Sequence-specific inhibition of RNA polymerase III-dependent transcription using Zorro locked nucleic acid (LNA)

Journal of Gene Medicine

Volumn 10, Issue 1, 2008, Pages 101-109

  Ge, Rongbin ^a   Svahn, Mathias G ^a   Simonson, Oscar E ^a   Mohamed, Abdalla J ^a   Lundin, Karin E ^a   Smith, C I Edvard ^a  

^a KAROLINSKA UNIVERSITY HOSPITAL   (Sweden)

Author keywords

Locked nucleic acid; RNA polymerase III; shRNA; Transcription

Indexed keywords

DNA DIRECTED RNA POLYMERASE III; LOCKED NUCLEIC ACID; LUCIFERASE; PLASMID VECTOR; RNA POLYMERASE; RNA POLYMERASE U6; SHORT HAIRPIN LUCIFERASE; SHORT HAIRPIN RNA; UNCLASSIFIED DRUG; ZORRO LOCKED NUCLEIC ACID;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BINDING SITE; CONTROLLED STUDY; ENZYME INHIBITION; FEMALE; GENE CONSTRUCT; GENE SILENCING; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; IN VIVO STUDY; MOUSE; NONHUMAN; NUCLEIC ACID HYBRIDIZATION; PRIORITY JOURNAL; RNA PROCESSING; TRANSCRIPTION REGULATION;

ANIMALS; BASE SEQUENCE; DNA; HUMANS; INJECTIONS, INTRAMUSCULAR; MICE; MODELS, ANIMAL; MOLECULAR SEQUENCE DATA; NIH 3T3 CELLS; OLIGONUCLEOTIDES; PROMOTER REGIONS (GENETICS); RNA POLYMERASE III; TRANSCRIPTION, GENETIC; TRANSFECTION;

MAMMALIA;

EID: 38349155431     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1124     Document Type: Article

References (41)

1. Schramm L, Hernandez N. Recruitment of RNA polymerase III to its target promoters. Genes Dev 2002; 16: 2593-2620.
2. Pombo A, Jackson DA, Hollinshead M, et al. Regional specialization in human nuclei: visualization of discrete sites of transcription by RNA polymerase III. EMBO J 1999; 18: 2241-2253.
3. Cairns CA, White RJ. p53 is a general repressor of RNA polymerase III transcription. EMBO J 1998; 17: 3112-3123.
4. Meister G, Tuschl T. Mechanisms of gene silencing by double-stranded RNA. Nature 2004; 431: 343-349.
5. Fire A, Xu S, Montgomery MK, et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998; 391: 806-811.
6. Elbashir SM, Harborth J, Lendeckel W, et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001; 411: 494-498.
7. Hannon GJ, Rossi JJ. Unlocking the potential of the human genome with RNA interference. Nature 2004; 431: 371-378.
8. Brummelkamp TR, Bernards R, Agami R. A system for stable expression of short interfering RNAs in mammalian cells. Science 2002; 296: 550-553.
9. McManus MT, Sharp PA. Gene silencing in mammals by small interfering RNAs. Nat Rev Genet 2002; 3: 737-747.
10. Miyagishi M, Taira K. U6 promoter-driven siRNAs with four uridine 3′ overhangs efficiently suppress targeted gene expression in mammalian cells. Nat Biotechnol 2002; 20: 497-500.
11. Paddison PJ, Caudy AA, Bernstein E, et al. Short hairpin RNAs (shRNAs) induce sequence-specific silencing in mammalian cells. Genes Dev 2002; 16: 948-958.
12. Paul CP, Good PD, Winer I, et al. Effective expression of small interfering RNA in human cells. Nat Biotechnol 2002; 20: 505-508.
13. Yu JY, DeRuiter SL, Turner DL. RNA interference by expression of short-interfering RNAs and hairpin RNAs in mammalian cells. Proc Natl Acad Sci U S A 2002; 99: 6047-6052.
14. Braasch DA, Corey DR. Locked nucleic acid (LNA): fine-tuning the recognition of DNA and RNA. Chem Biol 2001; 8: 1-7.
15. Koshkin AA, Wengel J. Synthesis of novel 2′,3′-linked bicyclic thymine ribonucleosides. J Org Chem 1998; 63: 2778-2781.
16. Obika S, Nanbu D, Hari Y, et al. Stability and structural features of the duplexes containing nucleoside analogues with a fixed N-type conformation, 2′-O,4′-C-methyleneribonucleosides. Tetrahedron Lett 1998; 39: 5401.
17. Singh SK, Kumar R, Wengel J. Synthesis of novel bicyclo[2.2.1] ribonucleosides: 2′-amino- and 2′-thio-LNA monomeric nucleosides. J Org Chem 1998; 63: 6078-6079.
18. Vester B, Wengel J. LNA (locked nucleic acid): high-affinity targeting of complementary RNA and DNA. Biochemistry 2004; 43: 13233-13241.
19. Hertoghs KM, Ellis JH, Catchpole IR. Use of locked nucleic acid oligonucleotides to add functionality to plasmid DNA. Nucleic Acids Res 2003; 31: 5817-5830.
20. Parekh-Olmedo H, Drury M, Kmiec EB. Targeted nucleotide exchange in Saccharomyces cerevisiae directed by short oligonucleotides containing locked nucleic acids. Chem Biol 2002; 9: 1073-1084.
21. Brunet E, Alberti P, Perrouault L, et al. Exploring cellular activity of locked nucleic acid-modified triplex-forming oligonucleotides and defining its molecular basis. J Biol Chem 2005; 280: 20076-20085.
22. Brunet E, Corgnali M, Perrouault L, et al. Intercalator conjugates of pyrimidine locked nucleic acid-modified triplex-forming oligonucleotides: improving DNA binding properties and reaching cellular activities. Nucleic Acids Res 2005; 33: 4223-4234.
23. Ge R, Heinonen JE, Svahn MG, et al. Zorro locked nucleic acid induces sequence-specific gene silencing. FASEB J 2007; 21: 1902-1914.
24. Lundin KE, Ge R, Svahn MG, et al. Cooperative strand invasion of supercoiled plasmid DNA by mixed linear PNA and PNA-peptide chimeras. Biomol Eng 2004; 21: 51-59.
25. Catapano CV, McGuffie EM, Pacheco D, et al. Inhibition of gene expression and cell proliferation by triple helix-forming oligonucleotides directed to the c-myc gene. Biochemistry 2000; 39: 5126-5138.
26. Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther 1999; 6: 1258-1266.
27. Zhang G, Budker V, Wolff JA. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum Gene Ther 1999; 10: 1735-1737.
28. Contag CH, Spilman SD, Contag PR, et al. Visualizing gene expression in living mammals using a bioluminescent reporter. Photochem Photobiol 1997; 66: 523-531.
29. Lundin KE, Hasan M, Moreno PM, et al. Increased stability and specificity through combined hybridization of peptide nucleic acid (PNA) and locked nucleic acid (LNA) to supercoiled plasmids for PNA-anchored "Bioplex" formation. Biomol Eng 2005; 22: 185.
30. Kurakin A, Larsen HJ, Nielsen PE. Cooperative strand displacement by peptide nucleic acid (PNA). Chem Biol 1998; 5: 81-89.
31. Peffer NJ, Hanvey JC, Bisi JE, et al. Strand-invasion of duplex DNA by peptide nucleic acid oligomers. Proc Natl Acad Sci U S A 1993; 90: 10648-10652.
32. Cherny DY, Belotserkovskii BP, Frank-Kamenetskii MD, et al. DNA unwinding upon strand-displacement binding of a thymine-substituted polyamide to double-stranded DNA. Proc Natl Acad Sci U S A 1993; 90: 1667-1670.
33. Braglia P, Percudani R, Dieci G. Sequence context effects on oligo(dT) termination signal recognition by Saccharomyces cerevisiae RNA polymerase III. J Biol Chem 2005; 280: 19551-19562.
34. Beane RL, Ram R, Gabillet S, et al. Inhibiting gene expression with locked nucleic acids (LNAs) that target chromosomal DNA. Biochemistry 2007; 46: 7572-7580.
35. Abes R, Arzumanov AA, Moulton HM, et al. Cell-penetrating-peptide-based delivery of oligonucleotides: an overview. Biochem Soc Trans 2007; 35: 775-779.
36. Branden LJ, Mohamed AJ, Smith CI. A peptide nucleic acid-nuclear localization signal fusion that mediates nuclear transport of DNA. Nat Biotechnol 1999; 17: 784-787.
37. Elmen J, Thonberg H, Ljungberg K, et al. Locked nucleic acid (LNA) mediated improvements in siRNA stability and functionality. Nucleic Acids Res 2005; 33: 439-447.
38. Schmidt KS, Borkowski S, Kurreck J, et al. Application of locked nucleic acids to improve aptamer in vivo stability and targeting function. Nucleic Acids Res 2004; 32: 5757-5765.
39. Swayze EE, Siwkowski AM, Wancewicz EV, et al. Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals. Nucleic Acids Res 2007; 35: 687-700.
40. Roberts J, Palma E, Sazani P, et al. Efficient and persistent splice switching by systemically delivered LNA oligonucleotides in mice. Mol Ther 2006; 14: 471-475.
41. McCaffrey AP, Meuse L, Karimi M, et al. A potent and specific morpholino antisense inhibitor of hepatitis C translation in mice. Hepatology 2003; 38: 503-508.