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

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Bibliographic Details
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
Database: MEDLINE Ultimate
Description
ISSN:2473-9537
DOI:10.1182/bloodadvances.2025017321