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Gene therapy: therapeutic gene causing lymphoma

Niels-Bjarne Woods1, Virginie Bottero, Manfred Schmidt

  • 1Laboratory of Genetics, The Salk Institute for Biological Studies, La Jolla, California 92037, USA.

Nature
|April 28, 2006
PubMed

Insights

Gene therapy for X-linked severe combined immune deficiency (X-SCID) using the IL2RG gene may pose risks. Studies show the therapeutic gene itself can contribute to T-cell lymphoma development in mouse models.

Area of Science:

  • Immunology
  • Gene Therapy
  • Oncology

Background:

  • Gene therapy using hematopoietic stem cells has shown promise for treating X-linked severe combined immune deficiency (X-SCID).
  • However, recent trials have seen the development of T-cell leukemia in treated patients, prompting a re-evaluation of the safety of this approach.

Purpose of the Study:

  • To investigate the potential oncogenic role of the IL2RG gene, the corrective therapeutic gene used in X-SCID gene therapy.
  • To assess the risk of T-cell lymphoma development associated with IL2RG gene therapy in a preclinical model.

Main Methods:

  • A mouse model was utilized to simulate gene therapy for X-SCID.
  • The study focused on the impact of the IL2RG gene as a therapeutic agent.

Main Results:

  • The IL2RG gene was found to contribute to the development of T-cell lymphomas in a significant portion (one-third) of the treated animals.
  • This finding challenges the previous assumption of IL2RG being minimally oncogenic.

Conclusions:

  • Gene therapy for X-SCID utilizing the IL2RG gene may carry an inherent risk of inducing T-cell lymphomas.
  • These findings necessitate a careful re-evaluation of the safety protocols and risk-benefit analysis for X-SCID gene therapy trials.

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