Carrier strategies boost the application of CRISPR/Cas system in gene therapy
Zunkai Xu,
Qingnan Wang,
Haiping Zhong,
Yaoyao Jiang,
Xiaoguang Shi,
Bo Yuan,
Na Yu,
Shubiao Zhang,
Xiaoyong Yuan,
Shutao Guo,
Yang Yang
Affiliations
Zunkai Xu
Key Laboratory of Functional Polymer Materials of Ministry of Education State Key Laboratory of Medicinal Chemical Biology and Institute of Polymer Chemistry College of Chemistry Nankai University Tianjin China
Qingnan Wang
State Key Laboratory of Biotherapy and Cancer Center West China Hospital Sichuan University and Collaborative Innovation Center Chengdu China
Haiping Zhong
Key Laboratory of Functional Polymer Materials of Ministry of Education State Key Laboratory of Medicinal Chemical Biology and Institute of Polymer Chemistry College of Chemistry Nankai University Tianjin China
Yaoyao Jiang
Key Laboratory of Functional Polymer Materials of Ministry of Education State Key Laboratory of Medicinal Chemical Biology and Institute of Polymer Chemistry College of Chemistry Nankai University Tianjin China
Xiaoguang Shi
Key Laboratory of Functional Polymer Materials of Ministry of Education State Key Laboratory of Medicinal Chemical Biology and Institute of Polymer Chemistry College of Chemistry Nankai University Tianjin China
Bo Yuan
School of Medicine Nankai University Tianjin China
Na Yu
Translational Medicine Center Key Laboratory of Molecular Target & Clinical Pharmacology School of Pharmaceutical Sciences and The Second Affiliated Hospital Guangzhou Medical University Guangzhou China
Shubiao Zhang
Key Laboratory of Biotechnology and Bioresources Utilization of Ministry of Education Dalian Minzu University Dalian China
Xiaoyong Yuan
Tianjin Key Laboratory of Ophthalmology and Visual Science Tianjin Eye Institute Tianjin Eye Hospital Tianjin China
Shutao Guo
Key Laboratory of Functional Polymer Materials of Ministry of Education State Key Laboratory of Medicinal Chemical Biology and Institute of Polymer Chemistry College of Chemistry Nankai University Tianjin China
Yang Yang
State Key Laboratory of Biotherapy and Cancer Center West China Hospital Sichuan University and Collaborative Innovation Center Chengdu China
Abstract Emerging clustered regularly interspaced short palindromic repeat/associated protein (CRISPR/Cas) genome editing technology shows great potential in gene therapy. However, proteins and nucleic acids suffer from enzymatic degradation in the physiological environment and low permeability into cells. Exploiting carriers to protect the CRISPR system from degradation, enhance its targeting of specific tissues and cells, and reduce its immunogenicity is essential to stimulate its clinical applications. Here, the authors review the state‐of‐the‐art CRISPR delivery systems and their applications, and describe strategies to improve the safety and efficacy of CRISPR mediated genome editing, categorized by three types of cargo formats, that is, Cas: single‐guide RNA ribonucleoprotein, Cas mRNA and single‐guide RNA, and Cas plasmid expressing CRISPR/Cas systems. The authors hope this review will help develop safe and efficient nanomaterial‐based carriers for CRISPR tools.
