The difficult translational pathway from animal models to patients.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 38579680.
- Also identified by DOI 10.1016/j.stem.2024.03.010.
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Abstract
Lee et al.<sup>1</sup> 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.
Medical subject headings
- Gene Editing
- Hematopoietic Stem Cell Transplantation