Novel viral vectors utilizing intron splice-switching to activate genome rescue, expression and replication in targeted cells

Virology Journal

Volumn 8, Issue , 2011, Pages

  Viru, Liane ^a   Heller, Gregory ^a   Lehto, Taavi ^a   Pärn, Kalle ^a   El Andaloussi, Samir ^a,b,c   Langel, Ülo ^a,b   Merits, Andres ^a  

^a UNIVERSITY OF TARTU   (Estonia)

^b STOCKHOLM UNIVERSITY   (Sweden)

^c KAROLINSKA INSTITUTE   (Sweden)

Author keywords

[No Author keywords available]

Indexed keywords

OLIGONUCLEOTIDE; VIRUS DNA; VIRUS RNA; VIRUS VECTOR;

ARTICLE; CANCER THERAPY; CELL CULTURE; CYTOTOXICITY; DNA SPLICING; GENE ACTIVATION; GENE EXPRESSION; GENE INSERTION; GENE REPLICATION; GENE THERAPY; GENETIC DISORDER; GENETIC TRANSFECTION; GENOME; INTRON; NONHUMAN; RNA SPLICING; VIRUS GENE; VIRUS INFECTION; VIRUS REPLICATION; ALTERNATIVE RNA SPLICING; BIOTECHNOLOGY; DRUG EFFECT; GENE EXPRESSION REGULATION; GENE VECTOR; GENETIC ENGINEERING; HELA CELL; HUMAN; METHODOLOGY; MOLECULAR BIOLOGY; VIROLOGY;

ADENOVIRIDAE; ALPHAVIRUS;

ALTERNATIVE SPLICING; BIOTECHNOLOGY; GENE EXPRESSION; GENE EXPRESSION REGULATION, VIRAL; GENETIC ENGINEERING; GENETIC VECTORS; HELA CELLS; HUMANS; INTRONS; MOLECULAR BIOLOGY; VIROLOGY; VIRUS REPLICATION;

EID: 79956034021     PISSN: None     EISSN: 1743422X     Source Type: Journal    
DOI: 10.1186/1743-422X-8-243     Document Type: Article

References (50)

1. Oncolytic (replication competent) adenoviruses as anti-cancer agents. K Toth D Dhar WS Wold, Expert Opin Biol Ther 2010 10 353 368 10.1517/ 14712590903559822 20132057
2. Oncolytic herpes simplex virus vectors and chemotherapy: are combinatorial strategies more effective for cancer? R Kanai H Wakimoto T Cheema SD Rabkin, Future Oncol 2010 6 619 634 10.2217/fon.10.18 20373873
3. Oncolytic parvoviruses as cancer therapeutics. J Rommelaere K Geletneky AL Angelova L Daeffler C Dinsart I Kiprianova RJ Schlehofer Z Raykov, Cytokine Growth Factor Rev 2010 21 185 195 10.1016/j.cytogfr.2010.02.011 20211577
4. Two-stage phase I dose-escalation study of intratumoral reovirus type 3 dearing and palliative radiotherapy in patients with advanced cancers. KJ Harrington EM Karapanagiotou V Roulstone KR Twigger CL White L Vidal D Beirne R Prestwich K Newbold M Ahmed K Thway CM Nutting M Coffey D Harris RG Vile HS Pandha JS Debono AA Melcher, Clin Cancer Res 2010 16 3067 3077 10.1158/1078-0432.CCR-10-0054 20484020
5. Vesicular stomatitis virus as an oncloytic vector. GN Barber, Viral Immunol 2004 17 516 527 10.1089/vim.2004.17.516 15671748 (Pubitemid 40054231)
6. Therapeutic and prophylactic applications of alphavirus vectors. GJ Atkins MN Fleeton GJ Sheahan, Expert Rev Mol Med 2008 11 33
7. ONYX-015, an E1B gene-attenuated adenovirus, causes tumor-specific cytolysis and antitumoral efficacy that can be augmented by standard chemotherapeutic agents. C Heise A Sampson-Johannes A Williams F McCormick DD Von Hoff DH Kirn, Nat Med 1997 3 639 645 10.1038/nm0697-639 9176490 (Pubitemid 27264973)
8. Genetic replacement of adenovirus shaft fiber reduces liver tropism in ovarian cancer gene therapy. M Breidenbach TD Rein M Wang DM Nettelbeck A Hemminki I Ulasov AR Rivera M Everts RD Alvarez JT Douglas DT Curiel, Hum Gene Ther 2004 15 509 518 10.1089/10430340460745829 15144580 (Pubitemid 38639621)
9. Re-targeted adenovirus vectors with dual specificity; binding specificities conferred by two different Affibody molecules in fiber. S Myhre P Henning M Freidman S Stahl L Lindholm MK Magnusson, Gene Ther 2009 16 252 261 10.1038/gt.2008.160 18946496
10. Prostate-specific antigene promoter/enhancer driven gene therapy for prostate cancer: construction and testing of tissue-specific adenovirus vector. JP Latham PF Searle V Mautner ND James, Cancer Res 2000 60 334 341 10667585 (Pubitemid 30070760)
11. Adenovirus-mediated suicide gene therapy under the control of Cox-2 promoter for colorectal cancer. ZX Wang HB Bian JS Yang W De XH Ji, Cancer Biol Ther 2009 8 1480 1488 10.4161/cbt.8.15.8940 19571664
12. A let-7 MicroRNA-sensitive vesicular stomatitis virus demonstrates tumor-specific replication. RE Edge TJ Falls CW Brown BD Lichty H Atkins JC Bell, Mol Ther 2008 16 1437 1443 10.1038/mt.2008.130 18560417
13. Oncolytic adenoviruses for treatment of brain tumors. C Gomez-Manzano J Fueyo, Curr Opin Mol Ther 2010 12 530 537 20886384
14. Adenoviridae: the viruses and their replication. AJ Berk, Fields Virology Lippincott Williams & Wilkins/Wolters Kulwer, Philadelphia, Knipe DM, Howley PM, fifth 2007 2 2355 2394
15. An adenovirus type 5 early gene function regulates expression of other early viral genes. N Jones T Shenk, Proc Natl Acad Sci USA 1979 76 3665 3669 10.1073/pnas.76.8.3665 291030 (Pubitemid 9246436)
16. The adenovirus type 5 E1A transcriptional control region contains a duplicated enhancer element. P Hearing T Shenk, Cell 1983 33 695 703 10.1016/0092-8674(83)90012-0 6871991
17. A simplified system for generating recombinant adenoviruses. TC He S Zhou LT da Costa J Yu KW Kinzler B Vogelstein, Proc Natl Acad Sci USA 1998 95 2509 2514 10.1073/pnas.95.5.2509 9482916 (Pubitemid 28145972)
18. Functional Sindbis virus replicative complexes are formed at the plasma membrane. EI Frolova R Gorchakov L Pereboeva S Atasheva I Frolov, J Virol 2010 84 11679 11695 10.1128/JVI.01441-10 20826696
19. Phosphatidylinositol 3-kinase-, actin-, and microtubule-dependent transport of Semliki Forest Virus replication complexes from plasma membrane to modified lysosomes. P Spuul G Balistreri L Kääriäinen T Ahola, J Virol 2010 84 7543 7557 10.1128/JVI.00477-10 20484502
20. Functions of alphavirus nonstructural proteins in RNA replication. L Kääriäinen T Ahola, Prog Nucleic Acid Res Mo Biol 2002 71 187 222
21. Properties and use of novel replication-competent vectors based on Semliki Forest virus. K Rausalu A Iofik L lper L Karo-Astover V Lulla A Merits, Virol J 2009 6 33 10.1186/1743-422X-6-33 19317912
22. A new generation of animal cell expression vectors based on the Semliki Forest virus replicon. P Liljeström H Garoff, Biotechnology (N Y) 1991 9 1356 1361 10.1038/nbt1291-1356
23. In vitro mutagenesis of a full-length cDNA clone of Semliki Forest virus: the small 6,000-molecular-weight membrane protein modulates virus release. P Liljeström S Lusa D Huylebroeck H Garoff, J Virol 1991 65 4107 4113 2072446
24. Sindbis virus DNA-based expression vectors: utility for in vitro and in vivo gene transfer. TW Dubensky Jr DA Driver JM Polo BA Belli EM Latham CE Ibanez S Chada D Brumm TA Banks SJ Mento DJ Jolly SMW Chang, J Virol 1996 70 508 519 8523564 (Pubitemid 126508819)
25. Therapeutic potential of splice-switching oligonucleotides. J Bauman N Jearawiriyapaisarn R Kole, Oligonucleotides 2009 19 1 13 10.1089/oli.2008.0161 19125639
26. Anti-tumor activity of splice-switching oligonucleotides. JA Bauman SD Li A Yang L Huang R Kole, Nucl Acids Res 2010 38 8348 8356 10.1093/nar/gkq731 20719743
27. An endogenous TNF-alpha antagonist induced by splice-switching oligonucleotides reduces inflammation in hepatitis and arthritis mouse models. MA Graziewicz TK Tarrant B Buckley J Roberts L Fulton H Hansen H rum R Kole P Sazani, Mol Ther 2008 16 1316 1322 10.1038/mt.2008.85 18461057 (Pubitemid 351866064)
28. The therapeutic potential of antisense-mediated exon skipping. GJ van Ommen J van Deutekom A Aartsma-Rus, Curr Opin Mol Ther 2008 10 140 149 18386226 (Pubitemid 351498876)
29. Construction, properties, and potential application of infectious plasmids containing Semliki Forest virus full-length cDNA with an inserted intron. L lper I Sarand K Rausalu A Merits, J Virol Methods 2008 148 265 270 10.1016/j.jviromet.2007.10.007 18054090 (Pubitemid 351248988)
30. Intron insertion facilitates amplification of clones virus cDNA in Escherichia coli while biological activity is re-established after transcription in vivo. IE Johansen, Proc Natl Acad Sci USA 1996 93 12400 12405 10.1073/pnas.93.22.12400 8901593 (Pubitemid 26367155)
31. Construction of full-length cDNA clones of lettuce mosaic virus (LMV) and the effects of intron-insertions on their viability in Escherichia coli and their infectivity to plants. SJ Yang F Revers S Souche H Lot O Le Gall T Candresse J Dunez, Arch Virol 1998 143 2443 2451 10.1007/s007050050474 9930200 (Pubitemid 29020117)
32. Construction of a stable and highly infectious intron-containing cDNA clone of plum pox potyvirus and its use to infect plants by particle bombardment. JJ Lopez-Moya JA Garcia, Virus Res 2000 68 99 107 10.1016/S0168-1702(00)00161-1 10958981 (Pubitemid 30665531)
33. Systemic Agrobacterium tumifaciens-mediated transfection of viral replicons for efficient transient expression in plants. S Marillonnet C Thoeringer R Kandzia V Klimyuk Y Gleba, Nature Biotechnol 2005 23 718 723 10.1038/nbt1094 (Pubitemid 41716362)
34. Stabilization of a full-length infectious cDNA clone of transmissible gastroenterits coronavirus by insertion an intron. JM Gonzalez Z Penzes F Almazan E Calvo L Enjuanes, J Virol 2002 76 4644 4661
35. Beta+thalassemia: aberrant splicing results from a single point mutation in an intron. M Busslinger N Moschonas RA Flavell, Cell 1981 27 289 298 10.1016/0092-8674(81)90412-8 6895866 (Pubitemid 12200498)
36. A case of theta-thalassemia with a C - - T substitution at position 654 of the second intervening sequence of the beta-globin gene. T Jo S Momita N Sadamori M Tomonaga S Fucharoem Y Fukumaki M Ichimaru, Intern Med 1992 31 269 272 10.2169/internalmedicine.31.269 1600278
37. Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides. Z Dominski R Kole, Proc Natl Acad Sci USA 1993 90 8673 8677 10.1073/pnas.90.18.8673 8378346 (Pubitemid 23277732)
38. Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development. SH Kang MJ Cho R Kole, Biochemistry 1998 37 6235 6239 10.1021/bi980300h 9572837 (Pubitemid 28213640)
39. Efficient and persistent splice switching by systemically delivered LNA oligonucleotides in mice. J Roberts E Palma P Sazani H rum M Cho R Kole, Mol Ther 2006 14 471 475 10.1016/j.ymthe.2006.05.017 16854630 (Pubitemid 44296096)
40. RNA repair restores hemoglobin expression in IVS2-641 thalassemic mice. S Svasti T Suwanmanee S Fucharoen HM Moulton MH Nelson N Maeda O Smithies R Kole, Proc Natl Acad Sci USA 2009 106 1205 1210 10.1073/pnas.0812436106 19164558
41. Insertion of EGFP into the replicase gene of Semliki Forest virus results in a novel, genetically stable marker virus. N Tamberg V Lulla R Fragkoudis A Lulla JK Fazakerley A Merits, J Gen Virol 2007 88 1225 1230 10.1099/vir.0.82436- 0 17374766 (Pubitemid 46523511)
42. Splice-switching efficiency and specificity for oligonucleotides with locked nucleic acid monomers. P Guterstam M Lindgren H Johansson U Tedebark J Wangel S El Andaloussi Langel, Biochem J 2008 412 307 313 10.1042/BJ20080013 18271753 (Pubitemid 351758236)
43. PKR-dependent and -independent mechanisms are involved in translational shutoff during Sindbis virus infection. R Gorchakov E Frolova BR Williams CM Rice I Frolov, J Virol 2004 78 8455 8467 10.1128/JVI.78.16.8455-8467.2004 15280454 (Pubitemid 39025111)
44. Novel functions of the alphavirus non-structural protein nsP3 C-terminal region. M Varjak E usinaite A Merits, J Virol 2010 84 2352 2364 10.1128/JVI.01540-09 20015978
45. Sindbis virus nonstructural protein nsP2 is cytotoxic and inhibits cellular transcription. N Garmashova R Gorchakov E Frolova I Frolov, J Virol 2006 80 5686 5696 10.1128/JVI.02739-05 16731907 (Pubitemid 43849146)
46. Alphavirus cDNA-based expression vectors: effects of RNA transcription and nuclear export. M Boorsma P Saudan H Pfruender JE Bailey S Schlesinger WA Renner FM Bachmann, Biotechnol Bioeng 2003 81 553 562 10.1002/bit.10496 12514804 (Pubitemid 36222314)
47. Switching on transgene expression by correcting aberrant splicing using multi-targeting steric-blocking oligonucleotides. S Resina R Kole A Travo B Lebleu AR Thierry, J Gen Med 2007 9 498 510 10.1002/jgm.1044 (Pubitemid 46993530)
48. The status of exon skipping as therapeutic approach to Duchenne muscular dystrophy. QL Lu T Yokota S Takeda L Garcia F Muntoni T Partridge, Mol Ther 2011 19 9 15 10.1038/mt.2010.219 20978473
49. Nucleic acid-based therapeutics in the battle against pathogenic viruses. J Haasnoot B Berkhout, Handb Exp Pharmacol 2009 189 243 263 10.1007/978-3-540-79086-0-9 19048203
50. Controlled propagation of replication-competent Sindbis viral vector using suicide gene strategy. JC Tseng G Daniels D Meruelo, Gene Ther 2009 16 291 296 10.1038/gt.2008.153 18818670