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LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.

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Affiliations

  • 1. INSERM Unit 429, Cedex 15, France.
      Authors
    • Hacein-Bey-Abina S 1
    (1 author)

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Science, 01 Oct 2003, 302(5644):415-419
https://doi.org/10.1126/science.1088547 PMID: 14564000 

This article has been corrected. See Science. 2003 Oct 24;302(5645):568. A comment on this article appears in "Medicine. Gene therapy--new challenges ahead." Science. 2003 Oct 17;302(5644):400-1.

Abstract 

We have previously shown correction of X-linked severe combined immunodeficiency [SCID-X1, also known as gamma chain (gamma(c)) deficiency] in 9 out of 10 patients by retrovirus-mediated gamma(c) gene transfer into autologous CD34 bone marrow cells. However, almost 3 years after gene therapy, uncontrolled exponential clonal proliferation of mature T cells (with gammadelta+ or alphabeta+ T cell receptors) has occurred in the two youngest patients. Both patients' clones showed retrovirus vector integration in proximity to the LMO2 proto-oncogene promoter, leading to aberrant transcription and expression of LMO2. Thus, retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.

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Read article at publisher's site: https://doi.org/10.1126/science.1088547

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