Treatment with rapamycin can restore regulatory T-cell function in IPEX patients

Laura Passerini, Federica Barzaghi, Rosalia Curto, Claudia Sartirana, Graziano Barera, Francesca Tucci, Luca Albarello, Alberto Mariani, Pier Alberto Testoni, Elena Bazzigaluppi, Emanuele Bosi, Vito Lampasona, Olaf Neth, Daniele Zama, Manfred Hoenig, Ansgar Schulz, Markus G Seidel, IVANA RABBONE, Sven Olek, Maria G RoncaroloMaria P Cicalese, Alessandro Aiuti, Rosa Bacchetta

Research output: Contribution to journalArticlepeer-review

Abstract

Immune-dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is a lethal disease caused by mutations in a transcription factor critical for the function of thymus-derived regulatory T (Treg) cells (ie, FOXP3), resulting in impaired Treg function and autoimmunity. At present, hematopoietic stem cell transplantation is the therapy of choice for patients with IPEX syndrome. If not available, multiple immunosuppressive regimens have been used with poor disease-free survival at long-term follow-up. Rapamycin has been shown to suppress peripheral T cells while sparing Treg cells expressing wild-type FOXP3, thereby proving beneficial in the clinical setting of immune dysregulation. However, the mechanisms of immunosuppression selective to Treg cells in patients with IPEX syndrome are unclear.
Original languageEnglish
Pages (from-to)1262-1271
Number of pages10
JournalJournal of Allergy and Clinical Immunology
Volume145
Issue number4
DOIs
Publication statusPublished - 2020

Keywords

  • Ebi3
  • FOXP3
  • GITR
  • IPEX
  • autoimmunity
  • mTOR
  • rapamycin
  • regulatory T cells
  • suppression

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