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Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side by Laura Garcia-Perez, Anita Ordas, Kirsten Canté-Barrett, Pauline Meij, Karin Pike-Overzet, Arjan Lankester, Frank J. T. Staal
Published 2020-06-01
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Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper‐IgM syndrome by Valentina Vavassori, Elisabetta Mercuri, Genni E Marcovecchio, Maria C Castiello, Giulia Schiroli, Luisa Albano, Carrie Margulies, Frank Buquicchio, Elena Fontana, Stefano Beretta, Ivan Merelli, Andrea Cappelleri, Paola MV Rancoita, Vassilios Lougaris, Alessandro Plebani, Maria Kanariou, Arjan Lankester, Francesca Ferrua, Eugenio Scanziani, Cecilia Cotta‐Ramusino, Anna Villa, Luigi Naldini, Pietro Genovese
Published 2021-03-01
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