从动物模型到患者的艰难转化途径。

The difficult translational pathway from animal models to patients.

机构信息

Université Paris Cité, Paris, France; Département de Biothérapie Hospital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France; Centre d'Investigation Clinique en Biothérapie, INSERM UMR1163, Paris, France; Imagine Institute, Paris, France.

Université Paris Cité, Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR1163, Paris, France.

出版信息

Cell Stem Cell. 2024 Apr 4;31(4):435-436. doi: 10.1016/j.stem.2024.03.010.

DOI:10.1016/j.stem.2024.03.010

PMID:38579680

Abstract

Lee et al. 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.

摘要

李等人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，与慢病毒载体介导的基因添加相比，同源定向修复编辑在体内效率较低，并且可能损害 HSPC 的植入和分化。