Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors

Gene Therapy

Volumn 13, Issue 21, 2006, Pages 1524-1533

  Schambach, A ^a   Mueller, D ^a   Galla, M ^a   Verstegen, M M A ^b   Wagemaker, G ^b   Loew, R ^c   Baum, C ^a,d   Bohne, J ^a  

^a HANNOVER MEDICAL SCHOOL   (Germany)

^b ERASMUS MEDICAL CENTER   (Netherlands)

^c BIONTECH AG   (Germany)

^d CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; DNA; ENVELOPE PROTEIN; LENTIVIRUS VECTOR; METHYLATED DNA PROTEIN CYSTEINE METHYLTRANSFERASE; PHOSPHOGLYCERATE KINASE; PURINE; RETROVIRUS VECTOR; TETRACYCLINE;

ALLELE; ANIMAL CELL; ARTICLE; CD34 SELECTION; CONTROLLED STUDY; DNA PACKAGING; FLOW CYTOMETRY; FLUORESCENCE ANALYSIS; GENE INSERTION; GENETIC TRANSCRIPTION; GENETIC TRANSFECTION; HEMATOPOIETIC CELL; HUMAN; HUMAN CELL; LENTIVIRINAE; LONG TERMINAL REPEAT; MALE; NONHUMAN; NORTHERN BLOTTING; NUCLEOTIDE SEQUENCE; PRIMATE; PRIORITY JOURNAL; PROMOTER REGION; RETROVIRUS; ROUS SARCOMA ONCOVIRUS; SUPERNATANT; UPREGULATION; VIRAL GENE DELIVERY SYSTEM; VIRUS INACTIVATION;

ANIMALS; ANTIGENS, CD34; CELL LINE; FLOW CYTOMETRY; GAMMARETROVIRUS; GENE EXPRESSION REGULATION; GENE THERAPY; GENETIC ENGINEERING; GENETIC VECTORS; MACACA MULATTA; MALE; PLASMIDS; PROMOTER REGIONS (GENETICS); PROMOTER REGIONS, GENETIC; T-LYMPHOCYTES; TERMINAL REPEAT SEQUENCES; TRANSDUCTION, GENETIC; TRANSFECTION; VIRUS INACTIVATION;

PRIMATES; ROUS SARCOMA VIRUS;

EID: 33750023988     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/sj.gt.3302807     Document Type: Article

References (46)

1. Yu SF, von Ruden T, Kantoff PW, Garber C, Seiberg M, Ruther U et al. Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc Natl Acad Sci USA 1986; 83: 3194-3198.
2. Zaiss AK, Son S, Chang LJ. RNA 3′ readthrough of oncore-trovirus and lentivirus: Implications for vector safety and efficacy. J Virol 2002; 76: 7209-7219.
3. Kraunus J, Schaumann DH, Meyer J, Modlich U, Fehse B, Brandenburg G et al. Self-inactivating retroviral vectors with improved RNA processing. Gene Therapy 2004; 11: 1568-1578.
4. Ailles LE, Naldini L. HIV-1-derived lentiviral vectors. Curr Top Microbiol Immunol 2002; 261: 31-52.
5. Trobridge G, Josephson N, Vassilopoulos G, Mac J, Russell DW. Improved foamy virus vectors with minimal viral sequences. Mol Ther 2002; 6: 321-328.
6. Hildinger M, Abel KL, Ostertag W, Baum C. Design of 5′ untranslated sequences in retroviral vectors developed for medical use. J Virol 1999; 73: 4083-4089.
7. Gaspar HB, Parsley KL, Howe S, King D, Gilmour KC, Sinclair J et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 2004; 364: 2181-2187.
8. Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 296: 2410-2413.
9. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000; 288: 669-672.
10. Bonini C, Ferrari G, Verzeletti S, Servida P, Zappone E, Ruggieri L et al. HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science 1997; 276: 1719-1724.
11. Werner M, Kraunus J, Baum C, Brocker T. B-cell-specific transgene expression using a self-inactivating retroviral vector with human CD19 promoter and viral post-transcriptional regulatory element. Gene Therapy 2004; 11: 992-1000.
12. Hope T. Improving the post-transcriptional aspects of lentiviral vectors. Curr Top Microbiol Immunol 2002; 261: 179-189.
13. Popa I, Harris ME, Donello JE, Hope TJ. CRM1-dependent function of a cis-acting RNA export element. Mol Cell Biol 2002; 22: 2057-2067.
14. Follenzi A, Ailles LE, Bakovic S, Geuna M, Naldini L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat Genet 2000; 25: 217-222.
15. Dull T, Zufferey R, Kelly M, Mandel RJ, Nguyen M, Trono D et al. A third-generation lentivirus vector with a conditional packaging system. J Virol 1998; 72: 8463-8471.
16. Schambach A, Bohne J, Chandra S, Will E, Margison GP, Williams DA et al. Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. Mol Ther 2006; 13: 391-400.
17. Ragg S, Xu-Welliver M, Bailey J, D'Souza M, Cooper R, Chandra S et al. Direct reversal of DNA damage by mutant methyl-transferase protein protects mice against dose-intensified chemotherapy and leads to in vivo selection of hematopoietic stem cells. Cancer Res 2000; 60: 5187-5195.
18. Schambach A, Wodrich H, Hildinger M, Bohne J, Krausslich HG, Baum C. Context dependence of different modules for post-transcriptional enhancement of gene expression from retroviral vectors. Mol Ther 2000; 2: 435-445.
19. Baum C, Hegewisch-Becker S, Eckert HG, Stocking C, Ostertag W. Novel retroviral vectors for efficient expression of the multidrug resistance (mdr-1) gene in early hematopoietic cells. J Virol 1995; 69: 7541-7547.
20. Kinsella TM, Nolan GP. Episomal vectors rapidly and stably produce high-titer recombinant retrovirus. Hum Gene Ther 1996; 7: 1405-1413.
21. Hanawa H, Kelly PF, Nathwani AC, Persons DA, Vandergriff JA, Hargrove P et al. Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood. Mol Ther 2002; 5: 242-251.
22. Morita S, Kojima T, Kitamura T. Plat-E: An efficient and stable system for transient packaging of retroviruses. Gene Therapy 2000; 7: 1063-1070.
23. Vogt B, Roscher S, Abel B, Hildinger M, Lamarre A, Baum C et al. Lack of superinfection interference in retroviral vector producer cells. Hum Gene Ther 2001; 12: 359-365.
24. Treisman R, Maniatis T. Simian virus 40 enhancer increases number of RNA polymerase II molecules on linked DNA. Nature 1985; 315: 73-75.
25. Dean DA, Dean BS, Muller S, Smith LC. Sequence requirements for plasmid nuclear import. Exp Cell Res 1999; 253: 713-722.
26. Galla M, Will E, Kraunus J, Chen L, Baum C. Retroviral pseudotransduction for targeted cell manipulation. Mol Cell 2004; 16: 309-315.
27. Bevis BJ, Glick BS. Rapidly maturing variants of the Discosoma red fluorescent protein (DsRed). Nat Biotechnol 2002; 20: 83-87.
28. Adhya S, Gottesman M. Promoter occlusion: Transcription through a promoter may inhibit its activity. Cell 1982; 29: 939-944.
29. Proudfoot NJ. Transcriptional interference and termination between duplicated alpha-globin gene constructs suggests a novel mechanism for gene regulation. Nature 1986; 322: 562-565.
30. Emerman M, Temin HM. Genes with promoters in retrovirus vectors can be independently suppressed by an epigenetic mechanism. Cell 1984; 39: 449-467.
31. Emerman M, Temin HM. Comparison of promoter suppression in avian and murine retrovirus vectors. Nucleic Acids Res 1986; 14: 9381-9396.
32. Emerman M, Temin HM. Quantitative analysis of gene suppression in integrated retrovirus vectors. Mol Cell Biol 1986; 6: 792-800.
33. Eggermont J, Proudfoot NJ. Poly(A) signals and transcriptional pause sites combine to prevent interference between RNA polymerase II promoters. EMBO J 1993; 12: 2539-2548
34. Gossen M, Bujard H. Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc Natl Acad Sci USA 1992; 89: 5547-5551.
35. Haack K, Cockrell AS, Ma H, Israeli D, Ho SN, McCown TJ et al. Transactivator and structurally optimized inducible lentiviral vectors. Mol Ther 2004; 10: 585-596.
36. Vigna E, Cavalieri S, Ailles L, Geuna M, Loew R, Bujard H et al. Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors. Mol Ther 2002; 5: 252-261.
37. Xu K, Ma H, McCown TJ, Verma IM, Kafri T. Generation of a stable cell line producing high-titer self-inactivating lentiviral vectors. Mol Ther 2001; 3: 97-104.
38. Milsom MD, Fairbairn LJ. Protection and selection for gene therapy in the hematopoietic system. J Gene Med 2004; 6: 133-146.
39. Egelhofer M, Brandenburg G, Martinius H, Schult-Dietrich P, Melikyan G, Kunert R et al. Inhibition of HIV-1 entry in cells expressing Gp41-derived peptides. J Virol 2004; 78: 568-575.
40. Engels B, Cam H, Schüler T, Indraccolo S, Gladow M, Baum C et al. Retroviral vectors for high transgene expression in T lymphocytes. Hum Gene Ther 2003; 14: 1155-1168.
41. Cullen BR, Lomedico PT, Ju G. Transcriptional interference in avian retroviruses - implications for the promoter insertion model of leukaemogenesis. Nature 1984; 307: 241-245.
42. Greger IH, Demarchi F, Giacca M, Proudfoot NJ. Transcriptional interference perturbs the binding of Sp1 to the HIV-1 promoter. Nucleic Acids Res 1998; 26: 1294-1301.
43. Yankulov K, Blau J, Purton T, Roberts S, Bentley DL. Transcriptional elongation by RNA polymerase II is stimulated by transactivators. Cell 1994; 77: 749-759.
44. Neelis KJ, Dubbelman YD, Wognum AW, Thomas GR, Eaton DL, Egeland T et al. Lack of efficacy of thrombopoietin and granulocyte colony-stimulating factor after high dose total-body irradiation and autologous stem cell or bone marrow transplantation in rhesus monkeys. Exp Hematol 1997; 25: 1094-1103.
45. van Hennik PB, Verstegen MM, Bierhuizen MF, Limon A, Wognum AW, Cancelas JA et al. Highly efficient transduction of the green fluorescent protein gene in human umbilical cord blood stem cells capable of cobblestone formation in long-term cultures and multilineage engraftment of immunodeficient mice. Blood 1998; 92: 4013-4022.
46. Moritz T, Dutt P, Xiao X, Carstanjen D, Vik T, Hanenberg H et al. Fibronectin improves transduction of reconstituting hematopoietic stem cells by retroviral vectors: Evidence of direct viral binding to chymotryptic carboxy-terminal fragments. Blood 1996; 88: 855-862.