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Towards a new era in medicine: therapeutic genome editing

机译:迈向医学新时代:治疗性基因组编辑

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摘要

Genome editing is the process of precisely modifying the nucleotide sequence of the genome. It has provided a powerful approach to research questions but, with the development of a new set of tools, it is now possible to achieve frequencies of genome editing that are high enough to be useful therapeutically. Genome editing is being developed to treat not only monogenic diseases but also infectious diseases and diseases that have both a genetic and an environmental component.
机译:基因组编辑是精确修饰基因组核苷酸序列的过程。它为研究问题提供了一种有力的方法,但是随着一套新工具的开发,现在有可能实现足够高的基因组编辑频率,从而在治疗上有用。正在开发基因组编辑以不仅治疗单基因疾病,而且还治疗传染病和具有遗传和环境成分的疾病。

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