title: Challenges in CRISPR/CAS9 Delivery: Potential Roles of Nonviral Vectors.
abstract: 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.
authors.affiliation: 1 Lab for Aging Research, State Key Laboratory of Biotherapy, West China Hospital, Sichuan University , Chengdu, Sichuan, PR China.
authors.forename: Ling
authors.initials: L
authors.lastname: Li
authors.affiliation: 1 Lab for Aging Research, State Key Laboratory of Biotherapy, West China Hospital, Sichuan University , Chengdu, Sichuan, PR China.
authors.forename: Zhi-Yao
authors.initials: ZY
authors.lastname: He
authors.affiliation: 1 Lab for Aging Research, State Key Laboratory of Biotherapy, West China Hospital, Sichuan University , Chengdu, Sichuan, PR China.
authors.forename: Xia-Wei
authors.initials: XW
authors.lastname: Wei
authors.affiliation: 2 Gene Therapy Center, University of Massachusetts Medical School , Worcester, Massachusetts.|3 Department of Microbiology and Physiology Systems, University of Massachusetts Medical School , Worcester, Massachusetts.
authors.forename: Guang-Ping
authors.initials: GP
authors.lastname: Gao
authors.affiliation: 1 Lab for Aging Research, State Key Laboratory of Biotherapy, West China Hospital, Sichuan University , Chengdu, Sichuan, PR China.
authors.forename: Yu-Quan
authors.initials: YQ
authors.lastname: Wei
mesh_terms: D064113:CRISPR-Cas Systems; D015316:Genetic Therapy / Q000639:trends*; D005822:Genetic Vectors; D006801:Humans; D014780:Viruses / Q000235:genetics