新型且有潜力的基因组工程工具包。
New and TALENted genome engineering toolbox.
机构信息
Center for Translational Science Activities, Mayo Clinic, Rochester, MN 55905, USA.
出版信息
Circ Res. 2013 Aug 16;113(5):571-87. doi: 10.1161/CIRCRESAHA.113.301765.
Abstract
Recent advances in the burgeoning field of genome engineering are accelerating the realization of personalized therapeutics for cardiovascular disease. In the postgenomic era, sequence-specific gene-editing tools enable the functional analysis of genetic alterations implicated in disease. In partnership with high-throughput model systems, efficient gene manipulation provides an increasingly powerful toolkit to study phenotypes associated with patient-specific genetic defects. Herein, this review emphasizes the latest developments in genome engineering and how applications within the field are transforming our understanding of personalized medicine with an emphasis on cardiovascular diseases.
摘要
近年来，基因组工程领域的新兴技术正在加速实现心血管疾病的个体化治疗。在后基因组时代，序列特异性基因编辑工具使我们能够对与疾病相关的遗传改变进行功能分析。与高通量模型系统相结合，高效的基因操作提供了一个日益强大的工具包，用于研究与患者特异性遗传缺陷相关的表型。本文强调了基因组工程的最新进展，以及该领域的应用如何改变我们对个性化医学的理解，重点是心血管疾病。
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本文引用的文献
1
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells.CRISPR-Cas 核酸酶在人类细胞中诱导的高频脱靶突变。
Nat Biotechnol. 2013 Sep;31(9):822-6. doi: 10.1038/nbt.2623. Epub 2013 Jun 23.
2
High efficiency In Vivo genome engineering with a simplified 15-RVD GoldyTALEN design.高效的体内基因组工程，采用简化的 15-RVD GoldyTALEN 设计。
PLoS One. 2013 May 29;8(5):e65259. doi: 10.1371/journal.pone.0065259. Print 2013.
3
Targeting human microRNA genes using engineered Tal-effector nucleases (TALENs).利用工程化 Tal 效应物核酸酶（TALENs）靶向人类 microRNA 基因。
PLoS One. 2013 May 7;8(5):e63074. doi: 10.1371/journal.pone.0063074. Print 2013.
4
Generation of gene disruptions by transcription activator-like effector nucleases (TALENs) in Xenopus tropicalis embryos.通过转录激活子样效应物核酸酶（TALENs）在非洲爪蟾胚胎中产生基因缺失。
Cell Biosci. 2013 May 10;3(1):21. doi: 10.1186/2045-3701-3-21.
5
High-efficiency and heritable gene targeting in mouse by transcription activator-like effector nucleases.转录激活因子样效应物核酸酶在小鼠中实现高效且可遗传的基因靶向。
Nucleic Acids Res. 2013 Jun;41(11):e120. doi: 10.1093/nar/gkt258. Epub 2013 Apr 28.
6
Quantitative assay for TALEN activity at endogenous genomic loci.内源性基因组靶位点 TALEN 活性的定量分析。
Biol Open. 2013 Feb 12;2(4):363-7. doi: 10.1242/bio.20133871. Print 2013 Apr 15.
7
A systematic genome-wide analysis of zebrafish protein-coding gene function.系统的全基因组分析斑马鱼蛋白质编码基因功能。
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Genome Res. 2013 Jul;23(7):1182-93. doi: 10.1101/gr.147314.112. Epub 2013 Apr 8.
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