Delivery methods for site-specific nucleases: Achieving the full potential of therapeutic gene editing

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• 作者：Jia Liu ; ^{liujia@shanghaitech.edu.cn} ; Sai-lan Shui
• 关键词：Site-specific nuclease ; Therapeutic gene editing ; Non-viral delivery ; Viral vectors
• 刊名：Journal of Controlled Release
• 出版年：2016
• 出版时间：28 December 2016
• 年：2016
• 卷：244
• 期：part_PA
• 页码：83-97
• 全文大小：1197 K
• 卷排序：244

文摘

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.