Stable Hematopoietic Cell Engraftment after Low-Intensity Nonmyeloablative Conditioning in Patients with Immune Dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) Syndrome

摘要

BackgroundImmune dysregulation, Polyendocrinopathy, Enteropathy, X-linked (IPEX) syndrome is characterized by severe systemic autoimmunity caused by mutations in the FOXP3 (Forkhead Box P3) gene. Hematopoietic cell transplantation is currently the only viable option for long-term survival, but patients are frequently very ill and may not tolerate traditional myeloablative conditioning regimens.