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  1. No Access

    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)

  2. No Access

    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)

  3. No Access

    Article

    Flow cytometric immunobead assay for the detection of BCR–ABL fusion proteins in leukemia patients

    BCR–ABL fusion proteins show increased signaling through their ABL tyrosine kinase domain, which can be blocked by specific inhibitors, thereby providing effective treatment. This makes detection of BCR–ABL aberr...

    F Weerkamp, E Dekking, Y Y Ng, V H J van der Velden, H Wai, S Böttcher… in Leukemia (2009)

  4. No Access

    Article

    Sola dosis facit venenum. Leukemia in gene therapy trials: a question of vectors, inserts and dosage?

    In clinical gene therapy trials for X-linked severe combined immunodeficiency, the development of leukemia has come up as a severe adverse effect. In all five cases, T-cell acute lymphoblastic leukemia (T-ALL)...

    F J T Staal, K Pike-Overzet, Y Y Ng, J J M van Dongen in Leukemia (2008)