A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice

Gene Therapy

Volumn 12, Issue 7, 2005, Pages 597-606

  Charrier, S ^a   Stockholm, D ^a   Seye, K ^a   Opolon, P ^b   Taveau, M ^a   Gross, D A ^a   Bucher Laurent, S ^a   Delenda, C ^a   Vainchenker, W ^b   Danos, O ^a   Galy, A ^a,b  

^a GÉNÉTHON   (France)

^b INSTITUT GUSTAVE ROUSSY   (France)

Author keywords

Bone marrow; Dendritic cells; Lentiviral vector; Transplantation; Wiskott Aldrich syndrome

Indexed keywords

LENTIVIRUS VECTOR; WISKOTT ALDRICH SYNDROME PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BIOLOGICAL ACTIVITY; BONE MARROW CELL; CELL TRANSPLANTATION; COLITIS; CONTROLLED STUDY; CYTOSKELETON; DATA ANALYSIS; DENDRITIC CELL; FEMALE; GENE EXPRESSION; GENE THERAPY; GENETIC CODE; GENETIC TRANSDUCTION; HUMAN IMMUNODEFICIENCY VIRUS 1; IMMUNOPATHOLOGY; IN VIVO STUDY; INFLAMMATORY DISEASE; IRRADIATION; KNOCKOUT MOUSE; LETHALITY; LYMPHOCYTE PROLIFERATION; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; RECIPIENT; SPLEEN CELL; STEM CELL; T LYMPHOCYTE; VIRUS INACTIVATION; WISKOTT ALDRICH SYNDROME;

ANIMALS; BONE MARROW TRANSPLANTATION; COLITIS; DENDRITIC CELLS; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; HIV-1; HUMANS; MICE; MICE, KNOCKOUT; PROTEINS; RNA, MESSENGER; T-LYMPHOCYTES; TRANSDUCTION, GENETIC; WISKOTT-ALDRICH SYNDROME; WISKOTT-ALDRICH SYNDROME PROTEIN;

HUMAN IMMUNODEFICIENCY VIRUS 1; VESPIDAE;

EID: 20144389599     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3302440     Document Type: Article

References (30)

1. Thrasher AJ. WASp in immune-system organization and function. Nat Rev Immunol 2002; 2: 635-646.
2. Badour K, Zhang J, Siminovitch KA. The Wiskott-Aldrich syndrome protein: forging the link between actin and cell activation. Immunol Rev 2003; 192: 98-112.
3. Notarangelo LD, Ochs HD. Wiskott-Aldrich syndrome: a model for defective actin reorganization, cell trafficking and synapse formation. Curr Opin Immunol 2003; 15: 585-591.
4. Dupuis-Girod S et al. Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients. Pediatrics 2003; 111: e622-e627.
5. Imai K et al. Clinical course of patients with WASP gene mutations. Blood 2004; 103: 456-464.
6. Derry JM et al. WASP gene mutations in Wiskott-Aldrich syndrome and X-linked thrombocytopenia. Hum Mol Genet 1995; 4 1127-1135.
7. Sasahara Y et al. Mechanism of recruitment of WASP to the immunological synapse and of its activation following TCR ligation. Mol Cell 2002; 10: 1269-1281.
8. Dupre L et al. Wiskott-Aldrich syndrome protein regulates lipid raft dynamics during immunological synapse formation. Immunity 2002; 17: 157-166.
9. Orange JS et al. Wiskott-Aldrich syndrome protein is required for NK cell cytotoxicity and colocalizes with actin to NK cell-activating immunologic synapses. Proc Natl Acad Sci USA 2002; 99: 11351-11356.
10. Jones GE et al. Restoration of podosomes and chemotaxis in Wiskott-Aldrich syndrome macrophages following induced expression of WASP. Int J Biochem Cell Biol 2002; 34: 806-815.
11. Calle Y et al. WASp deficiency in mice results in failure to form osteoclast sealing zones and defects in bone resorption. Blood 2004; 103: 3552-3561.
12. Snapper SB 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.
13. Zhang J et al. Antigen receptor-induced activation and cytoskeletal rearrangement are impaired in Wiskott-Aldrich syndrome protein-deficient lymphocytes. J Exp Med 1999; 190: 1329-4342.
14. Klein C 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.
15. Wada T, Jagadeesh GJ, Nelson DL, Candotti F. Retrovirus-mediated WASP gene transfer corrects Wiskott-Aldrich syndrome T-cell dysfunction. Hum Gene Ther 2002; 13: 1039-1046.
16. Strom TS et al. Functional correction of T cells derived from patients with the Wiskott-Aldrich syndrome (WAS) by transduction with an oncoretroviral vector encoding the WAS protein. Gene Therapy 2003; 10: 803-809.
17. Filipovich AH et al. Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program. Blood 2001; 97: 1598-1603.
18. Candotti F et al. Retrovirus-mediated WASP gene transfer corrects defective actin polymerization in B cell lines from Wiskott-Aldrich syndrome patients carrying 'null' mutations. Gene Therapy 1999; 6: 1170-1174.
19. Strom TS et al. Defects in T-cell-mediated immunity to influenza virus in murine Wiskott-Aldrich syndrome are corrected by oncoretroviral vector-mediated gene transfer into repopulating hematopoietic cells. Blood 2003; 102: 3108-3116.
20. Follenzi A et al. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat Genet 2000; 25: 217-222.
21. Linder S, Aepfelbacher M. Podosomes: adhesion hot-spots of invasive cells. Trends Cell Biol 2003; 13: 376-385.
22. Burns S et al. Configuration of human dendritic cell cytoskeleton by Rho GTPases, the WAS protein, and differentiation. Blood 2001; 98: 1142-1149.
23. Linder S et al. Macrophages of patients with X-linked thrombocytopenia display an attenuated Wiskott-Aldrich syndrome phenotype. Immunol Cell Biol 2003; 81: 130-136.
24. Biffi A et al. Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. J Clin Invest 2004; 113: 1118-1129.
25. Piacibello W et al. Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient. Blood 2002; 100: 4391-4400.
26. Levasseur DN, Ryan TM, Pawlik KM, Townes TM. Correction of a mouse model of sickle cell disease: lentiviral/anti-sickling {beta}-globin gene transduction of unmobilized, purified hematopoietic stem cells. Blood 2003; 102: 4312-4319.
27. Hacein-Bey-Abina S et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003; 348: 255-256.
28. Petrella A et al. A 5′ regulatory sequence containing two Ets motifs controls the expression of the Wiskott-Aldrich syndrome protein (WASP) gene in human hematopoietic cells. Blood 1998; 91 4554-4560.
29. Hagemann TL, Kwan SP. The identification and characterization of two promoters and the complete genomic sequence for the WiskottvAldrich syndrome gene. Biochem Biophys Res Commun 1999; 256: 104-109.
30. Towers GJ et al. One step screening of retroviral producer clones by real time quantitative PCR. J Gene Med 1999; 1: 352-359.