Stem Cell Therapy and Models for Autism Spectrum Disorder: Insights and Research

Abstract: Autism Spectrum Disorders (ASD) are complex neurodevelopmental conditions characterized by impaired social communication, repetitive behavior patterns, and atypical sensory perception. The Autism and Developmental Disabilities Monitoring Network reports that approximately 1 in 36 children are diagnosed with ASD, highlighting the increasing prevalence and the pressing need for innovative treatment approaches. Medications commonly used in ASD primarily aim to manage associated symptoms, as there are currently no FDA-approved medications specifically for treating ASD core symptoms. Stem cells have demonstrated significant potential in cell-based therapies for ASD and have been utilized in in vitro models to investigate the pathogenesis of the condition. This review focuses on the recent advancements in stem cell-based transplantation in animal models of ASD, aiming to explore the improvement of ASD symptoms and the underlying mechanisms involved. It also discussed the application of stem cell-based transplantation in pediatric and adolescent populations with ASD to evaluate treatment efficacy and potential preventive strategies. Furthermore, recent efforts are addressed in developing stem cell-based models for both syndromic and non-syndromic forms of ASD, emphasizing studies that utilize cerebral organoids for modeling ASD, which facilitate the exploration of disease mechanisms within a tissue-like environment.