Applications of CRISPR/CAS9 for therapy in human genetic diseases

The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) protein 9 systems, as a bacteria and archaea acquired immune system to resist against bacteriophages, provides a novel genome editing technology. This novel technology can precisely target a genomic site to disrupt or repair a specific gene, and has been successfully used in many types of cells for identifying therapeutic targets and gene functions, generating animal models, and developing gene therapies. Moreover, this system has been used to alleviate disease symptoms by correcting related genes. However, the safety and efficiency of CRISPR/Cas9 system remains a challenge that affects its clinical application. In this chapter, we reviewed the CRISPR/Cas9 potential clinical applications. Moreover, we discussed some essential clinical requirements for the efficiency and specificity in the gene therapy.