A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency
一种不依赖突变的 CRISPR-Cas9 介导的基因靶向方法,用于治疗鸟氨酸转氨甲酰酶缺乏症的小鼠模型
期刊:Science Advances
影响因子:11.7
doi:10.1126/sciadv.aax5701
Lili Wang, Yang Yang, Camilo Breton, Peter Bell, Mingyao Li, Jia Zhang, Yan Che, Alexei Saveliev, Zhenning He, John White, Caitlin Latshaw, Chenyu Xu, Deirdre McMenamin, Hongwei Yu, Hiroki Morizono, Mark L Batshaw, James M Wilson