Treatment of GATA2 deficiency by allele-specific CRISPR-Cas9-directed gene correction in hematopoietic stem cells
利用等位基因特异性 CRISPR-Cas9 介导的基因编辑技术治疗造血干细胞中的 GATA2 缺陷
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2025.07.038.
Thomas Wisbech Skov ,Jonas Holst Wolff ,Didde Haslund ,Anne Louise Revenfeld ,Lilly van de Venn ,Sofie Rahbek Dorset ,Sujan Ravendran ,Sofie Eg Jørgensen ,Martin Kristian Thomsen ,Mette Holm ,Jacob E Corn ,Bjarne Kuno Møller ,Trine H Mogensen ,Rasmus O Bak ,Jacob Giehm Mikkelsen