Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome

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Authors: Vavassori, Valentina; Mercuri, Elisabetta; Marcovecchio, Genni E.; Castiello, Maria C.; Schiroli, Giulia; Albano, Luisa; Margulies, Carrie; Buquicchio, Frank; Fontana, Elena; Beretta, Stefano; Merelli, Ivan; Cappelleri, Andrea; Rancoita, Paola M.V.; Lougaris, Vassilios; Plebani, Alessandro; Kanariou, Maria; Lankester, Arjan; Ferrua, Francesca; Scanziani, Eugenio; Cotta-Ramusino, Cecilia; Villa, An

Journal title: EMBO Molecular Medicine

Journal number: 13(3)

Journal publisher: John Wiley & Sons Ltd.

Published year: 2021

DOI identifier: 10.15252/emmm.202013545

ISSN: 1757-4676