Quantifying on- and off-target genome editing.

Quantifying on- and off-target genome editing.
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DOI:
10.1016/j.tibtech.2014.12.001
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发表时间:
2015-02
影响因子:
17.3
通讯作者:
Porteus, Matthew H.
Porteus, Matthew H.
中科院分区:
工程技术1区
文献类型:
--
作者:
Hendel, Ayal;Fine, Eli J.;Bao, Gang;Porteus, Matthew H.

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使用工程核酸酶进行基因组编辑是一个快速增长的领域,这要归功于变革性技术,这些技术允许研究人员为许多应用精确地改变基因组,包括基础研究、生物技术和人类基因治疗。尽管近年来在整个基因组的特定位置进行精确和可控改变的能力有了很大的提高,但我们仍然缺乏一套全面和标准化的分析方法来衡量在内源基因组位点产生的不同基因组编辑结果。在这里,我们回顾了现有的用于量化靶上和靶外基因组编辑的方法,并描述了它们在推进这项技术方面的效用。我们还强调了尚未满足的量化目标上和目标外基因组编辑结果的化验需求,并讨论了它们对基因组编辑领域的重要性。
Genome editing with engineered nucleases is a rapidly growing field, thanks to transformative technologies that allow researchers to precisely alter genomes for numerous applications, including basic research, biotechnology, and human gene therapy. While the capability to make precise and controlled changes at specified sites throughout the genome has grown tremendously in recent years, we still lack a comprehensive and standardized battery of assays for measuring the different genome editing outcomes created at endogenous genomic loci. Here, we review the existing assays for quantifying on and off-target genome editing and describe their utility in advancing the technology. We also highlight unmet assay needs for quantifying on and off-target genome editing outcomes and discuss their importance for the genome editing field.
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