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  1. No Access

    Article

    Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells

    Correction of patient-specific induced pluripotent stem cells (iPSC) upon gene delivery through retroviral vectors offers new treatment perspectives for monogenetic diseases. Gene-modified iPSC clones can be s...

    D Hoffmann, J W Schott, F K Geis, L Lange, F-J Müller, D Lenz, D Zychlinski… in Gene Therapy (2017)

  2. No Access

    Article

    Deoxycytidine-kinase knockdown as a novel myeloprotective strategy in the context of fludarabine, cytarabine or cladribine therapy

    N Lachmann, K Czarnecki, S Brennig, R Phaltane, M Heise, N Heinz, H Kempf… in Leukemia (2015)

  3. No Access

    Article

    Inducible T-cell receptor expression in precursor T cells for leukemia control

    Co-transplantation of hematopoietic stem cells with those engineered to express leukemia-reactive T-cell receptors (TCRs) and differentiated ex vivo into precursor T cells (preTs) may reduce the risk of leukemia ...

    S S Hoseini, M Hapke, J Herbst, D Wedekind, R Baumann, N Heinz, B Schiedlmeier… in Leukemia (2015)

  4. No Access

    Article

    Improved retroviral episome transfer of transcription factors enables sustained cell fate modification

    Retroviral vectors are versatile gene transfer vehicles widely used in basic research and gene therapy. Mutation of retroviral integrase converts these vectors into transient, integration-deficient gene delive...

    J W Schott, D Hoffmann, T Maetzig, F-J Müller, D Steinemann, D Zychlinski… in Gene Therapy (2014)

  5. No Access

    Article

    Activation of Evi1 inhibits cell cycle progression and differentiation of hematopoietic progenitor cells

    The transcription factor Evi1 has an outstanding role in the formation and transformation of hematopoietic cells. Its activation by chromosomal rearrangement induces a myelodysplastic syndrome with progression to...

    O S Kustikova, A Schwarzer, M Stahlhut, M H Brugman, T Neumann, M Yang, Z Li… in Leukemia (2013)

  6. No Access

    Article

    Efficient in vivo regulation of cytidine deaminase expression in the haematopoietic system using a doxycycline-inducible lentiviral vector system

    Regulated transgene expression may reduce transgene-specific and genotoxic risks associated with gene therapy. To prove this concept, we have investigated the suitability of doxycycline (Dox)-inducible human c...

    N Lachmann, S Brennig, N Pfaff, H Schermeier, J Dahlmann, R Phaltane… in Gene Therapy (2013)

  7. No Access

    Article

    MicroRNA-150-regulated vectors allow lymphocyte-sparing transgene expression in hematopoietic gene therapy

    Endogenous microRNA (miRNA) expression can be exploited for cell type-specific transgene expression as the addition of miRNA target sequences to transgenic cDNA allows for transgene downregulation specifically...

    N Lachmann, J Jagielska, D Heckl, S Brennig, N Pfaff, T Maetzig, U Modlich… in Gene Therapy (2012)

  8. No Access

    Article

    Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency

    Patients with X-linked severe combined immunodeficiency (SCID-X1) were successfully cured following gene therapy with a gamma-retroviral vector (gRV) expressing the common gamma chain of the interleukin-2 rece...

    J C M van der Loo, W P Swaney, E Grassman, A Terwilliger, T Higashimoto… in Gene Therapy (2012)

  9. No Access

    Article

    Epidermal growth factor improves lentivirus vector gene transfer into primary mouse hepatocytes

    Partial resistance of primary mouse hepatocytes to lentiviral (LV) vector transduction poses a challenge for ex vivo gene therapy protocols in models of monogenetic liver disease. We thus sought to optimize ex vi...

    M Rothe, I Rittelmeyer, M Iken, U Rüdrich, A Schambach, S Glage, M P Manns… in Gene Therapy (2012)

  10. No Access

    Article

    Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection

    The need for γ-retroviral (gRV) vectors with a self-inactivating (SIN) design for clinical application has prompted a shift in methodology of vector manufacturing from the traditional use of stable producer li...

    J C M van der Loo, W P Swaney, E Grassman, A Terwilliger, T Higashimoto… in Gene Therapy (2012)

  11. No Access

    Article

    CTF/NF1 transcription factors act as potent genetic insulators for integrating gene transfer vectors

    Gene transfer-based therapeutic approaches have greatly benefited from the ability of some viral vectors to efficiently integrate within the cell genome and ensure persistent transmission of newly acquired tra...

    A Gaussin, U Modlich, C Bauche, N J Niederländer, A Schambach, C Duros… in Gene Therapy (2012)

  12. 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)

  13. 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)

  14. No Access

    Article

    Mechanisms controlling titer and expression of bidirectional lentiviral and gammaretroviral vectors

    Bidirectional lentiviral vectors mediate expression of two or more cDNAs from a single internal promoter. In this study, we examined mechanisms that control titer and expression properties of this vector syste...

    T Maetzig, M Galla, M H Brugman, R Loew, C Baum, A Schambach in Gene Therapy (2010)

  15. No Access

    Article

    A new PG13-based packaging cell line for stable production of clinical-grade self-inactivating γ-retroviral vectors using targeted integration

    The clinical application of self-inactivating (SIN) retroviral vectors has been hampered by the lack of reliable and efficient vector production technologies. To enable production of SIN γ-retroviral vectors f...

    R Loew, Y Meyer, K Kuehlcke, L Gama-Norton, D Wirth, H Hauser, S Stein… in Gene Therapy (2010)

  16. No Access

    Article

    Zinc positive: tailored genome engineering meets reprogramming

    T Cathomen, A Schambach in Gene Therapy (2010)

  17. No Access

    Article

    Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells

    Gene therapy has proven to be of potential value for the correction of inherited hematopoietic disorders. However, the occurrence of severe side effects in some of the clinical trials has questioned the safety...

    B Moreno-Carranza, M Gentsch, S Stein, A Schambach, G Santilli, E Rudolf… in Gene Therapy (2009)

  18. No Access

    Article

    Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16

    Insertional activation of cellular proto-oncogenes by replication-defective retroviral vectors can trigger clonal dominance and leukemogenesis in animal models and clinical trials. Here, we addressed the leuke...

    U Modlich, A Schambach, M H Brugman, D C Wicke, S Knoess, Z Li, T Maetzig… in Leukemia (2008)

  19. No Access

    Article

    Gentherapie der SCID-X1

    SCID-X1 ist eine X-chromosomal vererbte Form der schweren kombinierten Immundefizienz, die durch inaktivierende Mutationen im IL2RG-Gen verursacht wird. Dieses Gen kodiert für die gemeinsame γ-Kette der Interl...

    Prof. Dr. Christopher Baum, A. Schambach… in Bundesgesundheitsblatt - Gesundheitsforsch… (2007)

  20. No Access

    Article

    Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors

    Retroviral vectors with self-inactivating (SIN) long-terminal repeats not only increase the autonomy of the internal promoter but may also reduce the risk of insertional upregulation of neighboring alleles. Ho...

    A Schambach, D Mueller, M Galla, M M A Verstegen, G Wagemaker, R Loew… in Gene Therapy (2006)

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