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GENE THERAPY

Gene therapy goes the distance in Wiskott–Aldrich syndrome

Nature Medicine volume 28, pages 24–25 (2022)Cite this article

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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.

Allogeneic hematopoietic stem-cell transplantation (HSCT) from HLA-matched donors has become the gold-standard treatment for patients with WAS3,4, offering a potential cure and correction of the underlying immunodeficiency and thrombocytopenia. However, as related identical donors are rare and the use of matched-unrelated donors (if available) carries a risk of complications, hematopoietic stem-cell (HSC) gene therapy has also been explored as a potential therapeutic strategy5. In this issue of Nature Medicine, Magnani et al. provide new evidence in support of the use of HSC gene therapy as curative option for patients with WAS6.

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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

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  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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