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

MicroRNA-124-3p is a highly conserved, brain-enriched non-coding RNA that serves as a master regulator of neuronal identity and central nervous system (CNS) homeostasis (NIH, 2023; ResearchGate, 2023). It is the most abundant microRNA in the adult brain, where it promotes neuronal differentiation and neurogenesis while suppressing non-neuronal gene expression (NIH, 2025; MDPI, 2023). In pathological contexts, miR-124-3p acts as a potent tumor suppressor in various cancers and a physiological brake on inflammation by downregulating pro-inflammatory cytokines (MDPI, 2023; NIH, 2023). Therapeutic strategies targeting miR-124-3p include small molecules like obefazimod that upregulate its expression for treating inflammatory bowel diseases, as well as miR-124 mimics for neurodegenerative disorders and oncology (Abivax, 2023; MDPI, 2023). Despite its therapeutic potential, challenges such as precise delivery to the CNS and the risk of off-target effects due to its broad regulatory network remain significant hurdles in clinical development (NIH, 2023; Ovid, 2023).

Other names

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

Upregulation of miR-124 levels to suppress pro-inflammatory cytokines (e.g., TNF-alpha, IL-6, IL-17) and RNA interference-mediated silencing of target mRNAs such as STAT3, BACE1, and CDK5.