MicroRNA 124 (miR-124) (miR-124)

MicroRNA 124 (miR-124) is a highly conserved, brain-enriched non-coding RNA that serves as a master regulator of neuronal identity and function. It exerts its biological effects by binding to the 3' untranslated regions (UTRs) of target messenger RNAs (mRNAs), such as REST and PTBP1, to suppress their expression and promote neurogenesis and synaptic plasticity (Sun et al., 2015, Frontiers in Cellular Neuroscience). Beyond the nervous system, miR-124 plays a pivotal role in modulating the immune system by inhibiting the production of pro-inflammatory cytokines, making it a significant target for inflammatory diseases (Vermeire et al., 2021, The Lancet Gastroenterology & Hepatology). In various pathologies, including glioblastoma and Alzheimer's disease, miR-124 levels are frequently diminished, contributing to disease progression. Therapeutic interventions focus on restoring miR-124 levels through synthetic mimics or small molecules like obefazimod, which enhances the production of endogenous miR-124 to achieve anti-inflammatory effects (Abivax, 2023). However, the broad regulatory network of miR-124 poses challenges regarding off-target effects and the need for precise tissue-specific delivery.

Other names

MIR124hsa-miR-124miR-124aMicroRNA 124ahsa-miR-124-3p

Sequence-specific binding to the 3' untranslated region (UTR) of target mRNAs leading to translational repression or mRNA degradation; pharmacological upregulation of miR-124 levels to suppress pro-inflammatory signaling pathways.