A humanized mouse model of X-linked severe combined immunodeficiency suggests that the safety of correcting the human IL2RG gene in haematopoietic stem cells depends on both gene-correction frequency and the conditioning regimen used before cell transplantation.
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Illuminating the genome-wide activity of genome editors for safe and effective therapeutics
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Tsai, S.Q. Towards safe therapy for immunodeficiency. Nat Biomed Eng 1, 937–938 (2017). https://doi.org/10.1038/s41551-017-0174-x