Towards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics.
Towards elucidating disease-relevant states of neurons and glia by CRISPR-based functional genomics.
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DOI：
10.1186/s13073-022-01134-7
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发表时间：
2022-11-18
期刊：
影响因子：
12.3
通讯作者：
中科院分区：
文献类型：
--
作者：
在过去的十年中，通过新技术的应用，我们对神经系统疾病的理解得到了极大的增强。全基因组关联研究强调神经胶质细胞是疾病的重要参与者。单细胞分析技术正在以前所未有的分子分辨率描述神经元和神经胶质细胞的疾病状态。然而，我们对驱动疾病相关细胞状态的机制以及这些状态如何导致疾病的理解仍然存在重大差距。我们理解中的这些差距可以通过基于 CRISPR 的功能基因组学来弥补，这是一种系统地询问基因功能的强大方法。在这篇综述中，我们将简要回顾当前关于神经系统疾病相关细胞状态的文献，并介绍基于 CRISPR 的功能基因组学。我们讨论基于 CRISPR 的筛选的进展，特别是在相关脑细胞类型或细胞环境中实施时，如何为揭示神经系统疾病相关细胞状态的潜在机制铺平道路。最后，我们将描述基于 CRISPR 的功能基因组学当前的挑战和未来的方向，以进一步了解神经系统疾病和潜在的治疗策略。
Our understanding of neurological diseases has been tremendously enhanced over the past decade by the application of new technologies. Genome-wide association studies have highlighted glial cells as important players in diseases. Single-cell profiling technologies are providing descriptions of disease states of neurons and glia at unprecedented molecular resolution. However, significant gaps remain in our understanding of the mechanisms driving disease-associated cell states, and how these states contribute to disease. These gaps in our understanding can be bridged by CRISPR-based functional genomics, a powerful approach to systematically interrogate gene function. In this review, we will briefly review the current literature on neurological disease-associated cell states and introduce CRISPR-based functional genomics. We discuss how advances in CRISPR-based screens, especially when implemented in the relevant brain cell types or cellular environments, have paved the way towards uncovering mechanisms underlying neurological disease-associated cell states. Finally, we will delineate current challenges and future directions for CRISPR-based functional genomics to further our understanding of neurological diseases and potential therapeutic strategies.
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影响因子：
5.9
作者：
Chen SW;Hung YS;Fuh JL;Chen NJ;Chu YS;Chen SC;Fann MJ;Wong YH
通讯作者：
Wong YH
影响因子：
64.5
作者：
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通讯作者：
Blainey, Paul C.
影响因子：
56.9
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通讯作者：
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64.5
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通讯作者：
Regev, Aviv
影响因子：
64.8
作者：
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通讯作者：
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