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Induced pluripotent stem cell-derived exosomes (iPSC-exosomes) are nano-sized extracellular vesicles harvested from the culture supernatant of induced pluripotent stem cells. These vesicles serve as a cell-free therapeutic modality, encapsulating a diverse cargo of proteins, lipids, and nucleic acids (such as miRNAs and mRNAs) that reflect the regenerative and immunomodulatory potential of their parent cells. Developed by The First Medical Center of the Chinese PLA General Hospital, this specific therapy is being investigated for the treatment of Autism Spectrum Disorder (ASD). The mechanism involves the intranasal delivery of these exosomes to the central nervous system, where they are thought to cross the blood-brain barrier and exert neuroprotective, anti-inflammatory, and pro-synaptogenic effects. By modulating the neuroimmune environment and promoting neuronal repair and synaptic plasticity, iPSC-exosomes aim to alleviate the core social and behavioral deficits associated with ASD.
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