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canada viagra online We developed a SIN lentiviral vector coding for human WASP* under the control of a 1.6 kb reconstituted WAS gene promoter (LV-w1.6W) (3). The use of this endogenous promoter ensures that the transgene is expressed in a physiological manner (4), restoring WASP expression and function in human and murine WAS cells (3, 30â34). Its moderate enhancer activity combined with the SIN LTR design reduces the risk of insertional mutagenesis (35), as shown by in vitro transformation assays (36) and preclinical in vivo studies in WASP-deficient mice (34, 37). These data provided the rationale for a phase I/II clinical trial in which LV-w1.6W was used as a gene therapy vector for treatment of patients with WAS.