Advances in CRISPR-Cas Gene Editing Systems and Novel Explorations in Disease Treatment Applications

Abstract

Developed from the bacteria’s immune system, CRISPR-Cas systems have become a critical basis for genome editing with high precision. In this review, we outline in detail the general architecture and mechanism underlying CRISPR-Cas, discuss the variety and evolutionary functions of Cas proteins, and highlight advancements made in improving editing accuracy via the creation of high-fidelity systems. More importantly, this review focuses on the latest clinical achievements made using this gene-editing tool to treat genetic and rare disorders, study multiplex editing and immune reprogramming in the realm of precision oncology, and conduct novel applications of stem cells and tissue repair therapy in treating complex diseases and regeneration. Current literature evidence demonstrates that technological refinement has promoted a number of clinical trials of genome editing in patients with hematologic genetic diseases, not only moving forward with in vitro editing therapy but also extending into in vivo intervention and personalized treatment in accordance with the principles of precision medicine. Future research should pay attention to the remaining technical barriers related to effective delivery, safe monitoring, and ethical regulation.