Fatal autoimmunity in mice reconstituted with human hematopoietic stem cells encoding defective FOXP3

Goettel JA1,2, Biswas S2,3, Lexmond WS1,2, Yeste A2,4, Passerini L5, Patel B2,4, Yang S6, Sun J2,3, Ouahed1,2, Shouval DS1,2, McCann KJ1, Horwitz BH2,7, Mathis D6, Milford EL2,8, Notarangelo LD2,9, Roncarolo MG5,10, Fiebiger E1,2, Marasco WA2,3, Bacchetta R5, Quintana FJ2,4, Pai SY2,11,12, Muise AM13, Snapper SB1,2,14 Department of Pediatrics, Division of Gastroenterology, Hepatology and Nutrition, Boston Children's Hospital, Boston MA1; Department of Medicine, Harvard Medical School, Boston MA2; Department of Cancer Immunology and AIDS, Dana-Farber Cancer Institute, Boston MA3; Center for Neurologic Diseases, Brigham and Women's Hospital, Boston MA4; Division of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Scientific Institute, San Raffaele Telethon Institute for Gene Therapy, Milan, Italy5; Division of Immunology, Department of Microbiology and Immunobiology, Harvard Medical School, Boston, MA6; Department of Pathology, Brigham and Women's Hospital, Boston, MA7; Renal Division of Tissue Typing Laboratory, Brigham and Women's Hospital, Boston, MA8; Division of Immunology and the Manton Center for Orphan Disease Research, Boston Children's Hospital, Boston, MA9; Vita-Salute San Raffaele University, Milan, Italy10; Division of Hematology-Oncology, Boston Children's Hospital, Boston, MA11; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA12; Division of Gastroenterology, Hepatology, and Nutrition, Department of Paediatrics, Hospital for Sick Children, Toronto, CA13; Division of Gastroenterology, Brigham and Women's Hospital, Boston, MA14