Gene Editing: An Effective Tool for the Future Treatment of Kidney Disease

Mei-Ling Cao,1,* Rui-Yi Han,2,* Si-Da Chen,3,* Dan-Yang Zhao,2 Ming-Yue Shi,2 Jia-Hui Zou,2 Lei Li,3 Hong-Kun Jiang2 1Department of Neonatology, The First Hospital of China Medical University, Shenyang, Liaoning, 110001, People’s Republic of China; 2Department of Pediatrics, The First Hospital of China Medical University, Shenyang, Liaoning, 110001, People’s Republic of China; 3Department of Orthopaedic Surgery, Shengjing Hospital of China Medical University, Shenyang, Liaoning, 110004, People’s Republic of China*These authors contributed equally to this studyCorrespondence: Hong-Kun Jiang, Department of Pediatrics, The First Hospital of China Medical University, No. 155 Nanjing North Street, Heping District, Liaoning, 110001, People’s Republic of China, Email [email protected] Lei Li, Department of Orthopedic Surgery, Shengjing Hospital of China Medical University, No. 36 Sanhao Street, Heping District, Liaoning, 110004, People’s Republic of China, Tel/Fax +86 02423896615, Email [email protected]: Gene editing technology involves modifying target genes to alter genetic traits and generate new phenotypes. Beginning with zinc-finger nucleases (ZFN) and transcription activator-like effector nucleases (TALEN), the field has evolved through the advent of clustered regularly interspaced short palindromic repeats and CRISPR-associated protein (CRISPR-Cas) systems, and more recently to base editors (BE) and prime editors (PE). These innovations have provided deep insights into the molecular mechanisms of complex biological processes and have paved the way for novel therapeutic strategies for a range of diseases. Gene editing is now being applied in the treatment of both genetic and acquired kidney diseases, as well as in kidney transplantation and the correction of genetic mutations. This review explores the current applications of mainstream gene editing technologies in biology, with a particular emphasis on their roles in kidney disease research and treatment of. It also addresses the limitations and challenges associated with these technologies, while offering perspectives on their future potential in this field.Keywords: clear cell renal cell carcinoma, ccRCC, clustered regularly interspaced short palindromic repeats, CRISPR, gene editing, genetic kidney diseases, polycystic kidney disease