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.

中文翻译：

---

应用 CRISPR 基因筛选研究神经系统疾病。


采用 CRISPR-Cas9 技术进行功能遗传筛选是一项革命性的进步。由于其模块化特性，该技术可以进行定制以解决无数问题。迄今为止，基因组规模的汇总研究已经发现了负责生存、增殖、耐药性、病毒易感性和许多其他功能的基因。该技术甚至已应用于非编码基因组的功能询问。然而，这项技术在神经系统疾病中的应用仍然很少。这一不足促使我们进行了一项综述，希望能够帮助朝这个方向前进的研究人员找到立足点。这里的重点是该方法论的设计考虑和概念。我们将重点介绍 CRISPR-Cas9 功能遗传学领域的突破性研究，并讨论该技术在神经系统疾病应用中的优势和局限性。最后，我们将为神经系统疾病研究实施 CRISPR-Cas9 功能遗传筛选时需要做出的许多选择提供实用指导。