Cell Therapies for Autism Spectrum Disorder Based on New Pathophysiology: A Review
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Autism spectrum disorder (ASD) is a complex neurodevelopmental disorder characterized by difficulties in social communication, repetitive behaviors, and restricted interests, with onset early in life. The prevalence of ASD has increased worldwide in the last two decades. However, there is currently no effective therapy for ASD. Therefore, it is important to develop new strategies for ASD treatment. Evidence for the relationship between ASD and neuroinflammation, ASD and microglia, and ASD and glucose metabolism has increased rapidly in recent decades. We reviewed 10 clinical studies on cell therapies for individuals with ASD. Almost all studies showed good outcomes and no remarkable adverse events. Over the past decades, the neurophysiological characteristics of ASD have been shown to be impaired communication, cognition, perception, motor skills, executive function, theory of mind, and control of emotions. Recent studies have focused on the roles of immune pathology, such as neuroinflammation, microglia, cytokines, and oxidative stress, in ASD. We also focused on glucose metabolism in patients with ASD. The significance of gap junction–mediated cell–cell interactions between the cerebral endothelium and transplanted cells was observed in both bone marrow mononuclear cells and mesenchymal stromal cells transplantation. Owing to the insufficient number of samples, cell therapies, such as umbilical cord blood cells, bone marrow mononuclear cells, and mesenchymal stromal cells, will be a major challenge for ASD. As a result of these findings, a new paradigm for cell therapy for autism may emerge.
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“Microglial priming and sustained cytokine exposure during development may create a neuroimmune environment conducive to ASD [60,135–139].”
Neuron-Specific Enolase (NSE) as a Biomarker for Autistic Spectrum Disease (ASD)
Stancioiu, F., Bogdan, R. D., & Dumitrescu, R. (2023) · Life · doi:10.3390/life13081736 · Cited by 14 · see citing works
Neural precursor cells rescue symptoms of Rett syndrome by activation of the Interferon γ pathway
Frasca, A., Miramondi, F., Butti, E., Indrigo, M., Balbontin Arenas, M., Postogna, F. M., Piffer, A., Bedogni, F., Pizzamiglio, L., Cambria, C., et al. (2024) · EMBO Molecular Medicine · doi:10.1038/s44321-024-00144-9 · Cited by 9 · see citing works
“Other stem cells, such as mesenchymal cells, were widely used with therapeutic purposes in the fi eld of neurodevelopmental and neurocognitive disorders, and a clinical trial on a small group of RTT patients treated with NPCs was reported on a Chinese journal (Liu et al, 2013; Nabetani et al, 2023).”
Kabataş, S., Çivelek, E., Savrunlu, E. C., Karaaslan, U., Yıldız, Ö., & Karaöz, E. (2024) · World Journal of Methodology · doi:10.5662/wjm.v15.i1.95857 · Cited by 5 · see citing works
Raja, S. A., Batool, A., Sana, M., et al. (2024) · Cureus · doi:10.7759/cureus.70913 · Cited by 3 · see citing works
“The neurophysiological functions of GDNF include enhancing neurogenesis, synaptic plasticity, and general brain function, all of which are essential for preserving behavioral and cognitive health in people with ASD [7].”
BackgroundStancioiu, F., Dumitrescu, R. M., Ivanescu, B., & Ivanescu, B. (2024) · World Journal of Clinical Pediatrics · doi:10.5409/wjcp.v14.i1.96643 · Cited by 3 · see citing works
The endocannabinoid system in autism spectrum disorder
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Advancing Cell Transplantation Research with Integrity and Rigor
Lou, Y. (2024) · Cell Transplantation · doi:10.1177/09636897241309996 · Cited by 1 · see citing works
Why retracted articles can have an influential afterlife—and what to do about it
Snyder, J., & Turner, L. (2026) · Stem Cells Translational Medicine · doi:10.1093/stcltm/szag080
“Instead, this retracted article is included as a normal part of the scientific literature in these articles, contributing to metaanalyses and reviews of the current state of stem cell science 19,20 or aiding the interpretation of newly reported data.”
Review of Cell Therapy Approaches to Autism: A Review of Clinical Trial
Amiri, D., Karlsson, B., & Briziarelli, L. (2023) · Journal of Vaccines Immunology and Immunopathology · doi:10.29011/2575-789x.0001103
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- Authors
- Nabetani, M., Mukai, T., & Taguchi, A.
- Affiliations
- Yodogawa Christian Hospital (JP)
- University of Tokyo Hospital (JP)
- Foundation for Biomedical Research and Innovation (JP)
- Corresponding author
- Nabetani, Makoto
- Venue
- Cell Transplantation
- Publisher
- SAGE Publications
- Published
- 2023-01
- Volume
- 32
- Pages
- 9636897231163217-9636897231163217
- ISSN
- 0963-6897 (ISSN-L)
- 1555-3892
- PMC
- PMC10069005
- OpenAlex
- W4361987374
- Semantic Scholar
- 0c0f868c201f55fafbfbfdeb4a4bbe7cf18f22ca
- Cited by
- 13
- References
- 88
- Citations by year
- 2026: 1 · 2025: 3 · 2024: 6 · 2023: 3
- FWCI
- 0.49
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Cell Transplantation (publishedVersion, cc-by-nc)
- PubMed Central (publishedVersion, cc-by-nc)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- the Japan Society for the Promotion of Science (Grant Number 21K15618)
- Topics
- Life Sciences › Neuroscience › Cognitive Neuroscience › Autism Spectrum Disorder Research
- Health Sciences › Medicine › Genetics › Mesenchymal stem cell research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
- Keywords
- autism spectrum disorder, cell therapy, neuroinflammation, microglia, glucose metabolism, bone marrow mononuclear cell transplantation, mesenchymal stem cells, umbilical cord blood cells, gap junctions, brain endothelium, immunopathology, oxidative stress, cytokines, neurodevelopmental disorders
- MeSH terms
- Autism Spectrum Disorder, Neuroinflammatory Diseases, Cognition, Glucose, Humans, Cytokines
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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