Int J Biol Sci 2020; 16(13):2283-2295. doi:10.7150/ijbs.33481


CRISPR/Cas9-related technologies in liver diseases: from feasibility to future diversity

Tao Xu1,2*, Li Li3,4*, Yu-chen Liu5*, Wei Cao6*, Jia-si Chen1,2, Shuang Hu1,2, Ying Liu7, Liang-yun Li1,2, Hong Zhou1,2,9, Xiao-ming Meng1,2, Cheng Huang1,2, Lei Zhang1,2, Jun Li1,2✉, Huan Zhou8✉

1. Inflammation and Immune Mediated Diseases Laboratory of Anhui Province, Anhui Institute of Innovative Drugs, School of Pharmacy, Anhui Medical University, 81 Meishan Road, Hefei 230032, Anhui Province, China.
2. Institute for Liver Diseases of Anhui Medical University, Anhui Medical University, Hefei 230032, China.
3. Department of Pathology and Pathophysiology, and Bone Marrow Transplantation Center of the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China.
4. Institute of Hematology, Zhejiang University & Zhejiang Engineering Laboratory for Stem Cell and Immunotherapy, Hangzhou, China.
5. Key Laboratory of Medical Reprogramming Technology, Shenzhen Second People's Hospital, First Affiliated Hospital of Shenzhen University, Shenzhen 518039, China.
6. Shenzhen Qianhai Icecold IT Co., Ltd. China.
7. Hefei Institutes of Physical Science, Chinese Academy of Sciences; Anhui Province Key Laboratory of Environmental Toxicology and Pollution Control Technology, Hefei, Anhui, PR China.
8. National Drug Clinical Trial Institution, the First Affiliated Hospital of Bengbu Medical College.
9. Anhui Provincial Cancer Hospital, West Branch of The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Anhui, 230031, P.R. China.
*These authors contributed equally to this work.

This is an open access article distributed under the terms of the Creative Commons Attribution License ( See for full terms and conditions.
Xu T, Li L, Liu Yc, Cao W, Chen Js, Hu S, Liu Y, Li Ly, Zhou H, Meng Xm, Huang C, Zhang L, Li J, Zhou H. CRISPR/Cas9-related technologies in liver diseases: from feasibility to future diversity. Int J Biol Sci 2020; 16(13):2283-2295. doi:10.7150/ijbs.33481. Available from

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Liver diseases are one of the leading causes of mortality in the world, mainly caused by different etiological agents, alcohol consumption, viruses, drug intoxication, and malnutrition. The maturation of gene therapy has heralded new avenues for developing effective interventions for these diseases. Derived from a remarkable microbial defense system, clustered regularly interspaced short palindromic repeats/CRISPR-associated proteins 9 system (CRISPR/Cas9 system) is driving innovative applications from basic biology to biotechnology and medicine. Recently, the mutagenic function of CRISPR/Cas9 system has been widely adopted for genome and disease research. In this review, we describe the development and applications of CRISPR/Cas9 system on liver diseases for research or translational applications, while highlighting challenges as well as future avenues for innovation.

Keywords: CRISPR/Cas9, viral hepatitis, hepatocellular carcinoma (HCC), clinical research