Direct thymine editing
A Deinococcus radiodurans UNG mutant fused with nCas9 enables efficient and low-toxicity thymine base editing, restoring IDUA enzyme activity in Hurler syndrome cells.
Yi, Zongyi · Li, Jiayi · Tang, Huixian · Yu, Ying · Wei, Wensheng
Nature Communications 2024
Minimal cellular toxicity
High editing specificity
Significantly restores IDUA enzyme activity in cells from Hurler syndrome patients