Efficacy of stem cell-derived extracellular vesicles in animal models of autism spectrum disorder: a systematic review and meta-analysis
- 1Department of Pediatrics, The Third Affiliated Hospital, Sun Yat-sen University, Guangzhou, China
Abstract
Background: Autism spectrum disorder (ASD) is a complicated neurodevelopmental condition that currently lacks a targeted therapy. Although stem cell-derived extracellular vesicles (EVs) represent a potential therapeutic approach for neurological disorders, their efficacy in ASD remains unclear. The objective of this study was to investigate the efficacy of stem cell-derived EVs on autistic-like behaviors in animal models of ASD and to explore the underlying mechanisms.
Methods: Relevant studies published up to April 20, 2026 were searched across five databases. Data were pooled using Review Manager 5.4. Outcome measures included behavioral tests and inflammatory cytokine levels.
Results: Out of 1,165 initially retrieved articles, five animal studies met the inclusion criteria. Pooled analyses demonstrated that EV treatment improved autistic-like behaviors in the self-grooming test, three-chamber social interaction test, and marble burying test, and decreased levels of proinflammatory cytokines IL-6 and IL-1β (all P < 0.05). No notable enhancements were observed for the open field test, Morris Water Maze, TNF-α or IL-10 (all P > 0.05).
Conclusion: Stem cell-derived EV therapy may improve repetitive behaviors and social deficits in ASD animal models, with observed associations with reduced IL-6 and IL-1β levels. These preclinical findings suggest promising translational potential of EV-based therapy for ASD.
Systematic Review Registration: https://osf.io/c78xm, identifier 10.17605/OSF.IO/C78XM.
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BibTeX
@article{zheng2026efficacy,
title = {{Efficacy of stem cell-derived extracellular vesicles in animal models of autism spectrum disorder: a systematic review and meta-analysis}},
author = {Zhen Zheng and Manfen Ji and Yayong Liang and Sinian Pan},
journal = {Frontiers in Psychiatry},
year = {2026},
month = oct,
doi = {10.3389/fpsyt.2026.1956214},
url = {https://doi.org/10.3389/fpsyt.2026.1956214},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Efficacy of stem cell-derived extracellular vesicles in animal models of autism spectrum disorder: a systematic review and meta-analysis
AU - Zhen Zheng
AU - Manfen Ji
AU - Yayong Liang
AU - Sinian Pan
PY - 2026
DA - 2026/10/12
JO - Frontiers in Psychiatry
DO - 10.3389/fpsyt.2026.1956214
UR - https://doi.org/10.3389/fpsyt.2026.1956214
AB - Background: Autism spectrum disorder (ASD) is a complicated neurodevelopmental condition that currently lacks a targeted therapy. Although stem cell-derived extracellular vesicles (EVs) represent a potential therapeutic approach for neurological disorders, their efficacy in ASD remains unclear. The objective of this study was to investigate the efficacy of stem cell-derived EVs on autistic-like behaviors in animal models of ASD and to explore the underlying mechanisms. Methods: Relevant studies published up to April 20, 2026 were searched across five databases. Data were pooled using Review Manager 5.4. Outcome measures included behavioral tests and inflammatory cytokine levels. Results: Out of 1,165 initially retrieved articles, five animal studies met the inclusion criteria. Pooled analyses demonstrated that EV treatment improved autistic-like behaviors in the self-grooming test, three-chamber social interaction test, and marble burying test, and decreased levels of proinflammatory cytokines IL-6 and IL-1β (all P < 0.05). No notable enhancements were observed for the open field test, Morris Water Maze, TNF-α or IL-10 (all P > 0.05). Conclusion: Stem cell-derived EV therapy may improve repetitive behaviors and social deficits in ASD animal models, with observed associations with reduced IL-6 and IL-1β levels. These preclinical findings suggest promising translational potential of EV-based therapy for ASD. Systematic Review Registration: https://osf.io/c78xm, identifier 10.17605/OSF.IO/C78XM.
SN - 1664-0640
ER -