Adenine base editing corrects point mutation in mitochondrial single-stranded binding protein (SSBP1) to improve mitochondrial function
Ju Hyuen Cha, Seok‐Hoon Lee, Yejin Yun, Won Hoon Choi, Hansol Koo, Sung Ho Jung, Ho Byung Chae, Dae Hee Lee, Seok Jae Lee, Dong Hyun Jo, Jeong Hun Kim, Jae‐Jin Song, Jong‐Hee Chae, Jun Ho Lee, Seung Ha Oh, , Sang‐Yeon Lee
This study corrects a SSBP1 mutation using adenine base editors, improving mitochondrial function but with trade-offs between editing efficacy and off-target risks.
medRxiv · Nov 2, 2023 · PreprintGenetic & Genomic Medicine