Probing disrupted neurodevelopment in autism using human stem cell-derived neurons and organoids: An outlook into future diagnostics and drug development.
Probing disrupted neurodevelopment in autism using human stem cell-derived neurons and organoids: An outlook into future diagnostics and drug development.
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DOI：
10.1002/dvdy.100
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发表时间：
2020-01
期刊：
影响因子：
--
通讯作者：
Shcheglovitov A
中科院分区：
文献类型：
--
作者：
Yang G;Shcheglovitov A
自闭症谱系障碍（ASD）是一系列神经发育障碍，其特征是社会互动受损，重复或限制性行为以及言语问题。根据疾病控制和预防中心最近的一份报告，美国每68名儿童中就有一名被诊断患有ASD。尽管近年来ASD相关的诊断和ASD相关遗传异常的知识有所改善，但我们对ASD中细胞和分子通路中断的理解仍然非常有限。因此，没有特定的治疗或药物可用于ASD患者。在这篇综述中，我们描述了ASD患者大脑中可能受到影响的神经发育过程，并讨论了患者特异性干细胞衍生的神经元和类器官如何用于在细胞和分子水平上研究这些过程。最后，我们提出了一个发现管道，用于在未来识别细胞和分子缺陷，并为特发性ASD患者开发新的个性化治疗。
Autism spectrum disorders (ASDs) represent a spectrum of neurodevelopmental disorders characterized by impaired social interaction, repetitive or restrictive behaviors, and problems with speech. According to a recent report by the Centers for Disease Control and Prevention, one in 68 children in the US is diagnosed with ASDs. Although ASD-related diagnostics and the knowledge of ASD-associated genetic abnormalities have improved in recent years, our understanding of the cellular and molecular pathways disrupted in ASD remains very limited. As a result, no specific therapies or medications are available for individuals with ASDs. In this review, we describe the neurodevelopmental processes that are likely affected in the brains of individuals with ASDs and discuss how patient-specific stem cell-derived neurons and organoids can be used for investigating these processes at the cellular and molecular levels. Finally, we propose a discovery pipeline to be used in the future for identifying the cellular and molecular deficits and developing novel personalized therapies for individuals with idiopathic ASDs.
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