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

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Title: Comparative analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model.
Authors: Butt H; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.; Center for Cancer and Blood Disorders, Children's National Hospital, Washington, DC., Sathish S; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD., London E; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD., Le A; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD., Li Q; Laboratory of Chemical Physics, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD., Gudmundsdottir B; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD., Lee DY; Laboratory of Chemical Physics, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD., Burke EV; Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD., Yates BP; Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD., Liu DR; Merkin Institute for Transformative Technologies in Healthcare, Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA., Hsieh M; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD., Leonard A; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Eaton WA; Laboratory of Chemical Physics, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD., Uchida N; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD., Pierciey FJ Jr; Bluebird Bio, Inc, Somerville, MA., Newby GA; Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD.; Merkin Institute for Transformative Technologies in Healthcare, Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA.; Institute for NanoBioTechnology, Johns Hopkins University, Baltimore, MD., Tisdale JF; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD., Demirci S; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.
Source: Blood advances [Blood Adv] 2026 Jan 27; Vol. 10 (2), pp. 289-300.
Publication Type: Journal Article; Comparative Study
Journal Info: Publisher: American Society of Hematology Country of Publication: United States NLM ID: 101698425 Publication Model: Print Cited Medium: Internet ISSN: 2473-9537 (Electronic) Linking ISSN: 24739529 NLM ISO Abbreviation: Blood Adv Subsets: MEDLINE
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  Data: Comparative analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model.
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  Data: <searchLink fieldCode="AU" term="%22Butt+H%22">Butt H</searchLink>; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.; Center for Cancer and Blood Disorders, Children's National Hospital, Washington, DC.<br /><searchLink fieldCode="AU" term="%22Sathish+S%22">Sathish S</searchLink>; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22London+E%22">London E</searchLink>; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Le+A%22">Le A</searchLink>; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Li+Q%22">Li Q</searchLink>; Laboratory of Chemical Physics, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Gudmundsdottir+B%22">Gudmundsdottir B</searchLink>; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Lee+DY%22">Lee DY</searchLink>; Laboratory of Chemical Physics, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Burke+EV%22">Burke EV</searchLink>; Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD.<br /><searchLink fieldCode="AU" term="%22Yates+BP%22">Yates BP</searchLink>; Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD.<br /><searchLink fieldCode="AU" term="%22Liu+DR%22">Liu DR</searchLink>; Merkin Institute for Transformative Technologies in Healthcare, Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA.<br /><searchLink fieldCode="AU" term="%22Hsieh+M%22">Hsieh M</searchLink>; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Leonard+A%22">Leonard A</searchLink>; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN.<br /><searchLink fieldCode="AU" term="%22Eaton+WA%22">Eaton WA</searchLink>; Laboratory of Chemical Physics, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Uchida+N%22">Uchida N</searchLink>; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Pierciey+FJ+Jr%22">Pierciey FJ Jr</searchLink>; Bluebird Bio, Inc, Somerville, MA.<br /><searchLink fieldCode="AU" term="%22Newby+GA%22">Newby GA</searchLink>; Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD.; Merkin Institute for Transformative Technologies in Healthcare, Broad Institute of Massachusetts Institute of Technology and Harvard, Cambridge, MA.; Institute for NanoBioTechnology, Johns Hopkins University, Baltimore, MD.<br /><searchLink fieldCode="AU" term="%22Tisdale+JF%22">Tisdale JF</searchLink>; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.<br /><searchLink fieldCode="AU" term="%22Demirci+S%22">Demirci S</searchLink>; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute/National Institutes of Health, Bethesda, MD.
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  Data: <searchLink fieldCode="JN" term="%22101698425%22">Blood advances</searchLink> [Blood Adv] 2026 Jan 27; Vol. 10 (2), pp. 289-300.
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        Value: 10.1182/bloodadvances.2025017321
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        Text: English
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