According to the attached PDF, what were the main gene editing approaches compared in the murine sickle cell disease model, and what was the key long-term hematopoiesis finding?
The murine sickle-cell model compared three ex vivo approaches in CD34+ haematopoietic stem and progenitor cells: CRISPR–Cas9 editing, lentiviral transduction, and base editing. The edited or transduced cells were transplanted into busulfan-conditioned NBSGW mice. [citation_18638e]
The key long-term haematopoiesis finding was that all three groups achieved broadly similar human CD45+ bone-marrow engraftment at 16 weeks (approximately 75–90%), and a secondary-transplantation experiment produced similar results, indicating persistence of long-term repopulating activity. In the competitive-transplant setting, however, CRISPR–Cas9 produced less BCL11A-enhancer editing than base editing or lentiviral transduction; base editing and lentiviral transduction also produced greater reduction in red-cell sickling than CRISPR–Cas9. [citation_18638e]