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首页> 美国政府科技报告 >Testing Brain Overgrowth and Synaptic Models of Autism Using NPCs and Neurons from Patient-Derived iPS Cells.
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Testing Brain Overgrowth and Synaptic Models of Autism Using NPCs and Neurons from Patient-Derived iPS Cells.

机译：使用来自患者衍生的ips细胞的NpC和神经元测试自闭症的脑过度生长和突触模型。

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Autism and autism spectrum disorders (ASD) are complex neurodevelopmental diseases that affect about 1% of children in the United States. Such disorders are characterized by deficits in verbal communication, impaired social interaction, and limited and repetitive interests and behavior. Recent studies have led to two major hypotheses for autism pathogenesis: early brain overgrowth and synaptogenesis defects. The goal of this project is to produce human cellular models of non-syndromic ASD. We used cellular reprogramming to develop iPSCs from ASD patients (and non-autistic controls) for the production of patient-derived neural progenitors (NPCs) and neurons to study cellular phenotypes that directly test whether brain overgrowth and/or synaptogenesis mechanisms are found in ASD NPCs and neurons. Patient-derived NPCs and neurons from these ASD and control individuals will be used for the functional characterization of iPSCs-derived NPCs and neurons from ASD and control individuals for potential autism-specific defects in proliferation, neural development and synaptogenesis, and for gene expression studies. We have made significant progress on these aims in the first year.

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