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Publication Detail
Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.
  • Publication Type:
    Journal article
  • Publication Sub Type:
    Article
  • Authors:
    Morris EC, Fox T, Chakraverty R, Tendeiro R, Snell K, Rivat C, Grace S, Gilmour K, Workman S, Buckland K, Butler K, Chee R, Salama AD, Ibrahim H, Hara H, Duret C, Mavilio F, Male F, Bushman FD, Galy A, Burns SO, Gaspar HB, Thrasher AJ
  • Publication date:
    17/07/2017
  • Journal:
    Blood
  • Medium:
    Print-Electronic
  • Print ISSN:
    0006-4971
  • Language:
    eng
  • Addresses:
    UCL Institute of Immunity and Transplantation, London, United Kingdom; e.morris@ucl.ac.uk.
Abstract
Until recently hematopoietic stem cell transplantation was the only curative option for Wiskott-Aldrich syndrome (WAS). The first attempts at gene therapy for WAS using a ϒ-retroviral vector improved immunological parameters substantially but were complicated by acute leukemia as a result of insertional mutagenesis in a high proportion of patients. More recently treatment of children with a state-of-the-art self-inactivating lentiviral vector (LV-w1.6 WASp) has resulted in significant clinical benefit without inducing selection of clones harbouring integrations near oncogenes. Here, we describe a case of a pre-splenectomised 30 year old patient with severe WAS manifesting as cutaneous vasculitis, inflammatory arthropathy, intermittent polyclonal lymphoproliferation, significant chronic kidney disease and requiring long-term immunosuppressive treatment. Following reduced intensity conditioning there was rapid engraftment and expansion of a polyclonal pool of transgene-positive functional T cells, and sustained gene marking in myeloid and B cell lineages up to 20 months of observation. The patient was able to discontinue immunosuppression and exogenous immunoglobulin support, with improvement in vasculitic disease and pro-inflammatory markers. Autologous gene therapy using a lentiviral vector is a viable strategy for adult WAS patients with severe chronic disease complications and for whom an allogeneic procedure could present an unacceptable risk. This trial was registered at ClinicalTrials.gov #NCT01347242.
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