Intranasal administration of exosomes derived from mesenchymal stem cells ameliorates autistic-like behaviors in mice

Mesenchymal stem cells (MSCs) have been regarded as candidate cell sources for therapeutic purposes of various neurological disorders, because of their self-renewal ability, plasticity in differentiation, neurotrophic characteristics, and immunomodulatory properties. More recently, MSC-derived exosomes are being examined for their role in MSC-based cellular therapy. Exosomes are endogenous nanovesicles that play critical roles in intercellular signaling by conveying functional genetic information and proteins between cells. Exosomes readily cross the blood-brain barrier and have promise as therapeutic delivery vehicles that have the potential to specifically deliver molecules to the central nervous system. Recently, we treated animal models of autism with exosomes isolated from MSC isolated from human bone marrow. We demonstrated increased male to male social interaction and reduced repetitive behaviors after intranasal administration. Moreover, the treatment led to increases of male to female ultrasonic vocalizations and significant improvement in maternal behaviors of pup retrieval. The marked beneficial effects of the exosomes in mice model is now translate to a novel, non-invasive therapeutic strategy to reduce the symptoms of autism.