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Other/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
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. (Butt, 2026)

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. (Butt, 2026)

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In one sentence, state which approach showed lower long-term hematopoietic contribution in the competitive transplant setting, using the attached PDF.

In the competitive-transplant setting, CRISPR–Cas9 editing showed the lower long-term haematopoietic contribution, reflected by less BCL11A-enhancer editing than base editing or lentiviral transduction. (Butt, 2026)

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Butt H, Sathish S, London E, Le A, Li Q, Gudmundsdottir B, Lee DY, Burke EV, Yates BP, Liu DR, Hsieh M, Leonard A, Eaton WA, Uchida N, Pierciey FJ, Newby GA, Tisdale JF, Demirci S. · 2026 ·

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Abstract
Source 1: Comparative analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model.

Butt H, Sathish S, +16 · 2026 · 1 citations

10.1182/bloodadvances.2025017321

Abstract

Sickle cell disease (SCD) is a red blood cell disorder caused by a mutation in the β-globin gene, leading to sickle hemoglobin polymerization under low oxygen conditions. Both CRISPR-Cas9 editing and lentiviral transduction have shown promising clinical outcomes, but it remains unclear which approach is superior. Alternatively, new editing tools such as base editing may also be pr…
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