Daring to learn from humanized mice

Abstract

In this issue of Blood, Goettel and colleagues introduce a novel humanized mouse model of immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome, whereby mice lacking murine major histocompatibility complex class II (MHC II) and expressing human HLA-DR1 (NOD. Prkdcscid Il2rg-/- H2-Ab1-/-; NSGAb°DR1) are reconstituted with hematopoietic stem cells (HSCs) from a patient with IPEX syndrome to generate a humanized model for primary immune deficiency presenting as fatal autoimmunity.