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Article

Towards gene therapy for IPEX syndrome.

European journal of immunology - 1 May 2022

Borna Simon, Lee Esmond, Sato Yohei, Bacchetta Rosa

Abstract excerpt

Immune dysregulation polyendocrinopathy enteropathy X linked (IPEX) syndrome is an uncurable disease of the immune system, with immune dysregulation that is caused by mutations in FOXP3. Current treatment options, such as pharmacological immune suppression and allogeneic hematopoietic stem cell transplantation, have been beneficial but present limitations, and their life-long consequences are ill-defined. Other...

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Identifiers

PMID: 35355253
DOI: 10.1002/eji.202149210
PMCID: PMC9322407

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

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