Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2006 Apr 27;440(7088):1123.
doi: 10.1038/4401123a.

Gene therapy: therapeutic gene causing lymphoma

Niels-Bjarne Woods  1 , Virginie Bottero, Manfred Schmidt, Christof von Kalle, Inder M Verma
Affiliations

Gene therapy: therapeutic gene causing lymphoma

Niels-Bjarne Woods et al. Nature. .

Abstract

The development of T-cell leukaemia following the otherwise successful treatment of three patients with X-linked severe combined immune deficiency (X-SCID) in gene-therapy trials using haematopoietic stem cells has led to a re-evaluation of this approach. Using a mouse model for gene therapy of X-SCID, we find that the corrective therapeutic gene IL2RG itself can act as a contributor to the genesis of T-cell lymphomas, with one-third of animals being affected. Gene-therapy trials for X-SCID, which have been based on the assumption that IL2RG is minimally oncogenic, may therefore pose some risk to patients.

PubMed Disclaimer

Comment in

  • Gene therapy: X-SCID transgene leukaemogenicity.
    Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, Otsu M, Sorrentino B, Scobie L, Cameron E, Blyth K, Neil J, Abina SH, Cavazzana-Calvo M, Fischer A. Thrasher AJ, et al. Nature. 2006 Sep 21;443(7109):E5-6; discussion E6-7. doi: 10.1038/nature05219. Nature. 2006. PMID: 16988659
  • Gene therapy: is IL2RG oncogenic in T-cell development?
    Pike-Overzet K, de Ridder D, Weerkamp F, Baert MR, Verstegen MM, Brugman MH, Howe SJ, Reinders MJ, Thrasher AJ, Wagemaker G, van Dongen JJ, Staal FJ. Pike-Overzet K, et al. Nature. 2006 Sep 21;443(7109):E5; discussion E6-7. doi: 10.1038/nature05218. Nature. 2006. PMID: 16988660

Substances