mRNA 编码的 ZFN、TALEN 和 Cas9 的基因组编辑。
Genome Editing with mRNA Encoding ZFN, TALEN, and Cas9.
机构信息
Department of Urology, Zhongnan Hospital of Wuhan University, 430071 Wuhan, China; Medical Research Institute, Wuhan University, 430071 Wuhan, China.
Medical Research Institute, Wuhan University, 430071 Wuhan, China.
出版信息
Mol Ther. 2019 Apr 10;27(4):735-746. doi: 10.1016/j.ymthe.2019.01.014. Epub 2019 Jan 25.
Genome-editing technologies based on programmable nucleases have significantly broadened our ability to make precise and direct changes in the genomic DNA of various species, including human cells. Delivery of programmable nucleases into the target tissue or cell is one of the pressing challenges in transforming the technology into medicine. In vitro-transcribed (IVT) mRNA-mediated delivery of nucleases has several advantages, such as transient expression with efficient in vivo and in vitro delivery, no genomic integration, a potentially low off-target rate, and high editing efficiency. This review focuses on key barriers related to IVT mRNA delivery, on developed modes of delivery, and on the application and future prospects of mRNA encoding nuclease-mediated genome editing in research and clinical trials.
基于可编程核酸酶的基因组编辑技术显著拓宽了我们在各种物种（包括人类细胞）的基因组 DNA 上进行精确和直接修饰的能力。将可编程核酸酶递送到靶组织或细胞是将该技术转化为医学应用的紧迫挑战之一。体外转录 (IVT) mRNA 介导的核酸酶递送具有许多优点，例如瞬时表达，具有高效的体内和体外递送效率、无基因组整合、潜在的低脱靶率和高效率的编辑。本综述重点介绍了与 IVT mRNA 递送相关的关键障碍、已开发的递送方式，以及 mRNA 编码核酸酶介导的基因组编辑在研究和临床试验中的应用和未来前景。
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