[Application of genome editing technology in gene therapy]

Abstract

Genome editing has been attracting increasing attention as a new treatment for several refractory diseases since the CRISPR-Cas discovery has facilitated easy modification of target chromosomal DNA. The concept of treating refractory diseases by genome editing has been achieved in various animal models, and genome editing has been applied to human clinical trials for β-thalassemia, sickle cell disease, mucopolysaccharidosis, transthyretin amyloidosis, HIV infection, and CAR-T therapy. The genome editing technology targets the germline in industrial applications in animals and plants and is directed at the chromosomal DNA of the somatic cells in human therapeutic applications. Genome editing therapy for germline cells is currently forbidden due to ethical and safety concerns. Concerns regarding genome editing technology include safety (off-target effects) as well as technical aspects (low homologous recombination). Various technological innovations for genome editing are expected to expand its clinical application to various diseases in the future.

Keywords: CRISPR-Cas; Genome editing; Zinc finger nuclease.