Exosomes present homing and therapeutic ability in both, genetic and idiopathic models of autism in mice.

Exosomes are small nanovesicles containing molecular information of proteins and RNAs. The capacity of the exosomes is mainly influenced by the cell type they are secreted from. It has been shown that exosomes derived from mesenchymal stem cells (MSC-Exo) are candidate sources for therapeutic purposes of various neurological disorders. Due to their ability to cross the blood-brain barrier via intranasal administration, they can be used as a noninvasive tool to treat neurological and psychiatric conditions. We have demonstrated that intranasal administration of MSC-Exo leads to significant amelioration of all autism core symptoms including a reduction in repetitive behavior, increased social interaction and significant improvement of ultrasonic vocalizations. This was done in both genetically modified mice SHANK3, and idiopathic model of BTBR mice. Furthermore, MSC-Exo seems to accumulate in the brain of pathologic mice, and evacuate from a healthy brain. Altogether we suggest that MSC-Exo can be used for a noninvasive and effective treatment for autism spectrum disorder, genetic and multifactorial.