21st Century FOX: Toward Gene Therapy for the Regulatory T Cell Deficiency Syndrome IPEX

IPEX is a fatal X-linked autoimmune disorder that results from mutations in the canonical regulatory T cell transcription factor FOXP3. In this issue of Cell Stem Cell, Masiuk et al. (2019) Masiuk K.E. Laborada J. Roncarolo M.G. Hollis R.P. Kohn D.B. Hematopoietic stem cell gene therapy for IPEX syndrome. Cell Stem Cell. 2019; 24 ( this issue) : 309-317 Google Scholar demonstrate an effective and potentially durable approach to eliminate the IPEX phenotype in murine models