Nanomolar cellular antisense activity of peptide nucleic acid (PNA) cholic acid ("Umbrella") and cholesterol conjugates delivered by cationic lipids

Bioconjugate Chemistry

Volumn 23, Issue 2, 2012, Pages 196-202

  Shiraishi, Takehiko ^a   Nielsen, Peter E ^a  

^a UNIVERSITY OF COPENHAGEN   (Denmark)

Author keywords

[No Author keywords available]

Indexed keywords

BIOCHEMISTRY; LANTHANUM COMPOUNDS; MOLECULAR BIOLOGY; NUCLEIC ACIDS; OLIGOMERS; PEPTIDES;

ANTIGENES; ANTISENSE; ANTISENSE ACTIVITY; CATIONIC LIPIDS; CELLULAR UPTAKE; CELLULARS; CHOLIC ACIDS; NANOMOLAR; NUCLEIC ACID OLIGOMERS; PEPTIDE NUCLEIC ACID;

CHOLESTEROL;

CHOLESTEROL; CHOLIC ACID; LIPID; OLIGOMER; PEPTIDE NUCLEIC ACID;

ANTISENSE THERAPY; ARTICLE; CELL SURFACE; CONJUGATION; CONTROLLED STUDY; GENETIC TRANSFECTION; PROTEIN FUNCTION; PROTEIN LIPID INTERACTION;

EID: 84856893727     PISSN: 10431802     EISSN: 15204812     Source Type: Journal    
DOI: 10.1021/bc200460t     Document Type: Article

References (28)

1. De Rosa, G. and La Rotonda, M. I. (2009) Nano and microtechnologies for the delivery of oligonucleotides with gene silencing properties Molecules 14, 2801-23
2. Shiraishi, T. and Nielsen, P. E. (2009) Cellular Biovailability of Peptide Nucleic Acids (PNAs) Conjugated to Cell Penetrating Peptides. In Delivery Technologies for Biopharmaceuticals, Peptides, Proteins, Nucleic Acids and Vaccines (Jørgensen, L. and Mørck Nielsen, H., Eds.) pp 305-338, Wiley, Chichester, UK.
3. Mae, M., Andaloussi, S. E., Lehto, T., and Langel, U. (2009) Chemically modified cell-penetrating peptides for the delivery of nucleic acids Expert Opin. Drug Delivery 6, 1195-205
4. Berthold, P. R., Shiraishi, T., and Nielsen, P. E. (2010) Cellular delivery and antisense effects of peptide nucleic acid conjugated to polyethyleneimine via disulfide linkers Bioconjugate Chem. 21, 1933-8
5. Shiraishi, T., Pankratova, S., and Nielsen, P. E. (2005) Calcium ions effectively enhance the effect of antisense peptide nucleic acids conjugated to cationic tat and oligoarginine peptides Chem. Biol. 12, 923-9 (Pubitemid 41206806)
6. Abes, S., Williams, D., Prevot, P., Thierry, A., Gait, M. J., and Lebleu, B. (2006) Endosome trapping limits the efficiency of splicing correction by PNA-oligolysine conjugates J. Controlled Release 110, 595-604 (Pubitemid 43163449)
7. Shiraishi, T. and Nielsen, P. E. (2006) Photochemically enhanced cellular delivery of cell penetrating peptide-PNA conjugates FEBS Lett. 580, 1451-6 (Pubitemid 43222014)
8. Shiraishi, T., Hamzavi, R., and Nielsen, P. E. (2008) Subnanomolar antisense activity of phosphonate-peptide nucleic acid (PNA) conjugates delivered by cationic lipids to HeLa cells Nucleic Acids Res. 36, 4424-32
9. Janout, V., Staina, I. V., Bandyopadhyay, P., and Regen, S. L. (2001) Evidence for an umbrella mechanism of bilayer transport J. Am. Chem. Soc. 123, 9926-7 (Pubitemid 32929412)
10. Ge, D., Wu, D., Wang, Z., Shi, W., Wu, T., Zhang, A., Hong, S., Wang, J., Zhang, Y., and Ren, L. (2009) Cellular uptake mechanism of molecular umbrella Bioconjugate Chem. 20, 2311-6
11. Bijsterbosch, M. K., Rump, E. T., De Vrueh, R. L., Dorland, R., van Veghel, R., Tivel, K. L., Biessen, E. A., van Berkel, T. J., and Manoharan, M. (2000) Modulation of plasma protein binding and in vivo liver cell uptake of phosphorothioate oligodeoxynucleotides by cholesterol conjugation Nucleic Acids Res. 28, 2717-25 (Pubitemid 30598756)
12. Joshi, R., Mishra, R., Pohmann, R., and Engelmann, J. (2010) MR contrast agent composed of cholesterol and peptide nucleic acids: design, synthesis and cellular uptake Bioorg. Med. Chem. Lett. 20, 2238-41
13. Bijsterbosch, M. K., Manoharan, M., Dorland, R., Van Veghel, R., Biessen, E. A., and Van Berkel, T. J. (2002) bis-Cholesteryl-conjugated phosphorothioate oligodeoxynucleotides are highly selectively taken up by the liver J. Pharmacol. Exp. Ther. 302, 619-26 (Pubitemid 34787228)
14. Soutschek, J, Akinc, A, Bramlage, B, Charisse, K, Constien, R, 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, and Vornlocher, H. P. (2004) Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs Nature 432, 173-8 (Pubitemid 39545841)
15. Kang, S. H., Cho, M. J., and Kole, R. (1998) Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development Biochemistry 37, 6235-9 (Pubitemid 28213640)
16. Christensen, L., Fitzpatrick, R., Gildea, B., Petersen, K. H., Hansen, H. F., Koch, T., Egholm, M., Buchardt, O., Nielsen, P. E., Coull, J., and Berg, R. H. (1995) Solid-phase synthesis of peptide nucleic acids J. Pept. Sci. 1, 175-83
17. Haaima, G., Lohse, A., Buchardt, O., and Nielsen, P. E. (1996) Peptide nucleic acids (PNAs) containing thymine monomers derived from chiral amino acids: hybridization and solubility properties of D-Lysine PNA Angew. Chem. 35, 1939-1941
18. Doyle, D. F., Braasch, D. A., Simmons, C. G., Janowski, B. A., and Corey, D. R. (2001) Inhibition of gene expression inside cells by peptide nucleic acids: effect of mRNA target sequence, mismatched bases, and PNA length Biochemistry 40, 53-64 (Pubitemid 32052677)
19. Shiraishi, T., Bendifallah, N., and Nielsen, P. E. (2006) Cellular delivery of polyheteroaromate-peptide nucleic acid conjugates mediated by cationic lipids Bioconjugate Chem. 17, 189-94 (Pubitemid 43157601)
20. Hallbrink, M., Oehlke, J., Papsdorf, G., and Bienert, M. (2004) Uptake of cell-penetrating peptides is dependent on peptide-to-cell ratio rather than on peptide concentration Biochim. Biophys. Acta 1667, 222-8 (Pubitemid 39576087)
21. Shiraishi, T. and Nielsen, P. E. (2006) Enhanced delivery of cell-penetrating peptide-peptide nucleic acid conjugates by endosomal disruption Nat. Protoc. 1, 633-6
22. Bendifallah, N., Rasmussen, F. W., Zachar, V., Ebbesen, P., Nielsen, P. E., and Koppelhus, U. (2006) Evaluation of cell-penetrating peptides (CPPs) as vehicles for intracellular delivery of antisense peptide nucleic acid (PNA) Bioconjugate Chem. 17, 750-758 (Pubitemid 43794386)
23. Koppelhus, U., Shiraishi, T., Zachar, V., Pankratova, S., and Nielsen, P. E. (2008) Improved cellular activity of antisense peptide nucleic acids by conjugation to a cationic peptide-lipid (CatLip) domain Bioconjugate Chem. 19, 1526-1534
24. Janout, V., Jing, B., and Regen, S. L. (2005) Molecular umbrella-assisted transport of an oligonucleotide across cholesterol-rich phospholipid bilayers J. Am. Chem. Soc. 127, 15862-70 (Pubitemid 41643296)
25. Cline, L. L., Janout, V, Fisher, M, Juliano, R. L., and Regen, S. L. (2011) A molecular umbrella approach to the intracellular delivery of small interfering RNA Bioconjugate Chem. 22, 2210-6
26. Ezzat, K., El Andaloussi, S., Zaghloul, E. M., Lehto, T., Lindberg, S., Moreno, P. M., Viola, J. R., Magdy, T., Abdo, R., Guterstam, P., Sillard, R., Hammond, S. M., Wood, M. J., Arzumanov, A. A., Gait, M. J., Smith, C. I., Hallbrink, M., and Langel, U. (2011) PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation Nucleic Acids Res. 39, 5284-98
27. Crombez, L. and Divita, G. (2011) A non-covalent peptide-based strategy for siRNA delivery Methods Mol. Biol. 683, 349-60
28. Betebenner, D. A., Carney, P. L., Zimmer, A. M., Kazikiewicz, J. M., Brücher, E, Sherry, A. D., and Johnson, D. K. (1991) Hepatobiliary delivery of polyaminopolycarboxylate chelates: synthesis and characterization of a cholic acid conjugate of EDTA and biodistribution and imaging studies with its indium-111 chelate Bioconjugate Chem. 2, 117-23