Down-modulation of cancer targets using locked nucleic acid (LNA)-based antisense oligonucleotides without transfection

Gene Therapy

Volumn 18, Issue 4, 2011, Pages 326-333

  Zhang, Y ^a   Qu, Z ^a   Kim, S ^a   Shi, V ^a   Liao, B ^a,b   Kraft, P ^a   Bandaru, R ^a   Wu, Y ^a   Greenberger, L M ^a   Horak, I D ^a  

^a Enzon Pharmaceuticals   (United States)

^b PFIZER INC   (United States)

Author keywords

antisense; down modulation; mRNA; oligonucleotide; siRNA

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; BETA CATENIN; EPIDERMAL GROWTH FACTOR RECEPTOR 3; EZN 3889; EZN 3920; EZN 4150; EZN 4516; HEAT SHOCK PROTEIN 27; LOCKED NUCLEIC ACID; MESSENGER RNA; PHOSPHATIDYLINOSITOL 3 KINASE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTISENSE THERAPY; ARTICLE; CANCER GENE THERAPY; CELL NUCLEUS; CELL PROLIFERATION; CONTROLLED STUDY; DOSE RESPONSE; DOWN REGULATION; DRUG POTENCY; DRUG TARGETING; FEMALE; GENE EXPRESSION; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; TUMOR XENOGRAFT;

ANIMALS; CELL LINE, TUMOR; GENE EXPRESSION REGULATION; GENE SILENCING; GENE TARGETING; HUMANS; NEOPLASMS; OLIGONUCLEOTIDES, ANTISENSE; RECEPTOR, ERBB-3; TRANSFECTION;

ANIMALIA;

EID: 79953767743     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2010.133     Document Type: Article

References (31)

1. Jason TL, Koropatnick J, Berg RW. Toxicology of antisense therapeutics. Toxicol Appl Pharmacol 2004; 201: 66-83. (Pubitemid 39446327)
2. Levin AA. A review of the issues in the pharmacokinetics and toxicology of phosphor-othioate antisense oligonucleotides. Biochimica et Biophysica Acta 1999; 1489: 69-84.
3. Crooke RM. In vitro toxicology and pharmacokinetics of antisense oligonucleotides. Anti-Cancer Drug Design 1991; 6: 609-646.
4. Potera C. Antisense-down, but not out. [see comment]. Nat Biotechnol 2007; 25: 497-499. (Pubitemid 46834827)
5. Rayburn ER, Wang H, Zhang R. Antisense-based cancer therapeutics: are we there yet? Exp Opin Emerg Drugs 2006; 11: 337-352. (Pubitemid 43723312)
6. Aleman LM, Doench J, Sharp PA. Comparison of siRNA-induced off-target RNA and protein effects. RNA-A Publ RNA Soc 2007; 13: 385-395. (Pubitemid 46327541)
7. Birmingham A, Anderson EM, Reynolds A, Ilsley-Tyree D, Leake D, Fedorov Y et al. 3¢ UTR seed matches, but not overall identity, are associated with RNAi off-targets [erratum appears in Nat Methods 2007;4(6):533]. Nat Methods 2006; 3: 199-204.
8. Jackson AL, Bartz SR, Schelter J, Kobayashi SV, Burchard J, Mao M et al. Expression profiling reveals off-target gene regulation by RNAi. Nat Biotechnol 2003; 21: 635-637. (Pubitemid 36638089)
9. Judge AD, Sood V, Shaw JR, Fang D, McClintock K, MacLachlan I. Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA. Nat Biotechnol 2005; 23: 457-462. (Pubitemid 41724921)
10. Scacheri PC, Rozenblatt-Rosen O, Caplen NJ, Wolfsberg TG, Umayam L, Lee JC et al. Short interfering RNAs can induce unexpected and divergent changes in the levels of untargeted proteins in mammalian cells. Proc Natl Acad Sci USA 2004; 101: 1892-1897. (Pubitemid 38228987)
11. Rabussay D. Applicator and electrode design for in vivo DNA delivery by electropora-tion. Methods Mol Biol 2008; 423: 35-59.
12. Cherry S. What have RNAi screens taught us about viral-host interactions? Curr Opin Microbiol 2009; 12: 446-452.
13. Veedu RN, Wengel J. Locked nucleic acid as a novel class of therapeutic agents. RNA Biol 2009; 6: 321-323.
14. Grunweller A, Hartmann RK. Locked nucleic acid oligonucleotides: the next generation of antisense agents? Biodrugs 2007; 21: 235-243. (Pubitemid 47104979)
15. Karkare S, Bhatnagar D. Promising nucleic acid analogs and mimics: characteristic features and applications of PNA, LNA, and morpholino. Appl Microbiol Biotechnol 2006; 71: 575-586. (Pubitemid 44114786)
16. Jepsen JS, Sorensen MD, Wengel J. Locked nucleic acid: a potent nucleic acid analog in therapeutics and biotechnology. Oligonucleotides 2004; 14: 130-146. (Pubitemid 38987382)
17. Orum H, Wengel J. Locked nucleic acids: a promising molecular family for gene-function analysis and antisense drug development. Curr Opin Mol Therapeut 2001; 3: 239-243. (Pubitemid 32624572)
18. Vester B, Wengel J. LNA (locked nucleic acid): high-affinity targeting of complementary RNA and DNA. Biochemistry 2004; 43: 13233-13241. (Pubitemid 39391124)
19. Li SM, Xu X, Liang HP, Li L. Progress in locked nucleic acid research. Sheng Li Ko Hsueh Chin Chan [Progr Physiol Sci] 2003; 34: 319-323.
20. Braasch DA, Corey DR. Locked nucleic acid (LNA): fine-tuning the recognition of DNA and RNA. Chem Biol 2001; 8: 1-7. (Pubitemid 32165807)
21. Greenberger LM, Horak ID, Filpula D, Sapra P, Westergaard M, Frydenlund HF et al. A RNA antagonist of hypoxia-inducible factor-1alpha, EZN-2968, inhibits tumor cell growth. Mol Cancer Therapeut 2008; 7: 3598-3608.
22. Hansen JB, Fisker N, Westergaard M, Kjaerulff LS, Hansen HF, Thrue CA et al. SPC3042: a proapoptotic survivin inhibitor. Mol Cancer Therapeut 2008; 7: 2736-2745.
23. Stein CA, Hansen JB, Lai J, Wu S, Voskresenskiy A, Hog A et al. Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents. Nucleic Acids Res 2010; 38: e3.
24. Castanotto D, Rossi JJ. The promises and pitfalls of RNA-interference- based therapeutics. Nature2009; 457: 426-433.
25. Jacobsen LB, Calvin SA, Lobenhofer EK. Transcriptional effects of transfection: the potential for misinterpretation of gene expression data generated from transiently transfected cells. BioTechniques 2009; 47: 617-624.
26. Abaza MS, Al-Saffar A, Al-Sawan S, Al-Attiyah R. c-myc antisense oligonucleotides sensitize human colorectal cancer cells to chemotherapeutic drugs. Tumour Biol 2008; 29: 287-303.
27. Salas PJ, Rodriguez ML, Viciana AL, Vega-Salas DE, Hauri HP. The apical submem-branecytoskeleton participates in the organization of the apical pole in epithelial cells. J Cell Biol 1997; 137: 359-375. (Pubitemid 27181290)
28. Baselga J, Swain SM. Novel anticancer targets: revisiting ERBB2 and discovering ERBB3. Nat Rev Cancer 2009; 9: 463-475.
29. Hsieh AC, Moasser MM. Targeting HER proteins in cancer therapy and the role of the non-target HER3. Br J Cancer 2007; 97: 453-457. (Pubitemid 47258458)
30. Ten Asbroek AL, Van Groenigen M, Nooij M, Baas F. The involvement of human ribonucleases H1 and H2 in the variation of response of cells to antisense phosphor-othioate oligonucleotides. Eur J Biochem 2002; 269: 583-592. (Pubitemid 34128016)
31. Lorenz P, Misteli T, Baker BF, Bennett CF, Spector DL. Nucleocytoplasmic shuttling: a novel in vivo property of antisense phosphorothioate oligodeoxynucleotides. Nucleic Acids Res 2000; 28: 582-592. (Pubitemid 30057999)