CRISPR Technology and Its Application in Therapeutics

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology is one of the most significant scientific breakthroughs of this century, revolutionizing therapeutics. A component of bacterial immune systems, CRISPR has been repurposed to precisely edit DNA sequences. The accuracy, efficiency, and simplicity of CRISPR technology have provided exciting new avenues to modify DNA and RNA for the treatment of human diseases. This includes both gene therapy, in which genes are edited in vivo in patients, and gene-edited cell therapy, in which cells are edited ex vivo and transfused into patients. This chapter is a comprehensive guide to CRISPR gene editing and its applications in biotherapeutics. It begins with an introduction to CRISPR-Cas9 technology, examining its discovery and adaptation, mode of action, advantages over previous tools, derivatives of the original CRISPR-Cas9 technology, and gene editing methods. This is followed by a review of the applications of CRISPR technology in biotherapeutics, including both gene therapies and gene-edited cell therapies for the treatment of three disease categories: genetic disorders, infectious diseases, and cancer. Relevant preclinical research and/or clinical trials are discussed for each disease category. The final section delves into the future of CRISPR therapeutics, including nascent applications, the industrialization and projected growth of the industry, safety and ethical considerations, and its transformative potential for human health.