Efficient retroviral transduction of human bone marrow progenitor and long-term culture-initiating cells: Partial reconstitution of cells from patients with X-linked chronic granulomatous disease by gp91-phox expression

Blood

Volumn 87, Issue 9, 1996, Pages 3722-3730

  Porter, Colin D ^a   Parkar, Mohamed H ^b   Collins, Mary K L ^b   Levinsky, Roland J ^b   Kinnon, Christine ^b  

^a INSTITUTE OF CANCER RESEARCH   (United Kingdom)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE;

ARTICLE; CELL TRANSFER; CHRONIC GRANULOMATOUS DISEASE; EXPRESSION VECTOR; GENE TARGETING; GENE THERAPY; GENETIC TRANSFECTION; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; MONONUCLEAR CELL; PRIORITY JOURNAL; RETROVIRUS; X CHROMOSOME LINKAGE;

BASE SEQUENCE; BONE MARROW CELLS; CELL DIFFERENTIATION; CELL DIVISION; CELLS, CULTURED; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GRANULOMATOUS DISEASE, CHRONIC; HEMATOPOIETIC STEM CELLS; HUMANS; MEMBRANE GLYCOPROTEINS; MOLECULAR SEQUENCE DATA; NADPH OXIDASE; RETROVIRIDAE;

EID: 0029876988     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v87.9.3722.bloodjournal8793722     Document Type: Article

References (44)

1. Blaese RM, Culver KW: Gene therapy for primary immunodeficiency disease. Immunodefic Rev 3:329, 1992
2. Bordignon C, Yu S-F, Smith CA, Hantzopoulos P, Ungers GE, Keever CA, O'Reilly RJ, Gilboa E: Retroviral vector-mediated high-efficiency expression of adenosine deaminase (ADA) in hematopoietic long-term cultures of ADA-deficient marrow cells. Proc Natl Acad Sci USA 86:6748, 1989
3. Lim B, Apperley JF, Orkin SH, Williams DA: Long-term expression of human adenosine deaminase in mice transplanted with retrovirus-infected hematopoietic stem cells. Proc Natl Acad Sci USA 86:8892, 1989
4. Cournoyer D, Scarpa M, Mitam K, Moore KA, Markowitz D, Bank A, Belmont JW, Caskey CT: Gene transfer of adenosine deaminase into primitive human progenitor cells. Hum Gene Ther 2:203, 1991
5. Ferrari G, Rossini S, Nadia N, Maggioni D, Garofalo A, Giavazzi R, Mavilio F, Bordignon C: Transfer of the ADA gene into human ADA-deficient T lymphocytes reconstitutes specific immune functions. Blood 80:1120, 1992
6. van Beusechem VW, Kukler A, Heidt PJ, Valerio D: Long-term expression of human adenosine deaminase in rhesus monkeys transplanted with retrovirus-infected bone-marrow cells. Proc Natl Acad Sci USA 89:7640, 1992
7. Mitani K, Wakamiya M, Caskey CT: Long-term expression of retroviral-transduced adenosine deaminase in human primitive hematopoietic progenitors. Hum Gene Ther 4.9, 1993
8. Fink JK, Correll PH, Perry LK, Brady RO, Karlsson S: Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease. Proc Natl Acad Sci USA 87:2334, 1990
9. Nolta JA, Yu XJ, Bahner I, Kohn DB: Retroviral-mediated transfer of the human glucocerebrosidase gene into cultured Gaucher bone marrow. J Clin Invest 90:342, 1992
10. Wolfe JH, Sands MS, Barker JE, Gwynn B, Rowe LB, Vogler CA. Birkenmeier EH: Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature 360:749, 1992
11. + cells Blood 84:1408, 1994
12. Thrasher AJ, Keep NH, Wientjes F, Segal AW: Review: Chronic granulomatous disease. Biochim Biophys Acta 1227:1, 1994
13. Finn A, Hadzik N, Morgan G, Strobel S, Levinsky RJ: Prognosis of chronic granulomatous disease. Arch Dis Child 65:942, 1990
14. Porter CD, Parkar MH, Levinsky RJ, Collins MKL, Kinnon C: X-linked chronic granulomatous disease: Correction of NADPH oxidase defect by retrovirus-mediated expression of gp91-phox. Blood 82:2196, 1993
15. Porter CD, Parkar MH, Verhoeven AJ, Levinsky RJ, Collins MKL, Kinnon C: p22-phox-deficient chronic granulomatous disease: Reconstitution by retrovirus-mediated expression and identification of a biosynthetic intermediate of gp91-phox. Blood 84:2767, 1994
16. 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
17. phox cDNA. Proc Natl Acad Sci USA 89:10174, 1992
18. Volpp B, Lin Y: In vitro molecular reconstitution of the respiratory burst in B lymphoblasts from p47-phox-deficient chronic granulomatous disease. J Clin Invest 91:201, 1993
19. phox cDNA. J Exp Med 178:2047, 1993
20. Porter CD, Parkar MH, Collins MKL, Levinsky RJ, Kinnon C: Superoxide production by normal and chronic granulomatous disease (CGD) patient-derived EBV-transformed B cell lines measured by chemiluminescence-based assays. J Immunol Methods 155:151, 1992
21. phox-deficient chronic granulomatous disease. Proc Natl Acad Sci USA 90:7446, 1993
22. 558 defective forms of chronic granulomatous disease. Blood 84:53, 1994
23. van Beusechem VW, Kukler A, Einerhand MPW, Bakx TA, van der Eb AJ, van Bekkum DW, Valerio D: Expression of human adenosine deaminase in mice transplanted with hemopoietic stem cells infected with amphotropic retroviruses. J Exp Med 172:729, 1990
24. Roe T, Reynolds TC, Yu G, Brown PO: Integration of murine leukemia virus DNA depends on mitosis. EMBO J 12:2099, 1993
25. Bodine DM, Karlsson S, Nienhuis AW: Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells. Proc Natl Acad Sci USA 86:8897, 1989
26. Yorifuji T, Wilson RW, Beaudet AL: Retroviral mediated expression of CD18 in normal and deficient human bone marrow progenitor cells. Hum Mol Genet 2:1443, 1993
27. Nolta JA, Kohn DB: Comparison of the effects of growth factors on retroviral vector-mediated gene transfer and the proiferative status of human progenitor cells. Hum Gene Ther 1:257, 1990
28. Hughes PFD, Eaves CJ, Hogge DE, Humphries RK: High-efficiency gene transfer to human hematopoietic cells maintained in long-term marrow culture. Blood 74:1915, 1989
29. + marrow cells, devoid of T and B lymphocytes, reconstitute stable lymphopoeisis and myelopoiesis in lethally irradiated allogeneic baboons. Blood 80:1693, 1992
30. Cassel A, Cottier-Fox M, Doren S, Dunbar CE: Retroviral-mediated gene transfer into CD34-enriched human peripheral blood stem cells. Exp Hematol 21:585, 1993
31. Markowitz D, Goff S, Bank A: Construction and use of a safe and efficient amphotropic packaging cell line. Virology 167:400, 1988
32. Ferry N, Duplessis O, Houssin D, Danos O, Heard J-M: Retroviral-mediated gene transfer into hepatocytes in vivo. Proc Natl Acad Sci USA 88:8377, 1991
33. Patel P, Flemming CL, Fisher C, Porter CD, Thomas JM, Gore ME, Collins MKL: Generation of interleukin-2-secreting melanoma cell populations from resected metastatic tumors. Hum Gene Ther 5:577, 1994
34. Danos O: Construction of retroviral packaging cell lines, in Collins MKL (ed): Methods in Molecular Biology, vol 8. Clifton, NJ, Humana, 1991, p 17
35. Hatzfeld J, Li M-L, Brown EL, Sookdeo H, Levesque J-P, O'Toole T, Gurney C, Clark SC, Hatzfeld A: Release of early human hematopoietic progenitors from quiescence by antisense transforming growth factor β1 or Rb oligonucleotides. J Exp Med 174:925, 1991
36. Sutherland HJ, Lansdorp PM, Henkelman DH, Eaves AC, Eaves CJ: Functional characterisation of individual human hematopoietic stem cells cultured at limiting dilution on supportive marrow stromal layers. Proc Natl Acad Sci USA 87:3584, 1990
37. Lipscombe RJ, Sumiya M, Hill AVS, Lau YL, Levinsky RJ, Summerfield JA, Turner MW: High frequencies in African and non-African populations of independent mutations in the mannose binding protein gene. Hum Mol Genet 1:709, 1992
38. Apperley JF, Luskey BD, Williams DA: Retroviral gene transfer of human adenosine deaminase in murine hematopoietic cells: Effect of selectable marker sequences on long-term expression. Blood 78:310, 1991
39. Moore KA, Deisseroth AB, Reading CL, Williams DE, Belmont JW: Stromat support enhances cell-free retroviral vector transduction of human bone marrow long-term culture-initiating cells. Blood 79:1393, 1992
40. 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
41. Xu LC, Young HA, Blanco M, Kessler S, Roberts AB, Karlsson S: Poor transduction efficiency of human hematopoietic progenitor cells by a high-titre amphotropic retrovirus producer cell clone. J Virol 68:7634, 1994
42. Germeraad WTV, Asami N, Fujimoto S, Mazda O, Katsura Y: Efficient retrovirus-mediated gene transduction into murine hematopoietic cells and long-lasting expression using a transwell coculture system. Blood 84:780, 1994
43. Hughes PFD, Thacker JD, Hogge D, Sutherland HJ, Thomas TE, Lansdorp PM, Eaves CJ, Humphries RK: Retroviral gene transfer to primitive normal and leukemic hematopoietic cells, using clinically applicable procedures. J Clin Invest 89:1817, 1992
44. Hoeben RC, Migchielsen AAJ, van der Jagt RCM, van Ormondt H, van der Eb A: Inactivation of the Moloney murine leukemia virus long terminal repeat in murine fibroblast cell lines is associated with methylation and dependent on its chromosomal position. J Virol 65:904, 1991