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Gene Therapy for Wiskott-Aldrich Syndrome—Long-Term Efficacy and Genotoxicity

12 Mar 2014

Abstract excerpt

Wiskott-Aldrich syndrome (WAS) is characterized by microthrombocytopenia, immunodeficiency, autoimmunity, and susceptibility to malignancies. In our hematopoietic stem cell gene therapy (GT) trial using a γ-retroviral vector, 9 of 10 patients showed sustained engraftment and correction of WAS protein (WASP) expression in lymphoid and myeloid cells and platelets. GT resulted in partial or complete resolution of...

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PMID: 24622513
DOI: 10.1126/scitranslmed.3007280

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