RNA interference is responsible for reduction of transgene expression after sleeping beauty transposase mediated somatic integration

PLoS ONE

Volumn 7, Issue 5, 2012, Pages

  Rauschhuber, Christina ^a   Ehrhardt, Anja ^a,b  

^a UNIVERSITY OF MUNICH   (Germany)

^b UNIVERSITY OF WITTEN HERDECKE   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; PROTEIN P19; SLEEPING BEAUTY TRANSPOSASE; SMALL INTERFERING RNA; TRANSPOSASE; UNCLASSIFIED DRUG;

ARTICLE; COLONY FORMING UNIT; CONTROLLED STUDY; DNA INTEGRATION; DNA TRANSPOSITION; GENE ACTIVITY; GENE FUNCTION; GENE REPRESSION; GENE SILENCING; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; PROTEIN BINDING; PROTEIN EXPRESSION; RNA INTERFERENCE; TRANSCRIPTION INITIATION; TRANSCRIPTION REGULATION; TRANSGENE; TRANSPOSON;

CELL LINE; DNA TRANSPOSABLE ELEMENTS; GENE EXPRESSION; GENE EXPRESSION REGULATION; GENE ORDER; GENE SILENCING; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; RNA INTERFERENCE; TRANSGENES; TRANSPOSASES; VIRAL PROTEINS;

EUKARYOTA; MAMMALIA; TOMATO BUSHY STUNT VIRUS;

EID: 84860520950     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0035389     Document Type: Article

References (56)

1. Groth AC, Olivares EC, Thyagarajan B, Calos MP, (2000) A phage integrase directs efficient site-specific integration in human cells. Proc Natl Acad Sci U S A 97: 5995-6000.
2. Ivics Z, Hackett PB, Plasterk RH, Izsvak Z, (1997) Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells. Cell 91: 501-510.
3. Kuhstoss S, Rao RN, (1991) Analysis of the integration function of the streptomycete bacteriophage phi C31. J Mol Biol 222: 897-908.
4. Miskey C, Izsvak Z, Plasterk RH, Ivics Z, (2003) The Frog Prince: a reconstructed transposon from Rana pipiens with high transpositional activity in vertebrate cells. Nucleic Acids Res 31: 6873-6881.
5. Ding S, Wu X, Li G, Han M, Zhuang Y, et al. (2005) Efficient transposition of the piggyBac (PB) transposon in mammalian cells and mice. Cell 122: 473-483.
6. Doherty JE, Huye L, Yusa K, Zhou L, Craig N, et al. (2011) Hyperactive piggyBac gene transfer in human cells and in vivo. Hum Gene Ther.
7. Olivares EC, Hollis RP, Chalberg TW, Meuse L, Kay MA, et al. (2002) Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol 20: 1124-1128.
8. Ehrhardt A, Yant SR, Giering JC, Xu H, Engler JA, et al. (2007) Somatic integration from an adenoviral hybrid vector into a hot spot in mouse liver results in persistent transgene expression levels in vivo. Mol Ther 15: 146-156.
9. Yusa K, Rad R, Takeda J, Bradley A, (2009) Generation of transgene-free induced pluripotent mouse stem cells by the piggyBac transposon. Nat Methods 6: 363-369.
10. Nakazawa Y, Huye LE, Salsman VS, Leen AM, Ahmed N, et al. (2011) PiggyBac-mediated Cancer Immunotherapy Using EBV-specific Cytotoxic T-cells Expressing HER2-specific Chimeric Antigen Receptor. Mol Ther.
11. Ehrhardt A, Xu H, Huang Z, Engler JA, Kay MA, (2005) A direct comparison of two nonviral gene therapy vectors for somatic integration: in vivo evaluation of the bacteriophage integrase phiC31 and the Sleeping Beauty transposase. Mol Ther 11: 695-706.
12. Yant SR, Ehrhardt A, Mikkelsen JG, Meuse L, Pham T, et al. (2002) Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo. Nat Biotechnol 20: 999-1005.
13. Yant SR, Meuse L, Chiu W, Ivics Z, Izsvak Z, et al. (2000) Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system. Nat Genet 25: 35-41.
14. Mates L, Chuah MK, Belay E, Jerchow B, Manoj N, et al. (2009) Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates. Nat Genet 41: 753-761.
15. Robert VJ, Vastenhouw NL, Plasterk RH, (2004) RNA interference, transposon silencing, and cosuppression in the Caenorhabditis elegans germ line: similarities and differences. Cold Spring Harb Symp Quant Biol 69: 397-402.
16. Vastenhouw NL, Fischer SE, Robert VJ, Thijssen KL, Fraser AG, et al. (2003) A genome-wide screen identifies 27 genes involved in transposon silencing in C. elegans. Curr Biol 13: 1311-1316.
17. Vastenhouw NL, Plasterk RH, (2004) RNAi protects the Caenorhabditis elegans germline against transposition. Trends Genet 20: 314-319.
18. Janowski BA, Huffman KE, Schwartz JC, Ram R, Nordsell R, et al. (2006) Involvement of AGO1 and AGO2 in mammalian transcriptional silencing. Nat Struct Mol Biol 13: 787-792.
19. Calero-Nieto FJ, Bert AG, Cockerill PN, (2010) Transcription-dependent silencing of inducible convergent transgenes in transgenic mice. Epigenetics Chromatin 3: 3.
20. Morris KV, (2005) siRNA-mediated transcriptional gene silencing: the potential mechanism and a possible role in the histone code. Cell Mol Life Sci 62: 3057-3066.
21. Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, et al. (2001) Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 411: 494-498.
22. Elbashir SM, Lendeckel W, Tuschl T, (2001) RNA interference is mediated by 21- and 22-nucleotide RNAs. Genes Dev 15: 188-200.
23. Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, et al. (1998) Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391: 806-811.
24. Hammond SM, Bernstein E, Beach D, Hannon GJ, (2000) An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells. Nature 404: 293-296.
25. Yang N, Kazazian HH Jr, (2006) L1 retrotransposition is suppressed by endogenously encoded small interfering RNAs in human cultured cells. Nat Struct Mol Biol 13: 763-771.
26. Moldt B, Yant SR, Andersen PR, Kay MA, Mikkelsen JG, (2007) Cis-acting gene regulatory activities in the terminal regions of sleeping beauty DNA transposon-based vectors. Hum Gene Ther 18: 1193-1204.
27. Walisko O, Schorn A, Rolfs F, Devaraj A, Miskey C, et al. (2008) Transcriptional activities of the Sleeping Beauty transposon and shielding its genetic cargo with insulators. Mol Ther 16: 359-369.
28. Djupedal I, Ekwall K, (2009) Epigenetics: heterochromatin meets RNAi. Cell Res 19: 282-295.
29. Morris KV, (2009) Long antisense non-coding RNAs function to direct epigenetic complexes that regulate transcription in human cells. Epigenetics 4: 296-301.
30. Scholthof HB, (2006) The Tombusvirus-encoded P19: from irrelevance to elegance. Nat Rev Microbiol 4: 405-411.
31. Voinnet O, Pinto YM, Baulcombe DC, (1999) Suppression of gene silencing: a general strategy used by diverse DNA and RNA viruses of plants. Proc Natl Acad Sci U S A 96: 14147-14152.
32. Dunoyer P, Lecellier CH, Parizotto EA, Himber C, Voinnet O, (2004) Probing the microRNA and small interfering RNA pathways with virus-encoded suppressors of RNA silencing. Plant Cell 16: 1235-1250.
33. Omarov R, Sparks K, Smith L, Zindovic J, Scholthof HB, (2006) Biological relevance of a stable biochemical interaction between the tombusvirus-encoded P19 and short interfering RNAs. J Virol 80: 3000-3008.
34. Silhavy D, Molnar A, Lucioli A, Szittya G, Hornyik C, et al. (2002) A viral protein suppresses RNA silencing and binds silencing-generated, 21- to 25-nucleotide double-stranded RNAs. EMBO J 21: 3070-3080.
35. Dunoyer P, Thomas C, Harrison S, Revers F, Maule A, (2004) A cysteine-rich plant protein potentiates Potyvirus movement through an interaction with the virus genome-linked protein VPg. J Virol 78: 2301-2309.
36. Kawasaki H, Taira K, (2004) MicroRNA-196 inhibits HOXB8 expression in myeloid differentiation of HL60 cells. Nucleic Acids Symp Ser (Oxf) pp. 211-212.
37. Mutskov VJ, Farrell CM, Wade PA, Wolffe AP, Felsenfeld G, (2002) The barrier function of an insulator couples high histone acetylation levels with specific protection of promoter DNA from methylation. Genes Dev 16: 1540-1554.
38. Li CL, Emery DW, (2008) The cHS4 chromatin insulator reduces gammaretroviral vector silencing by epigenetic modifications of integrated provirus. Gene Ther 15: 49-53.
39. Yi R, Qin Y, Macara IG, Cullen BR, (2003) Expotin-5 mediates the nuclear exprot of pre-miRNAs and short hairpin RNAs. Genes Dev 17: 3011-3016.
40. Zeng Y, Cullen BR, (2004) Structural requirements for pre-microRNA binding and nuclear export by Exportin 5. Nucleic Acids Res 32: 4776-4785.
41. Hutvagner G, McLachlan J, Pasquinelli AE, Balint E, Tuschl T, et al. (2001) A cellular function for the RNA-interference enzyme Dicer in the maturation of the let-7 small temporal RNA. Science 293: 834-838.
42. Ketting RF, Fischer SEJ, Bernstein E, Sijen T, Hannon GJ, et al. (2001) Dicer functions in RNA interference and in synthesis of small RNA involved in developmental timing in C.elegans. Genes Dev 15: 2654-2659.
43. Meister G, (2004) Human Argonaute2 mediates RNA cleavage targeted by miRNAs and siRNAs. Mol Cell 15: 185-197.
44. Mourelatos Z, Dostie J, Paushkin S, Sharma AK, Charroux B, et al. (2002) miRNPs: A novel class of ribonucleoproteins containing numerous microRNAs. Genes & Development 16: 720-728.
45. Morris KV, Chan SW, Jacobsen SE, Looney DJ, (2004) Small interfering RNA-induced transcriptional gene silencing in human cells. Science 305: 1289-1292.
46. Garrison BS, Yant SR, Mikkelsen JG, Kay MA, (2007) Postintegrative gene silencing within the Sleeping Beauty transposition system. Mol Cell Biol 27: 8824-8833.
47. Kim DH, Villeneuve LM, Morris KV, Rossi JJ, (2006) Argonaute-1 directs siRNA-mediated transcriptional gene silencing in human cells. Nat Struct Mol Biol 13: 793-797.
48. Skalsky RL, Cullen BR, (2010) Viruses, microRNAs, and host interactions. Annu Rev Microbiol 64: 123-141.
49. Andersson MG, Haasnoot PC, Xu N, Berenjian S, Berkhout B, et al. (2005) Suppression of RNA interference by adenovirus virus-associated RNA. J Virol 79: 9556-9565.
50. Aparicio O, Razquin N, Zaratiegui M, Narvaiza I, Fortes P, (2006) Adenovirus virus-associated RNA is processed to functional interfering RNAs involved in virus production. J Virol 80: 1376-1384.
51. Pfeffer S, Zavolan M, Grässer FA, Chien M, Russo JJ, et al. (2004) Identification of Virus- Encoded MicroRNAs. Science 304: 734-736.
52. Ehrhardt A, Kay MA, (2002) A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo. Blood 99: 3923-3930.
53. Uetz P, Dong YA, Zeretzke C, Atzler C, Baiker A, et al. (2006) Herpesviral protein networks and their interaction with the human proteome. Science 311: 239-242.
54. Vizoso Pinto MG, Pfrepper KI, Janke T, Noelting C, Sander M, et al. (2010) A systematic approach for the identification of novel, serologically reactive recombinant Varicella-Zoster Virus (VZV) antigens. Virol J 7: 165.
55. Shi R, Chiang VL, (2005) Facile means for quantifying microRNA expression by real-time PCR. Biotechniques 39: 519-525.
56. Yant SR, Huang Y, Akache B, Kay MA, (2007) Site-directed transposon integration in human cells. Nucleic Acids Res 35: e50.