Genome Editing with ZFN, TALEN and CRISPR/Cas Systems:The Applications and Future Prospects

With the rapid development of zinc finger nucleases (ZFN), transcription activator-like effector nuclease (TALEN) and Cluster regularly interspaced short palindromic repeat (CRISPR)/Cas systems, manipulating genome becomes relatively easy and efficient. There are two major purposes for genome manipulation: one is for understanding novel genes function and their regulation roles in cells.Recent genomic and genetic approaches for disease genes have identified many novel loci underlie the disease phenotypes. More than 2,000 robust associations have been found in more than 300 complex diseases and traitsin recent years by genome wide association studies (GWASs) [1]. Understudying the function roles of genetic loci, particular novel genes will bring new insight of the diseases mechanisms. The second major application for manipulating genome is providing a new therapeutic means for many genetic disorders.A disease causing mutation could be replaced with normal allele in several Mendelian monogenic diseases.