Efficacy assessment of lentiviral vector gene therapy in the immune dysregulated hypomorphic Rag1 mice

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Authors: V Capo, M C Castiello, E Draghici, S Penna, M Di Verniere, E Fontana, D Minuta, S Porcellini, P Uva, LD Notarangelo, K Pike-Overzet, FJT Staal, A Villa1

Journal title: Human Gene Therapy

Journal publisher: Mary Ann Liebert, Inc.

Published year: 2021

DOI identifier: 10.1089/hum.2021.29180.abstracts