CRISPR/Cas9 TECHNOLOGY- A NEW BOON IN GENOME EDITING

Targeted genome engineering provides the ability to precisely modify genetic information in order to study gene function, biological mechanisms, and disease pathology. Historically, random mutagenesis or low-efficiency homologous recombination were used to modify the genomes of cell lines or animal models. However, new advances in the design of sequence-specific endonucleases have enabled more effective, targeted editing of the genome. The most recent and fastest growing method for genome editing is based on the Clustered Regions of Interspersed Palindromic Repeats (CRISPR) viral defense system found in bacteria and archaea.  The CRISPR/Cas9 system is much easier to customize and optimize because the site selection for DNA cleavage is guided by a short sequence of RNA rather than an engineered protein as in the systems of zinc finger nucleases (ZFN), transcription activator–like effect or nucleases (TALEN), and meganucleases. Derived from a remarkable microbial defense system, Cas9 is driving innovative applications from basic biology to biotechnology and medicine. The simplicity of the CRISPR-Cas9 system has enabled its widespread applications in generating germline animal models, somatic genome engineering, and functional genomics screening and in treating genetic and infectious diseases. This technology will likely be used in all fields of biomedicine, ranging from basic research to human gene therapy.