Template-independent genome editing and repairing correct frameshift disease in vivo
Lian Liu, Kuan Li, Linzhi Zou, Hanqing Hou, Qun Hu, Shuang Liu, Shufeng Wang, Yangzhen Wang, Jie Li, Chenmeng Song, Jiaofeng Chen, Changri Li, Haibo Du, Jun-Liszt Li, Fangyi Chen, , Wei Xiong
This study demonstrates that NHEJ-mediated frame restoration can efficiently correct frameshift mutations in vivo, restoring function in a mouse model of Usher syndrome.
bioRxiv · Nov 13, 2020 · PreprintNeuroscience