Formulation of a peptide nucleic acid based nucleic acid delivery construct

Bioconjugate Chemistry

Volumn 21, Issue 3, 2010, Pages 445-455

  Millili, Peter G ^a,b   Yin, Daniel H ^c   Fan, Haihong ^c   Naik, Ulnas P ^a,b   Sullivan, Millicent O ^a,b  

^a University of Delaware   (United States)

^b UNIVERSITY OF DELAWARE   (United States)

^c MERCK RESEARCH LABORATORIES   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BIOMOLECULES; CYTOTOXICITY; EFFICIENCY; GENE TRANSFER; GENES; PEPTIDES; POLYETHYLENE GLYCOLS;

CELLULAR ACTIVITIES; DNA COMPLEX; GENE DELIVERY; IN-VIVO; NUCLEAR DELIVERY; NUCLEIC ACIDS DELIVERIES; PEPTIDE NUCLEIC ACID; PLASMID DNA; POLYCATIONS; TRANSFECTION EFFICIENCY;

DNA;

DNA; MACROGOL; NUCLEIC ACID; PEPTIDE NUCLEIC ACID; POLYETHYLENEIMINE; POLYETHYLENEIMINE DNA COMPLEX; POLYETHYLENEIMINE DNA PEPTIDE NUCLEIC ACID PEPTIDE POLYETHYLENEGLYCOL CONJUGATE; UNCLASSIFIED DRUG; MACROGOL DERIVATIVE;

ANIMAL CELL; ARTICLE; CHO CELL; DNA PACKAGING; DRUG CYTOTOXICITY; DRUG DELIVERY SYSTEM; DRUG FORMULATION; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; ANIMAL; CELL SURVIVAL; CHEMISTRY; CHO CELL LINE; CRICETULUS; DRUG EFFECTS; GENETIC TRANSFECTION; GENETICS; PARTICLE SIZE; PLASMID; PROCEDURES; STRUCTURE ACTIVITY RELATION; SURFACE PROPERTY; SYNTHESIS;

CRICETULUS GRISEUS;

ANIMALS; CELL SURVIVAL; CHO CELLS; CRICETULUS; DNA; PARTICLE SIZE; PEPTIDE NUCLEIC ACIDS; PLASMIDS; POLYETHYLENE GLYCOLS; POLYETHYLENEIMINE; STRUCTURE-ACTIVITY RELATIONSHIP; SURFACE PROPERTIES; TRANSFECTION;

EID: 77949811872     PISSN: 10431802     EISSN: 15204812     Source Type: Journal    
DOI: 10.1021/bc900328j     Document Type: Article

References (65)

1. Demeneix, B., Behr, J., Boussif, O., Zanta, M. A., Abdallah, B., and Remy, J. (1998) Gene transfer with lipospermines and polyethylenimines. Add. Drug Delivery Rev. 30, 85-95.
2. Ogris, M., Steinlein, P., Carotta, S., Brunner, S., and Wagner, E. (2001) DNA/polyethylenimine transfection particles: influence of ligands, polymer size, and PEGylation on internalization and gene expression. AAPS PharmSci 3, E21.
3. Garrett, S. W., Davies, O. R., Milroy, D. A., Wood, P. J., Pouton, C. W., and Threadgill, M. D. (2000) Synthesis and characterisation of polyamine-poly(ethylene glycol) constructs for DNA binding and gene delivery. Bioorg. Med. Chem. 8, 1779-1797.
4. Wolfert, M. A. S. L. W. (1996) Atomic force microscopic analysis of the influence of the molecular weight of poly(L)lysine on the size of polyelectrolyte complexes formed with DNA. Gene Ther. 3, 269-273.
5. Boussif, O., Lezoualc'h, F., Zanta, M. A., Mergny, M. D., Scherman, D., Demeneix, B., and Behr, J. P. (1995) A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl. Acad. Sci. U.S.A. 92, 7297-7301
6. Ogris, M., Brunner, S., Schuller, S., Kircheis, R., and Wagner, E. (1999) PEGylated DNA/transferrin-PEI complexes: reduced interaction with blood components, extended circulation in blood and potential for systemic gene delivery. Gene Ther. 6, 595-605.
7. Godbey WT, W. K., and Mikos, A. G. (1999) Poly(ethylenimine) and its role in gene delivery. J. Controlled Release 60, 149-160
8. Boussif, O., Lezoualc'h, F., Zanta, M. A., Mergny, M. D., Scherman, D., Demeneix, B., and Behr, J.-P. (1995) A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl. Acad. Sci. U.S.A. 92, 7297-7301.
9. Akinc, A., Thomas, M., Klibanov, A. M., and Langer, R. (2005) Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis. J. Gene Med., 7, 657-663.
10. Brunner, S., Sauer, T., Carotta, S., Gotten, M., Saltik, M., and Wagner, E. (2000) Cell cycle dependence of gene transfer by lipoplex, polyplex and recombinant adenovirus, Gene Ther. 7 401-407.
11. Chollet, P., Favrot, M., Hurbin, A., and Coll., J.-L. (2001) Sideeffects of a systemic injection of linear polyethylenimine-DNA complexes. J. Gene Med. 4, 84-91.
12. Dash, P., Read, M., Barrett, L., Wolfert, M., and Seymour, L. (1999) Factors affecting blood clearance and in vivo distribution of polyelectrolyte complexes for gene delivery. Gene Ther. 6, 643-650.
13. Nishikawa, M., Takemura, S., Takakura, Y., and Hashida, M. (1998) Targeted delivery of plasmid DNA to hepatocytes in vivo: optimization of the pharmacokinetics of plasmid DNA/galactosylated poly(L-lysine) complexes by controlling their physicochemical properties. J. Pharm. Exv. Ther. 287, 408-415.
14. Burke RS, P. S. (2008) Extracellular barriers to in Vivo PEI and PEGylated PEI polyplex-mediated gene delivery to the liver. Bioeoningate Chem. 19, 693-704.
15. Collard, W. T., Yang, Y., Kwok, K. Y., Park, Y., and Rice, K. G. (2000) Biodistribution, metabolism, and in vivo gene expression of low molecular weight, glycopeptide polyethylene glycol peptide DNA co-condensates. J. Pharm. Sci. 89, 499-512.
16. Ward, C. M., Read, M. L., and Seymour, L. W. (2001) Systemic circulation of poly(L-lysine)/DNA vectors is influenced by polycation molecular weight and type of DNA: differential, circulation in mice and rats and the implications for human gene therapy. Blood 97, 2221-2229.
17. Kircheis, R., Blessing, T., Brunner, S., Wightman, L., and Wagner, E. (2001) Tumor targeting with surface-shielded ligandpolycation DNA complexes. J. Controlled Release Release 72, 165-170
18. Bms, C. P. H., Aigner, A., Czubayko, F., and Kissel, T. (2004) Physicochemical and biological characterization of polyethylenimine-graft- poly(ethylene glycol) block copolymers as a delivery system for oligonucleotides and ribozymes Bioconjugate Chem. 15, 677-684
19. Merdan, T., K, K., Petersen, H., Bakowsky, U., Voigt, K. H., Kopeček, J., and Kissel, T. (2005) PEGylation of poly(ethylene imine) affects stability of complexes with plasmid DNA under in vivo conditions in a dose-dependent manner after intravenous injection into mice. Biconjugate Chem- 16, 785-792
20. Mishra, S., Webster, P., and Davis, M. E. (2004) PEGylation significantly affects cellular uptake and intracellular trafficking of non-viral gene delivery particles. Eur. J. Cell Biol. 83, 97-111.
21. Blessing, T., Kursa, M., Holzhauser, R., Kircheis, R., and Wagner, E. (2001) Different, strategies for formation of pegylated EGF-conjugated PEI/DNA complexes for targeted gene delivery. Bioconiusate Chem. 12, 529-537
22. Bartlett DW, D. M. (2007) Physicochemical and biological characterization of targeted, nucleic acid-containing nanoparticles. Bioconiusate Chem. 18, 456-468.
23. Wagner, E., Zenke, M., Gotten, M., Beug, H., and Birnstiel, M. L. (1990) Transferrin-polycation conjugates as earners for DNA uptake into cells. Proc. Natl. Acad. Sci. U.S.A. 87, 3410-3414
24. Varga, C. M., Wickham, T. J., and Lauffenburger, D. A. (2000) Receptor-mediated targeting of gene delivery vectors: insights from molecular mechanisms for improved vehicle design. Biotechnol Bioeng. 70, 593-605.
25. Rimann, M., Luhmann, T., Textor, M., Guerino, B., Ogier, J., and Hall, H. (2008) Characterization of PLL-g-PEG-DNA nanoparticles for the delivery of therapeutic DNA. Bioconjugate Chem. 19, 548-557
26. Choi, Y. H., Liu, F., Kim, J. S., Choi, Y. K., Park, J. S., and Kim, S. W. (1998) Polyethylene glycol-grafted poly-L-lysine as polymeric gene carrier. J. Controlled Release 54, 39-48.
27. Nguyen, H. K., Lemieux, P., Vinogradov, S. V., Gebhart, C. L., Guerin, N., Paradis, G., Bronich, T. K., Alakhov, V. Y., and Kabanov, A. V. (2000) Evaluation of polyether-polyethylenimine graft copolymers as gene transfer agents. Gene Ther. 7, 126-138.
28. Kaminskas LM., B. B., Kardias, P., Krippner, G. Y., Lessene, R., Kelly, B., and Porter, C. J. (2008) The impact, of molecular weight and PEG chain length on the systemic pharmacokinetics of PEGylated poly 1-lysine dendrimers. Mol. Pharm. 5, 449-463.
29. Walsh, M., Tangney, M., O'Neill, M. J., Larkin, J. O., Soden, D. M., McKenna, S. L., Darcy, R., O'Sullivan, G. C., and O'Driscoll, C. M. (2006) Evaluation, of cellular uptake and gene transfer efficiency of pegylated poly-L-lysine compacted DNA: implications for cancer gene therapy. Mol. Pharm. 3, 644-653.
30. Pun, S. H., and Davis, M. E. (2002) Development of a nonviral gene delivery vehicle for systemic application. Bioconjugate Chem. 13, 630-639
31. Pun, S. H., and Davis, M. E. (2002) Development, of a nonviral gene delivery vehicle for systemic application. Bioconjugate Chem. 13, 630-639.
32. Pun SH, B. N., Liu, A., Jensen, G., Machemer, T., Quijano, E., Schluep, T., Wen, S., Engler, H., Heidel, J., and Davis, M. E. (2004) Cyclodextrin-modified polyethylenimine polymers for gene delivery. Bioconjugate Chem. 15, 831-840.
33. Egholm, M., Christensen, L., Dueholm, K. L., Buchardt, O., Coull, J., and Nielsen, P. E. (1995) Efficient pH-independent sequence-specific DNA binding by pseudoisocytosine-containing bis-PNA. Nucleic Acids Res. 23, 217-222
34. Zelphati, O., Liang, X., Hobart, P., and Feigner, P. L. (1999) Gene chemistry: functionally and conformationally intact fluorescent, plasmid DNA. Hum. Gene Ther. 10, 15-24.
35. Egholm, M., Buchardt, O., Christensen, L., Behrens, C., Freier, S. M., Driver, D. A., Berg, R. H., Kim, S. K., Norden, B., and Nielsen, P. E. (1993) PNA hybridizes to complementary oligonucleotides obeying the Watson-Crick hydrogen-bonding rules. Nature 365, 566-568
36. Demidov, V. V., Potaman, V. N., Frank-Kamenetskii, M. D., Egholm, M., Buchard, O., Sonnichsen, S. H., and Nielsen, P. E. (1994) Stability of peptide nucleic acids in human semni and cellular extracts. Biochem. Pharmacol. 48, 1310-1313
37. Zelphati, O., Liang, X., Nguyen, C., Barlow, S., Sheng, S., Shao, Z., and Feigner, P. L. (2000) PNA-dependent gene chemistry: stable coupling of peptides and oligonucleotides to plasmid DNA. Biotechniques 28, 304-10, 312-4, 316.
38. Dean, D. A. (2000) Peptide nucleic acids: versatile tools for gene therapy strategies. Adv. Drug Delivery Rev. 44, 81-95.
39. Liang, K. W., Hoffman, E. P., and Huang, L. (2000) Targeted delivery of plasmid DNA to myogenic cells via transferrinconjugated peptide nucleic acid. Mol. Ther. 1, 236-243
40. Koppelhus, U., and Nielsen, P. E. (2003) Cellular delivery of peptide nucleic acid (PNA). Adv. Drug Delivery Rev. 55, 267-280
41. Yuan, F., Dellian, M., Fukumura, D., Leunig, M., Berk, D. A., Torchilin, V. P., and Jain, R. K. (1995) Vascular permeability in a human tumor xenograft: molecular size dependence and cutoff size, Cancer Res. 55, 3752-3756
42. Dvorak, H. F., Brown, L. F., Detmar, M., and Dvorak, A. M. (1995) Vascular permeability factor/vascular endothelial growth factor, microvascular hyperpermeability, and angiogenesis. Am. J. Pathol. 146, 1029-1039
43. Gerlowski, L. E., and Jain, R. K. (1986) Microvascular permeability of normal and neoplastic tissues. Microvasc. Res. 31, 288-305.
44. Sevick, E. M., and Jain, R. K. (1991) Measurement of capillary filtration coefficient in a solid tumor. Cancer Res. 51, 1352-1355
45. Maeda, H. (2001) The enhanced permeability and retention (EPR) effect in tumor vasculature: the key role of tumor-selective macromolecular drug targeting. Adv. Enzyme Regul. 41, 189-207.
46. Goodman, T. T., Ng, C. P., and Pun, S. H. (2008) 3-D tissue culture systems for the evaluation and optimization of nanoparticle-based drug carriers. Bioconjugate Chem. 19, 1951-1959
47. Enger, P. O., Thorsen, F., Lonning, P. E., Bjerkvig, R., and Hoover, F. (2002) Adeno-associated viral vectors penetrate human solid tumor tissue in vivo more effectively than adenoviral vectors. Hum. Gene Ther. 13, 1115-1125
48. Aoyama, Y., Kanamori, T., Nakai, T., Sasaki, T., Horiuchi, S., Sando, S., and Niidome, T. (2003) Artificial viruses and their application to gene delivery. Size-conttolled gene coating with glycocluster nanoparticles. J. Am. Chem. Soc. 125, 3455-3457
49. Nakai, T., Kanamori, T., Sando, S., and Aoyama, Y. (2003) Remarkably size-regulated cell invasion by artificial viruses. Saccharide-dependent self-aggregation of glycoviruses and its consequences in glycoviral gene delivery. J. Am. Chem. Soc. 125, 8465-8475
50. Osaki, F., Kanamori, T., Sando, S., Sera, T., and Aoyama, Y. (2004) A quantum, dot conjugated sugar ball, and its cellular uptake. On the size effects of endocytosis in the subviral region. J. Am. Chem. Soc. 126, 6520-6521
51. Chitlirani, B. D., Ghazani, A. A., and Chan, W. C. (2006) Determining the size and shape dependence of gold nanoparticle uptake into mammalian cells. Nano Lett. 6, 662-668
52. Wagner, E., Gotten, M., Foisner, R., and Birnstiel, M. L. (1991) Transferrin-polycation-DNA complexes: the effect of polycations on the structure of the complex and DNA delivery to cells. Proc. Natl. Acad. Sci. U.S.A. 88, 4255-4259
53. Perales, J. C., Ferkol, T., Beegen, H., Ratnoff, O. D., and Hanson, R. W. (1994) Gene transfer in vivo: sustained expression and regulation of genes introduced into the liver by receptortargeted uptake. Proc. Natl. Acad. Sci. U.S.A. 91, 4086-4090
54. Rippe, B., and Haraldsson, B. (1987) Fluid and protein fluxes across small and large pores in the microvasculature. Application of two-pore equations. Acta. Physiol. Stand- 131, 411-428
55. Melkko, S., Halin, C., Borsi, L., Zardi, L., and Neri, D. (2002) An antibody-calmodulin fusion protein, reveals a functional dependence between macromolecular isoelectric point and tumor targeting performance. Int. J. Radiat. Oncol. Biol. Phys. 54, 1485-1490
56. Dellian, M., Yuan, F., Trubetskoy, V. S., Torchilin, V. P., and Jain, R. K. (2000) Vascular permeability in a human tumour xenograft: molecular charge dependence. Br. J. Cancer 82, 1513-1518
57. Gao, H., Shi, W., and Freund, L. B. (2005) Mechanics of receptor-mediated endocytosis. Proc. Natl. Acad. U.S.A. 102, 9469-9474
58. Prabha, S., Zhou, W. Z., Panyani, J., and Labhasetwar, V. (2002) Size-dependency of nanoparticle-mediated gene transfection: studies with fractionated nanoparticles. Int. J. Pharm. 244, 105-115
59. Dauty, E., Remy, J. S., Zuber, G., and Behr, J. P. (2002) Inttacellular delivery of nanometric DNA particles via the folate receptor. Bioconjugate Chem. 13, 831-839
60. Lutolf MP, L.-F. J., Schmoekel, H. G., Metiers, A. T., Weber, F. E., Fields, G. B., and Hubbell, J. A. (2003) Synthetic matrix metalloproteinase- sensitive hydrogels for the conduction of tissue regeneration: engineering cell-invasion characteristics. Proc. Natl. Acad, Sci, U.S.A. 100, 5413-5418.
61. Hiemenez, P., and Rajagopalan, R. (1997) Principles of Colloid and Surface Chemistry, 3rd ed., CRC Press, Boca Raton, FL.
62. Vinogradov, S. V., Bronich, T. K., and Kabanov, A. V. (1998) Self-assembly of polyamine-poly(ethylene glycol) copolymers with phosphorothioate oligonucleotides. Bioconjugate Chem. 9, 805-812
63. Ferrari, S., Pettenazzo, A., Garbati, N., Zacchello, F., Behr, J. P., and Scarpa, M. (1999) Polyetliylenimine shows properties of interest, for cystic fibrosis gene therapy. Biochim. Biophys. Acta 1447, 219-225
64. Boeckle, S., von Gersdorff, K., van der Piepen, S., Culmsee, C. Wagner, E., and Ogris, M. (2004) Purification of polyethylenimine polyplexes highlights the role of free polycations in gene transfer. J. Gene Med. 6, 1102-1111
65. Choi, H. S., Liu, W., Misra, P., Tanaka, E., Zimmer, J. P., Itty Ipe, B., Bawendi, M. G., and Frangioni, J. V. (2007) Renal, clearance of quantum dots. Nat. Biotechnol. 25, 1165-1170