CRISPR-activated patient fibroblasts for modeling of familial Alzheimer's disease
CRISPR-activated patient fibroblasts for modeling of familial Alzheimer's disease
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DOI：
10.1016/j.neures.2021.03.008
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发表时间：
2021-09-14
影响因子：
2.9
通讯作者：
Inoue, Keiichi
中科院分区：
文献类型：
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作者：
Inoue, Keiichi
分析合适的疾病模型系统对开展疾病研究非常重要。分析从患者组织中获取的细胞不仅有助于阐明其病理机制，开发新的治疗方法，而且有助于未来个性化药物的开发。然而，从患有神经退行性疾病的患者身上收集和培养神经细胞通常是困难的。皮肤成纤维细胞比神经元更容易收集，但当疾病相关基因没有充分表达时，可能不会表现出预期的病理。在这篇文章中，我描述了一个体外模型系统，它能够通过CRISPR转录激活内源性疾病相关基因来方便地分析患者成纤维细胞培养中的神经疾病机制。该系统引入了一个额外的平台来分析神经退行性疾病。(C)2021年爱思唯尔公司和日本神经科学学会。版权所有。
Analyzing an appropriate disease model system is important to conduct disease research. Analyzing cells obtained from patient tissues could not only help elucidate the pathological mechanisms and to develop novel therapy but also lead to personalized medicine in the future. However, it is generally difficult to collect and culture neuronal cells from patients suffering from neurodegenerative disorders. Skin fibroblasts are easier to collect than neurons but may not show the expected pathology when disease relevant genes are not sufficiently expressed. In this article, I describe an in vitro model system that enables the facile analysis of neurological disease-mechanisms in patient fibroblast cultures by CRISPR transcriptional activation of endogenous disease-relevant genes. This system introduces an additional platform to analyze neurodegenerative disorders. (C) 2021 Elsevier B.V. and Japan Neuroscience Society. All rights reserved.