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Article

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

The Journal of allergy and clinical immunology - 1 Apr 2020

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

Abstract excerpt

BACKGROUND: 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...

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Identifiers

PMID: 31874182
DOI: 10.1016/j.jaci.2019.11.043

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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