CRISPR/Cas Systems for Therapeutic Purposes: Applications in Disease Prevention, Mechanism Study, and Treatment

Abstract

CRISPR/Cas systems were novel multifunctional genome editing techniques, and the modification of Cas proteins endowed them with more efficient and precise properties. Herein, we systematically outlined the principles and applications of CRISPR/Cas systems in disease prevention, mechanism research, and gene treatment of diseases. Specifically, we detailed the systems widely utilized, containing the Cas9, Cas12a, and Cas13a systems, and summarized the current status of the medical applications of these systems in terms of vaccine design, trait improvement, animal models of human diseases, organ transplantation, gene therapy, and gene detection. We concluded that CRISPR/Cas systems had great potential to advance medical research and practice but also should be carefully regulated and supervised to ensure their safety and efficacy.