Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations

IntroductionRecombination activating genes (RAG) 1 and 2 defects are the most frequent form of severe combined immunodeficiency (SCID). Patients with residual RAG activity have a spectrum of clinical manifestations ranging from Omenn syndrome to delayed-onset combined immunodeficiency, often associa...

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Autors principals:	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
Format:	Artigo
Idioma:	Inglês
Publicat:	Frontiers Media S.A. 2023-11-01
Col·lecció:	Frontiers in Immunology
Matèries:	RAG1 gene gene therapy immunodeficiency immune dysregulation lentiviral vectors autoimmunity
Accés en línia:	https://www.frontiersin.org/articles/10.3389/fimmu.2023.1268620/full
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