Clinical trial NCT05241444 - References

IPEX — CD4^LVFOXP3 in Participants With IPEX

Citation(s)

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  Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study. J Allergy Clin Immunol. 2018 Mar;141(3):1036-1049.e5. doi: 10.1016/j.jaci.2017.10.041. Epub 2017 Dec 11.
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  Demethylation analysis of the FOXP3 locus shows quantitative defects of regulatory T cells in IPEX-like syndrome. J Autoimmun. 2012 Feb;38(1):49-58. doi: 10.1016/j.jaut.2011.12.009. Epub 2012 Jan 20.
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  Rapamycin selectively expands CD4+CD25+FoxP3+ regulatory T cells. Blood. 2005 Jun 15;105(12):4743-8. doi: 10.1182/blood-2004-10-3932. Epub 2005 Mar 3.
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•   Consonni F, Ciullini Mannurita S, Gambineri E
  Atypical Presentations of IPEX: Expect the Unexpected. Front Pediatr. 2021 Feb 5;9:643094. doi: 10.3389/fped.2021.643094. eCollection 2021.
•   Cornetta K, Duffy L, Turtle CJ, Jensen M, Forman S, Binder-Scholl G, Fry T, Chew A, Maloney DG, June CH
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  A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndrome. Blood. 2021 Apr 29;137(17):2326-2336. doi: 10.1182/blood.2020009187.
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  Clinical, Immunological, and Molecular Heterogeneity of 173 Patients With the Phenotype of Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked (IPEX) Syndrome. Front Immunol. 2018 Nov 1;9:2411. doi: 10.3389/fimmu.2018.02411. eCollection 2018.
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  Treatment with rapamycin can restore regulatory T-cell function in IPEX patients. J Allergy Clin Immunol. 2020 Apr;145(4):1262-1271.e13. doi: 10.1016/j.jaci.2019.11.043. Epub 2019 Dec 23.
•   Passerini L, Rossi Mel E, Sartirana C, Fousteri G, Bondanza A, Naldini L, Roncarolo MG, Bacchetta R
  CD4(+) T cells from IPEX patients convert into functional and stable regulatory T cells by FOXP3 gene transfer. Sci Transl Med. 2013 Dec 11;5(215):215ra174. doi: 10.1126/scitranslmed.3007320.
•   Sakaguchi S, Sakaguchi N, Asano M, Itoh M, Toda M
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•   Sato Y, Passerini L, Piening BD, Uyeda MJ, Goodwin M, Gregori S, Snyder MP, Bertaina A, Roncarolo MG, Bacchetta R
  Human-engineered Treg-like cells suppress FOXP3-deficient T cells but preserve adaptive immune responses in vivo. Clin Transl Immunology. 2020 Nov 25;9(11):e1214. doi: 10.1002/cti2.1214. eCollection 2020.

Phase 1 Study of Autologous CD4^LVFOXP3 in Participants With Immune Dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) Syndrome

Details for clinical trial NCT05241444