Genome Editing in Gene and Cell-Based Therapies
Saranya Ganapathy and Sanjeev K Gupta
Journal Title:Acta Scientific Microbiology
Site-specific genetic modification , for effective treatment of human diseases, has become a realistic possibility after nearly 50 years of research in the field of gene editing/therapy. With the need for introducing exogenous fragment DNA or removing/modifying endogenous gene from/in a specific site in the genome of interest, precise genetic tools are being carefully designed for implementa-tion. Creating Double Stranded Breaks (DSBs) at targeted sites in the genome of interest followed by endogenous cell based repair (Homology Directed Repair (HDR) or Non-Homologous End Joining (NHEJ)) results in genomic modifications . With this approach, it is possible to carry out gene insertion, augmentation, deletion, gene sequence modification and mutation correction, aiding in the development of better animal disease models, ex vivo and in vivo gene repair and engineered cell based therapies.