Department of Laboratory Medicine & Pathobiology, University of Toronto, Medical Sciences Building, 6th Floor, 1 King's College Circle, Toronto, Ontario, M5S 1A8, Canada.
Tanz Centre for Research in Neurodegenerative Diseases, University of Toronto, Krembil Discovery Centre, 6th Floor60 Leonard Avenue, Toronto, Ontario, M5T 2S8, Canada.
Mol Neurodegener. 2019 Nov 14;14(1):41. doi: 10.1186/s13024-019-0343-3.
The adoption of CRISPR-Cas9 technology for functional genetic screens has been a transformative advance. Due to its modular nature, this technology can be customized to address a myriad of questions. To date, pooled, genome-scale studies have uncovered genes responsible for survival, proliferation, drug resistance, viral susceptibility, and many other functions. The technology has even been applied to the functional interrogation of the non-coding genome. However, applications of this technology to neurological diseases remain scarce. This shortfall motivated the assembly of a review that will hopefully help researchers moving in this direction find their footing. The emphasis here will be on design considerations and concepts underlying this methodology. We will highlight groundbreaking studies in the CRISPR-Cas9 functional genetics field and discuss strengths and limitations of this technology for neurological disease applications. Finally, we will provide practical guidance on navigating the many choices that need to be made when implementing a CRISPR-Cas9 functional genetic screen for the study of neurological diseases.
CRISPR-Cas9 技术在功能基因筛选中的应用是一项变革性的进展。由于其模块化的性质，该技术可以定制以解决无数的问题。迄今为止，大规模的 pooled 基因组研究已经揭示了与生存、增殖、耐药性、病毒易感性和许多其他功能相关的基因。该技术甚至已被应用于非编码基因组的功能研究。然而，该技术在神经疾病中的应用仍然很少。这种不足促使我们编写了这篇综述，希望能帮助从事这方面研究的研究人员找到立足点。这里的重点将放在该方法的设计考虑因素和概念上。我们将重点介绍 CRISPR-Cas9 功能遗传学领域的开创性研究，并讨论该技术在神经疾病应用中的优势和局限性。最后，我们将提供实用的指导，帮助您在为神经疾病研究实施 CRISPR-Cas9 功能基因筛选时，针对众多需要做出的选择进行导航。
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