Since their discovery, induced pluripotent stem cells (iPSCs) have paved a new path for regenerative medicine and the development of novel strategies for treating diseases. Conversely, iPSCs derived from patients with specific pathology offer fresh insights into understanding the intricate mechanisms of diseases and the development of personalized and more effective treatments. The research field is advancing rapidly, with multiple protocols being developed for the derivation, cultivation, and targeted differentiation of iPSCs. The approach of using patient-derived iPSCs enables the comparison of various cellular phenotypes between cell from diseased and healthy individuals. However, a significant challenge in modeling psychiatric disorders in vitro is to establish correlations between cellular and molecular findings and clinical symptoms. In this context, the utility and relevance of patient-derived iPSCs in autism spectrum disorders (ASD) research are explored. ASD pathogenesis involves disturbances in intercellular communication, imbalanced ratios of certain neuronal populations, and disrupted maturation/differentiation processes, which are closely tied to the three-dimensional (3D) organization within the brain. Thus, an overview of the cutting-edge 3D cell culture system known as brain organoids to assess its potential as a platform for studying brain development and related pathology is provided.

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Autism Spectrum Disorder Patients and Use of iPSC and Cerebral Organoids

  • Mirolyuba Simeonova Ilieva

摘要

Since their discovery, induced pluripotent stem cells (iPSCs) have paved a new path for regenerative medicine and the development of novel strategies for treating diseases. Conversely, iPSCs derived from patients with specific pathology offer fresh insights into understanding the intricate mechanisms of diseases and the development of personalized and more effective treatments. The research field is advancing rapidly, with multiple protocols being developed for the derivation, cultivation, and targeted differentiation of iPSCs. The approach of using patient-derived iPSCs enables the comparison of various cellular phenotypes between cell from diseased and healthy individuals. However, a significant challenge in modeling psychiatric disorders in vitro is to establish correlations between cellular and molecular findings and clinical symptoms. In this context, the utility and relevance of patient-derived iPSCs in autism spectrum disorders (ASD) research are explored. ASD pathogenesis involves disturbances in intercellular communication, imbalanced ratios of certain neuronal populations, and disrupted maturation/differentiation processes, which are closely tied to the three-dimensional (3D) organization within the brain. Thus, an overview of the cutting-edge 3D cell culture system known as brain organoids to assess its potential as a platform for studying brain development and related pathology is provided.