Malignant Tregs express low molecular splice forms of FOXP3 in Sézary syndrome

Leukemia. 2008 Dec;22(12):2230-9. doi: 10.1038/leu.2008.224. Epub 2008 Sep 4.

Abstract

Sézary syndrome (SS) is an aggressive variant of cutaneous T-cell lymphoma. During disease progression, immunodeficiency develops; however, the underlying molecular and cellular mechanisms are not fully understood. Here, we study the regulatory T cell (Treg) function and the expression of FOXP3 in SS. We demonstrate that malignant T cells in 8 of 15 patients stain positive with an anti-FOXP3 antibody. Western blotting analysis shows expression of two low molecular splice forms of FOXP3, but not of wild-type (wt) FOXP3. The malignant T cells produce interleukin-10 and TGF-beta and suppress the growth of non-malignant T cells. The Treg phenotype and the production of suppressive cytokines are driven by aberrant activation of Jak3 independent of the FOXP3 splice forms. In contrast to wt FOXP3, the low molecular splice forms of FOXP3 have no inhibitory effect on nuclear factor-kappaB (NF-kappaB) activity in reporter assays which is in keeping with a constitutive NF-kappaB activity in the malignant T cells. In conclusion, we show that the malignant T cells express low molecular splice forms of FOXP3 and function as Tregs. Furthermore, we provide evidence that FOXP3 splice forms are functionally different from wt FOXP3 and not involved in the execution of the suppressive function. Thus, this is the first description of FOXP3 splice forms in human disease.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Alternative Splicing
  • Antigens, CD / metabolism
  • CTLA-4 Antigen
  • Cell Line, Tumor
  • Female
  • Forkhead Transcription Factors / genetics*
  • Forkhead Transcription Factors / metabolism
  • Gene Expression Regulation, Neoplastic
  • Humans
  • Immunophenotyping
  • Interleukin-10 / metabolism
  • Interleukin-2 Receptor alpha Subunit / metabolism
  • Janus Kinase 3 / metabolism
  • Luciferases / genetics
  • Male
  • Middle Aged
  • NF-kappa B / metabolism
  • RNA, Small Interfering
  • STAT5 Transcription Factor / metabolism
  • Sezary Syndrome / genetics*
  • Sezary Syndrome / immunology*
  • Sezary Syndrome / pathology
  • Skin Neoplasms / genetics*
  • Skin Neoplasms / immunology*
  • Skin Neoplasms / pathology
  • T-Lymphocytes, Regulatory / pathology
  • T-Lymphocytes, Regulatory / physiology*
  • Transforming Growth Factor beta / metabolism

Substances

  • Antigens, CD
  • CTLA-4 Antigen
  • CTLA4 protein, human
  • FOXP3 protein, human
  • Forkhead Transcription Factors
  • IL10 protein, human
  • Interleukin-2 Receptor alpha Subunit
  • NF-kappa B
  • RNA, Small Interfering
  • STAT5 Transcription Factor
  • Transforming Growth Factor beta
  • Interleukin-10
  • Luciferases
  • JAK3 protein, human
  • Janus Kinase 3