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Gene therapy goes the distance in Wiskott–Aldrich syndrome

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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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Authors and Affiliations

  1. Pediatric Hematology, Oncology and Stem Cell Transplant Division, Padua University Hospital, Padua, Italy

    Alessandra Biffi

  2. Gene Therapy Program, Dana Farber–Boston Children’s Cancer and Blood Disorders Center, Boston, MA, USA

    Alessandra Biffi

Authors
  1. Alessandra Biffi
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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). https://doi.org/10.1038/s41591-021-01653-7

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  • DOI: https://doi.org/10.1038/s41591-021-01653-7

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