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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References
Urnov, F. D., Rebar, M. C., Holmes, M. C., Zhang, S. & Gregory, P. D. Nat. Rev. Genet.11, 636–646 (2010).
Joung, J. K. & Sander, J. D. Nat. Rev. Mol. Cell Biol.14, 49–55 (2012).
Sander, J. D. & Joung, J. K. Nat. Biotechnol.32, 347–355 (2014).
Schiroli, J. et al. Sci. Transl. Med.9, eaan0820 (2017).
Tsai, S. Q. et al. Nat. Biotechnol.33, 187–197 (2015).
Martins, V. C. et al. Nature509, 465–470 (2014).
Cavazzana, M., Six, E., Lagresle-Peyrou, C., André-Schmutz, S. & Hacein-Bey-Abina, S. Hum. Gene Ther.27, 108–116 (2016).
Hacein-Bey-Abina, S. et al. Science302, 415–419 (2003).
Aiuti, A. N. Engl. J. Med.360, 447–458 (2009).
Aiuti, A. Science296, 2410–2413 (2002).
Shaw, K. L. & Kohn, D. B. Sci. Transl. Med.3, 97ps36 (2011).
De Ravin, S. S. et al. Sci. Transl. Med.8, 335ra57 (2016).
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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
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DOI: https://doi.org/10.1038/s41551-017-0174-x
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