The Journal of allergy and clinical immunology, vol.141, no.3, 2018 (Journal Indexed in SCI)
Article / Article
Title of Journal :
The Journal of allergy and clinical immunology
IPEX, primary immune deficiency, FOXP3, Treg cells, hematopoietic stem cell transplantation, immunosuppression, rapamycin, enteropathy, neonatal diabetes, genetic autoimmunity, X-LINKED SYNDROME, STEM-CELL TRANSPLANTATION, REGULATORY T-CELLS, IMMUNE DYSREGULATION, POLYENDOCRINOPATHY, ENTEROPATHY, IMMUNODYSREGULATION, ENGRAFTMENT, MUTATIONS, SIROLIMUS
Background: Immunodysregulation polyendocrinopathy enteropathy x-linked(IPEX) syndrome is a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT) and immunosuppression (IS), have not been thoroughly examined.