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Gene therapy goes the distance in Wiskott–Aldrich syndrome

Long-term follow-up data reinforce the curative potential of hematopoietic stem-cell gene therapy for this rare primary immunodeficiency disorder.

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Correspondence to Alessandra Biffi.

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The author declares no competing interests.

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Biffi, A. Gene therapy goes the distance in Wiskott–Aldrich syndrome. Nat Med 28, 24–25 (2022).

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