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

Nicholas E Petty; Saar I Gill

doi:10.1016/j.stem.2018.12.016

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

Cell Stem Cell. 2019 Feb 7;24(2):208-209. doi: 10.1016/j.stem.2018.12.016.

Authors

Nicholas E Petty¹, Saar I Gill²

Affiliations

¹ Division of Hematology-Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, PA 19104, USA.
² Division of Hematology-Oncology, Department of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, PA 19104, USA; Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA 19104, USA. Electronic address: saar.gill@pennmedicine.upenn.edu.

PMID: 30735648
DOI: 10.1016/j.stem.2018.12.016

Abstract

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) demonstrate an effective and potentially durable approach to eliminate the IPEX phenotype in murine models.

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MeSH terms

Animals
Autoimmunity
Forkhead Transcription Factors / genetics
Genetic Diseases, X-Linked*
Genetic Therapy
Mice
Mutation
T-Lymphocytes, Regulatory*

Substances

Forkhead Transcription Factors
Foxp3 protein, mouse