Improved lentiviral vectors for Wiskott-Aldrich syndrome gene therapy mimic endogenous expression profiles throughout haematopoiesis

Gene Therapy

Volumn 15, Issue 12, 2008, Pages 930-941

  Frecha, C ^a,b   Toscano, M G ^a   Costa, C ^b   Saez Lara, M J ^c   Cosset, F L ^b   Verhoeyen, E ^b   Martin, F ^a  

^a INSTITUTO DE PARASITOLOGÍA Y BIOMEDICINA LÓPEZ NEYRA   (Spain)

^b UNIVERSITÉ DE LYON   (France)

^c UNIVERSITY OF GRANADA   (Spain)

Author keywords

[No Author keywords available]

Indexed keywords

ENHANCED GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR; WISKOTT ALDRICH SYNDROME PROTEIN;

ANIMAL EXPERIMENT; ARTICLE; CONTROLLED STUDY; ERYTHROID CELL; HEMATOPOIESIS; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; MACROPHAGE; MOUSE; MOUSE STRAIN; NEWBORN; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN EXPRESSION; VIRAL GENE DELIVERY SYSTEM; VIRUS EXPRESSION; VIRUS STRAIN; WISKOTT ALDRICH SYNDROME;

EID: 44849144691     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2008.20     Document Type: Article

References (44)

1. Standen GR. Wiskott-Aldrich syndrome: A multidisciplinary disease. J. Clin Pathol 1991; 44: 979-982.
2. Imai K, Morio, T, Zhu Y, jin Y, Itoh S, Kajiwara M et al. Clinical course of patients with WASP gene mutations. Blood 2004; 103: 456-464.
3. Thrasher AJ. WASp in immune-system organization and function. Nat Rev Immunol 2002; 2: 635-646.
4. Imai K, Nonoyama S, Ochs HD. WASP (Wiskott-Aldrich syndrome protein) gene mutations and phenotype. Curr Opin Allergy Clin Immunol 2003; 3: 427-436.
5. Wengler GS, Notarangelo LD, Berardelli S, Pollonni G, Mella P, Fasth A et al. High prevalence of nonsense, frame shift, and splice-site mutations in 16 patients with full-blown Wiskott-Aldrich syndrome. Blood 1995; 86: 3648-3654.
6. Greer WL, Shehabeldin A, Schuhnan J, Junker A, Siminovitch KA. Identification of WASP mutations, mutation hotspots and genotype-phenotype disparities in 24 patients with the Wiskott-Aldrich syndrome. Hum Genet 1996; 98: 685-690.
7. Shcherbina A, Rosen FS, Remold-O'Donnell E. WASP levels in platelets and lymphocytes of Wiskott-Aldrich syndrome patients correlate with cell dysfunction. J Immunol 1999; 163: 6314-6320.
8. Stewart DM, Treiber-Held S, Kurman CC, Facchetti F, Notarangelo LD, Nelson DL. Studies of the expression of the Wiskott-Aldrich syndrome protein. J Clin Invest 1996; 97: 2627-2634.
9. Derry JM, Ochs HD, Francke U. Isolation of a novel gene mutated in Wiskott-Aldrich syndrome. Cell 1994; 78: 635-644.
10. Parolini O, Berardelli S, Riedl E, Bello-Fernandez C, Strobl H, Majdic O et al. Expression of Wiskott-Aldrich syndrome protein (WASP) gene during hematopoietic differentiation. Blood 1997; 90: 70-75.
11. 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.
12. Molina IJ, Kenney DM, Rosen FS, Remold-O'Donnell E. T cell lines characterize events in the pathogenesis of the Wiskott-Aldrich syndrome. J Exp Med 1992; 176: 867-874.
13. Gallego MD, Santamaria M, Pena J, Molina IJ. Defective actin reorganization and polymerization of Wiskott-Aldrich T cells in response to CB3-mediated stimulation. Blood 1997; 90: 3089-3097.
14. Dupre L, Aiuti A, Trifari S, Martino S, Saracco P, Bordignon C et al Wiskott-Aldrich syndrome protein regulates lipid raft dynamics during immunological synapse formation. Immunity 2002; 17: 157-166.
15. Linder S, Nelson D, Weiss M, Aepfelbacher M. Wiskott-Aldrich syndrome protein regulates podosomes in primary human macrophages. Proc Natl Acad Sci USA 1999; 96: 9648-9653.
16. Oda A, Ochs HD. Wiskott-Aldrich syndrome protein and platelets. Immunol Rev 2000; 178: 111-117.
17. Thrasher AJ, Jones GE, Kinnon C, Brickell PM, Katz DR. Is Wiskott-Aldrich syndrome a cell trafficking disorder? Immunol Today 1998; 19: 537-539.
18. Takenawa T, Miki H. WASP and WAVE family proteins: Key molecules for rapid rearrangement of cortical actin filaments and cell movement. J Cell Sci 2001; 114: 1801-1809.
19. Munoz A, Olive T, Martinez A, Bureo E, Maldonado MS, Diaz de Heredia C et al. Allogeneic hemopoietic stem cell transplantation (HSCT) for Wiskott-Aldrich syndrome: A report of the Spanish Working Party for Blood and Marrow Transplantation in Children (GETMON). Pediatr Hematol Oncol 2007; 24: 393-402.
20. Kobayashi R, Ariga T, Nonoyama S, Kanegane H, Tsuchiya S, Morio T et al. Outcome in patients with Wiskott-Aldrich syndrome following stem cell transplantation: An analysis of 57 patients in Japan. Br J Haematol 2006; 135: 362-366.
21. Knutsen AP, Steffen M, Wassmer K, Wall DA. Umbilical cord blood transplantation in Wiskott Aldrich syndrome. J Pediatr 2003; 142: 519-523.
22. Toscano MG, Frecha C, Benabdellah K, Cobo M, Blundell M, Thrasher AJ et al. Hematopoictic-specific lentiviral vectors circumvent cellular toxicity due to ectopic expression of the Wiskott-Aldrich syndrome protein. Hum Gene Ther 2008; 19: 179-197.
23. Marx J. Cell biology. Podosomes and invadopodia help mobile cells step lively. Science 2006; 312: 1868-1869.
24. Martin F, Toscano, MG, Blundell M, Frecha C, Srivastava GK, Santamaria. M et al. Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences. Gene Therapy 2005; 12: 715-723.
25. Dupre L, Trifari S, Follenzi A, Marangoni F, Lain de Lera T, Bernad A et al. Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction. Mol Ther 2004; 10: 903-915.
26. 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.
27. 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.
28. 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-contaming lentiviral vector and nonlethal irradiation. Hum Gene Ther 2006; 17: 303-313.
29. Hagemann TL, Kwan SP. The identification and Characterization of two promoters and the complete genomic sequence for the Wiskott-Aldrich syndrome gene. Biochem Biophys Res Commun 1999; 256: 104-109.
30. Hagemann TL, Mares D, Kwan S. Gene regulation of Wiskott-Aldrich syndrome protein and the human homolog of the Drosophila Su(var)3-9: WASP and SUV39H1, two adjacent genes at Xp11.23. Biochim Biophys Acta 2000; 1493: 368-372.
31. Demaison C, Parsley K, Brouns G, Scherr M, Battmer K, Kinnon C et al High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency (correction of immunodeficiency) virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. Hum Gene Ther 2002; 13: 803-813.
32. Traggiai E, Chicha L, Mazzucchelli L, Bronz L, Piffaretti JC, Lanzavecchia A et al. Development of a human adaptive immune system in cord blood cell-transplanted mice. Science 2004; 304 104-107.
33. -/-mice: functional inactivation of p53 in developing T cells. Blood 2004; 104: 3886-3893.
34. Mikkola HK, Orkin SH. The journey of developing hematopoietic stem cells. Development 2006; 133: 3733-3744.
35. Petrella A, Doti I, Agosti V, Giarrusso PC, Vitale D, Bond HM 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.
36. Liu H, Keefer JR, Wang QF, Friedman AD. Reciprocal effects of C/EBPalpha and PKCdelta on JunB expression and monocytic differentiation depend upon the C/EBPalpha basic region. Blood 2003; 101: 3885-3892.
37. Iwasaki H, Somoza C, Shigematsu H, Duprez EA, Iwasaki-Arai J, Mizuno S et al. Distinctive and indispensable roles of PU.1 in maintenance of hematopoietic stem cells and their differentiation. Blood 2005; 106: 1590-1600.
38. Friedman AD, Keefer JR, Kummalue T, Liu H, Wang QF, Cleaves R. Regulation of granulocyte and morlocyte differentiation by CCAAT/ enhancer binding protein alpha. Blood Cells Mol Dis 2003; 31 338-341.
39. Passlick B, Flieger D, Ziegler-Heitbrock HW. Identification and characterization of a novel monocyte subpopulation in human peripheral blood. Blood 1980; 74: 2527-2534.
40. Gordon S, Taylor PR. Monocyte and macrophage heterogeneity. Nat Rev Immunol 2005; 5: 953-964.
41. Gonda TJ. The c-Myb oncoprotein. Int J Biochem Cell Biol 1998; 30: 547-551.
42. Hohaus S, Petrovick MS, Voso MT, Sun Z, Zhang DE, Tenen DG. PU.1 (Spi-1) and C/EBP alpha regulate expression of the granulocyte-macrophage colony-stimulating factor receptor alpha gene. Mol Cell Biol 1995; 15:5830-5845.
43. Zhang DE Hethenngton CJ, Meyers S, Rhoades KL, Larson CJ, Chen HM-et al. CCAAT enhancer-binding protein (C/EBP) and AML1 (CBF alpha2) synergistically activate the macrophage colony-stimulating factor receptor promoter. Mol Cell Biol. 1996; 16: 1231-1240.
44. Toscano MG, Frecha C, Ortega C, Santamaria M, Martin F, Molina IJ. Efficient lentiviral transduction of Herpesvirus; saimiri immortalized T cells as a model for gene therapy in primary immunodeficiencies. Gene Therapy 2004; 11: 956-961.