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通过简单的核酶诱导的双链断裂进行精确的治疗基因校正

Sukanya Iyer1, Sneha Suresh1, Dongsheng Guo2,3

  • 1Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, USA.

Nature
|April 5, 2019
PubMed
概括

这项研究引入了微复制突变的新基因校正方法. 通过创建DNA断裂, 它有效地将引起疾病的序列逆转为野生类型,

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