Comparative analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model.
在小鼠模型中对 CRISPR-Cas9、慢病毒转导和碱基编辑治疗镰状细胞病进行比较分析。
期刊:Blood Advances
影响因子:7.1
doi:10.1182/bloodadvances.2025017321
Butt Henna, Sathish Shruti, London Evan, Le Anh, Li Quan, Gudmundsdottir Bjorg, Lee Duck-Yeon, Burke Emma V, Yates Bradley P, Liu David R, Hsieh Matthew, Leonard Alexis, Eaton William A, Uchida Naoya, Pierciey Francis J Jr, Newby Gregory A, Tisdale John F, Demirci Selami