Nonviral base editing of KCNJ13 mutation preserves vision in an inherited retinal channelopathy
Meha Kabra, Pawan Kumar Shahi, Yuyuan Wang, Divya Sinha, Allison Spillane, Gregory A. Newby, Shivani Saxena, Yao Tong, Yu Chang, Amr A. Abdeen, Kimberly L. Edwards, Cole O. Theisen, David R. Liu, David M. Gamm, Shaoqin Gong, , Bikash Ranjan Pattnaik
This paper demonstrates nonviral base editing of a KCNJ13 mutation in a mouse model of retinal degeneration, preserving vision through functional rescue of Kir7.1 channels.
bioRxiv · Jul 12, 2022 · PreprintGenetics