title: Delivery methods for site-specific nucleases: Achieving the full potential of therapeutic gene editing.
abstract: The advent of site-specific nucleases, particularly CRISPR/Cas9, provides researchers with the unprecedented ability to manipulate genomic sequences. These nucleases are used to create model cell lines, engineer metabolic pathways, produce transgenic animals and plants, perform genome-wide functional screen and, most importantly, treat human diseases that are difficult to tackle by traditional medications. Considerable efforts have been devoted to improving the efficiency and specificity of nucleases for clinical applications. However, safe and efficient delivery methods remain the major obstacle for therapeutic gene editing. In this review, we summarize the recent progress on nuclease delivery methods, highlight their impact on the outcomes of gene editing and discuss the potential of different delivery approaches for therapeutic gene editing.
authors.affiliation: Shanghai Institute for Advanced Immunochemical Studies, ShanghaiTech University, 99 Haike Road, Shanghai 201210, China. Electronic address: liujia@shanghaitech.edu.cn.
authors.forename: Jia
authors.initials: J
authors.lastname: Liu
authors.affiliation: Shanghai Institute for Advanced Immunochemical Studies, ShanghaiTech University, 99 Haike Road, Shanghai 201210, China.
authors.forename: Sai-Lan
authors.initials: SL
authors.lastname: Shui
mesh_terms: D000818:Animals; D064113:CRISPR-Cas Systems; D064112:Clustered Regularly Interspaced Short Palindromic Repeats; D003851:Deoxyribonucleases / Q000737:chemistry / Q000494:pharmacology*; D000072669:Gene Editing / Q000379:methods*; D018390:Gene Targeting / Q000379:methods*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans
chemical_list: D003851:Deoxyribonucleases
keywords: Non-viral delivery; Site-specific nuclease; Therapeutic gene editing; Viral vectors