CRISPR Editing in Biological and Biomedical Investigation.

The revolutionary technology for genome editing known as the clustered regularly interspaced short palindromic repeat (CRISPR)-CRISPR-associated protein 9 (Cas9) system has sparked advancements in biological and biomedical research. The scientific breakthrough of the development of CRISPR-Cas9 technology has allowed us to recapitulate human diseases by generating animal models of interest ranging from zebrafish to non-human primates. The CRISPR-Cas9 system can also be used to delineate the mechanisms underlying the development of human disorders and to precisely correct disease-causing mutations. Repurposing this technology enables wider applications in transcriptome and epigenome manipulation and holds promise to reach the clinic. In this review, we highlight the latest advances of the CRISPR-Cas9 system in different platforms and discuss the hurdles and challenges this technology is facing. J. Cell. Biochem. 118: 4152-4162, 2017. © 2017 Wiley Periodicals, Inc.