LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

  title={LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1},
  author={S. Hacein-Bey-Abina and C. von Kalle and M. Schmidt and M. McCormack and N. Wulffraat and P. Leboulch and A. Lim and C. Osborne and R. Pawliuk and E. Morillon and R. Sorensen and A. Forster and P. Fraser and J. Cohen and G. de Saint Basile and I. Alexander and U. Wintergerst and T. Frebourg and A. Aurias and D. Stoppalyonnet and S. Romana and I. Radford-Weiss and F. Gross and F. Valensi and E. Delabesse and E. Macintyre and F. Sigaux and J. Soulier and L. Leiva and M. Wissler and C. Prinz and T. Rabbitts and F. Le Deist and A. Fischer and M. Cavazzana‐Calvo},
  pages={415 - 419}
  • S. Hacein-Bey-Abina, C. von Kalle, +32 authors M. Cavazzana‐Calvo
  • Published 2003
  • Biology, Medicine
  • Science
  • We have previously shown correction of X-linked severe combined immunodeficiency [SCID-X1, also known as γ chain (γc) deficiency] in 9 out of 10 patients by retrovirus-mediated γc gene transfer into autologous CD34 bone marrow cells. However, almost 3 years after gene therapy, uncontrolled exponential clonal proliferation of mature T cells (with γδ+ or αβ+ T cell receptors) has occurred in the two youngest patients. Both patients' clones showed retrovirus vector integration in proximity to the… CONTINUE READING
    3,269 Citations
    Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.
    • 1,395
    • PDF
    Safety Modality for X-linked Severe Combined Immunodeficiency Gene
    • 2
    • PDF
    Assessing the risk of T-cell malignancies in mouse models of SCID-X1.
    • B. Sorrentino
    • Biology, Medicine
    • Molecular therapy : the journal of the American Society of Gene Therapy
    • 2010
    • 6
    Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?
    • 50
    [Gene therapy of SCID-X1].
    • 6


    T cell tumours of disparate phenotype in mice transgenic for Rbtn-2.
    • 83
    Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer
    • 529
    • PDF
    The T cell leukemia LIM protein Lmo2 is necessary for adult mouse hematopoiesis.
    • 328
    • PDF