Autism Spectrum Disorder and Exosome Therapy

autism spectrum disorder

Autism spectrum disorder is a complex and difficult issue to address for individuals suffering from this disease and those that must support the individual afflicted. While science struggles to find a definitive solution, technology continues to improve. One of these improvements is the development of exosome therapies which may pave the way for many novel disease treatments including Autism, cancer, and neurodegenerative diseases. This treatment modality is paving the way for a large number of individuals being helped.

Autism spectrum disorder cases are on the rise with 2014 estimations of a 16.8% prevalence for children 8 years of age. When compared to data collected for the year 2000 the prevalence is up 10.1% using the same age group. This dramatic rise should grab the attention of parents, legislatures, and healthcare professionals to work together to find both preventative measures and treatments for this costly and harmful issue many children are facing in their lives.1 Studies using BTBR mice (mice that have been specifically bred to have the three core symptoms of Autism which include the severe impairment of social interactions including

communication skills, the increase of repetitive behaviors, and presentation of cognitive inflexibility.) 2

Exosomes are tiny particles invisible to the human eye. These particles contain genetic information (e.g. miRNA),3 cell signaling particles, and other constituents that allow an individual to function normally. For certain individuals that are in a state of dysfunction, the artificial addition of healthy exosomes could be used to help alleviate symptoms stemming from their dysfunction. Numerous subtypes of exosomes have been identified with the mesenchymal stem cell exosomes (MSC-exo) subtype being highlighted in regards to Autism treatment.2

BTBR mice based models have been used to measure the efficacy of MSC-exo treatment. The exosomes are given through intranasal delivery which appears to be a highly acceptable and non-invasive delivery method. Upon completing the exosome treatment these mice are put into social situations where their interactions are measured. Perets et. al collected data showing improved social function including increased male to male interaction, reduced repetitive behaviors, increased male to female vocalization (related to breeding), and increased pup retrieval of BTBR female mice. The exact mechanism of this treatment is currently unknown due to the complex nature of exosome interactions in vivo.

autism spectrum disorder

Exosomes have a bright and promising future in regards to various treatment modalities including Autism spectrum disorder. With the MSC-exo subtype being at the forefront for Autism specifically. Further evidence is needed to ensure the safe application in human subjects. For now, there appears to be a good trajectory for individuals suffering from Autism and exosome therapy.

1. Report MW. Prevalence of Autism Spectrum Disorder Among Children Aged 8 Years — Autism and Developmental Disabilities Monitoring Network , 11 Sites , United States , 2014. 2018;67(6).

2. Perets N, Hertz S, London M, Offen D. Intranasal administration of exosomes derived from mesenchymal stem cells ameliorates autistic-like behaviors of BTBR mice. 2018:1-12.

3. Nisim Perets, Oshra Betzer, Ronit Shapira, Shmuel Brenstein, Ariel Angel, Tamar Sadan, Uri Ashery, Rachela Popovtzer  and DO. Golden Exosomes Selectively Target Brain Pathologies in Neurodegenerative Diseases and Neurodevelopmental Disorders. Nano Lett. 2019;19(6):3422-3431.


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