Peptide nanoparticles for oligonucleotide delivery

Progress in Molecular Biology and Translational Science

Volumn 104, Issue , 2011, Pages 397-426

  Lehto, Taavi ^a   Ezzat, Kariem ^b   Langel, Ülo ^a,b  

^a UNIVERSITY OF TARTU   (Estonia)

^b STOCKHOLM UNIVERSITY   (Sweden)

Author keywords

Antisense oligonucleotides; Cell penetrating peptides; Endosomal escape; Gene delivery; Nanoparticles; Noncovalent nanoparticle formation; Oligonucleotide delivery; Plasmid delivery; siRNA delivery; Splice switching

Indexed keywords

EID: 81255166873     PISSN: 18771173     EISSN: None     Source Type: Book Series    
DOI: 10.1016/B978-0-12-416020-0.00010-3     Document Type: Chapter

References (85)

1. C.E. Thomas, A. Ehrhardt, and M.A. Kay Progress and problems with the use of viral vectors for gene therapy Nat Rev Genet 4 2003 346 358 (Pubitemid 36538359)
2. D.W. Pack, A.S. Hoffman, S. Pun, and P.S. Stayton Design and development of polymers for gene delivery Nat Rev Drug Discov 4 2005 581 593 (Pubitemid 40975711)
3. K.A. Whitehead, R. Langer, and D.G. Anderson Knocking down barriers: advances in siRNA delivery Nat Rev Drug Discov 8 2009 129 138
4. M. Lindgren, and Ü. Langel Classes and prediction of cell-penetrating peptides Methods Mol Biol 683 2011 3 19
5. S. El-Andaloussi, T. Holm, and Ü. Langel Cell-penetrating peptides: mechanisms and applications Curr Pharm Des 11 2005 3597 3611 (Pubitemid 41630987)
6. M. Mäe, and Ü. Langel Cell-penetrating peptides as vectors for peptide, protein and oligonucleotide delivery Curr Opin Pharmacol 6 2006 509 514 (Pubitemid 44340662)
7. R. Brasseur, and G. Divita Happy birthday cell penetrating peptides: already 20 years Biochim Biophys Acta 1798 2010 2177 2181
8. S.B. Fonseca, M.P. Pereira, and S.O. Kelley Recent advances in the use of cell-penetrating peptides for medical and biological applications Adv Drug Deliv Rev 61 2009 953 964
9. F.S. Hassane, A.F. Saleh, R. Abes, M.J. Gait, and B. Lebleu Cell penetrating peptides: overview and applications to the delivery of oligonucleotides Cell Mol Life Sci 67 2010 715 726
10. P. Lundin, H. Johansson, P. Guterstam, T. Holm, M. Hansen, and Ü. Langel Distinct uptake routes of cell-penetrating peptide conjugates Bioconjug Chem 19 2008 2535 2542
11. M. Zorko, and Ü. Langel Cell-penetrating peptides: mechanism and kinetics of cargo delivery Adv Drug Deliv Rev 57 2005 529 545 (Pubitemid 40255559)
12. S. Futaki, I. Nakase, A. Tadokoro, T. Takeuchi, and A.T. Jones Arginine-rich peptides and their internalization mechanisms Biochem Soc Trans 35 2007 784 787 (Pubitemid 47310368)
13. A. El-Sayed, S. Futaki, and H. Harashima Delivery of macromolecules using arginine-rich cell-penetrating peptides: ways to overcome endosomal entrapment AAPS J 11 2009 13 22
14. M. Mäe, S.E. Andaloussi, T. Lehto, and Ü. Langel Chemically modified cell-penetrating peptides for the delivery of nucleic acids Expert Opin Drug Deliv 6 2009 1195 1205
15. L. Crombez, M.C. Morris, S. Deshayes, F. Heitz, and G. Divita Peptide-based nanoparticle for ex vivo and in vivo dug delivery Curr Pharm Des 14 2008 3656 3665
16. M. Mäe, S. El Andaloussi, P. Lundin, N. Oskolkov, H.J. Johansson, and P. Guterstam A stearylated CPP for delivery of splice correcting oligonucleotides using a non-covalent co-incubation strategy J Control Release 134 2009 221 227
17. S. Hacein-Bey-Abina, A. Garrigue, G.P. Wang, J. Soulier, A. Lim, and E. Morillon Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1 J Clin Invest 118 2008 3132 3142
18. M. Cavazzana-Calvo Basic research tries to decrease the risks of translational medicine Gene Ther 16 2009 309 310
19. S.J. Howe, M.R. Mansour, K. Schwarzwaelder, C. Bartholomae, M. Hubank, and H. Kempski Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients J Clin Invest 118 2008 3143 3150
20. M.A. Kay, J.C. Glorioso, and L. Naldini Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics Nat Med 7 2001 33 40 (Pubitemid 32095639)
21. C.R. Dass Lipoplex-mediated delivery of nucleic acids: factors affecting in vivo transfection J Mol Med 82 2004 579 591 (Pubitemid 39312663)
22. P.E. Rakoczy Antisense DNA technology Methods Mol Med 47 2001 89 104
23. N.M. Bell, and J. Micklefield Chemical modification of oligonucleotides for therapeutic, bioanalytical and other applications Chembiochem 10 2009 2691 2703
24. S.M. Elbashir, J. Harborth, W. Lendeckel, A. Yalcin, K. Weber, and T. Tuschl Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells Nature 411 2001 494 498 (Pubitemid 32494397)
25. J. Bauman, N. Jearawiriyapaisarn, and R. Kole Therapeutic potential of splice-switching oligonucleotides Oligonucleotides 19 2009 1 13
26. T.W. Nilsen, and B.R. Graveley Expansion of the eukaryotic proteome by alternative splicing Nature 463 2010 457 463
27. P. Sazani, and R. Kole Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing J Clin Invest 112 2003 481 486 (Pubitemid 38056330)
28. M.J. Wood, M.J. Gait, and H. Yin RNA-targeted splice-correction therapy for neuromuscular disease Brain 133 2010 957 972
29. G.D. Ivanova, A. Arzumanov, R. Abes, H. Yin, M.J. Wood, and B. Lebleu Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle Nucleic Acids Res 36 2008 6418 6428
30. J.C. van Deutekom, A.A. Janson, I.B. Ginjaar, W.S. Frankhuizen, A. Aartsma-Rus, and M. Bremmer-Bout Local dystrophin restoration with antisense oligonucleotide PRO051 N Engl J Med 357 2007 2677 2686
31. M. Kinali, V. Arechavala-Gomeza, L. Feng, S. Cirak, D. Hunt, and C. Adkin Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study Lancet Neurol 8 2009 918 928
32. Z. Oren, and Y. Shai Mode of action of linear amphipathic alpha-helical antimicrobial peptides Biopolymers 47 1998 451 463
33. D. Derossi, S. Calvet, A. Trembleau, A. Brunissen, G. Chassaing, and A. Prochiantz Cell internalization of the third helix of the Antennapedia homeodomain is receptor-independent J Biol Chem 271 1996 18188 18193 (Pubitemid 26250811)
34. J.P. Richard, K. Melikov, E. Vives, C. Ramos, B. Verbeure, and M.J. Gait Cell-penetrating peptides - a reevaluation of the mechanism of cellular uptake J Biol Chem 278 2003 585 590 (Pubitemid 36043612)
35. I.A. Khalil, S. Futaki, M. Niwa, Y. Baba, N. Kaji, and H. Kamiya Mechanism of improved gene transfer by the N-terminal stearylation of octaarginine: enhanced cellular association by hydrophobic core formation Gene Ther 11 2004 636 644 (Pubitemid 38455982)
36. A. Eguchi, and S.F. Dowdy Efficient siRNA delivery by novel PTD-DRBD fusion proteins Cell Cycle 9 2010 424 425
37. S. EL Andaloussi, T. Lehto, I. Mager, K. Rosenthal-Aizman, I.I. Oprea, and O.E. Simonson Design of a peptide-based vector, PepFect6, for efficient delivery of siRNA in cell culture and systemically in vivo Nucleic Acids Res 39 2011 3972 3987
38. K. Ezzat, S. El Andaloussi, E.M. Zaghloul, T. Lehto, S. Lindberg, and P.M. Moreno PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation Nucleic Acids Res 39 2011 5284 5298
39. T. Lehto, O.E. Simonson, I. Mager, K. Ezzat, H. Sork, and D.M. Copolovici A peptide-based vector for efficient gene transfer in vitro and in vivo Mol Ther 19 2011 1457 1467
40. K. Ezzat, S. El Andaloussi, R. Abdo, and U. Langel Peptide-based matrices as drug delivery vehicles Curr Pharm Des 16 2010 1167 1178
41. S. Deshayes, K. Konate, G. Aldrian, L. Crombez, F. Heitz, and G. Divita Structural polymorphism of non-covalent peptide-based delivery systems: highway to cellular uptake Biochim Biophys Acta 1798 2010 2304 2314
42. I. Lynch, K.A. Dawson, and S. Linse Detecting cryptic epitopes created by nanoparticles Sci STKE 2006 2006 pe14
43. H. Jatzkewitz Incorporation of physiologically-active substances into a colloidal blood plasma substitute. I. Incorporation of mescaline peptide into polyvinylpyrrolidone Hoppe Seylers Z Physiol Chem 297 1954 149 156
44. M.E. Davis, Z.G. Chen, and D.M. Shin Nanoparticle therapeutics: an emerging treatment modality for cancer Nat Rev Drug Discov 7 2008 771 782
45. L. Zhang, F.X. Gu, J.M. Chan, A.Z. Wang, R.S. Langer, and O.C. Farokhzad Nanoparticles in medicine: therapeutic applications and developments Clin Pharmacol Ther 83 2008 761 769 (Pubitemid 351556021)
46. J.R. Viola, S. El-Andaloussi, I.I. Oprea, and C.I.E. Smith Non-viral nanovectors for gene delivery: factors that govern successful therapeutics Expert Opin Drug Deliv 7 2010 721 735
47. D.J. Glover, H.J. Lipps, and D.A. Jans Towards safe, non-viral therapeutic gene expression in humans Nat Rev Genet 6 2005 299 310 (Pubitemid 40469049)
48. M.C. Morris, P. Vidal, L. Chaloin, F. Heitz, and G. Divita A new peptide vector for efficient delivery of oligonucleotides into mammalian cells Nucleic Acids Res 25 1997 2730 2736 (Pubitemid 27299785)
49. B.R. Meade, and S.F. Dowdy Enhancing the cellular uptake of siRNA duplexes following noncovalent packaging with protein transduction domain peptides Adv Drug Deliv Rev 60 2008 530 536
50. V.A. Bloomfield DNA condensation by multivalent cations Biopolymers 44 1997 269 282
51. D.V. Schaffer, N.A. Fidelman, N. Dan, and D.A. Lauffenburger Vector unpacking as a potential barrier for receptor-mediated polyplex gene delivery Biotechnol Bioeng 67 2000 598 606 (Pubitemid 30096964)
52. O. Zelphati, and F.C. Szoka Jr. Mechanism of oligonucleotide release from cationic liposomes Proc Natl Acad Sci USA 93 1996 11493 11498 (Pubitemid 26347149)
53. I. Nakase, T. Takeuchi, G. Tanaka, and S. Futaki Methodological and cellular aspects that govern the internalization mechanisms of arginine-rich cell-penetrating peptides Adv Drug Deliv Rev 60 2008 598 607 (Pubitemid 351215576)
54. I. Nakase, A. Tadokoro, N. Kawabata, T. Takeuchi, H. Katoh, and K. Hiramoto Interaction of arginine-rich peptides with membrane-associated proteoglycans is crucial for induction of actin organization and macropinocytosis Biochemistry 46 2007 492 501 (Pubitemid 46105438)
55. M.M. Patel, and T.J. Anchordoquy Contribution of hydrophobicity to thermodynamics of ligand-DNA binding and DNA collapse Biophys J 88 2005 2089 2103 (Pubitemid 40976218)
56. M.C. Morris, J. Depollier, J. Mery, F. Heitz, and G. Divita A peptide carrier for the delivery of biologically active proteins into mammalian cells Nat Biotechnol 19 2001 1173 1176 (Pubitemid 33115705)
57. T. Lehto, R. Abes, N. Oskolkov, J. Suhorutšenko, D.M. Copolovici, and I. Mäger Delivery of nucleic acids with a stearylated (RxR)(4) peptide using a non-covalent co-incubation strategy J Control Release 141 2010 42 51
58. Y. Nakamura, K. Kogure, S. Futaki, and H. Harashima Octaarginine-modified multifunctional envelope-type nano device for siRNA J Control Release 119 2007 360 367 (Pubitemid 46770881)
59. S. EL Andaloussi, T. Lehto, P. Lundin, and Ü. Langel Application of PepFect peptides for the delivery of splice-correcting oligonucleotides Methods Mol Biol 683 2011 361 373
60. K. Konate, L. Crombez, S. Deshayes, M. Decaffmeyer, A. Thomas, and R. Brasseur Insight into the cellular uptake mechanism of a secondary amphipathic cell-penetrating peptide for siRNA delivery Biochemistry 49 2010 3393 3402
61. L. Crombez, G. Aldrian-Herrada, K. Konate, Q.N. Nguyen, G.K. McMaster, and R. Brasseur A new potent secondary amphipathic cell-penetrating peptide for siRNA delivery into mammalian cells Mol Ther 17 2009 95 103
62. F. Simeoni, M.C. Morris, F. Heitz, and G. Divita Insight into the mechanism of the peptide-based gene delivery system MPG: implications for delivery of siRNA into mammalian cells Nucleic Acids Res 31 2003 2717 2724 (Pubitemid 37442116)
63. L. Crombez, M.C. Morris, S. Dufort, G. Aldrian-Herrada, Q. Nguyen, and G. Mc Master Targeting cyclin B1 through peptide-based delivery of siRNA prevents tumour growth Nucleic Acids Res 37 2009 4559 4569
64. M.C. Morris, L. Chaloin, M. Choob, J. Archdeacon, F. Heitz, and G. Divita Combination of a new generation of PNAs with a peptide-based carrier enables efficient targeting of cell cycle progression Gene Ther 11 2004 757 764 (Pubitemid 38659754)
65. M.C. Morris, E. Gros, G. Aldrian-Herrada, M. Choob, J. Archdeacon, and F. Heitz A non-covalent peptide-based carrier for in vivo delivery of DNA mimics Nucleic Acids Res 35 2007 e49
66. K. Rittner, A. Benavente, A. Bompard-Sorlet, F. Heitz, G. Divita, and R. Brasseur New basic membrane-destabilizing peptides for plasmid-based gene delivery in vitro and in vivo Mol Ther 5 2002 104 114 (Pubitemid 34144826)
67. S. Futaki, W. Ohashi, T. Suzuki, M. Niwa, S. Tanaka, and K. Ueda Stearylated arginine-rich peptides: a new class of transfection systems Bioconjug Chem 12 2001 1005 1011 (Pubitemid 33136872)
68. W.J. Kim, L.V. Christensen, S. Jo, J.W. Yockman, J.H. Jeong, and Y.H. Kim Cholesteryl oligoarginine delivering vascular endothelial growth factor siRNA effectively inhibits tumor growth in colon adenocarcinoma Mol Ther 14 2006 343 350 (Pubitemid 44184964)
69. U. Soomets, M. Lindgren, X. Gallet, M. Hällbrink, A. Elmquist, and L. Balaspiri Deletion analogues of transportan Biochim Biophys Acta 1467 2000 165 176 (Pubitemid 30621595)
70. M.C. Morris, L. Chaloin, J. Mery, F. Heitz, and G. Divita A novel potent strategy for gene delivery using a single peptide vector as a carrier Nucleic Acids Res 27 1999 3510 3517 (Pubitemid 29414679)
71. S. Veldhoen, S.D. Laufer, A. Trampe, and T. Restle Cellular delivery of small interfering RNA by a non-covalently attached cell-penetrating peptide: quantitative analysis of uptake and biological effect Nucleic Acids Res 34 2006 6561 6573 (Pubitemid 46017951)
72. H. Hatakeyama, E. Ito, H. Akita, M. Oishi, Y. Nagasaki, and S. Futaki A pH-sensitive fusogenic peptide facilitates endosomal escape and greatly enhances the gene silencing of siRNA-containing nanoparticles in vitro and in vivo J Control Release 139 2009 127 132
73. P. Kumar, H. Wu, J.L. McBride, K.E. Jung, M.H. Kim, and B.L. Davidson Transvascular delivery of small interfering RNA to the central nervous system Nature 448 2007 39 43 (Pubitemid 47037001)
74. A. Eguchi, B.R. Meade, Y.C. Chang, C.T. Fredrickson, K. Willert, and N. Puri Efficient siRNA delivery into primary cells by a peptide transduction domain-dsRNA binding domain fusion protein Nat Biotechnol 27 2009 567 571
75. H. Michiue, A. Eguchi, M. Scadeng, and S.F. Dowdy Induction of in vivo synthetic lethal RNAi responses to treat glioblastoma Cancer Biol Ther 8 2009 2306 2313
76. R. Abes, H.M. Moulton, P. Clair, S.T. Yang, S. Abes, and K. Melikov Delivery of steric block morpholino oligomers by (R-X-R)4 peptides: structure-activity studies Nucleic Acids Res 36 2008 6343 6354
77. S. Abes, H.M. Moulton, P. Clair, P. Prevot, D.S. Youngblood, and R.P. Wu Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents J Control Release 116 2006 304 313 (Pubitemid 44827406)
78. N. Jearawiriyapaisarn, H.M. Moulton, B. Buckley, J. Roberts, P. Sazani, and S. Fucharoen Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice Mol Ther 16 2008 1624 1629
79. H.M. Moulton, S. Fletcher, B.W. Neuman, G. McClorey, D.A. Stein, and S. Abes Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo Biochem Soc Trans 35 2007 826 828 (Pubitemid 47310377)
80. M. Pooga, M. Hällbrink, M. Zorko, and Ü. Langel Cell penetration by transportan FASEB J 12 1998 67 77 (Pubitemid 28060272)
81. M. Pooga, U. Soomets, M. Hallbrink, A. Valkna, K. Saar, and K. Rezaei Cell penetrating PNA constructs regulate galanin receptor levels and modify pain transmission in vivo Nat Biotechnol 16 1998 857 861 (Pubitemid 28405857)
82. S. EL Andaloussi, H. Johansson, A. Magnusdottir, P. Järver, P. Lundberg, and Ü. Langel TP10, a delivery vector for decoy oligonucleotides targeting the Myc protein J Control Release 110 2005 189 201 (Pubitemid 41713942)
83. K. Kilk, S. El Andaloussi, P. Järver, A. Meikas, A. Valkna, and T. Bartfai Evaluation of transportan 10 in PEI mediated plasmid delivery assay J Control Release 103 2005 511 523 (Pubitemid 40342545)
84. P. Lundberg, S. El Andaloussi, T. Sütlü, H. Johansson, and Ü. Langel Delivery of short interfering RNA using endosomolytic cell-penetrating peptides FASEB J 21 2007 2664 2671 (Pubitemid 47372734)
85. E. Ramsay, and M. Gumbleton Polylysine and polyornithine gene transfer complexes: a study of complex stability and cellular uptake as a basis for their differential in-vitro transfection efficiency J Drug Target 10 2002 1 9 (Pubitemid 34223844)