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To cleave or not to cleave: therapeutic gene editing with and without programmable nucleases
Nature Reviews Drug Discovery ( IF 122.7 ) Pub Date : 2017-03-17 , DOI: 10.1038/nrd.2017.42
Tod M. Woolf , Channabasavaiah B. Gurumurthy , Frederick Boyce , Eric B. Kmiec

In recent years, pioneering clinical studies involving the use of programmable nucleases to achieve gene editing have begun to evaluate the therapeutic potential of such approaches. For example, Sangamo BioSciences has reported successful proof-of-concept clinical studies to treat HIV infection using an engineered zinc-finger nuclease

中文翻译:

切割或不切割:带有和不带有可编程核酸酶的治疗性基因编辑

近年来,涉及使用可编程核酸酶实现基因编辑的开创性临床研究已开始评估此类方法的治疗潜力。例如,Sangamo BioSciences报告了成功的概念验证临床研究,该研究使用工程化的锌指核酸酶治疗HIV感染
更新日期:2017-04-11
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