CRISPR/Cas9 for Genome Engineering: the Next Genomic Revolution

Abstract

Genetically modified cells and animals represent a crucial tool for understanding gene function in development and diseases. The recently developed simple-to-design, easy-to-use and multiplexing CRISPR/Cas9 system is an efficient gene-targeting technology that has already stimulated innovative applications in biology and enabled researchers to make changes in the sequence or expression of any gene in virtually any cell type or organism of interest. When combined with large libraries of sgRNAs, CRISPR/Cas9 enables facile comprehensive forward genetic screens both in vitro and in vivo. Although challenges still remain regarding the off-target mutations, delivery methods as well as the frequency of homology-directed repair, the rapid advance in CRISPR/Cas9 technology opens the door for gene function revealing and genome and epigenome editing. It can be optimistically anticipated that, in the future, CRISPR/Cas9 technology may revolutionize gene therapy research and become a convenient and versatile tool to treat a wide variety of human diseases.