Development and challenges of using CRISPR-Cas9 system in mammalians

Abstract Currently, the type II CRISPR-Cas9 system is used for genome editing in number of organisms. It has recently gained much interest due to easy to use and precisely manipulate organisms. CRISPR-Cas9 is used for the correction of gene repaired, gene therapy and replacement or insertion of desired gene. We explore and explain the conceptual ways about how CRISPR-Cas9 technology has made us move a step ahead in order to achieve gene therapy. This chapter covers a wide range of experimental evidences regarding the caliber of CRISPR-Cas9 for its use as therapeutic agent against number of diseases including cancer, duchenne muscular dystrophy, tyrosinemia type-1 and many more. Further, we also present a glimpse about functional challenges that need to be overcome as well as the ethical and the regulatory issues that engenders use of the mentioned entity.