ABSTRACT
CRISPR-Cas system has a potential role in diverse scientific fields such as agriculture, environment, and human health affected by familial or non-familial diseases. Interestingly, the absence of CRISP-Cas system in eukaryotes makes it a powerful tool for gene manipulation and editing in humans. Due to its low cost, easy manipulation, and straightforward mechanism, researchers find CRISPR-Cas systems the most advanced tool for gene therapeutics. Several clinical trials for the cure of human genetic disorders using CRISPR-Cas system are completed or underway. There are, however, some challenges with this technology, such as delivery of foreign DNA in the human body, host immune response, and risk of off-target mutations. However, we are very hopeful that in the near future scientists all over the world will find solutions for these hindrances and we can easily cure human genetic disorders. In this chapter we systematically explain the origin, mechanism of action, and application of CRISPR-Cas in human genetic disorders. The chapter will be very helpful for young researchers and students working in the field of gene therapeutics.
