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The difficult translational pathway from animal models to patients
Cell Stem Cell ( IF 23.9 ) Pub Date : 2024-04-04 , DOI: 10.1016/j.stem.2024.03.010
Marina Cavazzana , Annarita Miccio

Lee et al.1 analyzed the impacts of lentiviral vector transduction and CRISPR-Cas9/homology-directed repair editing on hematopoietic stem and progenitor cell (HSPC) engraftment and clonal dynamics. The study suggests that relative to lentiviral-vector-mediated gene addition, homology-directed repair editing is inefficient in vivo and might impair the engraftment and differentiation of HSPCs.



中文翻译:

从动物模型到患者的艰难转化途径

李等人。图 1分析了慢病毒载体转导和 CRISPR-Cas9/同源定向修复编辑对造血干细胞和祖细胞 (HSPC) 植入和克隆动力学的影响。该研究表明,相对于慢病毒载体介导的基因添加,同源定向修复编辑在体内效率低下,可能会损害 HSPC 的植入和分化。

更新日期:2024-04-04
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