Research ArticleGENE EDITING

Gene editing to induce FOXP3 expression in human CD4+ T cells leads to a stable regulatory phenotype and function

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Science Translational Medicine  03 Jun 2020:
Vol. 12, Issue 546, eaay6422
DOI: 10.1126/scitranslmed.aay6422

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Enforced editing

Various autoimmune diseases could potentially be treated with regulatory T cells (Tregs), but there are many hurdles between this idea and clinical execution. Honaker et al. devised a gene-editing strategy to enforce expression of FOXP3, the master Treg transcription factor, in CD4+ T cells isolated from human peripheral blood, thereby overcoming limitations of Treg isolation and expansion. Resulting stable FOXP3 expression enabled a suppressive phenotype in vitro, and the edited cells were also functional in a xenogeneic graft-versus-host disease model and an experimental autoimmune encephalitis model. This approach has the potential to rapidly translate to clinical use.

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