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Engineering nucleases for gene targeting: Safety and regulatory considerations (Review)

机译:用于基因靶向的工程核酸酶:安全性和监管考虑(综述)

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摘要

Nuclease-based gene targeting (NBGT) represents a significant breakthrough in targeted genome editing since it is applicable from single-celled protozoa to human, including several species of economic importance. Along with the fast progress in NBGT and the increasing availability of customized nucleases, more data are available about off-target effects associated with the use of this approach. We discuss how NBGT may offer a new perspective for genetic modification, we address some aspects crucial for a safety improvement of the corresponding techniques and we also briefly relate the use of NBGT applications and products to the regulatory oversight.
机译:基于核酸酶的基因靶向(NBGT)代表了靶向基因组编辑的重大突破,因为它适用于从单细胞原生动物到人类,包括几种具有经济重要性的物种。随着NBGT的快速发展以及定制核酸酶的可用性不断提高,可获得更多与使用这种方法相关的脱靶效应的数据。我们讨论了NBGT如何为基因修饰提供新的视角,我们讨论了对改进相应技术的安全性至关重要的某些方面,并且还将NBGT应用程序和产品的使用与监管进行了简要的联系。

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