Concise Review: Progress and Challenges in Using Human Stem Cells for Biological and Therapeutics Discovery: Neuropsychiatric Disorders.
Concise Review: Progress and Challenges in Using Human Stem Cells for Biological and Therapeutics Discovery: Neuropsychiatric Disorders.
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DOI：
10.1002/stem.2295
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发表时间：
2016-03
期刊：
影响因子：
--
通讯作者：
Panchision DM
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文献类型：
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作者：
Panchision DM
面对使用人类胚胎干细胞和诱导多能干细胞（hESC，hiPSC）研究复杂的神经回路障碍，如精神分裂症（SCZ），情绪和焦虑障碍以及自闭症谱系障碍（ASD）的艰巨挑战，2012年国家精神卫生研究所研讨会提出了一系列建议，以推进基础研究并使行业参与神经精神疾病的细胞研究。本文综述了满足这些建议的进展，包括开发新的工具，在概括相关的细胞和组织类型，这些疾病的遗传基础，允许整合风险相关的基因调控网络与细胞/电路表型的见解的进步，和有前途的发现患者控制的差异，使用基于细胞的检测。然而，许多挑战仍在解决，需要进一步的技术发展，解决疾病异质性的方法和不同学科研究人员的合作结构。此外，由于迄今为止获得的数据是在小样本量上，因此需要在更大的样本集中进行复制。一些成功的案例指出了一条将发现科学转化为治疗学开发的检测方法的前进道路。
In facing the daunting challenge of using human embryonic and induced pluripotent stem cells (hESCs, hiPSCs) to study complex neural circuit disorders such as schizophrenia (SCZ), mood and anxiety disorders and autism spectrum disorders (ASDs), a 2012 National Institute of Mental Health workshop produced a set of recommendations to advance basic research and engage industry in cell-based studies of neuropsychiatric disorders. This review describes progress in meeting these recommendations, including the development of novel tools, strides in recapitulating relevant cell and tissue types, insights into the genetic basis of these disorders that permit integration of risk-associated gene regulatory networks with cell/circuit phenotypes, and promising findings of patient-control differences using cell-based assays. However, numerous challenges are still being addressed, requiring further technological development, approaches to resolve disease heterogeneity and collaborative structures for investigators of different disciplines. Additionally, since data obtained so far is on small sample sizes, replication in larger sample sets is needed. A number of individual success stories point to a path forward in developing assays to translate discovery science to therapeutics development.