Specific Micro RNA-Regulated TetR-KRAB Transcriptional Control of Transgene Expression in Viral Vector-Transduced Cells

PLoS ONE

Volumn 7, Issue 12, 2012, Pages

  Pichard, Virginie ^a,d   Aubert, Dominique ^b   Boni, Sebastien ^a   Battaglia, Severine ^c   Ivacik, Dejana ^e   Nguyen, Tuan Huy ^a,d   Arbuthnot, Patrick ^e   Ferry, Nicolas ^a  

^a INSERM   (France)

^b UNIVERSITÉ DE NANTES   (France)

^c Universite de Nantes   (France)

^d Centre National de la Recherche Scientifique   (France)

^e UNIVERSITY OF THE WITWATERSRAND   (South Africa)

Author keywords

[No Author keywords available]

Indexed keywords

DOXYCYCLINE; KRUPPEL LIKE FACTOR; LENTIVIRUS VECTOR; MICRORNA; TETRACYCLINE;

ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; FLUORESCENCE ACTIVATED CELL SORTING; GENE EXPRESSION REGULATION; GENE EXPRESSION SYSTEM; GENE SILENCING; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; IN VIVO STUDY; LIVER CELL; MACROPHAGE; MOUSE; MUSCLE CELL; NONHUMAN; PROMOTER REGION; REPRESSOR GENE; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; RNA ANALYSIS; TISSUE SPECIFICITY; TISSUES; TRANSCRIPTION REGULATION; TRANSGENE;

ANIMALS; CELL LINE; CELL LINE, TUMOR; GENE SILENCING; GENETIC VECTORS; HEK293 CELLS; HUMANS; KRUPPEL-LIKE TRANSCRIPTION FACTORS; LIVER; MACROPHAGES; MICE; MICE, INBRED BALB C; MICRORNAS; MUSCLE CELLS; PROMOTER REGIONS, GENETIC; REPRESSOR PROTEINS; TRANSCRIPTION, GENETIC; TRANSGENES;

MURINAE;

EID: 84871338759     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0051952     Document Type: Article

References (33)

1. Di Domenico C, Di Napoli D, Gonzalez YRE, Lombardo A, Naldini L, et al. (2006) Limited transgene immune response and long-term expression of human alpha-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy. Hum Gene Ther 17: 1112-1121.
2. Pastore L, Morral N, Zhou H, Garcia R, Parks RJ, et al. (1999) Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors. Hum Gene Ther 10: 1773-1781.
3. Nicklin SA, Reynolds PN, Brosnan MJ, White SJ, Curiel DT, et al. (2001) Analysis of cell-specific promoters for viral gene therapy targeted at the vascular endothelium. Hypertension 38: 65-70.
4. Brown BD, Shi CX, Rawle FE, Tinlin S, McKinven A, et al. (2004) Factors influencing therapeutic efficacy and the host immune response to helper-dependent adenoviral gene therapy in hemophilia A mice. J Thromb Haemost 2: 111-118.
5. Schmitt F, Remy S, Dariel A, Flageul M, Pichard V, et al. (2010) Lentiviral vectors that express UGT1A1 in liver and contain miR-142 target sequences normalize hyperbilirubinemia in Gunn rats. Gastroenterology 139: 999-1007, 1007 e1001-1002.
6. Gossen M, Bujard H, (1992) Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc Natl Acad Sci U S A 89: 5547-5551.
7. Gossen M, Freundlieb S, Bender G, Muller G, Hillen W, et al. (1995) Transcriptional activation by tetracyclines in mammalian cells. Science 268: 1766-1769.
8. Deuschle U, Meyer WK, Thiesen HJ, (1995) Tetracycline-reversible silencing of eukaryotic promoters. Mol Cell Biol 15: 1907-1914.
9. Schultz DC, Friedman JR, Rauscher FJ, 3rd (2001) Targeting histone deacetylase complexes via KRAB-zinc finger proteins: the PHD and bromodomains of KAP-1 form a cooperative unit that recruits a novel isoform of the Mi-2alpha subunit of NuRD. Genes Dev 15: 428-443.
10. Underhill C, Qutob MS, Yee SP, Torchia J, (2000) A novel nuclear receptor corepressor complex, N-CoR, contains components of the mammalian SWI/SNF complex and the corepressor KAP-1. J Biol Chem 275: 40463-40470.
11. Groner AC, Meylan S, Ciuffi A, Zangger N, Ambrosini G, et al. (2010) KRAB-zinc finger proteins and KAP1 can mediate long-range transcriptional repression through heterochromatin spreading. PLoS Genet 6: e1000869.
12. Wiznerowicz M, Jakobsson J, Szulc J, Liao S, Quazzola A, et al. (2007) The Kruppel-associated box repressor domain can trigger de novo promoter methylation during mouse early embryogenesis. J Biol Chem 282: 34535-34541.
13. Laurenti E, Barde I, Verp S, Offner S, Wilson A, et al. (2010) Inducible gene and shRNA expression in resident hematopoietic stem cells in vivo. Stem Cells 28: 1390-1398.
14. Szulc J, Wiznerowicz M, Sauvain MO, Trono D, Aebischer P, (2006) A versatile tool for conditional gene expression and knockdown. Nat Methods 3: 109-116.
15. Brown BD, Venneri MA, Zingale A, Sergi Sergi L, Naldini L, (2006) Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat Med 12: 585-591.
16. Bovia F, Salmon P, Matthes T, Kvell K, Nguyen TH, et al. (2003) Efficient transduction of primary human B lymphocytes and nondividing myeloma B cells with HIV-1-derived lentiviral vectors. Blood 101: 1727-1733.
17. Landgraf P, Rusu M, Sheridan R, Sewer A, Iovino N, et al. (2007) A mammalian microRNA expression atlas based on small RNA library sequencing. Cell 129: 1401-1414.
18. Barde I, Laurenti E, Verp S, Groner AC, Towne C, et al. (2009) Regulation of episomal gene expression by KRAB/KAP1-mediated histone modifications. J Virol 83: 5574-5580.
19. Rittner K, Schultz H, Pavirani A, Mehtali M, (1997) Conditional repression of the E2 transcription unit in E1-E3-deleted adenovirus vectors is correlated with a strong reduction in viral DNA replication and late gene expression in vitro. J Virol 71: 3307-3311.
20. Proudfoot NJ, (1986) Transcriptional interference and termination between duplicated alpha-globin gene constructs suggests a novel mechanism for gene regulation. Nature 322: 562-565.
21. Greger IH, Demarchi F, Giacca M, Proudfoot NJ, (1998) Transcriptional interference perturbs the binding of Sp1 to the HIV-1 promoter. Nucleic Acids Res 26: 1294-1301.
22. Amendola M, Venneri MA, Biffi A, Vigna E, Naldini L, (2005) Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters. Nat Biotechnol 23: 108-116.
23. Andrianaki A, Siapati EK, Hirata RK, Russell DW, Vassilopoulos G, (2010) Dual transgene expression by foamy virus vectors carrying an endogenous bidirectional promoter. Gene Ther 17: 380-388.
24. Di Domenico C, Villani GR, Di Napoli D, Reyero EG, Lombardo A, et al. (2005) Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector. Hum Gene Ther 16: 81-90.
25. Aubert D, Menoret S, Chiari E, Pichard V, Durand S, et al. (2002) Cytotoxic immune response blunts long-term transgene expression after efficient retroviral-mediated hepatic gene transfer in rat. Mol Ther 5: 388-396.
26. Kootstra NA, Matsumura R, Verma IM, (2003) Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther 7: 623-631.
27. Wang L, Calcedo R, Wang H, Bell P, Grant R, et al. (2010) The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther 18: 126-134.
28. Hoffman BE, Martino AT, Sack BK, Cao O, Liao G, et al. (2011) Nonredundant roles of IL-10 and TGF-beta in suppression of immune responses to hepatic AAV-factor IX gene transfer. Mol Ther 19: 1263-1272.
29. Gao G, Wang Q, Calcedo R, Mays L, Bell P, et al. (2009) Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses. Hum Gene Ther 20: 930-942.
30. Brown BD, Cantore A, Annoni A, Sergi LS, Lombardo A, et al. (2007) A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood 110: 4144-4152.
31. Matsui H, Hegadorn C, Ozelo M, Burnett E, Tuttle A, et al. (2011) A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A. Mol Ther. 19: 723-730.
32. Latta-Mahieu M, Rolland M, Caillet C, Wang M, Kennel P, et al. (2002) Gene transfer of a chimeric trans-activator is immunogenic and results in short-lived transgene expression. Hum Gene Ther 13: 1611-1620.
33. Le Guiner C, Stieger K, Snyder RO, Rolling F, Moullier P, (2007) Immune responses to gene product of inducible promoters. Curr Gene Ther 7: 334-346.