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Background <p>Autism spectrum disorder (ASD) is a complex neurodevelopmental condition affecting millions of people worldwide, with limited therapeutic options and significant heterogeneity in presentation and underlying pathophysiology. This review comprehensively examines the current state of induced pluripotent stem cell (iPSC) applications in ASD research, evaluates recent advances, identifies key limitations, and outlines future directions for clinical translation.</p> Methods <p>We conducted a narrative review of peer-reviewed literature examining iPSCs applications in ASD research, including disease modeling, drug discovery, and therapeutic development, with a particular focus on recent technological advances and clinical translation challenges.</p> Results <p>iPSC applications in ASD research have shown significant promise through personalized cellular models that have uncovered key mechanisms underlying synaptic defects, neuronal network changes, and neurotransmitter disruptions. Advanced three-dimensional organoid systems derived from patient-derived iPSCs offer enhanced physiological relevance for studying brain development and ASD pathogenesis. Nevertheless, critical obstacles remain, particularly safety risks, including tumor formation from incompletely differentiated cells, genetic alterations in cell lines, and marked inconsistency in laboratory protocols. Furthermore, the absence of standardized manufacturing guidelines and prohibitive treatment costs present substantial challenges to widespread clinical implementation and patient access.</p> Conclusions <p>Although iPSCs offer transformative potential for ASD research and therapy, significant obstacles must be overcome to achieve clinical success. Critical needs include enhanced safety measures, uniform manufacturing standards, improved cell viability, and robust regulatory oversight. Achieving clinical translation will depend on ongoing partnerships among scientists, healthcare providers, regulatory bodies, and autism communities to ensure that research breakthroughs result in safe, effective, and equitable treatments.</p>

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Induced pluripotent stem cells in autism research: a review of current advances and future directions

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年2月24日

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  • Armin Ariaei,
  • Arsh Haj Mohamad Ebrahim Ketabforoush,
  • Sanaz Bordbar,
  • Shirin Barati,
  • Abolfazl Lotfi,
  • Forough Azam Sayahpour,
  • Faezeh Faghihi,
  • Saeid Abediankenari

摘要

Background

Autism spectrum disorder (ASD) is a complex neurodevelopmental condition affecting millions of people worldwide, with limited therapeutic options and significant heterogeneity in presentation and underlying pathophysiology. This review comprehensively examines the current state of induced pluripotent stem cell (iPSC) applications in ASD research, evaluates recent advances, identifies key limitations, and outlines future directions for clinical translation.

Methods

We conducted a narrative review of peer-reviewed literature examining iPSCs applications in ASD research, including disease modeling, drug discovery, and therapeutic development, with a particular focus on recent technological advances and clinical translation challenges.

Results

iPSC applications in ASD research have shown significant promise through personalized cellular models that have uncovered key mechanisms underlying synaptic defects, neuronal network changes, and neurotransmitter disruptions. Advanced three-dimensional organoid systems derived from patient-derived iPSCs offer enhanced physiological relevance for studying brain development and ASD pathogenesis. Nevertheless, critical obstacles remain, particularly safety risks, including tumor formation from incompletely differentiated cells, genetic alterations in cell lines, and marked inconsistency in laboratory protocols. Furthermore, the absence of standardized manufacturing guidelines and prohibitive treatment costs present substantial challenges to widespread clinical implementation and patient access.

Conclusions

Although iPSCs offer transformative potential for ASD research and therapy, significant obstacles must be overcome to achieve clinical success. Critical needs include enhanced safety measures, uniform manufacturing standards, improved cell viability, and robust regulatory oversight. Achieving clinical translation will depend on ongoing partnerships among scientists, healthcare providers, regulatory bodies, and autism communities to ensure that research breakthroughs result in safe, effective, and equitable treatments.