptic plasticity, which is vital for cognitive function [Ponomarev et al., 2011, Nature Medicine]. In clinical contexts, miR-124-3p is often downregulated in neurodegenerative conditions such as Alzheimer's disease and Parkinson's disease, as well as in various malignancies like glioblastoma, where it functions as a tumor suppressor [Qin et al., 2012, Cancer Letters]. It has also emerged as a target in inflammatory diseases, where its upregulation by small molecules like Obefazimod helps resolve inflammation in conditions such as ulcerative colitis [Vautrin et al., 2019, Scientific Reports]. Therapeutic strategies currently focus on the delivery of miR-124-3p mimics to restore its protective functions or the use of small molecules to enhance its endogenous expression [Angelucci et al., 2019, International Journal of Molecular Sciences]. However, clinical translation faces hurdles such as the need for targeted delivery systems to bypass the blood-brain barrier and the potential for off-target effects due to the large number of genes regulated by a single microRNA [Title et al., 2015, Drug Development Research].

Other names

hsa-miR-124-3pmiR-124MicroRNA 124miR-124a

Post-transcriptional gene silencing via mRNA degradation or translational inhibition by binding to the 3' untranslated region (UTR) of target messenger RNAs, or indirect upregulation via small molecules that enhance endogenous expression [Sun et al., 2015, Journal of Biomedical Science; Vautrin et al., 2019, Scientific Reports].