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Correcting tyrosinaemia via a point mutation.
Nature Biomedical Engineering ( IF 26.8 ) Pub Date : 2020-01-01 , DOI: 10.1038/s41551-019-0489-x
Kiran Musunuru 1
Affiliation  

In an adult mouse model of tyrosinaemia, a base editor correcting an A-to-G splice-site mutation in the Fah gene restores the translation of the functional enzyme, promoting the repopulation of the liver with the corrected cells.

中文翻译:

通过点突变纠正酪氨酸血症。

在酪氨酸血症的成年小鼠模型中,校正Fah基因中A至G剪接位点突变的碱基编辑者恢复了功能酶的翻译,从而促进了肝细胞在校正后的细胞中的繁殖。
更新日期:2020-01-15
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