The difficult translational pathway from animal models to patients

Cell Stem Cell. 2024 Apr 4;31(4):435-436. doi: 10.1016/j.stem.2024.03.010.ABSTRACTLee 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.PMID:38579680 | DOI:10.1016/j.stem.2024.03.010
Source: Cell Stem Cell - Category: Stem Cells Authors: Source Type: research