Abstract
A Aiuti, L Biasco, S Scaramuzza. Science. 2013;341(6148):1233151 The goal of this study was to develop a clinical protocol for Wiskott-Aldrich syndrome (WAS) by using a lentiviral vector (LV) to introduce a functional WAS gene into hematopoietic stem and progenitor cells (HSPCs) for autologous transplantation. Three patients who had WAS with a severe clinical condition or severe mutation/absent WAS protein expression without a suitable matched donor for allogeneic transplant or ineligible for HSPC transplantation were enrolled. Patients were pretreated with a reduced-intensity myeloablative regimen. Afterward, they received autologous HSPCs that were transduced …
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