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Correction of persistent thrombocytopenia by a boost of CD133+ selected stem cells in a patient transplanted for Wiskott-Aldrich syndrome 10 years ago

Bone Marrow Transplantation volume 33, pages 879–880 (2004)Cite this article

Wiskott-Aldrich syndrome is a hematological disorder with impaired function and number of platelets and lymphocytes. The typical symptoms comprise eczema, thrombopenia and recurrent infections. Mutations in the WAS gene at XP11.22, primarily expressed in hematopoietic cells, have been shown to cause this X-linked immunodeficiency.1,2

Transplantation of allogeneic stem cells is the only curative approach to date. Thus, for patients who lack an identical sibling or unrelated donor, transplants from mismatched related (haploidentical) donors are increasingly used. However, graft failure, EBV-LPD and severe viral infections remain major obstacles, especially when direct T-cell depletion methods are used.1,3,4

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  1. Children's University Hospital, Tuebingen, Germany

    P Lang, T Klingebiel, M Schumm, R Handgretinger, J Greil, J F Beck, D Niethammer & P Bader

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  1. P Lang
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  2. T Klingebiel
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Lang, P., Klingebiel, T., Schumm, M. et al. Correction of persistent thrombocytopenia by a boost of CD133+ selected stem cells in a patient transplanted for Wiskott-Aldrich syndrome 10 years ago. Bone Marrow Transplant 33, 879–880 (2004). https://doi.org/10.1038/sj.bmt.1704434

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