Partial Correction of Immunodeficiency by Lentiviral Vector Gene Therapy in Mouse Models Carrying Rag1 Hypomorphic Mutations

Maria Carmina Castiello,Martina Di Verniere,Elena Draghici,Elena Fontana,Sara Penna,Lucia Sereni, Alessandra Zecchillo,Denise Minuta,Paolo Uva,Marco Zahn,Irene Gil-Farina,Andrea Annoni, Silvia Iaia,Lisa M. Ott de Bruin,Luigi D. Notarangelo,Karin Pike-Overzet,Frank J. T. Staal,Anna Villa,Valentina Capo

Frontiers in immunology（2023）

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关键词
RAG1 gene,gene therapy,immunodeficiency,immune dysregulation,lentiviral vectors,autoimmunity,leaky SCID
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