Genetic correction of p67(phox) deficient chronic granulomatous disease using peripheral blood progenitor cells as a target for retrovirus mediated gene transfer

Blood

Volumn 89, Issue 5, 1997, Pages 1754-1761

  Weil, Wayne M ^a   Linton, Gilda F ^a   Whiting Theobald, Narda ^a   Vowells, Sarah J ^a   Rafferty, Steven P ^a   Li, Fei ^a   Malech, Harry L ^a  

^a NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIOXIDANT ACTIVITY; ARTICLE; CHRONIC GRANULOMATOUS DISEASE; GENE TRANSFER; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; PRIORITY JOURNAL; RETROVIRUS; CELL CULTURE; FLOW CYTOMETRY; GENE THERAPY; GENE VECTOR; GENETICS; IMMUNOBLOTTING; METABOLISM;

NEUTROPHIL CYTOSOL FACTOR 67K; PHOSPHOPROTEIN;

CELLS, CULTURED; FLOW CYTOMETRY; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GRANULOMATOUS DISEASE, CHRONIC; HEMATOPOIETIC STEM CELLS; HUMANS; IMMUNOBLOTTING; PHOSPHOPROTEINS; RETROVIRIDAE;

EID: 0030614975     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v89.5.1754.1754_1754_1761     Document Type: Article

References (61)

1. Malech HL, Gallin JI: Current concepts: Immunology. Neutrophils in human diseases. N Engl J Med 317:687, 1987
2. Gallin JI, Malech HL: Update on chronic granulomatous diseases of childhood. Immunotherapy and potential for gene therapy. JAMA 263:1533, 1990
3. Curautte JT: Chronic Granulomatous Disease: The solving of a clinical riddle at the molecular level. Clin Immunol Irnmunopathol 67:S2, 1993 (suppl)
4. Malech HL: Phagocyte oxidative mechanisms. Curr Opin Hematol 1993:123, 1993
5. Abo A, Pick E, Hall A, Totty N, Teahan CG, Segal AW: Activation of the NADPH oxidase involves the small GTP-binding protein p21racl. Nature 353:668, 1991
6. Knaus UG, Heyworth PG, Evans T, Curnutte JT, Bokoch GM: Regulation of phagocyte oxygen radical production by the GTP-binding protein Rac 2, Science 254:1512, 1991
7. 558: The flavin binding component of the phagocyte NADPH oxidase. Science 256:1459, 1992
8. Kwong CH, Malech HL, Rotrosen D, Leto TL: Regulation of the human neutrophil NADPH oxidase by rho-related G-proteins. Biochemistry 32:5711, 1993
9. Clark RA, Malech HL, Gallin JI, Nunoi H, Volpp BD, Pearson DW, Nauseef WM, Curnette JT: Genetic variants of chronic granulomatous disease: Prevalence of deficiencies of two cytosolic components of the NADPH oxidase system. N Engl J Med 321:647, 1989
10. Roos D, de Boer M, Kuribayashi F, Meischl C, Weening RS, Segal AW, Ahlin A, Nernet K, Hossle JP, Bernatowska-Matuszkiewicz E, Middleton-Price H: Mutations in the X-linked and autosomal recessive forms of chronic granulomatous disease. Blood 87:1663, 1996
11. Wientjes FB, Hsuan JJ, Totty NF, Segal AW: p40phox a third cytosolic component of the activation complex of the NADPH oxidase to contain src homology 3 domains. J Biochem 296:557, 1993
12. Royer-Pokora B, Kunkel LM, Monaco AP, Goff SC, Newburger PE, Baehner RL, Cole FS, Curnutte JT, Orkin SH: Cloning the gene for an inherited human disorder-chronic granulomatous disease-on the basis of its chromosomal location. Nature 322:32, 1986
13. Dinauer MC, Orkin SH, Brown R, Jesaitis AJ, Parkos CA: The glycoprotein encoded by the X-linked chronic granulomatous disease locus is a component of the neutrophil cytochrome b complex. Nature 327:717, 1987
14. Parkos CA, Dinauer MC, Walker LE, Allen RA, Jesaitis AJ, Orkin SH: Primary structure and unique expression of the 22-kilodalton light chain of human neutrophil cytochrome b. Proc Natl Acad Sci USA 85:3319, 1988
15. Lomax KJ, Leto TL, Nunoi H, Gallin JI, Malech HL: Recombinant 47-kilodalton cytosol factor restores NADPH oxidase in chronic granulomatous disease. Science 245:409, 1989
16. Volpp BD, Nauseef WM, Donelson JE, Moser DR, Clark RA: Cloning of the cDNA and functional expression of the 47-kilodalton cytosolic component of human neutrophil respiratory burst oxidase. Proc Natl Acad Sci USA 86:7195, 1989
17. Leto TL, Lomax KJ, Volpp BD, Nunoi H, Sechler JM, Nauseef WM, Clark RA, Gallin JI, Malech HL: Cloning of a 67-kD neutrophil oxidase factor with similarity to a noncatalytic region of p60c-src. Science 248:727, 1990
18. phox cDNA. Proc Natl Acad Sci USA 89:10174, 1992
19. phox protein by Epstein-Barr virus-transformed B lymphocytes from a patient with autosomal chronic granulomatous disease. Blood 79:1829, 1992
20. Thrasher A, Chetty M, Casimir C, Segal AW: Restoration of Superoxide generation to a chronic granulomatous disease-derived B-cell line by retrovirus mediated gene transfer. Blood 80:1125, 1992
21. phox deficient chronic granulomatous disease. J Clin Invest 91:201, 1993
22. phox-deficient chronic granulomatous disease. Proc Natl Acad Sci USA 90:7446, 1993
23. phox. Blood 82:2196, 1993
24. phox cDNA. J Exp Med 178:2047, 1993
25. de Mendez I, Adams AG, Sokolic RA, Malech HL, Leto TL: Multiple SH3 domain interactions regulate NADPH oxidase assembly in whole cells. EMBO J 15:1211, 1996
26. phox. Proc Natl Acad Sci USA 90:9832, 1993
27. 558 defective forms of chronic granulomatous disease. Blood 84:53, 1994
28. Cassel A, Cottler-Fox M, Doren S, Dunbar CE: Retroviral-mediated gene transfer into CD34-enriched human peripheral blood stem cells. Exp Hematol 21:585, 1993
29. Kessinger A: Is blood or bone marrow better? Stem Cells 11:290, 1993
30. Bregni M, Magni M, Siena S, Di Nicola M, Bonadonna G, Gianni AM: Human peripheral blood hematopoietic progenitors are optimal targets of retroviral-mediated gene transfer. Blood 80:1418, 1992
31. Dunbar CE, Cottler-Fox M, O'Shaughnessy JA, Doren S, Carter C, Berenson R, Brown S, Moen RC, Greenblatt J, Stewart FM, Leitman SF, Wilson WH, Cowan K, Young NS, Nienhuis AW: Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood 85:3048, 1995
32. Rappaport JM, Newburger PE, Golblum RM, Goldman AS, Nathan DG, Parkman R: Allogeneic bone marrow transplantation for chronic granulomatous disease. J Pediatr 101:952, 1982
33. Hobbs JR, Monteil M, McCluskey DR, Jurges E, el Tumi M: Chronic granulomatous disease 100% corrected by displacement bone marrow transplantation from a volunteer unrelated donor. Eur J Pediatr 151:806, 1992
34. Dranoff G, Jaffee E, Lazenby A, Golumbek P, Levitsky H, Brose K, Jackson V, Hamada H, Pardoll D, Mulligan RC: Vaccination with irradiated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulates potent, specific, and long-lasting anti-tumor immunity. Proc Natl Acad Sci USA 90:3539, 1993
35. Riviere I, Brose K, Mulligan RC: Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells. Proc Natl Acad Sci USA 92:6733, 1995
36. Danos O, Mulligan RC: Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc Natl Acad Sci USA 85:6460, 1988
37. Miller AD, Rosman GJ: Improved retroviral vectors for gene transfer and expression. BioTechniques 7:980, 1989
38. + progenitors to peripheral blood: Impaired mobilization in chronic granulomatous disease and adenosine deaminase-deficient severe combined immunodeficiency disease patients: Blood 88:1104, 1996
39. Van Hoef MEHM: Review of the literature on PBPC mobilization: Recommendations for efficient PBPC collection. Blood 84:739a, 1994 (abstr, suppl 1)
40. Winter J, Jahnke L, Rademaker A, Tallman M, Gordon L, Newman S, Kaminer L, Villa M, Bauman A, Byrd K: Mobilization of peripheral blood progenitor cells (PBPC) by cyclophosphamide (cyclo) and/or colony stimulating factor (CSF): A comparative analysis. Blood 84:739a, 1994 (abstr, suppl 1)
41. Lane TA, Law P, Marruyama M, Young D, Burgess J, Mullen M, Mealiffe M, Terstappen LW, Hardwick A, Moubayed M: Harvesting and enrichment of hematopoietic progenitor cells mobilized into the peripheral blood of normal donors by granulocyte-macrophage colony-stimulating factor (GM-CSF) or G-CSF: Potential role in allogeneic marrow transplantation. Blood 85:275, 1995
42. Dreger P, Viehmann K, Steinmann J, Eckstein V, Muller-Ruchholtz W, Loffler H, Schmitz N: G-CSF mobilized peripheral blood progenitor cells for allogeneic transplantation: Comparison of T cell depletion strategies using different CD34+ selection systems or CAMPATH-1. Exp Hematol 23:147, 1995
43. Scadden DT, Berardi AC, Wang A, Lopez P, Levine JD: The functional isolation and characterization of human hematopoietic stem cells. Blood 84:542a, 1994 (abstr, suppl 1)
44. + cells and expansion of neutrophil precursors in PIXY321 for clinical application. Blood 84:542a, 1994 (abstr, suppl 1)
45. Sandstrom CE, McAdams TA, Collins PC, Bender JG, Papoutsakis ET, Miller WM: Comparison of eight serum-free media for expansion of cord blood and mobilized blood mononuclear cells. Blood 84:739a, 1994 (abstr, supp 1)
46. Thorsteinsdottir U, Humphries RK: Survival and proliferation in vitro of long-term hematopoietic repopulating cells. Blood 84:542a, 1994 (abstr, suppl 1)
47. Malech HL, Sekhsaria S, Whiting-Theobald N, Linton GF, Vowells SJ, Li F, Miller JA, Holland SM, Leitman SF, Carter CS, Read EJ, Butz R, Wannebo C, Fleisher TA, Deans RJ, Spratt SK, Maack CA, Rokovich JA, Cohen LK, Maples PB, Gallin JI: Prolonged detection of oxidase-positive neutrophils in the peripheral blood of five patients following a single cycle of gene therapy for chronic granulomatous disease. Blood 88:486a, 1996 (abstr, suppl 1)
48. Kotani H, Newton PB III, Zhang S, Chiang YL, Otto E, Weaver L, Blaese RM, Anderson WF, McGarrity GJ: Improved methods of retroviral vector transduction and production for gene threrapy. Hum Gene Ther 5:19, 1994
49. phox from recombinant baculoviruses. J Biol Chem 266:19812, 1991
50. Vowells SJ, Sekhsaria S, Malech HL, Shalit M, Fleisher TA: Flow cytometric analysis of the granulocyte respitory burst: A comparison of fluorescent probes. J Immunol Meth 178:89, 1994
51. Vowells SJ, Fleisher TA, Sekhsaria S, Ailing DW, Maguire TE, Malech HL: Genotype-dependent variability in flow cytometric evaluation of reduced nicotinamide adenine dinucleotide phosphate oxidase function in patients with chronic granulomatous disease. J Pediatr 128:104, 1996
52. Sekhsaria S, Malech HL: Recombinant human stem cell factor enhances myeloid colony growth from human peripheral blood progenitors. Blood 81:2125, 1993
53. Zakhireh B, Root RK: Development of oxidase activity by human bone marrow granulocytes. Blood 54:429, 1979
54. Ho AD, Haas R, Korbling M, Dietz M, Hunstein W: Utilization of recombinant human GM-CSF to enhance peripheral progenitor cell yield for autologous transplantation. Bone Marrow Transplant 7:13, 1991
55. Ravagnani F, Siena S, Bregni M, Sciorelli G, Gianni AM, Pellegris G: Large-scale collection of circulating haematopoietic progenitors in cancer patients treated with high-dose cyclophosphamide and recombinant human GM-CSF. Eur J Cancer 26:562, 1990
56. Haas R, Ho Ad, Bredthauer U, Cayeux S, Egerer G, Knauf W, Hunstein W: Successful autologous transplantation of blood stem cells mobilized with recombinant human granulocyte-macrophage colony-stimulating factor. Exp Hematol 18:94, 1990
57. Pileri A, Tarella C, Bregni M, Boccadoro M, Siena S, Caracciolo D, Bonadonna G, Gianni AM: GM-CSF-exposed peripheral blood progenitors as sole source of stem cells for autologous transplantation in two patients with multiple myeloma. Haematologica 75:79, 1990 (suppl 1)
58. Sokolic RA, Sekhsaria S, Sugimoto Y, Whiting-Theobald N, Linton GF, Li F, Gottesman M, Malech HL: A bicistronic retrovirus vector containing a picornavirus internal ribosome entry site allows for correction of X-linked CGD by selection for MDR1 expression. Blood 87:42, 1996
59. Rex JH, Bennett JE, Gallin JI, Malech HL, Melnick DA: Normal and deficient neutrophils can cooperate to damage Aspergillus fumigatus hyphae. J Infect Dis 162:523, 1990
60. Ohno Y, Gallin JI: Diffusion of extracellular hydrogen peroxide into intracellular compartments of human neutrophils. Studies utilizing the inactivation of myeloperoxidase by hydrogen peroxide and azide. J Biol Chem 260:8438, 1985
61. Buescher ES, Alling DW, Gallin JI: Use of an X-linked human neutrophil marker to estimate timing of lyonization and size of the dividing stem cell pool. J Clin Invest 76:1581, 1985