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    Article

    Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer

    Severe combined immunodeficiency (SCID) patients with an inactivating mutation in recombination activation gene 1 (RAG1) lack B and T cells due to the inability to rearrange immunoglobulin (Ig) and T-cell recepto...

    K Pike-Overzet, M Rodijk, Y-Y Ng, M R M Baert, C Lagresle-Peyrou, A Schambach in Leukemia (2011)

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    Article

    Correction of B-cell development in Btk-deficient mice using lentiviral vectors with codon-optimized human BTK

    X-linked agammaglobulinemia (XLA) is the most common primary immunodeficiency (PID) in man and caused by mutations in the Bruton’s tyrosine kinase (BTK) gene. XLA is characterized by a B-cell differentiation arre...

    Y Y Ng, M R M Baert, K Pike-Overzet, M Rodijk, M H Brugman, A Schambach, C Baum in Leukemia (2010)