Genome Editing with mRNA Encoding ZFN, TALEN, and Cas9

被引：160
|
作者：
Zhang, Hong-Xia [1 ,2 ]
Zhang, Ying [2 ]
Yin, Hao [1 ,2 ]
机构：
[1] Wuhan Univ, Zhongnan Hosp, Dept Urol, Wuhan 430071, Hubei, Peoples R China
[2] Wuhan Univ, Med Res Inst, Donghu Rd 115,Bldg 8, Wuhan 430071, Hubei, Peoples R China
来源：
MOLECULAR THERAPY | 2019年 / 27卷 / 04期
基金：
中国国家自然科学基金;
关键词：
ONE-STEP GENERATION; IN-VIVO; HEMATOPOIETIC STEM; GENE DELIVERY; RESTRICTION ENZYMES; GUIDED ENDONUCLEASE; CATIONIC POLYMERS; NONVIRAL VECTORS; DNA; EFFICIENT;
D O I：
10.1016/j.ymthe.2019.01.014
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
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.
引用
收藏
页码：735 / 746
页数：12
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