microRNA-124-enriched mesenchymal stem cell-derived exosomes

microRNA-124-enriched mesenchymal stem cell-derived exosomes (miR-124-MSC-Exos) are an engineered exosome-based therapy designed to deliver neuroprotective and anti-inflammatory microRNA cargo directly to the central nervous system. These exosomes are typically harvested from mesenchymal stem cells (MSCs) that have been genetically modified or transfected to overexpress miR-124. As natural extracellular vesicles, they possess the ability to cross the blood-brain barrier and protect their genetic cargo from enzymatic degradation. Once internalized by target cells—such as neurons, microglia, or neural stem cells—miR-124 acts via RNA interference to downregulate target mRNAs like SOX9, STAT3, and Jagged1. This modulation promotes an anti-inflammatory (M2) microglial phenotype, inhibits neuronal apoptosis, and stimulates neurogenesis. The therapy is primarily being investigated for the treatment of ischemic stroke, spinal cord injury, and various neurodegenerative conditions where neuroinflammation and neuronal loss are key drivers of pathology.

Other names

microRNA-124-enriched MSC-derived exosomesmicroRNA124-enriched MSC-derived exosomesmicroRNA 124-enriched MSC-derived exosomesmiR-124 loaded mesenchymal stem cell exosomesmiR124 loaded mesenchymal stem cell exosomesmiR 124 loaded mesenchymal stem cell exosomesmiR-124-overexpressing MSC-derived exosomesmiR124-overexpressing MSC-derived exosomesmiR 124-overexpressing MSC-derived exosomes