Lipid-conjugated oligonucleotides via "click chemistry" efficiently inhibit hepatitis C virus translation

Journal of Medicinal Chemistry

Volumn 51, Issue 15, 2008, Pages 4374-4376

  Godeau, Guilhem ^a,b   Staedel, Cathy ^a,b   Barthélémy, Phílippe ^a,b  

^a INSERM U869   (France)

^b UNIVERSITÉ DE BORDEAUX   (France)

Author keywords

[No Author keywords available]

Indexed keywords

OLIGONUCLEOTIDE; OLIGONUCLEOTIDE PLUS LIPID CONJUGATE; UNCLASSIFIED DRUG;

ANTIVIRAL ACTIVITY; ARTICLE; CELL LINE; CHEMISTRY; CLICK CHEMISTRY; DRUG CONJUGATION; DRUG SYNTHESIS; HEPATITIS C VIRUS; HUH 7 CELL; HUMAN; HUMAN CELL; INTERNAL RIBOSOME ENTRY SITE; LIVER CELL CULTURE; STRUCTURE ACTIVITY RELATION; VIRUS INHIBITION; VIRUS TRANSCRIPTION;

CELL LINE, TUMOR; CELL SURVIVAL; HEPACIVIRUS; HUMANS; LIPIDS; MOLECULAR STRUCTURE; OLIGONUCLEOTIDES; VIRUS REPLICATION;

EID: 49449098341     PISSN: 00222623     EISSN: None     Source Type: Journal    
DOI: 10.1021/jm800518u     Document Type: Article

References (26)

1. (a) Verma, I. M.; Somia, N. Gene therapy - promises, problems and prospects. Nature 1997, 389, 239-242.
2. (b) Soutschek, J.; Akinc, A.; Bramlage, B.; Charisse, K.; Rainer, C.; Donoghue, M.; Elbashir, S.; Geick, A.; Hadwiger, P.; Harborth, J.; John, M.; Kesavan, V.; Lavine, G.; Pandey, R. K.; Racie, T.; Rajeev, K. G.; Röhl, I.; Toudjarska, I.; Wang, G.; Wuschko, S.; Bumcrot, D.; Koteliansky, V.; Limmer, S.; Manoharan, M.; Vornlocher, H.-P. Therapeutic silencing of an endogenous gene by systemic administration of modified si RNAs. Nature 2004, 432, 173-178.
3. (a) Prakash, T. P.; Kawasaki, A. M.; Wancewicz, E. V.; Shen, L.; Monia, B. P.; Ross, B. S.; Baht, B.; Manoharan, M. Comparing in vitro and in vivo activity of 2′-O-[2-(methylamino)-2-oxoethyl]- and 2′-O-methoxyethyl-modified antisense oligonucleotides. J. Med. Chem. 2008, 51, 2766-2776.
4. (b) Opalinska, J. B.; Gewirtz, A. M. Nucleic-acid therapeutics: basic principles and recent applications. Nat. Rev. Drug Discovery 2002, 1, 503-514.
5. (a) Mukherjee, K.; Bhattacharyya, J.; Sen, J.; Sistla, R.; Chaudhuri, A. Covalent grafting of common trihydroxymethylaminomethane in the headgroup region imparts high serum compatibility and mouse lung transfection property to cationic amphiphile. J. Med. Chem. 2008, 51, 1967.
6. (b) Li, S. D.; Huang, L. Gene therapy progress and prospects: non-viral gene therapy by systemic delivery. Gene Ther. 2006, 13, 1313-1319.
7. (c) Fraley, A. W.; Pons, B.; Dalkara, D.; Nullans, G.; Behr, J.-P.; Zuber, G. Cationic oligonucleotide - peptide conjugates with aggregating properties enter efficiently into cells while maintaining hybridization properties and enzymatic recognition. J. Am. Chem. Soc. 2006, 128, 10763.
8. (a) Sundaram, S.; Lee, L. K.; Roth, C. M. Interplay of polyethyleneimine molecular weight and oligonucleotide backbone chemistry in the dynamics of antisense activity. Nucleic Acids Res. 2007, 35, 4396-4408.
9. (b) Roth, C. M. Quantitative measurements and rational materials design for intracellular delivery of oligonucleotides. Biotechnol. Prog. 2008, 24, 23-28.
10. (a) Letsinger, R. L.; Zhang, G.; Sun, D. K.; Ikeuchi, T.; Sarin, P. S. Cholesteryl-conjugated oligonucleotides: synthesis, properties, and activity as inhibitors of replication of human immunodeficiency virus in cell culture. Proc. Natl. Acad. Sci. U.S.A. 1989, 86, 6553-6556.
11. (b) Manoharan, M.; Tivel, K. L.; Condon, T. P.; Andrade, L. K.; Barber-Peoch, I.; Inamati, G.; Shah, S.; Mohan, V.; Graham, M. J.; Bennett, F. C.; Crooke, S. T.; Cook, P. D. Conjugated antisense oligonucleotides. Nucleosides Nucleotides 1997, 16, 1129-1138.
12. Gissot, A.; Camplo, M.; Grinstaff, M. W.; Barthélémy, P. Nucleoside, nucleotide and oligonucleotide based amphiphiles: a successful marriage of nucleic acids with lipids. Org. Biomol. Chem 2008, 6, 1324-1333.
13. Huisgen, R. 1,3-Dipolar Cycloaddition Chemistry; Wiley: New York, 1984; pp 1-176.
14. Rostovtsev, V. V.; Green, L. G.; Fokin, V. V.; Sharpless, K. B. A stepwise Huisgen cycloaddition process: copper(I)-catalyzed regioselective "ligation" of azides and terminal alkynes. Angew. Chem., Int. Ed. 2002, 41, 2596-2599.
15. (a) Bouillon, C.; Meyer, A.; Vidal, S.; Jochum, A.; Chevolot, Y.; Cloarec, J.-P.; Praly, J.-P.; Morvan, F. Microwave assisted "click" chemistry for the synthesis of multiple labeled-carbohydrate oligonucleotides on solid support. J. Org. Chem. 2006, 71, 4700-4702.
16. (b) Chevolot, Y.; Bouillon, C.; Vidal, S.; Morvan, F.; Meyer, A.; Cloarec, J.-P.; Jochum, A.; Souteyrand, E. DNA-based carbohydrate biochips: a platform for surface glyco-engineering. Angew. Chem., Int. Ed. 2007, 46, 2398-2402.
17. Gogoi, K.; Mane, M. V.; Kunte, S. S.; Kumar, V. A. A versatile method for the preparation of conjugates of peptides with DNA/PNA/analog by employing chemo-selective click reaction in water. Nucleic Acids Res. 2007, 35, e139.
18. Humenik, M.; Huang, Y.; Wang, Y.; Sprinzl, M. C-Terminal incorporation of bio-orthogonal azide groups into a protein and preparation of protein-oligodeoxynucleotide conjugates by CuI-catalyzed cycloaddition. ChemBioChem 2007, 8, 1103-1106.
19. Seo, T. S.; Li, Z.; Ruparel, H.; Ju, J. Click chemistry to construct fluorescent oligonucleotides for DNA sequencing. J. Org. Chem. 2003, 68, 609-612.
20. Devaraj, N. K.; Miller, G. P.; Ebina, W.; Kakaradov, B.; Collman, J. P.; Kool, E. T.; Christopher, E. D.; Chidsey; C. E, D. Chemoselective covalent coupling of oligonucleotide probes to self-assembled monolayers. J. Am. Chem. Soc. 2005, 127, 8600-8601.
21. (a) Kumar, R.; El-Sagheer, A.; Tumpane, J.; Lincoln, P.; Wilhelmsson, L. M.; Brown, T. Template-directed oligonucleotide strand ligation, covalent intramolecular DNA circularization and catenation using click chemistry. J. Am. Chem. Soc. 2007, 129, 6859-6864.
22. (b) Lietard, J.; Meyer, A.; Vasseur, J.-J.; Morvan, F. New strategies for cyclization and bicyclization of oligonucleotides by click chemistry assisted by microwaves. J. Org. Chem. 2008, 73, 191-200.
23. Gramlich, P. M. E.; Warncke, S.; Gierlich, J.; Carell, T. Click-click-click: single to triple modification of DNA. Angew. Chem., Int. Ed. 2008, 47, 3442-3444.
24. Tallet-Lopez, B.; Aldaz-Carroll, L.; Chabas, S.; Dausse, E.; Staedel, C.; Toulmé, J. J. Antisense oligonucleotides targeted to the domain IIId of the hepatitis C virus IRES compete with 40S ribosomal subunit binding and prevent in vitro translation. Nucleic Acids Res. 2003, 31, 734-742.
25. De Jong, L.; Moreau, X.; Thiéry, A.; Godeau, G.; Grinstaff, M. W.; Barthélémy, P. Amphiphilic copolymer for delivery of xenobiotics: in vivo studies in a freshwater invertebrate, a mesostominae flatworm. Bioconjugate Chem. 2008, 19, 891-898.
26. Richard, J. P.; Melikov, K.; Vives, E.; Ramos, C.; Verbeure, B.; Gait, M. J.; Chernomordik, L. V.; Lebleu, B. Cell-penetrating peptides. A reevaluation of the mechanism of cellular uptake. J. Biol. Chem. 2003, 278, 585-590.