Challenges in CRISPR/CAS9 Delivery: Potential Roles of Nonviral Vectors

被引:156
作者
Li, Ling [1 ]
He, Zhi-Yao [1 ]
Wei, Xia-Wei [1 ]
Gao, Guang-Ping [2 ,3 ]
Wei, Yu-Quan [1 ]
机构
[1] Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Lab Aging Res, Chengdu 610041, Sichuan, Peoples R China
[2] Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USA
[3] Univ Massachusetts, Sch Med, Dept Microbiol & Physiol Syst, Worcester, MA USA
关键词
MOLECULAR-WEIGHT POLYETHYLENIMINE; MEDIATED DNA-TRANSFECTION; GENE DELIVERY; IN-VITRO; PHYSICOCHEMICAL PROPERTIES; PLASMID DNA; CHITOSAN/DNA NANOPARTICLES; QUATERNIZED CHITOSAN; EXTENDED CIRCULATION; HIGHLY EFFICIENT;
D O I
10.1089/hum.2015.069
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
CRISPR/Cas9 genome editing platforms are widely applied as powerful tools in basic research and potential therapeutics for genome regulation. The appropriate alternative of delivery system is critical if genome editing systems are to be effectively performed in the targeted cells or organisms. To date, the in vivo delivery of the Cas9 system remains challenging. Both physical methods and viral vectors are adopted in the delivery of the Cas9-based gene editing platform. However, physical methods are more applicable for in vitro delivery, while viral vectors are generally concerned with safety issues, limited packing capacities, and so on. With the robust development of nonviral drug delivery systems, lipid- or polymer-based nanocarriers might be potent vectors for the delivery of CRISPR/Cas9 systems. In this review, we look back at the delivery approaches that have been used for the delivery of the Cas9 system and outline the recent development of nonviral vectors that might be potential carriers for the genome editing platform in the future. The efforts in optimizing cationic nanocarriers with structural modification are described and promising nonviral vectors under clinical investigations are highlighted.
引用
收藏
页码:452 / 462
页数:11
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