Application of CRISPR genetic screens to investigate neurological diseases

Molecular Neurodegeneration

卷:14

Raphaella W. L. So¹  Zaid A. M. Al-Azzawi¹  Jossana Bishay¹  Sai Wai Chung¹  Lilian Tsai-Wei Lin¹  Xinzhu Wang¹  Erin Gaudette¹  Heather H. C. Lau¹  Gerold Schmitt-Ulms¹  Jeremy J. Watts²  Julia Joung³ 

[1] Department of Laboratory Medicine & Pathobiology, University of Toronto, Medical Sciences Building;

[2] Department of Pharmacology & Toxicology, University of Toronto, Medical Sciences Building;

[3] Departments of Biological Engineering and Brain and Cognitive Science, and McGovern Institute for Brain Research at MIT;

关键词: CRISPR-Cas9;    Functional genetics;    sgRNA;    CRISIPR KO;    CRISPRi;    CRISPRa;   

DOI  :  10.1186/s13024-019-0343-3

来源: DOAJ

【 摘 要 】

Abstract 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.

【 授权许可】

Unknown