CRISPR/Cas9-mediated genome editing: From basic research to translational medicine

The recent development of the CRISPR/Cas9 system as an efficient and accessi-

ble programmable genome-editing tool has revolutionized basic science research.

CRISPR/Cas9 system-based technologies have armed researchers with new power-

ful tools to unveil the impact of genetics on disease development by enabling the

creation of precise cellular and animal models of human diseases. The therapeutic

potential of these technologies is tremendous, particularly in gene therapy, in which

a patient-specific mutation is genetically corrected in order to treat human dis-

eases that are untreatable with conventional therapies. However, the translation of

CRISPR/Cas9 into the clinics will be challenging, since we still need to improve the

efficiency, specificity and delivery of this technology. In this review, we focus on

several in vitro, in vivo and ex vivo applications of the CRISPR/Cas9 system in human

disease-focused research, explore the potential of this technology in translational

medicine and discuss some of the major challenges for its future use in patients