Donello JE, Loeb JE, Hope TJ . Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element. J Virol 1998; 72: 5085–5092.
Zufferey R, Donello JE, Trono D, Hope TJ . Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J Virol 1999; 73: 2886–2892.
Schambach A, Wodrich H, Hildinger M, Bohne J, Krausslich HG, Baum C . Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors. Mol Ther 2000; 2: 435–445.
Salmon P, Kindler V, Ducrey O, Chapuis B, Zubler RH, Trono D . High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors. Blood 2000; 96: 3392–3398.
CAS PubMed Google Scholar
Callendret B, Lorin V, Charneau P, Marianneau P, Contamin H, Betton JM et al. Heterologous viral RNA export elements improve expression of severe acute respiratory syndrome (SARS) coronavirus spike protein and protective efficacy of DNA vaccines against SARS. Virology 2007; 363: 288–302.
Higashimoto T, Urbinati F, Perumbeti A, Jiang G, Zarzuela A, Chang LJ et al. The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors. Gene Therapy 2007; 14: 1298–1304.
Popa I, Harris ME, Donello JE, Hope TJ . CRM1-dependent function of a cis-acting RNA export element. Mol Cell Biol 2002; 22: 2057–2067.
Article CAS PubMed PubMed Central Google Scholar
Schambach A, Galla M, Maetzig T, Loew R, Baum C . Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors. Mol Ther 2007; 15: 1167–1173.
Charrier S, Dupre L, Scaramuzza S, Jeanson-Leh L, Blundell MP, Danos O et al. Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients. Gene Therapy 2007; 14: 415–428.
Dupre L, Marangoni F, Scaramuzza S, Trifari S, Hernandez RJ, Aiuti A et al. Efficacy of gene therapy for Wiskott–Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation. Hum Gene Ther 2006; 17: 303–313.
Kingsman SM, Mitrophanous K, Olsen JC . Potential oncogene activity of the woodchuck hepatitis post-transcriptional regulatory element (WPRE). Gene Therapy 2005; 12: 3–4.
Wei Y, Etiemble J, Fourel G, Vitvitski-Trepo L, Buendia MA . Hepadna virus integration generates virus-cell cotranscripts carrying 3′ truncated X genes in human and woodchuck liver tumors. J Med Virol 1995; 45: 82–90.
Tu H, Bonura C, Giannini C, Mouly H, Soussan P, Kew M et al. Biological impact of natural COOH-terminal deletions of hepatitis B virus X protein in hepatocellular carcinoma tissues. Cancer Res 2001; 61: 7803–7810.
CAS PubMed Google Scholar
Themis M, Waddington SN, Schmidt M, von Kalle C, Wang Y, Al-Allaf F et al. Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol Ther 2005; 12: 763–771.
Gaspar HB, Bjorkegren E, Parsley K, Gilmour KC, King D, Sinclair J et al. Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol Ther 2006; 14: 505–513.
Charrier S, Stockholm D, Seye K, Opolon P, Taveau M, Gross DA et al. A lentiviral vector encoding the human Wiskott–Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice. Gene Therapy 2005; 12: 597–606.
Calle Y, Chou HC, Thrasher AJ, Jones GE . Wiskott–Aldrich syndrome protein and the cytoskeletal dynamics of dendritic cells. J Pathol 2004; 204: 460–469.
Westerberg L, Larsson M, Hardy SJ, Fernandez C, Thrasher AJ, Severinson E . Wiskott–Aldrich syndrome protein deficiency leads to reduced B-cell adhesion, migration, and homing, and a delayed humoral immune response. Blood 2005; 105: 1144–1152.
Snapper SB, Rosen FS, Mizoguchi E, Cohen P, Khan W, Liu CH et al. Wiskott–Aldrich syndrome protein-deficient mice reveal a role for WASP in T but not B cell activation. Immunity 1998; 9: 81–91.
Nguyen DD, Maillard MH, Cotta-de-Almeida V, Mizoguchi E, Klein C, Fuss I et al. Lymphocyte-dependent and Th2 cytokine-associated colitis in mice deficient in Wiskott–Aldrich syndrome protein. Gastroenterology 2007; 133: 1188–1197.
Schambach A, Bohne J, Baum C, Hermann FG, Egerer L, von Laer D et al. Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression. Gene Therapy 2006; 13: 641–645.
Zychlinski D, Schambach A, Modlich U, Maetzig T, Meyer J, Grassman E et al. Physiological promoters reduce the genotoxic risk of integrating gene vectors. Mol Ther 2008; 16: 718–725.
Maillard MH, Cotta-de-Almeida V, Takeshima F, Nguyen DD, Michetti P, Nagler C et al. The Wiskott–Aldrich syndrome protein is required for the function of CD4(+)CD25(+)Foxp3(+) regulatory T cells. J Exp Med 2007; 204: 381–391.
Klein C, Nguyen D, Liu CH, Mizoguchi A, Bhan AK, Miki H et al. Gene therapy for Wiskott–Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice. Blood 2003; 101: 2159–2166.
Meyer-Bahlburg A, Becker-Herman S, Humblet-Baron S, Khim S, Weber M, Bouma G et al. Wiskott–Aldrich syndrome protein deficiency in B cells results in impaired peripheral homeostasis. Blood 2008; 112: 4158–4169.
Montini E, Cesana D, Schmidt M, Sanvito F, Ponzoni M, Bartholomae C et al. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol 2006; 24: 687–696.
Marangoni F, Bosticardo M, Charrier S, Draghici E, Locci M, Scaramuzza S et al. Evidence for long-term efficacy and safety of gene therapy for Wiskott–Aldrich Syndrome in preclinical models. Mol Ther 2009 (in press).
Zufferey R, Nagy D, Mandel RJ, Naldini L, Trono D . Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat Biotechnol 1997; 15: 871–875.
Towers GJ, Stockholm D, Labrousse-Najburg V, Carlier F, Danos O, Pages JC . One step screening of retroviral producer clones by real time quantitative PCR. J Gene Med 1999; 1: 352–359.