Targeted Editing of Zebrafish Genes to Understand Gene Function and Human Disease Pathology

Abstract Researchers often attempt to understand biological phenomena, particularly disease pathology, in an organism by manipulating a biological process to observe and interpret the effects. Although human diseases have mostly been modeled in mice, in the last decades, the zebrafish model system caught the attention of an increasing number of researchers and gradually becoming popular. A series of different advantages make the zebrafish model system well suited to be used in genetic studies. Zebrafish presents a high gene homology to humans, a complete genomic sequence, and transparency during embryo development. Moreover, the zebrafish system excels in both manipulating the genome and in observing what happens afterward: zebrafish produce large numbers of externally fertilized eggs, making them easily accessible for both manipulation and observation. In this chapter, we will focus on how the zebrafish genome can be edited and modified in order to study gene function and develop new models of human diseases.