DNA-PK inhibition enhances gene editing efficiency in HSPCs for CRISPR-based treatment of X-linked hyper IgM syndrome.
DNA-PK 抑制剂可提高 HSPC 中基因编辑的效率,从而利用 CRISPR 技术治疗 X 连锁高 IgM 综合征
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2024.101297
Pugliano Cole M, Berger Mason, Ray Roslyn M, Sapkos Kai, Wu Betty, Laird Aidan, Ye Yidian, Thomson Daniel, DeGottardi M Quinn, Khan Iram F, Tatiossian Kristina, Miles Brodie A, Aeschimann Florian, Pasquier Jerome, Kim Mihee M, Rawlings David J