Genetic & Genomic MedicinemedRxivPreprint
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…·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.
DOI 10.1101/2023.11.02.23297943