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20 years of gene therapy for SCID

Severe combined immunodeficiency conditions are devastating disorders of adaptive immunity. Although these diseases were initially treated by transplantation of allogeneic hematopoietic stem cells, the past 20 years has shown that these conditions are correctable by gene therapy.

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Figure 1: Block in lymphocyte differentiation resulting from a deficiency in γc (SCID-X1) and ADA.
Figure 2: Gene therapy for SCID-X1 and ADA deficiency.

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Acknowledgements

We thank many colleagues who have taken part in this research, notably G. de Saint Basile, J. Di Santo, A. Durandy, C von Kalle and R. Bushman; A. Aiuti, F. Candotti, D.B. Kohn and A. Thrasher for providing unpublished data on their ongoing research; and the many colleagues who have contributed to the field whose important publications could not be cited here because of space constraints.

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Correspondence to Alain Fischer.

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Fischer, A., Hacein-Bey-Abina, S. & Cavazzana-Calvo, M. 20 years of gene therapy for SCID. Nat Immunol 11, 457–460 (2010). https://doi.org/10.1038/ni0610-457

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