Hydrosoluble polymers for muscular gene delivery

European Journal of Pharmaceutics and Biopharmaceutics

Volumn 72, Issue 2, 2009, Pages 378-390

  Roques, Caroline ^a,b,c,d   Fromes, Yves ^c,d   Fattal, Elias ^a,b  

^a UNIVERSITÉ PARIS SACLAY   (France)

^b CNRS   (France)

^c INSERM   (France)

^d SORBONNE UNIVERSITÉ   (France)

Author keywords

Amphiphilic copolymers; Cationic polymers; Gene delivery; Hydrosoluble polymers; Striated muscles

Indexed keywords

COPOLYMER; DEXTRAN SPERMINE; DYSTROPHIN; ION; LIPOPOLYMER; NAKED DNA; PLASMID DNA; POLOXAMER; POLY(2 METHYL 2 OXAZIDINE); POLY(METHYLOXAZOLINE); POLY(PROPYLENEOXYDE); POLYAMIDOETHYLENEDIAMINE; POLYCATION; POLYETHYLENE OXIDE 13; POLYETHYLENEIMINE; POLYGLACTIN 10; POLYMER; POLYPHOSPHOESTER; POLYVINYL ALCOHOL; POVIDONE; SOMATOMEDIN C; SP 1017; TETRONIC ACID DERIVATIVE; UNCLASSIFIED DRUG;

BIODEGRADABLE IMPLANT; CARDIOMYOPATHY; CLINICAL TRIAL; CONGESTIVE CARDIOMYOPATHY; DRUG EFFICACY; DUCHENNE MUSCULAR DYSTROPHY; GENE EXPRESSION; GENE LOCATION; GENE TARGETING; GENE TRANSFER; GENETIC TRANSFECTION; HEART MUSCLE; HUMAN; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; POLYMERIZATION; REVIEW; SKELETAL MUSCLE; SOLUBILITY;

ANIMALS; MUSCLE, SKELETAL; POLYMERS; SOLUBILITY; TRANSFECTION;

EID: 67349088739     PISSN: 09396411     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ejpb.2008.11.006     Document Type: Review

References (160)

1. Wolff J.A., Malone R.W., Williams P., Chong W., Acsadi G., Jani A., and Felgner P.L. Direct gene transfer into mouse muscle in vivo. Science 247 (1990) 1465-1468
2. Sanger J.W., Sanger J.M., and Franzini-Armstrong C. Assembly of the skeletal muscle cell. In: Engel A.G., and Franzini-Armstrong C. (Eds). Myology vol. 1 (2004), McGraw-Hill, New York 45-65
3. Burke R.E. The structure and function of motor units. In: Engel A.G., and Franzini-Armstrong C. (Eds). Myology vol. 1 (2004), McGraw-Hill, New York 104-118
4. Sanes J.R. The extracellular matrix. In: Engel A.G., and Franzini-Armstrong C. (Eds). Myology vol. 1 (2004), McGraw-Hill, New York 471-487
5. Bischoff R., and Franzini-Armstrong C. Satellite and stem cells in muscle regeneration. In: Engel A.G., and Franzini-Armstrong C. (Eds). Myology vol. 1 (2004), McGraw-Hill, New York 66-86
6. Banker B.Q., and Engel A.G. Basic reactions of muscle. In: Engel A.G., and Franzini-Armstrong C. (Eds). Myology vol. 1 (2004), McGraw-Hill, New York 691-747
7. Engel A.G., and Banker B.Q. Ultrastructural changes in diseased muscle. In: Engel A.G., and Franzini-Armstrong C. (Eds). Myology vol. 1 (2004), McGraw-Hill, New York 749-887
8. Emery A.E. The muscular dystrophies. Lancet 359 (2002) 687-695
9. Katz A.M. Structure of the heart, myocardial cells, and biological membranes and biological membranes. In: Katz A.M. (Ed). Physiology of the Heart (2001), Lippincott, Williams & Wilkins, Philadelphia 1-38
10. LeGrice I., Pope A., and Smaill B. The architecture of the heart: myocyte organization and the cardiac extracellular matrix. In: Villareal F.J. (Ed). Interstitial Fibrosis in Heart Failure (2005), Springer, New York 3-21
11. Jugdutt B.I. Extracellular matrix and cardiac remodelling. In: Villareal F.J. (Ed). Interstitial Fibrosis in Heart Failure (2005), Springer, New York 23-55
12. Robinson T.F., Cohen-Gould L., and Factor S.M. Skeletal framework of mammalian heart muscle. Arrangement of inter- and pericellular connective tissue structures. Lab. Invest. 49 (1983) 482-498
13. Katz A.M. Proliferative signalling: regulation of gene expression, protein synthesis, cell growth and proliferation, and apoptosis. In: Katz A.M. (Ed). Physiology of the Heart (2001), Lippincott, Williams & Wilkins, Philadelphia 312-367
14. Katz A.M. Heart failure. In: Katz A.M. (Ed). Physiology of the Heart (2001), Lippincott, Williams & Wilkins, Philadelphia 658-694
15. Brown R.D., Mitchell M.D., and Long C.S. Pro-inflammatory cytokines and cardiac extracellular matrix: regulation of fibroblast phenotype. In: Villareal F.J. (Ed). Interstitial Fibrosis in Heart Failure (2005), Springer, New York 58-81
16. Lu Q.L., Bou-Gharios G., and Partridge T.A. Non-viral gene delivery in skeletal muscle: a protein factory. Gene Ther. 10 (2003) 131-142
17. Ratanamart J., and Shaw J.A. Plasmid-mediated muscle-targeted gene therapy for circulating therapeutic protein replacement: a tale of the tortoise and the hare?. Curr. Gene. Ther. 6 (2006) 93-110
18. Croze F., and Prud'homme G.J. Gene therapy of streptozotocin-induced diabetes by intramuscular delivery of modified preproinsulin genes. J. Gene Med. 5 (2003) 425-437
19. Pierce G.F., Lillicrap D., Pipe S.W., and Vandendriessche T. Gene therapy bioengineered clotting factors and novel technologies for hemophilia treatment. J. Thromb. Haemost. 5 (2007) 901-906
20. Rabinovsky E.D., and Draghia-Akli R. Insulin-like growth factor I plasmid therapy promotes in vivo angiogenesis. Mol. Ther. 9 (2004) 46-55
21. Sun X., Qiao H., Jiang H., Zhi X., Liu F., Wang J., Liu M., Dong D., Kanwar J.R., Xu R., and Krissansen G.W. Intramuscular delivery of antiangiogenic genes suppresses secondary metastases after removal of primary tumors. Cancer Gene Ther. 12 (2005) 35-45
22. Ulmer J.B., Donnelly J.J., Parker S.E., Rhodes G.H., Felgner P.L., Dwarki V.J., Gromkowski S.H., Deck R.R., DeWitt C.M., Friedman A., Hawe L.A., Leander K.R., Martinez D., Perry H.C., Shiver J.W., Montgomery D.L., and Liu M.A. Heterologous protection against influenza by injection of DNA encoding a viral protein. Science 259 (1993) 1745-1749
23. Prud'homme G.J., Lawson B.R., Chang Y., and Theofilopoulos A.N. Immunotherapeutic gene transfer into muscle. Trends Immunol. 22 (2001) 149-155
24. Prud'homme G.J. DNA vaccination against tumors. J. Gene Med. 7 (2005) 3-17
25. Prud'homme G.J., Draghia-Akli R., and Wang Q. Plasmid-based gene therapy of diabetes mellitus. Gene Ther. 14 (2007) 553-564
26. Michel M.L., and Mancini-Bourgine M. Therapeutic vaccination against chronic hepatitis B virus infection. J. Clin. Virol. 34 Suppl. 1 (2005) S108-S114
27. Romero N.B., Braun S., Benveniste O., Leturcq F., Hogrel J.Y., Morris G.E., Barois A., Eymard B., Payan C., Ortega V., Boch A.L., Lejean L., Thioudellet C., Mourot B., Escot C., Choquel A., Recan D., Kaplan J.C., Dickson G., Klatzmann D., Molinier-Frenckel V., Guillet J.G., Squiban P., Herson S., and Fardeau M. Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Hum. Gene Ther. 15 (2004) 1065-1076
28. Rando T.A. Non-viral gene therapy for Duchenne muscular dystrophy: progress and challenges. Biochim. Biophys. Acta 1772 (2007) 263-271
29. Traktuev D.O., Tsokolaeva Z.I., Shevelev A.A., Talitskiy K.A., Stepanova V.V., Johnstone B.H., Rahmat-Zade T.M., Kapustin A.N., Tkachuk V.A., March K.L., and Parfyonova Y.V. Urokinase gene transfer augments angiogenesis in ischemic skeletal and myocardial muscle. Mol. Ther. 15 (2007) 1939-1946
30. Anghel T.M., and Pogwizd S.M. Creating a cardiac pacemaker by gene therapy. Med. Biol. Eng. Comput. 45 (2007) 145-155
31. Marban E., and Cho H.C. Creation of a biological pacemaker by gene- or cell-based approaches. Med. Biol. Eng. Comput. 45 (2007) 133-144
32. Ashikaga H., and Villareal F.J. Prospects for gene therapy for the fibrosed heart: targeting regulators of extracellular matrix turnover. In: Villareal F.J. (Ed). Interstitial Fibrosis in Heart Failure (2005), Springer, New York 343-354
33. Matsumura S., Iwanaga S., Mochizuki S., Okamoto H., Ogawa S., and Okada Y. Targeted deletion or pharmacological inhibition of MMP-2 prevents cardiac rupture after myocardial infarction in mice. J. Clin. Invest. 115 (2005) 599-609
34. Serose A., Prudhon B., Salmon A., Doyennette M.A., Fiszman M.Y., and Fromes Y. Administration of insulin-like growth factor-1 (IGF-1) improves both structure and function of delta-sarcoglycan deficient cardiac muscle in the hamster. Basic Res. Cardiol. 100 (2005) 161-170
35. Serose A., Salmon A., Fiszman M.Y., and Fromes Y. Short-term treatment using insulin-like growth factor-1 (IGF-1) improves life expectancy of the delta-sarcoglycan deficient hamster. J. Gene Med. 8 (2006) 1048-1055
36. Acsadi G., Dickson G., Love D.R., Jani A., Walsh F.S., Gurusinghe A., Wolff J.A., and Davies K.E. Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs. Nature 352 (1991) 815-818
37. Acsadi G., Jiao S.S., Jani A., Duke D., Williams P., Chong W., and Wolff J.A. Direct gene transfer and expression into rat heart in vivo. New Biol. 3 (1991) 71-81
38. Wolff J.A., Ludtke J.J., Acsadi G., Williams P., and Jani A. Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum. Mol. Genet. 1 (1992) 363-369
39. Wolff J.A., Dowty M.E., Jiao S., Repetto G., Berg R.K., Ludtke J.J., Williams P., and Slautterback D.B. Expression of naked plasmids by cultured myotubes and entry of plasmids into T tubules and caveolae of mammalian skeletal muscle. J. Cell Sci. 103 Pt 4 (1992) 1249-1259
40. Buttrick P.M., Kass A., Kitsis R.N., Kaplan M.L., and Leinwand L.A. Behavior of genes directly injected into the rat heart in vivo. Circ. Res. 70 (1992) 193-198
41. Jiao S., Williams P., Berg R.K., Hodgeman B.A., Liu L., Repetto G., and Wolff J.A. Direct gene transfer into nonhuman primate myofibers in vivo. Hum. Gene Ther. 3 (1992) 21-33
42. Cao B., and Huard J. Gene transfer to skeletal muscle using herpes simplex virus-based vectors. Methods Mol. Biol. 246 (2004) 301-308
43. van Deutekom J.C., Hoffman E.P., and Huard J. Muscle maturation: implications for gene therapy. Mol. Med. Today 4 (1998) 214-220
44. Fromes Y., Salmon A., Wang X., Collin H., Rouche A., Hagege A., Schwartz K., and Fiszman M.Y. Gene delivery to the myocardium by intrapericardial injection. Gene Ther. 6 (1999) 683-688
45. Hagstrom J.E., Hegge J., Zhang G., Noble M., Budker V., Lewis D.L., Herweijer H., and Wolff J.A. A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs. Mol. Ther. 10 (2004) 386-398
46. Zhang G., Ludtke J.J., Thioudellet C., Kleinpeter P., Antoniou M., Herweijer H., Braun S., and Wolff J.A. Intraarterial delivery of naked plasmid DNA expressing full-length mouse dystrophin in the mdx mouse model of duchenne muscular dystrophy. Hum. Gene Ther. 15 (2004) 770-782
47. Fromes Y., terKeurs H., Schwartz K., and Fiszman M.Y. ECG-synchronized arterial injections improve in vivo cardiac gene transfer. J. Mol. Cell. Cardiol. 30 (1998) A48
48. Beeri R., Guerrero J.L., Supple G., Sullivan S., Levine R.A., and Hajjar R.J. New efficient catheter-based system for myocardial gene delivery. Circulation 106 (2002) 1756-1759
49. Hayase M., Del Monte F., Kawase Y., Macneill B.D., McGregor J., Yoneyama R., Hoshino K., Tsuji T., De Grand A.M., Gwathmey J.K., Frangioni J.V., and Hajjar R.J. Catheter-based antegrade intracoronary viral gene delivery with coronary venous blockade. Am. J. Physiol. Heart Circ. Physiol. 288 (2005) H2995-3000
50. Roques C., Salmon A., Fiszman M.Y., Fattal E., and Fromes Y. Intrapericardial administration of novel DNA formulations based on thermosensitive Poloxamer 407 gel. Int. J. Pharm. 331 (2007) 220-223
51. Boussif O., Lezoualc'h F., Zanta M.A., Mergny M.D., Scherman D., Demeneix B., and Behr J.P. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl. Acad. Sci. USA 92 (1995) 7297-7301
52. Huang H., Tang G., Wang Q., Li D., Shen F., Zhou J., and Yu H. Two novel non-viral gene delivery vectors: low molecular weight polyethylenimine cross-linked by (2-hydroxypropyl)-beta-cyclodextrin or (2-hydroxypropyl)-gamma-cyclodextrin. Chem. Commun. (Camb.) 5 (2006) 2382-2384
53. Tang M.X., and Szoka F.C. The influence of polymer structure on the interactions of cationic polymers with DNA and morphology of the resulting complexes. Gene Ther. 4 (1997) 823-832
54. Suh J., Paik H.J., and Hwang B.K. Ionization of poly(ethylenimine) and poly(allylamine) at various pH's. Bioorg. Chem. 22 (1994) 318-327
55. Lungwitz U., Breunig M., Blunk T., and Gopferich A. Polyethylenimine-based non-viral gene delivery systems. Eur. J. Pharm. Biopharm. 60 (2005) 247-266
56. Choosakoonkriang S., Lobo B.A., Koe G.S., Koe J.G., and Middaugh C.R. Biophysical characterization of PEI/DNA complexes. J. Pharm. Sci. 92 (2003) 1710-1722
57. Bronich T., Kabanov A.V., and Marky L.A. A thermodynamic characterization of the interaction of a cationic copolymer with DNA. J. Phys. Chem. B 105 (2001) 6042-6050
58. Dunlap D.D., Maggi A., Soria M.R., and Monaco L. Nanoscopic structure of DNA condensed for gene delivery. Nucleic Acids Res. 25 (1997) 3095-3101
59. Hsu C.Y., and Uludag H. Effects of size and topology of DNA molecules on intracellular delivery with non-viral gene carriers. BMC Biotechnol. 8 (2008) 23
60. Boeckle S., von Gersdorff K., van der Piepen S., Culmsee C., Wagner E., and Ogris M. Purification of polyethylenimine polyplexes highlights the role of free polycations in gene transfer. J. Gene Med. 6 (2004) 1102-1111
61. Finsinger D., Remy J.S., Erbacher P., Koch C., and Plank C. Protective copolymers for nonviral gene vectors: synthesis, vector characterization and application in gene delivery. Gene Ther. 7 (2000) 1183-1192
62. Sharma V.K., Thomas M., and Klibanov A.M. Mechanistic studies on aggregation of polyethylenimine-DNA complexes and its prevention. Biotechnol. Bioeng. 90 (2005) 614-620
63. Behr J.P. Gene transfer with synthetic cationic amphiphiles: prospects for gene therapy. Bioconjug. Chem. 5 (1994) 382-389
64. Remy J.S., Abdallah B., Zanta M.A., Boussif O., Behr J.P., and Demeneix B. Gene transfer with lipospermines and polyethylenimines. Adv. Drug Deliv. Rev. 30 (1998) 85-95
65. Labat-Moleur F., Steffan A.M., Brisson C., Perron H., Feugeas O., Furstenberger P., Oberling F., Brambilla E., and Behr J.P. An electron microscopy study into the mechanism of gene transfer with lipopolyamines. Gene Ther. 3 (1996) 1010-1017
66. Mislick K.A., and Baldeschwieler J.D. Evidence for the role of proteoglycans in cation-mediated gene transfer. Proc. Natl. Acad. Sci. USA 93 (1996) 12349-12354
67. Godbey W.T., Wu K.K., and Mikos A.G. Poly(ethylenimine) and its role in gene delivery. J. Control. Release 60 (1999) 149-160
68. Remy-Kristensen A., Clamme J.P., Vuilleumier C., Kuhry J.G., and Mely Y. Role of endocytosis in the transfection of L929 fibroblasts by polyethylenimine/DNA complexes. Biochim. Biophys. Acta 1514 (2001) 21-32
69. Rejman J., Bragonzi A., and Conese M. Role of clathrin- and caveolae-mediated endocytosis in gene transfer mediated by lipo- and polyplexes. Mol. Ther. 12 (2005) 468-474
70. von Gersdorff K., Sanders N.N., Vandenbroucke R., De Smedt S.C., Wagner E., and Ogris M. The internalization route resulting in successful gene expression depends on both cell line and polyethylenimine polyplex type. Mol. Ther. 14 (2006) 745-753
71. Behr J.P. The proton sponge: a trick to enter cells the viruses did not exploit. Chimia 51 (1997) 34-36
72. Akinc A., Thomas M., Klibanov A.M., and Langer R. Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis. J. Gene Med. 7 (2005) 657-663
73. Sonawane N.D., Szoka Jr. F.C., and Verkman A.S. Chloride accumulation and swelling in endosomes enhances DNA transfer by polyamine-DNA polyplexes. J. Biol. Chem. 278 (2003) 44826-44831
74. Clamme J.P., Krishnamoorthy G., and Mely Y. Intracellular dynamics of the gene delivery vehicle polyethylenimine during transfection: investigation by two-photon fluorescence correlation spectroscopy. Biochim. Biophys. Acta 1617 (2003) 52-61
75. Godbey W.T., Wu K.K., and Mikos A.G. Tracking the intracellular path of poly(ethylenimine)/DNA complexes for gene delivery. Proc. Natl. Acad. Sci. USA 96 (1999) 5177-5181
76. Akinc A., and Langer R. Measuring the pH environment of DNA delivered using nonviral vectors: implications for lysosomal trafficking. Biotechnol. Bioeng. 78 (2002) 503-508
77. Melchior F., and Gerace L. Mechanisms of nuclear protein import. Curr. Opin. Cell Biol. 7 (1995) 310-318
78. Brunner S., Furtbauer E., Sauer T., Kursa M., and Wagner E. Overcoming the nuclear barrier: cell cycle independent nonviral gene transfer with linear polyethylenimine or electroporation. Mol. Ther. 5 (2002) 80-86
79. Kawakami S., Ito Y., Charoensit P., Yamashita F., and Hashida M. Evaluation of proinflammatory cytokine production induced by linear and branched polyethylenimine/plasmid DNA complexes in mice. J. Pharmacol. Exp. Ther. 317 (2006) 1382-1390
80. Kunath K., von Harpe A., Fischer D., Petersen H., Bickel U., Voigt K., and Kissel T. Low-molecular-weight polyethylenimine as a non-viral vector for DNA delivery: comparison of physicochemical properties, transfection efficiency and in vivo distribution with high-molecular-weight polyethylenimine. J. Control. Release 89 (2003) 113-125
81. Fischer D., Bieber T., Li Y., Elsasser H.P., and Kissel T. A novel non-viral vector for DNA delivery based on low molecular weight, branched polyethylenimine: effect of molecular weight on transfection efficiency and cytotoxicity. Pharm. Res. 16 (1999) 1273-1279
82. Werth S., Urban-Klein B., Dai L., Hobel S., Grzelinski M., Bakowsky U., Czubayko F., and Aigner A. A low molecular weight fraction of polyethylenimine (PEI) displays increased transfection efficiency of DNA and siRNA in fresh or lyophilized complexes. J. Control. Release 112 (2006) 257-270
83. Demeneix B., and Behr J.-P. Polyethylenimine (PEI). In: Huang L., Hung M.-C., and Wagner E. (Eds). Advances in Genetics vol. 53 (2005), Academic Press, New York 215-230
84. Jeong G.J., Byun H.M., Kim J.M., Yoon H., Choi H.G., Kim W.K., Kim S.J., and Oh Y.K. Biodistribution and tissue expression kinetics of plasmid DNA complexed with polyethylenimines of different molecular weight and structure. J. Control. Release 118 (2007) 118-125
85. Anderson D.G., Peng W., Akinc A., Hossain N., Kohn A., Padera R., Langer R., and Sawicki J.A. A polymer library approach to suicide gene therapy for cancer. Proc. Natl. Acad. Sci. USA 101 (2004) 16028-16033
86. Lee M., Rentz J., Han S.O., Bull D.A., and Kim S.W. Water-soluble lipopolymer as an efficient carrier for gene delivery to myocardium. Gene Ther. 10 (2003) 585-593
87. Chang C.W., Choi D., Kim W.J., Yockman J.W., Christensen L.V., Kim Y.H., and Kim S.W. Non-ionic amphiphilic biodegradable PEG-PLGA-PEG copolymer enhances gene delivery efficiency in rat skeletal muscle. J. Control. Release 118 (2007) 245-253
88. Ruponen M., Yla-Herttuala S., and Urtti A. Interactions of polymeric and liposomal gene delivery systems with extracellular glycosaminoglycans: physicochemical and transfection studies. Biochim. Biophys. Acta 1415 (1999) 331-341
89. Ruponen M., Ronkko S., Honkakoski P., Pelkonen J., Tammi M., and Urtti A. Extracellular glycosaminoglycans modify cellular trafficking of lipoplexes and polyplexes. J. Biol. Chem. 276 (2001) 33875-33880
90. Oh Y.K., Park J.S., Kang M.J., Ko J.J., Kim J.M., and Kim C.K. Enhanced adjuvanticity of interleukin-2 plasmid DNA administered in polyethylenimine complexes. Vaccine 21 (2003) 2837-2843
91. Bos G.W., Kanellos T., Crommelin D.J., Hennink W.E., and Howard C.R. Cationic polymers that enhance the performance of HbsAg DNA in vivo. Vaccine 23 (2004) 460-469
92. Wang S., Ma N., Gao S.J., Yu H., and Leong K.W. Transgene expression in the brain stem effected by intramuscular injection of polyethylenimine/DNA complexes. Mol. Ther. 3 (2001) 658-664
93. Schaffer D.V., Fidelman N.A., Dan N., and Lauffenburger D.A. Vector unpacking as a potential barrier for receptor-mediated polyplex gene delivery. Biotechnol. Bioeng. 67 (2000) 598-606
94. Varga C.M., Hong K., and Lauffenburger D.A. Quantitative analysis of synthetic gene delivery vector design properties. Mol. Ther. 4 (2001) 438-446
95. Prasad T.K., Gopal V., and Madhusudhana Rao N. Structural changes in DNA mediated by cationic lipids alter in vitro transcriptional activity at low charge ratios. Biochim. Biophys. Acta 1619 (2003) 59-69
96. Okuda T., Niidome T., and Aoyagi H. Cytosolic soluble proteins induce DNA release from DNA-gene carrier complexes. J. Control. Release 98 (2004) 325-332
97. Florea B.I., Meaney C., Junginger H.E., and Borchard G. Transfection efficiency and toxicity of polyethylenimine in differentiated Calu-3 and nondifferentiated COS-1 cell cultures. AAPS Pharm. Sci. 4 (2002) E12
98. Zou S.M., Erbacher P., Remy J.S., and Behr J.P. Systemic linear polyethylenimine (L-PEI)-mediated gene delivery in the mouse. J. Gene Med. 2 (2000) 128-134
99. Chollet P., Favrot M.C., Hurbin A., and Coll J.L. Side-effects of a systemic injection of linear polyethylenimine-DNA complexes. J. Gene Med. 4 (2002) 84-91
100. S.M. Moghimi, P. Symonds, J.C. Murray, A.C. Hunter, G. Debska, A. Szewczyk, A two-stage poly(ethylenimine)-mediated cytotoxicity: implications for gene transfer/therapy. Mol. Ther. 11 (2005) 990-995.
101. Hong S., Leroueil P.R., Janus E.K., Peters J.L., Kober M.M., Islam M.T., Orr B.G., Baker Jr. J.R., and Banaszak Holl M.M. Interaction of polycationic polymers with supported lipid bilayers and cells: nanoscale hole formation and enhanced membrane permeability. Bioconjug. Chem. 17 (2006) 728-734
102. Clamme J.P., Azoulay J., and Mely Y. Monitoring of the formation and dissociation of polyethylenimine/DNA complexes by two photon fluorescence correlation spectroscopy. Biophys. J. 84 (2003) 1960-1968
103. Fahrmeir J., Gunther M., Tietze N., Wagner E., and Ogris M. Electrophoretic purification of tumor-targeted polyethylenimine-based polyplexes reduces toxic side effects in vivo. J. Control. Release 122 (2007) 236-245
104. Neu M., Fischer D., and Kissel T. Recent advances in rational gene transfer vector design based on poly(ethylene imine) and its derivatives. J. Gene Med. 7 (2005) 992-1009
105. Lee M., Rentz J., Bikram M., Han S., Bull D.A., and Kim S.W. Hypoxia-inducible VEGF gene delivery to ischemic myocardium using water-soluble lipopolymer. Gene Ther. 10 (2003) 1535-1542
106. Hosseinkhani H., Azzam T., Tabata Y., and Domb A.J. Dextran-spermine polycation: an efficient nonviral vector for in vitro and in vivo gene transfection. Gene Ther. 11 (2004) 194-203
107. Wang J., Zhang P.C., Mao H.Q., and Leong K.W. Enhanced gene expression in mouse muscle by sustained release of plasmid DNA using PPE-EA as a carrier. Gene Ther. 9 (2002) 1254-1261
108. Xu P., Li S.Y., Li Q., Ren J., Van Kirk E.A., Murdoch W.J., Radosz M., and Shen Y. Biodegradable cationic polyester as an efficient carrier for gene delivery to neonatal cardiomyocytes. Biotechnol. Bioeng. 95 (2006) 893-903
109. Christensen L.V., Chang C.W., Yockman J.W., Conners R., Jackson H., Zhong Z., Feijen J., Bull D.A., and Kim S.W. Reducible poly(amido ethylenediamine) for hypoxia-inducible VEGF delivery. J. Control. Release 118 (2007) 254-261
110. Alvarez-Lorenzo C., Gonzalez-Lopez J., Fernandez-Tarrio M., Sandez-Macho I., and Concheiro A. Tetronic micellization, gelation and drug solubilization: influence of pH and ionic strength. Eur. J. Pharm. Biopharm. 66 (2007) 244-252
111. Armstrong J.K., Chowdhry B.Z., Snowden M.J., Dong J., and Leharne S.A. The effect of pH and concentration upon aggregation transitions in aqueous solutions of poloxamine T701. Int. J. Pharm. 229 (2001) 57-66
112. Fernandez-Tarrio M., Alvarez-Lorenzo C., and Concheiro A. Calorimetric approach to tetronic/water interactions. J. Ther. Anal. Calorimetry 87 (2007) 171-178
113. Habas J.P., Pavie E., Perreur C., Lapp A., and Peyrelasse J. Nanostructure in block copolymer solutions: rheology and small-angle neutron scattering. Phys. Rev. E Stat. Nonlin. Soft Matter Phys. 70 (2004) 061802
114. Perreur C., Habas J.P., Francois J., Peyrelasse J., and Lapp A. Determination of the structure of the organized phase of the block copolymer PEO-PPO-PEO in aqueous solutions under flow by small-angle neutron scattering. Phys. Rev. E Stat. Nonlin. Soft Matter Phys. 65 (2002) 041802
115. Sosnik A., and Sefton M.V. Semi-synthetic collagen/poloxamine matrices for tissue engineering. Biomaterials 26 (2005) 7425-7435
116. Cappel M.J., and Kreuter J. Effect of nonionic surfactants on transdermal drug delivery: II. Poloxamer and poloxamine surfactants. J. Pharm. 69 (1991) 155-167
117. Chiappetta D.A., Degrossi J., Teves S., D'Aquino M., Bregni C., and Sosnik A. Triclosan-loaded poloxamine micelles for enhanced topical antibacterial activity against biofilm. Eur. J. Pharm. Biopharm. (2007)
118. Csaba N., Sanchez A., and Alonso M.J. PLGA: poloxamer and PLGA: poloxamine blend nanostructures as carriers for nasal gene delivery. J. Control. Release 113 (2006) 164-172
119. Moghimi S.M., and Hunter A.C. Poloxamers and poloxamines in nanoparticle engineering and experimental medicine. Trends Biotechnol. 18 (2000) 412-420
120. Prokop A., Kozlov E., Moore W., and Davidson J.M. Maximizing the in vivo efficiency of gene transfer by means of nonviral polymeric gene delivery vehicles. J. Pharm. Sci. 91 (2002) 67-76
121. Pitard B., Bello-Roufai M., Lambert O., Richard P., Desigaux L., Fernandes S., Lanctin C., Pollard H., Zeghal M., Rescan P.Y., and Escande D. Negatively charged self-assembling DNA/poloxamine nanospheres for in vivo gene transfer. Nucleic Acids Res. 32 (2004) e159
122. Richard P., Bossard F., Desigaux L., Lanctin C., Bello-Roufai M., and Pitard B. Amphiphilic block copolymers promote gene delivery in vivo to pathological skeletal muscles. Hum Gene Ther. 16 (2005) 1318-1324
123. Budker V., Trubetskoy V., and Wolff J.A. Condensation of nonstochiometric DNA/polycation complexes by divalent cations. Biopolymers 83 (2006) 646-657
124. Mumper R.J., Duguid J.G., Anwer K., Barron M.K., Nitta H., and Rolland A.P. Polyvinyl Derivatives as Novel Interactive Polymers for Controlled Gene Delivery to Muscle. Pharm. Res. 13 (1996) 701-709
125. Mumper R.J., Wang J., Klakamp S.L., Nitta H., Anwer K., Tagliaferri F., and Rolland A.P. Protective interactive noncondensing (PINC) polymers for enhanced plasmid distribution and expression in rat skeletal muscle. J. Control. Release 52 (1998) 191-203
126. Mumper R.J., and Rolland A.P. Plasmid delivery to muscle: Recent advances in polymer delivery systems. Adv. Drug Deliv. Rev. 30 (1998) 151-172
127. Quezada A., Horner M.J., Loera D., French M., Pericle F., Johnson R., Perrard J., Jenkins M., and Coleman M. Safety toxicity study of plasmid-based IL-12 therapy in Cynomolgus monkeys. J. Pharm. Pharmacol. 54 (2002) 241-248
128. Fewell J.G., MacLaughlin F., Mehta V., Gondo M., Nicol F., Wilson E., and Smith L.C. Gene therapy for the treatment of hemophilia B using PINC-formulated plasmid delivered to muscle with electroporation. Mol. Ther. 3 (2001) 574-583
129. Sheu S.J., Chou L.C., Bee Y.S., Chen J.F., Lin H.C., Lin P.R., Lam H.C., and Tai M.H. Suppression of choroidal neovascularization by intramuscular polymer-based gene delivery of vasostatin. Exp. Eye Res. 81 (2005) 673-679
130. Alila H., Coleman M., Nitta H., French M., Anwer K., Liu Q., Meyer T., Wang J., Mumper R., Oubari D., Long S., Nordstrom J., and Rolland A. Expression of biologically active human insulin-like growth factor-I following intramuscular injection of a formulated plasmid in rats. Hum. Gene Ther. 8 (1997) 1785-1795
131. Anwer K., Shi M., French M.F., Muller S.R., Chen W., Liu Q., Proctor B.L., Wang J., Mumper R.J., Singhal A., Rolland A.P., and Alila H.W. Systemic effect of human growth hormone after intramuscular injection of a single dose of a muscle-specific gene medicine. Hum. Gene Ther. 9 (1998) 659-670
132. Oga M., Takenaga K., Sato Y., Nakajima H., Koshikawa N., Osato K., and Sakiyama S. Inhibition of metastatic brain tumor growth by intramuscular administration of the endostatin gene. Int. J. Oncol. 23 (2003) 73-79
133. Zimecki M., Webb D.R., and Rogers T.J. The regulation of the immune response to polyvinylpyrollidone: antigen induced changes in prostaglandin and cyclic nucleotide levels. Arch. Immunol. Ther. Exp. (Warsz) 28 (1980) 179-197
134. Anwer K., Earle K.A., Shi M., Wang J., Mumper R.J., Proctor B., Jansa K., Ledebur H.C., Davis S., Eaglstein W., and Rolland A.P. Synergistic effect of formulated plasmid and needle-free injection for genetic vaccines. Pharm. Res. 16 (1999) 889-895
135. Mortensen K. Structural studies of aqueous solutions of PEO-PPO-PEO triblock copolymers, their micellar aggregates and mesophases; a small-angle neutron scattering study. J. Phys. Condens. Matter 8 (1996) A103-A124
136. Alexandridis P., and Holzwarth J.F. Differential scanning calorimetry investigation of the effect of salts on aqueous solution properties of an amphiphilic block copolymer (poloxamer). Langmuir 13 (1997) 6074-6082
137. Alexandridis P., Holzwarth J.F., and Hatton T.A. Micellization of poly(ethylene oxide)-poly(propylene oxide)-poly(ethylene oxide) triblock copolymers in aqueous solutions: thermodynamics of copolymer association. Macromolecules 27 (1994) 2414-2425
138. Ma J.h., Guo C., Tang Y.l., and Liu H.z. 1H NMR spectroscopic investigations on the micellization and gelation of PEO-PPO-PEO block copolymers in aqueous solutions. Langmuir 23 (2007) 9596-9605
139. Mata J.P., Majhi P.R., Guo C., Liu H.Z., and Bahadur P. Concentration, temperature, and salt-induced micellization of a triblock copolymer Pluronic L64 in aqueous media. J. Colloid Interface Sci. 292 (2005) 548-556
140. Wanka G., Hoffmann H., and Ulbricht W. Phase Diagrams and Aggregation Behavior of Poly(oxyethylene)-Poly(oxypropylene)-Poly(oxyethylene) Triblock Copolymers in Aqueous Solutions. Macromolecules 27 (1994) 4145-4159
141. Zheng L., Guo C., Wang J., Liang X., Bahadur P., Chen S., Ma J., and Liu H.z. Micellization of Pluronic L64 in salt solution by FTIR spectroscopy. Vib. Spectrosc. 39 (2005) 157-162
142. Nagarajan R. Solubilization of hydrocarbons and resulting aggregate shape transitions in aqueous solutions of Pluronic(R) (PEO-PPO-PEO) block copolymers. Colloids Surf. B Biointerfaces 16 (1999) 55-72
143. Schillen K., Brown W., and Johnsen R.M. Micellar sphere-to-rod transition in an aqueous triblock copolymer system. A dynamic light scattering study of translational and rotational diffusion. Macromolecules 27 (1994) 4825-4832
144. Chiappetta D.A., and Sosnik A. Poly(ethylene oxide)-poly(propylene oxide) block copolymer micelles as drug delivery agents: improved hydrosolubility, stability and bioavailability of drugs. Eur. J. Pharm. Biopharm. 66 (2007) 303-317
145. Lemieux P., Guerin N., Paradis G., Proulx R., Chistyakova L., Kabanov A., and Alakhov V. A combination of poloxamers increases gene expression of plasmid DNA in skeletal muscle. Gene Ther. 7 (2000) 986-991
146. Pitard B., Pollard H., Agbulut O., Lambert O., Vilquin J.T., Cherel Y., Abadie J., Samuel J.L., Rigaud J.L., Menoret S., Anegon I., and Escande D. A nonionic amphiphile agent promotes gene delivery in vivo to skeletal and cardiac muscles. Hum. Gene Ther. 13 (2002) 1767-1775
147. Yang Z., Zhu J., Sriadibhatla S., Gebhart C., Alakhov V., and Kabanov A. Promoter- and strain-selective enhancement of gene expression in a mouse skeletal muscle by a polymer excipient Pluronic P85. J. Control. Release 108 (2005) 496-512
148. Sriadibhatla S., Yang Z., Gebhart C., Alakhov V.Y., and Kabanov A. Transcriptional activation of gene expression by pluronic block copolymers in stably and transiently transfected cells. Mol. Ther. 13 (2006) 804-813
149. Alakhov V., Lemieux P., Klinski E., Kabanov A., and Pietrzynski G. Block copolymeric biotransport carriers as versatile vehicles for drug delivery. Expert Opin. Biol. Ther. 1 (2001) 583-602
150. Richard P., Pollard H., Lanctin C., Bello-Roufai M., Desigaux L., Escande D., and Pitard B. Inducible production of erythropoietin using intramuscular injection of block copolymer/DNA formulation. J. Gene Med. 7 (2005) 80-86
151. Johnston T.P., and Miller S.C. Toxicological evaluation of poloxamer vehicles for intramuscular use. J. Parenter. Sci. Technol. 39 (1985) 83-89
152. Gau-Racine J., Lal J., Zeghal M., and Auvray L. PEO-PPO block copolymer vectors do not interact directly with DNA but with lipid membranes. J. Phys. Chem. B 111 (2007) 9900-9907
153. Bello-Roufai M., Lambert O., and Pitard B. Relationships between the physicochemical properties of an amphiphilic triblock copolymers/DNA complexes and their intramuscular transfection efficiency. Nucleic Acids Res. 35 (2007) 728-739
154. Evdokimov Y.M., Platonov A.L., Tikhonenko A.S., and Varshavsky Y.M. A compact form of double-stranded DNA in solution. FEBS Lett. 23 (1972) 180-184
155. Evdokimov Y.M., Pyatigorskaya T.L., Polyvtsev O.F., Akimenko N.M., Kadykov V.A., Tsvankin D.Y., and Varshavsky Y.M. A comparative X-ray diffraction and circular dichroism study of DNA compact particles formed in water-salt solutions, containing poly(ethylene glycol). Nucleic Acids Res. 3 (1976) 2353-2366
156. Kabanov A.V., Lemieux P., Vinogradov S., and Alakhov V. Pluronic block copolymers: novel functional molecules for gene therapy. Adv. Drug Deliv. Rev. 54 (2002) 223-233
157. Batrakova E.V., Li S., Vinogradov S.V., Alakhov V.Y., Miller D.W., and Kabanov A.V. Mechanism of pluronic effect on P-glycoprotein efflux system in blood-brain barrier: contributions of energy depletion and membrane fluidization. J. Pharmacol. Exp. Ther. 299 (2001) 483-493
158. Lavigne M.D., Pohlschmidt M., Novo J.F., Higgins B., Alakhov V., Lochmuller H., Sakuraba H., Goldspink G., MacDermot K., and Gorecki D.C. Promoter dependence of plasmid-pluronics targeted alpha galactosidase A expression in skeletal muscle of Fabry mice. Mol. Ther. 12 (2005) 985-990
159. Lavigne M.D., Yates L., Coxhead P., and Gorecki D.C. Nuclear-targeted chimeric vector enhancing nonviral gene transfer into skeletal muscle of Fabry mice in vivo. FASEB J. 22 (2008)
160. Brissault B., Kichler A., Leborgne C., Jarroux N., Cheradame H., and Guis C. Amphiphilic poly[(propylene glycol)-block-(2-methyl-2-oxazoline)] copolymers for gene transfer in skeletal muscle. ChemMedChem 2 (2007) 1202-1207