In ß-thalassemia, either -globin induction to form fetal hemoglobin (a22) or ß-globin repair to restore adult hemoglobin (a2ß2) could be therapeutic. ABE8e, a recently evolved adenine base editor variant, can achieve efficient adenine conversion, yet...
Authors: Liao J, Chen S, Hsiao S, Jiang Y, Yang Y, Zhang Y, Wang X, Lai Y, Bauer DE, Wu Y
SST Peer Reviewed