SunnyTALEN: A Second-Generation TALEN System for Human Genome Editing

被引：20

作者：

Sun, Ning ^{[1
]}

Bao, Zehua ^{[1
]}

Xiong, Xiong ^{[2
]}

Zhao, Huimin ^{[1
,2
,3
,4
]}

机构：

[1] Univ Illinois, Dept Biochem, Urbana, IL 61801 USA

[2] Univ Illinois, Dept Chem & Biomol Engn, Urbana, IL 61801 USA

[3] Univ Illinois, Dept Bioengn, Dept Chem, Ctr Biophys & Computat Biol, Urbana, IL 61801 USA

[4] Univ Illinois, Inst Genom Biol, Urbana, IL 61801 USA

来源：

BIOTECHNOLOGY AND BIOENGINEERING | 2014年 / 111卷 / 04期

关键词：

directed evolution; double-strand break; genome editing; gene therapy; synthetic biology; ZINC-FINGER NUCLEASES; EFFECTOR NUCLEASES; GENE-THERAPY; HIGHLY EFFICIENT; DISEASE-MODELS; CELLS; SPECIFICITIES; MUTATIONS; TOXICITY; TOOLS;

D O I：

10.1002/bit.25154

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：

Transcription activator-like effector nucleases (TALENs) have rapidly emerged as a powerful genome editing tool. The site-specific DNA double-strand breaks generated by TALENs in the human chromosome can induce homologous recombination or non-homologous end joining, resulting in desired genetic modifications. In this study, we report the development of a TALEN variant, SunnyTALEN, with >2.5-fold improved genome editing efficacy in human cells. The corresponding scaffold increases the rate of genetic modification at all the 13 tested loci of human genome and is compatible with heterodimer TALEN architectures. This enhanced and high-efficiency TALEN variant represents a novel second-generation TALEN system and has great potential for biological and therapeutic applications. Biotechnol. Bioeng. 2014;111: 683-691. (c) 2013 Wiley Periodicals, Inc.

引用

页码：683 / 691

页数：9

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