Biblio
Autoantibodies to Harmonin and Villin Are Diagnostic Markers in Children with IPEX Syndrome. PLoS One. 2013;8(11):e78664.
. Fatal autoimmunity in mice reconstituted with human hematopoietic stem cells encoding defective FOXP3. Blood. 2015.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. N Engl J Med. 2021;385(21):1929-1940.
Treatment with Rapamycin Can Restore Regulatory T Cell Function in IPEX Patients. J Allergy Clin Immunol. 2019.