OtherMolecular autism2020

Exosomes derived from mesenchymal stem cells improved core symptoms of genetically modified mouse model of autism Shank3B.

N Perets, O Oron, S Herman and 2 others

PMID 32807217

WHAT IT FOUND

In mice with a genetic mutation linked to autism, nasal drops of stem-cell exosomes improved social interaction and reduced repetitive behaviors.

Vocalization rates increased but did not reach statistical significance. This is preclinical animal data only.

Key findings

01Treated mice spent significantly more time in social interaction compared to saline-treated controls, with results similar to healthy wild-type mice.

02Repetitive behaviors like grooming and digging were significantly reduced in treated mice compared to controls.

03While no significant difference was found in the number of vocalizations, the percentage of mice that vocalized at all increased from 10% in controls to 40% in the treated group.

STILL TO COME

How it was doneWhat they found

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What it does not show

This is a preclinical study in mice, not a clinical trial in humans. The sample size was very small (10 mice in the treatment group). The mechanism of action is not fully understood; while social behavior improved, the specific biological pathways remain unclear. The study did not test whether the effects last long-term beyond the 3-week post-treatment window.

Declared interests

The authors declare no competing interests. The study was supported by non-US government grants.

The easy way to misread this

Do not interpret the improvement in social and repetitive behaviors in mice as evidence that exosome therapy works for human autism. This is a small animal study with a very different biological model, and no human patients were treated.

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The study

Participants
10 Shank3B KO male mice treated; 5 Shank3B KO and 5 WT mice for brain analysis
Certainty of evidence
Low

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    Cite

    N Perets, O Oron, S Herman, et al. Exosomes derived from mesenchymal stem cells improved core symptoms of genetically modified mouse model of autism Shank3B. Molecular autism. 2020.

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