The difficult translational pathway from animal models to patients
CELL STEM CELL(2024)
摘要
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.
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