Direct delivery of stabilized Cas-embedded base editors achieves efficient and accurate editing of clinically relevant targets
Jeong Min Lee, Jing Zeng, Pengpeng Liu, My Anh Nguyen, Diego Suchenski Loustaunau, Daniel E. Bauer, Nese Kurt Yilmaz, Scot A. Wolfe, Celia A. Schiffer
This study develops stabilized Cas-embedded base editors (sCE_CBEs) that enable efficient and accurate C-to-T editing with minimal off-target effects in hematopoietic stem and progenitor cells.
bioRxiv · Feb 8, 2024 · PreprintBiochemistry