Article
Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations.
Frontiers in immunology - 1 Jan 2023
Castiello Maria Carmina, Di Verniere Martina, Draghici Elena, Fontana Elena, Penna Sara, Sereni Lucia, Zecchillo Alessandra, Minuta Denise, Uva Paolo, Zahn Marco, Gil-Farina Irene, Annoni Andrea, Iaia Silvia, Ott de Bruin Lisa M, Notarangelo Luigi D, Pike-Overzet Karin, Staal Frank J T, Villa Anna, Capo Valentina
Abstract excerpt
Introduction: Recombination 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 associated with granulomas and/or autoimmunity (CID-G/AI). Lentiviral vector (LV) gene therapy (GT) has been proposed as...
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