Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy
#CHIR 99021 ; #2520691 ; J Clin Invest. 2023 Aug 10;e171356. doi: 10.1172/JCI171356. Meha Kabra 1 2, Pawan K Shahi 1 2, Yuyuan Wang 3 4, Divya Sinha 2 5, Allison Spillane 1, Gregory A Newby 6 7 8, Shivani Saxena 2 3 4, Yao Tong 3 4, Yu Chang 1, Amr A Abdeen 2 3 4, Kimberly L Edwards 2 5, Cole O Theisen 5, David R Liu 6 7 8, David M Gamm 2 5 9, Shaoqin Gong 2 3 4 9, Krishanu Saha 1 2 3 4 10, Bikash R Pattnaik 1 2 9 Affiliations expand Free PMC article Abstract Clinical genome editing is emerging for rare disease treatment, but one …