A bicistronic retrovirus vector containing a picornavirus internal ribosome entry site allows for correction of X-linked CGD by selection for MDR1 expression

Blood

Volumn 87, Issue 1, 1996, Pages 42-50

  Sokolic, Robert A ^a   Sekhsaria, Sudhir ^c   Sugimoto, Yoshikazu ^c   Whiting Theobald, Narda ^c   Linton, Gilda F ^c   Li, Fei ^c   Gottesman, Michael M ^c   Malech, Harry L ^b  

^a BROWN UNIVERSITY   (United States)

^b NATIONAL INSTITUTES OF HEALTH   (United States)

^c NONE

Author keywords

[No Author keywords available]

Indexed keywords

ANTIBIOTIC G 418; GLYCOPROTEIN P; MESSENGER RNA; OXIDOREDUCTASE; SUPEROXIDE; VINCRISTINE;

ANIMAL CELL; ARTICLE; CELL SELECTION; CHEMOLUMINESCENCE; CHRONIC GRANULOMATOUS DISEASE; CISTRON; CONTROLLED STUDY; ENCEPHALOMYOCARDITIS VIRUS; EXPRESSION VECTOR; GENE EXPRESSION; GENE THERAPY; HUMAN; HUMAN CELL; LONG TERMINAL REPEAT; MOUSE; MULTIDRUG RESISTANCE; NONHUMAN; PHAGOCYTE; PICORNAVIRUS; PRIORITY JOURNAL; PROMOTER REGION; RECURRENT INFECTION; RETROVIRUS; RIBOSOME; X CHROMOSOME LINKED DISORDER;

EID: 0030045657     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v87.1.42.42     Document Type: Article

References (55)

1. Chen C-J, Chin JE, Kazumitsu U, Clark DP, Pastan I, Gottesman MM, Roninson IB: Internal duplication and homology with bacterial transport proteins in the mdr1 (P-glycoprotein) gene from multidrug-resistant human cells. Cell 47:381, 1986
2. Gottesman MM, Pastan I: Biochemistry of multidrug resistance mediated by the multidrug transporter. Annu Rev Biochem 62:385, 1993
3. Endicott JA, Ling V: The biochemistry of P-glycoprotein-mediated multidrug resistance. Annu Rev Biochem 58:137, 1989
4. de la Flor-Weiss E, Richardson C, Ward M, Himelstein A, Smith L, Podda S, Gottesman M, Pastan I, Bank A: Transfer and expression of the human multidrug resistance gene in mouse erythroleukemia cells. Blood 80:3106, 1992
5. Pastan I, Gottesman MM, Kazumitsu U, Lovelace E, Rutherford AV, Willingham MC: A retrovirus carrying an MDR1 cDNA confers multidrug resistance and polarized expression of P-glycoprotein in MDCK cells. Proc Natl Acad Sci USA 85:4486, 1988
6. Podda S, Ward M, Himelstein A, Richardson C, de la Flor-Weiss E, Smith L, Gottesman M, Pastan I, Bank A: Transfer and expression of the human multiple drug resistance gene into live mice. Proc Natl Acad Sci USA 86:9670, 1992
7. Mickisch GH, Licht T, Merlino GT, Gottesman MM, Pastan I: Chemotherapy and chemosensitization of transgenic mice which express the human multidrug resistance gene in bone marrow: Efficacy, potency, and toxicity. Cancer Res 51:5417, 1991
8. Mickisch GH, Merlino GT, Galski H, Gottesman MM, Pastan I: Transgenic mice that express the human multidrug-resistance gene in bone marrow enable a rapid identification of agents that reverse drug resistance. Proc Natl Acad Sci USA 88:547, 1991
9. Sorrentino BP, Brandt SJ, Bodine D, Gottesman MM, Paslan I, Cline A, Nienhuis AW: Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1. Science 257:99, 1992
10. Sugimoto Y, Aksentijevich I, Gottesman MM, Pastan I: Efficient expression of drug-selectable genes in retroviral vectors under control of an internal ribosome entry site. BioTechnology 12:694, 1994
11. Licht T, Aksentijevich I, Gottesman MM, Pastan I: Efficient expression of functional human MDR1 gene in murine bone marrow after retroviral transduction of purified hematopoietic stem cells. Blood 86:111, 1995
12. Hanania EG, Deisseroth AB: Serial transplantation shows that early hematopoietic precursor cells are transduced by MDR-1 retroviral vector in a mouse gene therapy model. Cancer Gene Therapy 1:21, 1994
13. Aran JM, Gottesman MM, Pastan I: Drug-selected coexpression of human glucocerebrosidase and P-glycoprotein using a bicistronic vector. Proc Natl Acad Sci USA 91:3176, 1994
14. Gottesman MM, Germann UA, Aksentijevich I, Sugimoto Y, Cardarelli CO, Pastan I: Gene transfer of drug resistance genes. Implications for cancer therapy. Ann NY Acad Sci 716:126, 1994
15. Sorrentino BP, McDonagh KT, Woods D, Orlic D: Expression of retroviral vectors containing the human multidrug resistance 1 cDNA in hematopoietic cells of transplanted mice. Blood 86:491, 1995
16. Miller AD, Rosman GJ: Improved retroviral vectors for gene transfer and expression. BioTechniques 7:980, 1989
17. Germann UA, Chin K-V, Pastan I, Gottesman MM: Retroviral transfer of a chimeric multidrug resistance-adenosine deaminase gene. FASEB J 4:1501, 1990
18. Davies M, Kaufman RJ: The sequence of the initiation codon in the encephalomyocarditis virus leader modulates efficiency of internal translation initiation J Virol 66.1924, 1992
19. Evstafieva AG, Ugarova TY, Chernov BK, Shatsky IN: A complex RNA sequence determines the internal initiation of encephalomyocarditis virus RNA translation. Nucleic Acids Res 16:665, 1990
20. Jang SK, Wimmer E: Cap-independent translation of encephalomyocarditis virus RNA: Structural elements of the internal ribosomal entry site and involvement of a cellular 57-kD RNA-binding protein. Genes Dev 4:1560, 1990
21. Jang SK, Pestova TV, Hellen CUT, Witherell GW, Wimmer E: Cap-independent translation of picornavirus RNAs: Structure and function of the internal ribosome entry site. Enzyme 44:292, 1990
22. Martínez-Salas E, Sáiz J-C, Dávila M, Belsham GJ, Domingo E: A single nucleotide substitution in the internal ribosome entry site of foot-and-mouth disease virus leads to enhanced cap-independent translation in vivo. J Virol 67:3748, 1993
23. Skinner MA, Racaniello VR, Dunn G, Cooper J, Minor PD, Almond JW: New model for the secondary structure of the 5′ non-coding RNA of poliovirus is supported by biochemical and genetic data that also show that RNA secondary structure is important in neurovirulence. J Molec Biol 207:379, 1989
24. Sonenberg N, Meerovitch K: Translation of poliovirus mRNA. Enzyme 44:278, 1990
25. Chen B-F, Hwang L-H, Chen D-S: Characterization of a bicistronic retroviral vector composed of the swine vesicular disease virus internal ribosome entry site. J Virol 67:2142, 1993
26. Ghattas IR, Sanes JR, Majors JE: The encephalomyocarditis virus internal ribosome entry site allows efficient coexpression of two genes from a recombinant provirus in cultured cells and in embryos. Molec Cell Biol 11:5848, 1991
27. Kim DG, Kang HM, Jang SK, Shin H-S: Construction of a bifunctional mRNA in the mouse by using the internal ribosomal entry site of the encephalomyocarditis virus. Mol Cell Biol 12:3636, 1992
28. Morgan RA, Couture L, Elroy-Stein O, Ragheb J, Moss B, Anderson WF: Retroviral vectors containing putative internal ribosome entry sites: Development of a polycistronic gene transfer system and applications to human gene transfer Nucleic Acids Res 20:1293, 1992
29. Malech HL, Gallin JI: Current concepts: Immunology. Neutrophils in human diseases. N Engl J Med 317:687, 1987
30. Gallin JI, Malech HL: Update on chronic granulomatous diseases of childhood. Immunotherapy and potential for gene therapy. JAMA 263:1533, 1990
31. Curnutte JT: Chronic granulomatous disease: The solving of a clinical riddle at the molecular level. Clin Immunol Immunopathol 67:S2, 1993 (suppl)
32. Malech HL: Phagocyte oxidative mechanisms. Curr Opin Hematol 1993:123, 1993
33. Maly FE, Schuerer-Maly CC, Quilliam L, Cochrane CG, Newburger PE, Curnutte JT, Gifford M, Dinauer MC: Restitution of superoxide generation in autosomal cytochrome-negative chronic granulomatous disease (A22(0) CGD)-derived B lymphocyte cell lines by transfection with p22phox cDNA. J Exp Med 178:2047, 1993
34. 2 production by B lymphocytes lacking the respiratory burst oxidase subunit p47phox after transfection with an expression vector containing a p47phox cDNA. Proc Natl Acad Sci USA 89:10174, 1992
35. Cobbs CS, Malech HL, Leto TL, Freeman SM, Blaese RM, Gallin JI, Lomax KJ: Retroviral expression of recombinant p47phox protein by Epstein-Barr virus-transformed B lymphocytes from a patient with autosomal chronic granulomatous disease. Blood 79:1829, 1992
36. 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
37. Volpp BD, 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
38. Sekhsaria S, Gallin JI, Linton GF, Mallory RM, Mulligan RC, Malech HL: Peripheral blood progenitors as a target for genetic correction of p47phox-deficient chronic granulomatous disease. Proc Natl Acad Sci USA 90:7446, 1993
39. Porter CD, Parkar MH, Levinsky RJ, Collins MK, Kinnon C: X-linked chronic granulomatous disease: Correction of NADPH oxidase defect by retrovirus-mediated expression of gp91-phox. Blood 82:2196, 1993
40. de Mendez I, Adams AG, Leto TL: In vivo reconstitution of NADPH oxidase deficient B lymphocytes with full length and deleted forms of p47 and p67-phox. Clin Res 41:278a, 1993 (abstr)
41. 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
42. 558 defective forms of chronic granulomatous disease. Blood 84:53, 1994
43. Miller AD, Buttimore C: Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production. Mol Cell Biol 6:2895, 1986
44. Kotani H, Newton PB 3rd, Zhang S, Chiang YL, Otto E, Weaver L, Blaese RM, Anderson WF, McGarrity GJ: Improved methods of retroviral vector transduction and production for gene therapy. Hum Gene Therapy 5:19, 1994
45. Blaese RM: Development of gene therapy for immunodeficiency: Adenosine deaminase deficiency. Pediatr Res 33.S49, 1993 (suppl)
46. Blaese RM, Culver KW: Gene therapy for primary immunodeficiency disease. Immunodeficiency Rev 3:329, 1992
47. Culver KW, Anderson WF, Blaese RM: Lymphocyte gene therapy Hum Gene Ther 2:107, 1991
48. 558 leading to X-linked chronic granulomatous disease. Blood 77:2482, 1991
49. Casimir C, Chetty M, Bohler M-C, Garcia R, Fischer A, Griscelli C, Johnson B, Segal AW: Identification of the defective NADPH-oxidase component in chronic granulomatous disease: A study of 57 European families. Eur J Clin Invest 22:403, 1992
50. Curnutte JT: Classification of chronic granulomatous disease. Hematol Oncol Clin North Am 2:241, 1988
51. Dinauer MC, Curnutte JT, Rosen H, Orkin SH: A missense mutation in the neutrophil cytochrome b heavy chain in cytochrome-positive X-linked chronic granulomatous disease. J Clin Invest 84:2012, 1989
52. Schapiro BL, Newburger PE, Klempner MS, Dinauer MC: Chronic granulomatous disease presenting in a 69-year-old man. N Engl J Med 325:1786, 1991
53. Chaudhary PM, Roninson IB: Expression and activity of P-glycoprotein, a multidrug efflux pump, in human hematopoietic stem cells. Cell 66:85, 1991
54. Drenou B, Fardel O, Amiot L, Fauchet R: Detection of P glycoprotein on normal and leukemic CD34+ cells. Leuk Res 17:1031, 1993
55. Kume A, Dinauer MC: Retrovirus-mediated reconstitution of respiratory burst activity in X-linked chronic granulomatous disease cells. Blood 84:3311, 1994