CRISPR: a revolutionary tool for modeling and treating cancer and Duchenne muscular dystrophy

Abstract

The novel CRISPR/Cas system is an effective and widely used gene-editing tool that has created the opportunity to study gene function and understand the pathogenic mechanisms underlying various genetic disorders. It has uncovered the pathophysiology of several previously unsolved medical conditions and is currently an essential genome-editing tool being employed to develop new drugs and innovative gene therapies for potentially life-threatening diseases such as, cancer and Duchenne muscular dystrophy (DMD). The specificity, accuracy, high probability of successful outcomes, cost and time effectiveness of CRISPR/Cas systems have made it a reliable means to treat cancer and DMD over other previously used genetic modification tools. This review will discuss how CRISPR/Cas can be utilized to design in vivo and in vitro disease models, target cancer cells, and aid in the identification of novel drug targets to treat cancer and DMD patients.