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Lunch & Learn: IPEX syndrome

View a PDF of this presentation: https://primaryimmune.org/sites/defau... Rosa Bachetta, MD provides an over view of IPEX syndrome. To learn more, volunteer, or donate, visit primaryimmune.org/donate. The information, terminology, and opinions presented in this forum do not necessarily reflect the views of IDF, its Board of Trustees, sponsors, or donors. LINKS: Stanford clinical trial: https://clinicaltrials.gov/study/NCT0... CRISPR-based gene editing enables FOXp3 gene repair in IPEX patient cells: https://www.science.org/doi/full/10.1.... Regulatory T cells and human disease: https://pubmed.ncbi.nlm.nih.gov/32017... Regulatory T cells: the many faces of FOXp3: https://pubmed.ncbi.nlm.nih.gov/31478... Emerging functions of regulatory t cells in tissue homeostasis:https://pubmed.ncbi.nlm.nih.gov/29887... Towards gene therapy for IPEX syndrome: https://pmc.ncbi.nlm.nih.gov/articles... From IPEX syndrome to FOXp3 mutation: a lesson on immune dysregulation: https://pubmed.ncbi.nlm.nih.gov/26918... FOXp3 deficiency, from the mechanisms of the disease to curative strategies: https://pubmed.ncbi.nlm.nih.gov/37994... Genetic engineering of regulatory T cells for treatment of autoimmune disorders including type 1 diabetes: https://pubmed.ncbi.nlm.nih.gov/38236... Treg cell-based therapies: challenges and perspectives: https://pubmed.ncbi.nlm.nih.gov/31811...