The difficult translational pathway from animal models to patients

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.