title: In Vivo Delivery Systems for Therapeutic Genome Editing.
abstract: Therapeutic genome editing technology has been widely used as a powerful tool for directly correcting genetic mutations in target pathological tissues and cells to cure of diseases. The modification of specific genomic sequences can be achieved by utilizing programmable nucleases, such as Meganucleases, zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and the clustered regularly-interspaced short palindromic repeat-associated nuclease Cas9 (CRISPR/Cas9). However, given the properties, such as large size, negative charge, low membrane penetrating ability, as well as weak tolerance for serum, and low endosomal escape, of these nucleases genome editing cannot be successfully applied unless in vivo delivery of related programmable nucleases into target organisms or cells is achieved. Here, we look back at delivery strategies having been used in the in vivo delivery of three main genome editing nucleases, followed by methodologies currently undergoing testing in clinical trials, and potential delivery strategies provided by analyzing characteristics of nucleases and commonly used vectors.
authors.affiliation: Institute for Advancing Translational Medicine in Bone &amp; Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China. luyaoben@126.com.
authors.forename: Luyao
authors.initials: L
authors.lastname: Wang
authors.affiliation: Institute for Advancing Translational Medicine in Bone &amp; Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China. fayebalaba@live.com.
authors.forename: Fangfei
authors.initials: F
authors.lastname: Li
authors.affiliation: Institute for Advancing Translational Medicine in Bone &amp; Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China. danglei_hkbu@163.com.
authors.forename: Lei
authors.initials: L
authors.lastname: Dang
authors.affiliation: Institute for Advancing Translational Medicine in Bone &amp; Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China. liangchao512@163.com.
authors.forename: Chao
authors.initials: C
authors.lastname: Liang
authors.affiliation: Institute for Advancing Translational Medicine in Bone &amp; Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China. wangchao@hkbu.edu.hk.
authors.forename: Chao
authors.initials: C
authors.lastname: Wang
authors.affiliation: Institute for Advancing Translational Medicine in Bone &amp; Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China. hebinghb@gmail.com.
authors.forename: Bing
authors.initials: B
authors.lastname: He
authors.affiliation: Institute for Advancing Translational Medicine in Bone &amp; Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China. liujin_hkbu@163.com.
authors.forename: Jin
authors.initials: J
authors.lastname: Liu
authors.affiliation: Institute for Advancing Translational Medicine in Bone &amp; Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China. lidefang@163.com.
authors.forename: Defang
authors.initials: D
authors.lastname: Li
authors.affiliation: Institute for Advancing Translational Medicine in Bone &amp; Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China. wxho0606@163.com.
authors.forename: Xiaohao
authors.initials: X
authors.lastname: Wu
authors.affiliation: Central Laboratory, Zheng Zhou Hospital of Traditional Chinese Medicine, Zhengzhou 450000, China. xuxg1115@126.com.
authors.forename: Xuegong
authors.initials: X
authors.lastname: Xu
authors.affiliation: Institute for Advancing Translational Medicine in Bone &amp; Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China. aipinglu@hkbu.edu.hk.
authors.forename: Aiping
authors.initials: A
authors.lastname: Lu
authors.affiliation: Institute for Advancing Translational Medicine in Bone &amp; Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong 00852, China. zhangge@hkbu.edu.hk.
authors.forename: Ge
authors.initials: G
authors.lastname: Zhang
mesh_terms: D000256:Adenoviridae / Q000235:genetics; D064113:CRISPR-Cas Systems / Q000235:genetics; D004260:DNA Repair; D000072669:Gene Editing; D005822:Genetic Vectors / Q000235:genetics / Q000378:metabolism; D016086:Lentivirus / Q000235:genetics; D000069896:Transcription Activator-Like Effector Nucleases / Q000235:genetics; D016335:Zinc Fingers / Q000235:genetics
chemical_list: D000069896:Transcription Activator-Like Effector Nucleases
keywords: genome editing; in vivo delivery systems; programmable nucleases; vectors