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

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, hemat...

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Veröffentlicht in:	Journal of allergy and clinical immunology 2020-04, Vol.145 (4), p.1262-1271.e13
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	autoimmunity Cell Movement Cells, Cultured Child Diabetes Mellitus, Type 1 - congenital Diabetes Mellitus, Type 1 - drug therapy Diabetes Mellitus, Type 1 - genetics Diabetes Mellitus, Type 1 - immunology Diarrhea - drug therapy Diarrhea - genetics Diarrhea - immunology Ebi3 Forkhead Transcription Factors - genetics FOXP3 Gene Expression Regulation Genetic Diseases, X-Linked - drug therapy Genetic Diseases, X-Linked - genetics Genetic Diseases, X-Linked - immunology GITR Glucocorticoid-Induced TNFR-Related Protein - metabolism Humans Immune System Diseases - congenital Immune System Diseases - drug therapy Immune System Diseases - genetics Immune System Diseases - immunology Immune Tolerance Immunosuppressive Agents - therapeutic use Interleukins - genetics Interleukins - metabolism IPEX Lymphocyte Activation Male Minor Histocompatibility Antigens - genetics Minor Histocompatibility Antigens - metabolism mTOR Mutation - genetics rapamycin regulatory T cells Sirolimus - therapeutic use suppression T-Lymphocytes, Regulatory - immunology
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