Genetic Manipulation/Modulation for Target Discovery and Validation

Identifying and validating a molecular target remains a major challenge in drug discovery. The use of genetic manipulation technologies has increased constantly and is now of fundamental value for the identification as well as validation of novel drug targets and for the whole process of drug discovery. Among available technologies, the CRISPR/Cas technology is at present the tool of choice, and its flexibility enables researchers to explore novel biomedical terrain. This chapter discusses the development and principles of modern genetic manipulation technologies with a focus on the CRISPR/Cas technology. Current methods and applications of the CRISPR/Cas technology will be outlined followed by key considerations for successful genetic manipulation experiments. Further technology development will be covered, and the impact of the CRISPR/Cas technology on target discovery and validation will be highlighted.