Targeting of cancer-related proteins with PNA oligomers

Current Cancer Drug Targets

Volumn 1, Issue 3, 2001, Pages 231-239

  Poogai, Margus ^b   Langel, Ülo ^a,b  

^a UNIVERSITY OF TARTU   (Estonia)

^b STOCKHOLM UNIVERSITY   (Sweden)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; PEPTIDE NUCLEIC ACID; TUMOR PROTEIN;

ANIMAL; CHEMISTRY; DRUG EFFECT; GENE THERAPY; GENETIC TRANSCRIPTION; GENETICS; HUMAN; METHODOLOGY; MOLECULAR MIMICRY; NEOPLASM; NUCLEIC ACID PROBE; PROTEIN SYNTHESIS; REVIEW;

ANIMALS; ANTISENSE ELEMENTS (GENETICS); GENE THERAPY; HUMANS; MOLECULAR MIMICRY; NEOPLASM PROTEINS; NEOPLASMS; OLIGONUCLEOTIDES, ANTISENSE; PEPTIDE NUCLEIC ACIDS; PROTEIN BIOSYNTHESIS; TRANSCRIPTION, GENETIC;

EID: 0035502362     PISSN: 15680096     EISSN: None     Source Type: Journal    
DOI: 10.2174/1568009013334142     Document Type: Article

References (64)

1. Pawlak, W.; Zolnierek, J.; Sarosiek, T.; Szczylik, C. Antisense Therapy in Cancer. Cancer Treat. Rev. 2000, 26, 333-350.
2. Williams, S. A.; Chang, L.; Buzby, J. S.; Suen, Y.; Cairo, M. S. Cationic Lipids Reduce Time and Dose of c-myc Antisense Oligodeoxynucleotides Required to Specifically Inhibit Burkitt's Lymphoma Cell Growth. Leukemia. 1996,10, 1980-1989.
3. Kronenwett, R.; Steidl, U.; Kirsch, M.; Sczakiel, G.; Haas, R. Oligodeoxyribonucleotide Uptake in Primary Human Hematopoietic cells is Enhanced by Cationic Lipids and Depends on the Hematopoietic Cell Subset. Blood. 1998, 91, 852-862.
4. Yuen, A. R.; Sikic, B.I. Clinical Studies of Antisense Therapy in Cancer. Front. Biosci. 2000, 5, D588-593.
5. Monia, B. P.; Johnston, J.E.; Geiger, T.; Muller, M.; Fabbro, D. Antitumor Activity of a Phosphorothioate Antisense Oligodeoxynucleotide Targeted against C-raf Kinase. Nat. Med. 1996, 2, 668-675.
6. Monia, B. P.; Sasmor,'H.; Johnston, J. F.; Freier, S. M.; Lesnik, E. A.; Muller, M.; Geiger, T.; Altmann, K. H.; Moser, H.; Fabbro, D. Sequence-specific Antitumor Activity of a Phosphorothioate Oligodeoxyribonucleotide Targeted to Human C-raf Kinase Supports an Antisense Mechanism of Action in vivo. Proc. Natl. Acad. Sei. USA 1996,93, 1548115484.
7. Cunningham, C. C.; Holmlund, J. T.; Schiller, J. H.; Geary, R. S.; Kwoh, T. J.; Dorr, A.; Nemunaitis, J. A Phase I Trial of c-Raf Kinase Antisense Oligonucleotide ISIS 5132 Administered as a Continuous Intravenous Infusion in Patients with Advanced Cancer. Clin. Cancer Res. 2000, 6, 1626-1631.
8. Stevenson, J. P.; Yao, K. S.; Gallagher, M.; Friedland, D.; Mitchell, E. P.; Cassella, A.; Monia, B.; Kwoh, T. J.; Yu, R.; Holmlund, J.; Dorr, F. A.; O'Dwyer, P. J. Phase I Clinical/pharmacokinetic and Pharmacodynamic Trial of the c-raf- 1 Antisense Oligonucleotide ISIS 5132 (CGP 69846A). J. Clin. Oncol. 1999, 17, 2227-2236.
9. Rudin, C. M; Holmlund, J.; Fleming, G. F.; Mani, S.; Stadler, W. M.; Schumm, P.; Monia, B. P.; Johnston, J. F.; Geary, R.; Yu, R. Z.; Kwoh, T. J.; Dorr, F. A.; Ratain, M. J. Phase I Trial.of ISIS 5132, an Antisense Oligonucleotide Inhibitor, of c- raf-1, Administered by 24-Hour Weekly Infusion to Patients with Advanced Cancer. Clin. Cancer Res..2001, 7, 1214-1220.
10. Toulme, J. J. New Candidates for True Antisense. Nat. Biotechnol. 2001, 19, 17-18.
11. Nielsen, P. E.; Egholm, M.; Berg, R. H.; Buchardt, O. Sequence-Selective Recognition of DNA by Strand Displacement with a Thymine-substituted Polyamide. Science 1991,254, 1497-1500.
12. Egholm, M.; Buchardt, O.; Christensen, L.; Behrens, C.; Freier, S. M.; Driver, D. A.; Berg, R. H.; Kim, S. K.; Norden, B.; Nielsen, P. E. PNA Hybridizes to Complementary Oligonucleotides Obeying the WatsonCrick Hydrogen-bonding Rules. Nature 1993, 365, 566568.
13. Demidov, V. V.; Potaman, V. N.; Frank-Kamenetskii, M. D.; Egholm, M.; Buchard, O.; Sonnichsen, S. H.; Nielsen, P. E. Stability of Peptide Nucleic Acids in Human Serum and Cellular Extracts. Biochem. Pharmacol. 1994,45, 1310-1313.
14. Egholm, M.; Christensen, L.; Dueholm, K.L.; Buchardt, O.; Coull, J.; Nielsen, P.E. Efficient pH-independent Sequence-specific DNA Binding by PseudoisocytosineContaining bis-PNA. Nucleic Acids Res. 1995,23, 217222.
15. Betts, L.; Josey, J. A.; Veal, J. M.; Jordan, S. R. A nucleic Acid Triple Helix Formed by a Peptide Nucleic AcidDNA Complex. Science 1995,270, 1838-1841.
16. Hanvey, J. C.; Peffer, N. J.; Bisi, J. E.; Thomson, S. A.; Cadilla, R.; Josey, J. A.; Ricca, D. J.; Hassman, C. F.; Bonham, M. A.; Au, K. G.; Carter, S. G.; Bruckenstein, D. A.; Boyd, A. L.; Noble, S. A.; Babiss, E. L. Antisense and Antigène Properties of Peptide Nucleic Acids. Science 1992,258, 1481-1485.
17. Bonham, M. A.; Brown, S.; Boyd, A. L.; Brown, P. H.; Bruckenstein, D. A.; Hanvey, J. C.; Thomson, S. A.; Pipe, A.; Hassman, F.; Bisi, J. E.; Froehler, B, C.; Mateucci, M. D.; Wagner, R. W.; Noble, S. A.; Babiss, L. E. An assessment of the Antisense Properties of RNase Hcompetent and Steric-blocking Oligomers. Nucleic Acids Res. 1995,23, 1197-1203.
18. Knudsen, H.; Nielsen, P. E. Antisense Properties of Duplex- and Triplex-forming PNAs. Nucleic Acids Res. 1996, 24, 494-500.
19. Koppelhus, U.; Zachar, V.; Nielsen, P. E.; Liu, X.; Eugen-Olsen, J.; Ebbesen, P. Efficient in vitro Inhibition of HIV-1 gag Reverse Transcription by Peptide Nucleic Acid (PNA) at Minimal Ratios of PNA/RNA. Nucleic Acids Res. 1997,25, 2167-2173.
20. Larsen, H. J.; Bentin, T.; Nielsen, P. E. Antisense Properties of Peptide Nucleic Acid. Biochim. Biophys. Acta. 1999,1489, 159-166.
21. Giovine, M.; Gasparini, A.; Scarfi, S.; Damonte, G.; Sturla, L.; Millo, E.; Tonetti, M.; Benatti, U. Synthesis and Characterization of a Specific Peptide Nucleic Acid that Inhibits Expression of Inducible NO Synthase. FEBS Lett. 1998, 426, 33-36.
22. Mologni, L.; leCoutre, P.; Nielsen, P. E.; GambacortiPasserini, C. Additive Antisense Effects of Different PNAs on the in vitro Translation of the PML/RARalpha Cms. Nucleic Acids Res. 1998,26, 1934-1938.
23. Gambacorti-Passerini, C.; Mologni, L.; Bertazzoli, C.; le Coutre, P.; Marches!, E.; Grignani, F.; Nielsen, P. E. In Vitro Transcription and Translation Inhibition by Antipromyelocytic Leukemia (PML)/Retinoic Acid Receptor alpha and Anti-PML Peptide Nucleic Acid. Blood 1996, 88, 1411-1417.
24. Dias, N.; Dheur, S.; Nielsen, P.'E.; Gryaznov, S.; Van Aerschot, A.; Herdevvijn, P.; Hélène, C.; SaisonBehmoaras, T.E. Antisense PNA Tridecamers Targeted to the Coding Region of Ha-ras mRNA Arrest Polypeptide Chain Elongation. J. Mol. Biol. 1999,294, 403-416.
25. Mologni, L.; Nielsen, P. E.; Gambacorti-Passerini, C. In vitro Transcriptional and Translational Block of the bcl2 Gene Operated by Peptide Nucleic Acid. Biochem Biophys. Res. Commun. 1999, 264, 537-543.
26. Herbert, B.; Puts, A. E.; Baker, S. I.; Hamilton, S. E.; Wright, W. E.; Shay, J. W.; Corey, D. R. Inhibition of Human Telomerase in Immortal Human Cells Leads to Progressive Telomere Shortening and Cell Death. Proc. Natl. Acad. Sei. U. S. A. 1999, 96, 14276-14281.
27. Shammas, M. A.; Simmons, C. G.; Corey, D. R.; Reis, R. J. Telomerase Inhibition by Peptide Nucleic Acids Reverses 'Immortality1 of Transformed Human Cells. Oncogens 1999, 18, 6191-6200.
28. Chinnery, P. F.; Taylor, R. W.; Diekert, K.; Lill, R.; Turnbull, D. M.; Lightowlers, R. N. Peptide Nucleic Acid Delivery to Human Mitochondria Gene Ther. 1999, 6, 1919-1928.
29. [Published Erratum Appears in Gene Ther. 2000, 7, 813]
30. Sei, S.; Yang, Q. E.; O'Neill, D.; Yoshimura, K.; Nagashima, K.; Mitsuya, H. Identification of a Key Target Sequence to Block Human Immunodeficiency Virus Type 1 Replication within the gag-pol Transframe Domain. J. Viral 2000, 74, 4621-4633.
31. Tyler, B. M.; McCormick, D. J.; Hoshall, C. V.; Douglas, C. L.; Jansen, K.; Lacy, B. W.; Cusack, B.; Richelson, E. Specific Gene Blockade Shows that Peptide Nucleic Acids Readily Enter Neuronal Cells in vivo. FEBS Lett. 1998, 421, 280-284.
32. Tyler, B. M.; Jansen, K.; McCormick, D. J.; Douglas, C. L.; Boules, M.; Stewart, J. A.; Zhao, L.; Lacy, B.; Cusack, B.; Fauq, A.; Richelson, E. Peptide Nucleic Acids Targeted to the Neurotensin Receptor and Administered i.p. Cross the Blood-Brain Barrier and Specifically Reduce Gene Expression. Proc. Natl. Acad. Sei. USA 1999, 96, 7053-7058.
33. Fraser, G. L.; Holmgren, J.; Clarke, P. B.; Wahlestedt, C. Antisense Inhibition of Delta-Opioid Receptor Gene Function In Vivo by Peptide Nucleic Acids. Mol. Pharmacol. 2000, 57, 725-731.
34. Rezaei, K.; Xu, I. S.; Wu, W. P.; Shi, T. J.; Soomets, U.; Land, T.; Xu, X. J.; Wiesenfeld-Hallin, Z.; Hökfelt, T.; Bartfai, T.; Langel, 0. Intrathecal Administration of PNA Targeting Galanin Receptor Reduces Galanin-Mediated Inhibitory Effect in the Rat Spinal Cord. Neuroreport. 2001,72, 317-320.
35. Karras, J.G.; Maier, M.A.; Lu, T.; Watt, A.; Manoharan, M. Peptide Nucleic Acids are Potent Modulators of Endogenous pre-mrna Splicing of the Murine InterIeukin-5 Receptor-alpha Chain. Biochemistry 2001, 40, 7853-7859.
36. Doyle, D. F.; Braasch, D. A.; Simmons, C. G.; Janovvski, B. A.; Corey, D. R. Inhibition of Gene Expression Inside Cells by Peptide Nucleic Acids: Effect of mRNA Target Sequence, Mismatched Bases, and PNA Length. Biochemistry 2001,40, 53-64.
37. Uhlmann, E. Peptide Nucleic acids (PNA) and PNADNA Chimeras: from High Binding Affinity towards Biological Function. Biol. Chem. 1998, 379, 1045-1052.
38. Nastruzzi, C.; Cortesi, R.; Esposito, E.; Gambari, R.; Borgatti, M.; Bianchi, N.; Feriotto, G.; Mischiati, C. Liposomes as carriers for DNA-PNA hybrids. J. Control. Release 2000, 68, 237-249.
39. Basu, S.; Wickström, E. Synthesis and Characterization of a Peptide Nucleic Acid Conjugated to a D-peptide Analog of Insulin-like Growth Factor 1 for Increased Cellular Uptake. Bioconjug. Chem. 1991,8, 481-488.
40. Zhang, X.; Simmons, C. G.; Corey, D. R. Liver Cell Specific Targeting of Peptide Nucleic Acid Oligomers. Bioorg. Med. Chem. Lett. 2001,11, 1269-1272.
41. Lebedeva, I.; Benimetskaya, L.; Stein, C.A.; Vilenchik, M. Cellular Delivery of Antisense Oligonucleotides. Eur. J. Pharm. Biopharm. 2000, 50, 101-119.
42. Boffa, L. C.; Scarfi, S.; Mariani, M. R.; Damonte, G.; Allfrey, V. G.; Benatti, U.; Morris, P. L. Dihydrotestosterone as a Selective Cellular/Nuclear Localization Vector for Anti-gene Peptide Nucleic Acid in Prostatic Carcinoma Cells. Cancer Res. 2000, 60, 2258-2262.
43. Mann, D. A.; Frankel, A. D. Endocytosis and Targeting of Exogenous HIV-1 Tat Protein. EMBO J. 1991, 10, 1733-1739.
44. Joliot, A.; Pernelle, C.; Deagostini-Bazin, H.; Prochiantz, A. Antennapedia Homeobox Peptide Regulates Neural Morphogenesis. Proc. Natl. Acad. Sei. USA 1991, 88, 1864-1868.
45. Derossi, D.; Joliot, A. H.; Chassalng, G.; Prochiantz, A. The Third Helix of the Antennapedia Homeodomain Translocates through Biological Membranes. J. Biol. Chem. 1994,269, 10444-10450.
46. Vivés, E.; Brodin, P.; Lebleu, B. A truncated HIV-1 Tat Protein Basic Domain Rapidly Translocates through the Plasma Membrane and Accumulates in the Cell Nucleus. J. Biol Chem. 1997,272, 16010-16017.
47. Derossi, D.; Calvet, S.; Trembleau, A.; Brunissen, A.; Chassaing, G.; Prochiantz, A. Cell Internalization of the Third Helix of the Antennapedia Homeodomain is Receptor-Independent. J. Biol. Chem. 1996,271, 18188-18193.
48. Pooga, M.; Hällbrink, M.; Zorko, M.; Langel, Ü. Cell Penetration by Transportas FASEB J. 1998, 12, 67-77.
49. Lin, Y. Z.; Yao, S. Y.; Veach, R. A.; Torgerson, T. R.; Hawiger, J. Inhibition of Nuclear Translocation of Transcription Factor NF-kappa B by a Synthetic Peptide Containing a Cell Mmembrane-permeable Motif and Nuclear Localization Sequence. J. Biol. Chem. 1995, 270, 14255-14258.
50. Oehlke, J.; Krause, E.; Wiesner, B.; Beyermann, M.; Bienert, M. Extensive Cellular Uptake into Endothelial Cells of an Amphipathic beta- sheet Forming Peptide. FEES Lett. 1997,415, 196-199.
51. Lindgren, M.; Hällbrink, M.; Prochiantz, A.; Langel, Ü. Cell-penetrating Peptides. Trends Pharmacol. Sei. 2000,27, 99-103.
52. Simmons, C. G.; Pitts, A.E.; Mayfield, L. D.; Shay, J. W.; Corey, D. R. Synthesis and Membrane Permeability of PNA-Peptide Conjugates. Bioorg.Med.Chem.Lett. 1997, 7, 3001-3006.
53. Scarfi, S.; Giovine, M.; Gasparini, A.; Damonte, G.; Millo, E.; Pozzolini, M.; Benatti, U. Modified Peptide Nucleic Acids are Internalized in Mouse Macrophages RAW 264.7 and Inhibit inducible Nitric Oxide Synthase. FEBS Lett. 1999, 451, 264-268.
54. Pooga, M.; Soomets, U.; Hällbrink, M.; Valkna, A.; Saar, K.; Rezaei, K.; Kahl, U.; Hao, J. X.; Xu, X .J.; WiesenfeldHallin, Z.; Hökfelt, T.; Bartfai, T.; Langel, Ü. Cell Penetrating PNA Constructs Regulate Galanin Receptor Levels and Modify Pain Transmission in vivo. Nat. Biotechnol. 1998, 16, 857-861.
55. Wittau, N.; Grosse, R.; Kalkbrenner, F.; Gohla, A.; Schultz, G.; Gudermann, T. The Galanin Receptor Type 2 Initiates Multiple Signaling Pathways in Small Cell Lung Cancer Cells by Coupling to G(q), G(i) and G(12) Proteins. Oncogene 2000, 19, 4199-4209.
56. Hyde, J. F.; Moore, J. P., Jr.; Cai, A. Galanin in Normal and Hyperplastic Anterior Pituitary Cells. From Pituitary Tumor Cell Lines to Transgenic Mice. Ann. N. Y. Acad. Sei. 1998, 863, 48-55.
57. Ormandy, C. J.; Lee, C. S.; Ormandy, H. F.; Fantl, V.; Shine, J.; Peters, G.; Sutherland, R. L. Amplification, Expression, and Steroid Regulation of the Preprogalanin Gene in Human Breast Cancer. Cancer Res. 1998,55, 1353-1357.
58. Aldrian-Herrada, G.; Desarmenien, M. G.; Orcel, H.; Boissin-Agasse, L.; Mery, J.; Brugidou, J.; Rabie, A. A Peptide Nucleic Acid (PNA) is More Rapidly Internalized in Cultured Neurons when Coupled to a Retro-inverso Delivery Peptide. The Antisense Activity Depresses the Target mRNA and Protein in Magnocellular Oxytocin Neurons. Nucleic Acids Res. 1998,26. 4910-4916.
59. Cutrona, G.; Carpaneto, E. M.; Ulivi, M; Roncella, S.; Landt, O.; Ferrarini, M.; Boffa, L. C. Effects in Live Cells of a c-myc Anti-gene PNA Linked to a Nuclear Localization Signal. Nat. Biolechnol. 2000, 18, 300-303.
60. Branden, L. J.; Mohamed, A. J.; Smith, C. I. A Peptide Nucleic Acid-Nuclear Localization Signal Fusion that Mediates Nuclear Transport of DNA. Nat. Biotechnol. 1999,17, 784-787.
61. Astriab-Fisher, A.; Sergueev, D. S.; Fisher, M.; Shaw, B. R.; Juliano, R. L. Antisense Inhibition of P-glycoprotein Expression Using Peptide- Oligonucleotide Conjugates. Biochem. Pharmacol. 2000, 60, 83-90.
62. Summerton, J. Morpholino Antisense Oligomers: the Case for an RNase H-independent Structural Type. Biochim. Biophys. Acta 1999, 1489, 141-158.
63. Villa, R.; Folini, M.; Lualdi, S.; Veronese, S.; Daidone, M.G.; Zaffaroni, N. Inhibition of Telomerase Activity by a Cell-Penetrating Peptide Nucleic Acid Construct in Human Melanoma Cells. FEES Lett. 2000, 473, 241-248.
64. Gorre, M. E.; Mohammed, M.; Elhvood, K.; Hsu, N.; Paquette, R.; Rao, P. N.; Sawyers, C.L. Clinical Resistance to STI-571 Cancer Therapy Caused by BCRABL Gene Mutation or Amplification. Science 2001, 293, 876-80.