Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

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Authors: Laura Garcia-Perez, Marja van Eggermond, Lieke van Roon, Sandra A. Vloemans, Martijn Cordes, Axel Schambach, Michael Rothe, Dagmar Berghuis, Chantal Lagresle-Peyrou, Marina Cavazzana, Fang Zhang, Adrian J. Thrasher, Daniela Salvatori, Pauline Meij, Anna Villa, Jacques J.M. Van Dongen, Jaap-Jan Zwaginga, Mirjam van der Burg, H. Bobby Gaspar, Arjan Lankester, Frank J.T. Staal, Karin Pike-Overzet

Journal title: Molecular Therapy - Methods & Clinical Development

Journal number: 17

Journal publisher: Cell press

Published year: 2020

Published pages: 666-682

DOI identifier: 10.1016/j.omtm.2020.03.016

ISSN: 2329-0501