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Systemic delivery of phagocytosis-shielded retroviral vectors enables in vivo HSC gene therapy for sickle cell disease

Journal content Created on 24 Sep 2026 Cell Stem Cell

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Cell Stem Cell, Cell Press (Reed Elsevier)

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Denise Klatt, Adele Mucci, Boya Liu, Marie Peschers, Giulia Schiroli, Axel Schambach, John Manis, Myriam Armant, David T. Scadden, Els Verhoeyen, Danilo Pellin, David A. Williams, Christian Brendel

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