Carbohydrate polymers for nonviral nucleic acid delivery

Topics in Current Chemistry

Volumn 296, Issue , 2010, Pages 131-190

  Sizovs, Antons ^a   McLendon, Patrick M ^d   Srinivasachari, Sathya ^b,c   Reineke, Theresa M ^a  

^a VIRGINIA POLYTECHNIC INSTITUTE AND STATE UNIVERSITY   (United States)

^b UNIVERSITY OF CINCINNATI   (United States)

^c Institute for Stem Cell Biology and Regenerative Medicine   (India)

^d CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

Author keywords

Carbohydrate; DNA; Nucleic acid delivery; polysaccharide; Transfection

Indexed keywords

CARBOHYDRATE; NUCLEIC ACID; POLYMER;

ARTICLE; CHEMISTRY; GENETIC TRANSFECTION; GENETICS; HUMAN; METABOLISM; METHODOLOGY;

CARBOHYDRATES; HUMANS; NUCLEIC ACIDS; POLYMERS; TRANSFECTION;

EID: 78649276922     PISSN: 03401022     EISSN: None     Source Type: Book Series    
DOI: 10.1007/128_2010_68     Document Type: Article

References (186)

1. Pickler RH, Munro CL (1995) Gene therapy for inherited disorders. J Pediatr Nurs 10:40-47
2. Anderson WF (1992) Human gene therapy. Science 256:803-813
3. Mulligan RC (1993) The basic science of gene therapy. Science 260:926-932
4. Wiethoff CM, Middaugh CR (2003) Barriers to nonviral gene delivery. J Pharm Sci 92 (2):203-217
5. Braun CS, Vetro JA, Tomalia DA et al (2005) Structure/function relationships of polyami-doamine/DNA dendrimers as gene delivery vehicles. J Pharm Sci 94(2):423-436
6. Behr J-P (1993) Synthetic gene-transfer vectors. Acc Chem Res 26:274-279
7. Luo D, Saltzman WM (2000) Synthetic DNA delivery systems. Nat Biotechnol 18:33-37
8. Davis ME (2002) Non-viral gene delivery systems. Curr Opin Biotechnol 13(2):128-131
9. Kircheis R, Wagner E (2000) Polycation/DNA complex for in vivo gene delivery. Gene Ther Regul 1:95-114
10. Godbey WT, Wu KK, Mikos AG (1999) Poly(ethylenimine) and its role in gene delivery. J Control Release 60(2-3):149-160
11. Godbey WT, Wu KK, Mikos AG (1999) Size matters: molecular weight affects the efficiency of poly(ethylenimine) as a gene delivery vehicle. J Biomed Mater Res B 45 (3):268-275
12. Mannisto M, Vanderkerken S, Toncheva V et al (2002) Structure-activity relationships of poly(L-lysines): effects of pegylation and molecular shape on physicochemical and biological properties in gene delivery. J Control Release 83(1):169-182
13. Kim SW (2007) Polylysine copolymers for gene delivery. Gene Transfer 461-471
14. Kwoh DY, Coffin CC, Lollo CP et al (1999) Stabilization of poly-L-lysine/DNA polyplexes for in vivo gene delivery to the liver. Biochim Biophys Acta 1444(2):171-190
15. Smith DK (2006) Dendritic supermolecules-towards controllable nanomaterials. Chem Commun 1:34-44
16. Eliyahu H, Siani S, Azzam T et al (2006) Relationships between chemical composition, physical properties and transfection efficiency of polysaccharide-spermine conjugates. Biomaterials 27:1646-1655
17. Hong S, Bielinska AU, Mecke A et al (2004) Interaction of poly(amidoamine) dendrimers with supported lipid bilayers and cells: hole formation and the relation to transport. Bio-conjug Chem 15(4):774-782
18. Kraemer M (2004) Dendritic polyamines: simple access to new materials with defined treelike structures for application in nonviral gene delivery. ChemBioChem 5(8):1081
19. Svenson S, Tomalia DA (2005) Dendrimers in biomedical applications - reflections on the field. Adv Drug Deliv Rev 57(15):2106-2129
20. Mintzer MA, Simanek EE (2009) Nonviral vectors for gene delivery. Chem Rev 109:259-303
21. Liu Y, Reineke TM (2005) Hydroxyl stereochemistry and amine number within poly(gly-coamidoamine)s affect intracellular DNA delivery. J Am Chem Soc 127(9):3004-3015
22. Velter I, La Ferla B, Nicotra F (2006) Carbohydrate-based molecular scaffolding. J Carbohydr Chem 25:97-138
23. Varma AJ, Kennedy JF, Galgali P (2004) Synthetic polymers functionalized by carbohydrates: a review. Carbohydr Polym 56:429-445
24. Yudovin-Farber I, Domb AJ (2007) Cationic polysaccharides for gene delivery. Mat Sci Eng 27:595-598
25. Miyata T, Nakamae K (1997) Polymers with pendant saccharides - 'glycopolymers'. Trends Polym Sci 5(6):198-205
26. Ren T, Zhang G, Liu D (2001) Synthesis of galactosyl compounds for targeted gene delivery. Bioorg Med Chem 9(11):2969-2978
27. Lundquist JJ, Toone EJ (2002) The cluster glycoside effect. Chem Rev 102(2):555-578
28. Ladmiral V, Melia E, Haddleton DM (2004) Synthetic glycopolymers: an overview. Eur Polym J 40:431-449
29. Mehvar R (2000) Dextrans for targeted and sustained delivery of therapeutic and imaging agents. J Control Release 69:1-25
30. Sakurai K, Uezu K, Numata M et al (2005) b-1,3-Glucan polysaccharides as novel one-dimensional hosts for DNA/RNA, conjugated polymers and nanoparticles. Chem Commun 35:4383-4398
31. Borchard G (2001) Chitosans for gene delivery. Adv Drug Deliv Rev 52(2):145-150
32. Yun YH, Goetz DJ, Yellen P et al (2004) Hyaluronan microspheres for sustained gene delivery and site-specific targeting. Biomaterials 25(1):147-157
33. Gupta M, Gupta AK (2004) Hydrogel pullulan nanoparticles encapsulating pBUDLacZ plasmid as an efficient gene delivery carrier. J Control Release 99(1):157-166
34. Vaheri A, Pagano JS (1965) Infectious poliovirus RNA: a sensitive method of assay. Virology 27(3):434-436
35. Mack KD, Wei R, Elbagarri A et al (1998) A novel method for DEAE-dextran mediated transfection of adherent primary cultured human macrophages. J Immunol Methods 211:79-86
36. Onishi Y, Eshita Y, Murashita A et al (2005) Synthesis and characterization of 2-diethyl-aminoethyl-dextran-methyl methacrylate graft copolymer for nonviral gene delivery vector. J Appl Polym Sci 98:9-14
37. Azzam T, Eliyahu H, Makocitzki A et al (2003) Dextran-spermine conjugate: an efficient vector for gene delivery. Macromol Symp 195:247-261
38. Azzam T, Raskin A, Makovitzki A et al (2002) Cationic polysaccharides for gene delivery. Macromolecules 35(27):9947-9953
39. Hosseinkhani H, Azzam T, Tabata Y et al (2004) Dextran-spermine polycation: an efficient nonviral vector for in vitro and in vivo gene transfection. Gene Ther 11:194-203
40. Eliyahu H, Joseph A, Schillemans JP et al (2007) Characterization and in vivo performance of dextran-spermine polyplexes and DOTAP/cholesterol lipoplexes administered locally and systemically. Biomaterials 28(14):2339-2349
41. Eliyahu H, Joseph A, Azzam T et al (2006) Dextran-spermine-based polyplexes - evaluation of transgene expression and of local and systemic toxicity in mice. Biomaterials 27 (8):1636-1645
42. Singh A, Suri S, Roy K (2009) In-situ crosslinking hydrogels for combinatorial delivery of chemokines and siRNA-DNA carrying microparticles to dendritic cells. Biomaterials 30 (28):5187-5200
43. Beaudette TT, Cohen JA, Bachelder EM et al (2009) Chemoselective ligation in the functionalization of polysaccharide-based particles. J Am Chem Soc 131(30):10360-10361
44. Bachelder EM, Beaudette TT, Broaders KE et al (2008) Acetal-derivatized dextran: an acid-responsive biodegradable material for therapeutic applications. J Am Chem Soc 130 (32):10494-10495
45. Sakurai K, Iguchi R, Mizu M et al (2003) Polysaccharide-polynucleotide complexes. Part 7. Hydrogen-ion and salt concentration dependence of complexation between schizophyllan and single-stranded homo RNAs. Bioorg Chem 31(3):216-226
46. Nagasaki T, Hojo M, Uno A et al (2004) Long-term expression with a cationic polymer derived from a natural polysaccharide: schizophyllan. Bioconjug Chem 15:249-259
47. Hasegawa T, Fujisawa T, Haraguchi S et al (2005) Schizophyllan-folate conjugate as a new non-cytotoxic and cancer-targeted antisense carrier. Bioorg Med Chem Lett 15(2):327-330
48. Sakurai K, Shinkai S (2000) Molecular recognition of adenine, cytosine, and uracil in a single-strand RNA by a natural polysaccharide: schizophyllan. J Am Chem Soc 122:4520-4521
49. Sakurai K, Mizu M, Shinkai S (2001) Polysaccharide-polynucleotide complexes. 2. Complementary polynucleotide mimic behavior of a natural polysaccharide: schizophyllan in the macromolecular comple with a single strand RNA: poly(C). Biomacromolecules 2:641-650
50. Sletmoen M, Naess SN, Stokke BT (2009) Structure and stability of polynucleotide-(1, 3)-[beta]-D-glucan complexes. Carbohydr Polym 76(3):389-399
51. Hasegawa T, Umeda M, Matsumoto T et al (2004) Lactose-appended schizophyllan is a potential candidate as a hepatocyte-targeted antisense carrier. Chem Commun 4:382-383
52. Mizu M, Koumoto K, Anada T et al (2004) Antisense oligonucleotides bound in the polysaccharide complex and the enhanced antisense effect due to the low hydrolysis. Biomaterials 25:3117-3123
53. Koumoto K, Mizu M, Sakurai K et al (2004) Polysaccharide/polynucleotide complexes. Part 6. Chem Biodivers 1(3):520-529
54. Anada T, Karinaga R, Koumoto K et al (2005) Linear double-stranded DNA that mimics an infective tail of virus genome to enhance transfection. J Control Release 108(2-3):529-539
55. Takeda Y, Shimada N, Kaneko K et al (2007) Ternary complex consisting of DNA, polycation, and a natural polysaccharide of schizophyllan to induce cellular uptake by antigen presenting cells. Biomacromolecules 8(4):1178-1186
56. Hasegawa T, Fujisawa T, Numata M et al (2004) Schizophyllans carrying oligosaccharide appendages as potential candidates for cell-targeted antisense carrier. Org Biomol Chem 2 (21):3091-3098
57. Matsumoto T, Numata M, Anada T et al (2004) Chemically modified polysaccharide schizophyllan for antisense oligonucleotides delivery to enhance the cellular uptake efficiency. Biochem Biophys Acta 1670:91-104
58. Karinaga R, Koumoto K, Mizu M et al (2005) PEG-appended beta-(1->3)-D-glucan schizophyllan to deliver antisense-oligonucleotides with avoiding lysosomal degradation. Biomaterials 26(23):4866-4873
59. Karinaga R, Anada T, Minari J et al (2006) Galactose-PEG dual conjugation of beta-(1->3)-D-glucan schizophyllan for antisense oligonucleotides delivery to enhance the cellular uptake. Biomaterials 27(8):1626-1635
60. Mizu M, Koumoto K, Anada T et al (2004) A polysaccharide carrier for immunostimulatory CpG DNA to enhance cytokine secretion. J Am Chem Soc 126(27):8372-8373
61. Klinman DM, Klaschik S, Sato T et al (2009) CpG oligonucleotides as adjuvants for vaccines targeting infectious diseases. Adv Drug Deliv Rev 61(3):248-255
62. Shimada N, Coban C, Takeda Y et al (2007)Apolysaccharide carriertoeffectivelydeliver native phosphodiester CpG DNA to antigen-presenting cells. Bioconjug Chem 18(4):1280-1286
63. Dias N, Stein CA (2002) Antisense oligonucleotides: basic concepts and mechanisms. Mol Cancer Ther 1(5):347-355
64. Pouyani T, Prestwich GD (1994) Functionalized derivatives of hyaluronic acid oligosacchar-ides: drug carriers and novel biomaterials. Bioconjug Chem 5(4):339-347
65. Kim A, Checkla DM, Dehazya P et al (2003) Characterization of DNA-hyaluronan matrix for sustained gene transfer. J Control Release 90:81-95
66. Kim AP, Yellen P, Yun YH et al (2005) Delivery of a vector encoding mouse hyaluronan synthase 2 via a crosslinked hyaluronan film. Biomaterials 26:1585-1593
67. Yun YH, Chen W (2005) Microspheres formulated from native hyaluronan for applications in gene therapy. In: Mansoor MA (ed) Polymeric gene delivery: principles and applications, CRC Press LLC, USA, pp 475-486
68. de la Fuente M, Seijo B, Alonso MJ (2008) Bioadhesive hyaluronan-chitosan nanoparticles can transport genes across the ocular mucosa and transfect ocular tissue. Gene Ther 15 (9):668-676
69. de la Fuente M, Seijo B, Alonso MJ (2008) Design of novel polysaccharidic nanostructures for gene delivery. Nanotechnology 19(7):075105/1-075105/9
70. Duceppe N, Tabrizian M (2009) Factors influencing the transfection efficiency of ultra low molecular weight chitosan/hyaluronic acid nanoparticles. Biomaterials 30(13):2625-2631
71. Wieland JA, Houchin-Ray TL, Shea LD (2007) Non-viral vector delivery from poly(ethyl-ene glycol)-hyaluronic acid hydrogels. J Control Release 120(3):233-241
72. Saraf A, Hacker MC, Sitharaman B et al (2008) Synthesis and conformational evaluation of a novel gene delivery vector for human mesenchymal stem cells. Biomacromolecules 9 (3):818-827
73. Shen Y, Li Q, Tu J et al (2009) Synthesis and characterization of low molecular weight hyaluronic acid-based cationic micelles for efficient siRNA delivery. Carbohydr Polym 77 (1):95-104
74. Bruneel D, Schacht E (1995) End group modification of pullulan. Polymer 36:169-172
75. Kaneo Y, Tanaka T, Nakano T et al (2001) Evidence for receptor-mediated hepatic uptake of pullulan in rats. J Control Release 70:365-373
76. Nabi IR, Le PU (2003) Caveolae/raft-dependent endocytosis. J Cell Biol 161(4):673-677
77. Na K, Lee ES, Bae YH (2003) Self-assembled nanoparticles of hydrophobically-modified polysaccharide bearing vitamin H as a targeted anti-cancer drug delivery system. Eur J Pharm Biopharm 18(2):165-173
78. Akiyoshi K, Kobayashi S, Shichibe S et al (1998) Self-assembled hydrogel nanoparticle of cholesterol-bearing pullulan as a carrier of protein drugs: complexation and stabilization of insulin. J Control Release 54(3):313-320
79. Hosseinkhani H, Aoyama T, Ogawa O et al (2002) Liver targeting of plasmid DNA by pullulan conjugation based on metal coordination. J Control Release 83(2):287-302
80. Kanatani I, Ikai T, Okazaki A et al (2006) Efficient gene transfer by pullulan-spermine occurs through both clathrin- and raft/caveolae-dependent mechanisms. J Control Release 116(1):75-82
81. Jo J, Ikai T, Okazaki A, Nagane K, Yamamoto M, Hirano Y, Tabata Y (2007) Expression profile of plasmid DNA obtained using spermine derivatives of pullulan with different molecular weights. J Biomater Sci Polym Ed 18(7):883-899
82. San Juan A, Hlawaty H, Chaubet F et al (2007) Cationized pullulan 3D matrices as new materials for gene transfer. J Biomed Mater Res A 82A(2):354-362
83. Imai T, Shiraishi S, Saito H et al (1991) Interaction of indomethacin with low molecular weight chitosan, and improvements of some pharmacuetical properties of indomethacin by low molecular weight chitosans. Int J Pharm 67:11-20
84. Takayama K, Hirata M, Machida Y et al (1990) Effect of interpolymer complex formation on bioadhesive property and drug release phenomenon of compressed tablet consisting of chitosan and sodium hyaluronate. Chem Pharm Bull 38:1993-1997
85. Meshali MM, Gabr KE (1993) Effect of interpolymer complex formation of chitosan with pectin or acacia on the release behavior of chlorpromazine HCl. Int J Pharm 89:177-181
86. Mao H-Q (2001) Chitosan-DNA nanoparticles as gene carriers: synthesis, characterization and transfection efficiency. J Control Release 70(3):399-421
87. Mumper R, Wang JJ, Claspell JM et al (1995) Novel polymeric condensing carriers for gene delivery. Proc Intl Sym Control Release Bioact Mater 22:178-179
88. MacLaughlin FC, Mumper RJ, Wang J et al (1998) Chitosan and depolymerized chitosan oligomers as condensing carriers for in vivo plasmid delivery. J Control Release 56:259-272
89. Dodane V, Vilivalam VD (1998) Pharmaceutical applications of chitosan. Pharm Sci Technol Today 1(6):246-253
90. Aranaz I, Mengibar M, Harris R et al (2009) Functional characterization of chitin and chitosan. Curr Chem Biol 3:203-230
91. Aiba S-i (1991) Studies on chitosan: 3. Evidence for the presence of random and block copolymer structures in partially N-acetylated chitosans. Int J Biol Macromol 13(1):40-44
92. Mima S, Miya M, Iwamoto R et al (1983) Highly deacetylated chitosan and its properties. J Appl Polym Sci 28(6):1909-1917
93. Nguyen S, Hisiger S, Jolicoeur M et al (2009) Fractionation and characterization of chitosan by analytical SEC and 1H NMR after semi-preparative SEC. Carbohydr Polym 75(4):636-645
94. Köping-Höggard M, Tubulekas I, Guan H, Edwards K, Nilsson M, Varum KM, Artursson P (2001) Chitosan as a nonviral gene delivery system. Structure-property relationships and characteristics compared with polyethylenimine in vitro and after lung administration in vivo. Gene Ther 8:1108-1121
95. Kiang T, Wen J, Lim HW et al (2004) The effect of the degree of chitosan deacetylation on the efficiency of gene transfection. Biomaterials 25:5293-5301
96. Huang M, Fong C-W, Khor E et al (2005) Transfection efficiency of chitosan vectors: effect of polymer molecular weight and degree of deacetylation. J Control Release 106(3):391-406
97. Liu X, Howard KA, Dong M et al (2007) The influence of polymeric properties on chitosan/ siRNA nanoparticle formulation and gene silencing. Biomaterials 28(6):1280-1288
98. Rejman J, Oberle V, Zuhorn IS et al (2004) Size-dependent internalization of particles via the pathways of clathrin- and caveolae-mediated endocytosis. Biochem J 377(1):159-169
99. Choksakulnimitr S, Masuda S, Tokuda H et al (1995) In vitro cytotoxicity of macromole-cules in different cell culture systems. J Control Release 34(3):233-241
100. Erbacher P, Zou S, Bettinger T et al (1998) Chitosan-based vector/DNA complexes for gene delivery: biophysical characteristics and transfection ability. Pharm Res 15(9):1332-1339
101. Richardson SCW, Kolbe HVJ, Duncan R (1999) Potential of low molecular mass chitosan as a DNA delivery system: biocompatibility, body distribution and ability to complex and protect DNA. Int J Pharm 178(2):231-243
102. Murata J, Ohya Y, Ouchi T (1996) Possibility of application of quaternary chitosan having pendant galactose residues as gene delivery tool. Carbohydr Polym 29(1):69-74
103. Murata J, Ohya Y, Ouchi T (1997) Design of quaternary chitosan conjugate having antennary galactose residues as a gene delivery tool. Carbohydr Polym 32(2):105-109
104. Thanou M, Florea BI, Geldof M et al (2002) Quaternized chitosan oligomers as novel gene delivery vectors in epithelial cell lines. Biomaterials 23:153-159
105. Kean T, Roth S, Thanou M (2005) Trimethylated chitosans as non-viral gene delivery vectors: cytotoxicity and transfection efficiency. J Control Release 103:643-653
106. Kim TH, Kim SI, Akaike T et al (2005) Synergistic effect of poly(ethylenimine) on the transfection efficiency of galactosylated chitosan/DNA complexes. J Control Release 105(3):354-366
107. Wong K, Sun G, Zhang X et al (2006) PEI-g-chitosan, a novel gene delivery system with transfection efficiency comparable to polyethylenimine in vitro and after liver administration in vivo. Bioconjug Chem 17:152-158
108. Jiang H-L, Kim Y-K, Arote R et al (2007) Chitosan-graft-polyethylenimine as a gene carrier. J Control Release 117:273-280
109. Kim T-H, Jiang H-L, Jere D et al (2007) Chemical modification of chitosan as a gene carrier in vitro and in vivo. Prog Polym Sci 32(7):726-753
110. Nicolet BH, Shinn LA (1939) The action of periodic acid on a-amino alcohols. J Am Chem Soc 61(6):1615
111. Vold IMN, Christensen BE (2005) Periodate oxidation of chitosans with different chemical compositions. Carbohydr Res 340(4):679-684
112. Jiang H, Kwon J, Kim Y et al (2007) Galactosylated chitosan-graft- polyethylenimine as a gene carrier for hepatocyte targeting. Gene Ther 14(19):1389-1398
113. Jiang H-L, Kwon J-T, Kim E-M et al (2008) Galactosylated poly(ethylene glycol)-chitosan-graft-polyethylenimine as a gene carrier for hepatocyte-targeting. J Control Release 131 (2):150-157
114. Lu B, Xu X-D, Zhang X-Z et al (2008) Low molecular weight polyethylenimine grafted N-maleated chitosan for gene delivery: properties and in vitro transfection studies. Bioma-cromolecules 9(10):2594-2600
115. Lou Y-L, Peng Y-S, Chen B-H et al (2009) Poly(ethylene imine)-g-chitosan using EX-810 as a spacer for nonviral gene delivery vectors. J Biomed Mater Res A 88A(4):1058-1068
116. Wu Y, Liu C, Zhao X et al (2008) A new biodegradable polymer: PEGylated chitosan-g-PEI possessing a hydroxyl group at the PEG end. J Polym Res 15(3):181-185
117. Jiang H-L, Kim Y-K, Arote R et al (2009) Mannosylated chitosan-graft-polyethylenimine as a gene carrier for Raw 264.7 cell targeting. Int J Pharm 375(1-2):133-139
118. Jiang H-L, Xu C-X, Kim Y-K et al (2009) The suppression of lung tumorigenesis by aerosol-delivered folate-chitosan-graft-polyethylenimine/Akt1 shRNA complexes through the Akt signaling pathway. Biomaterials 30(29):5844-5852
119. Jere D, Jiang H-L, Kim Y-K et al (2009) Chitosan-graft-polyethylenimine for Akt1 siRNA delivery to lung cancer cells. Int J Pharm 378(1-2):194-200
120. Pack DW, Putnam D, Langer R (2000) Design of imidazole-containing endosomolytic biopolymers for gene delivery. Biotechnol Bioeng 67(2):217-223
121. Muzzarelli RAA, Mattioli-Belmonte M, Tietz C et al (1994) Stimulatory effect on bone formation exerted by a modified chitosan. Biomaterials 15(13):1075-1081
122. Midoux P, Monsigny M (1999) Efficient gene transfer by histidylated polylysine/pDNA complexes. Bioconjug Chem 10(3):406-411
123. Kim TH, Ihm JE, Choi YJ et al (2003) Efficient gene delivery by urocanic acid-modified chitosan. J Control Release 93(3):389-402
124. Jin H, Kim T, Hwang S et al (2006) Aerosol delivery of urocanic acid-modified chitosan/ programmed cell death 4 complex regulated apoptosis, cell cycle, and angiogenesis in lungs of K-ras null mice. Mol Cancer Ther 5(4):1041
125. Jin H, Xu CX, Kim HW et al (2008) Urocanic acid-modified chitosan-mediated PTEN delivery via aerosol suppressed lung tumorigenesis in K-rasLA1 mice. Cancer Gene Ther 15(5):275-283
126. Bauhuber S, Hozsa C, Breunig M et al (2009) Delivery of nucleic acids via disulfide-based carrier systems. Adv Mater 21(32-33):3286-3306
127. Bernkop-Schnürch A, Hornof M, Guggi D (2004) Thiolated chitosans. Eur J Pharm Biopharm 57(1):9-17
128. Schmitz T, Bravo-Osuna I, Vauthier C et al (2007) Development and in vitro evaluation of a thiomer-based nanoparticulate gene delivery system. Biomaterials 28(3):524-531
129. Martien R, Loretz B, Thaler M et al (2007) Chitosan-thioglycolic acid conjugate: an alternative carrier for oral nonviral gene delivery? J Biomed Mater Res A 82A(1):1-9
130. Park IK, Ihm JE, Park YH et al (2003) Galactosylated chitosan (GC)-graft-poly(vinyl pyrrolidone) (PVP) as hepatocyte-targeting DNA carrier. Preparation and physicochemical characterization of GC-graft-PVP/DNA complex (1). J Control Release 86:349-359
131. Park YK, Park YH, Shin BA et al (2000) Galactosylated chitosan-graft-dextran as hepato-cyte-targeting DNA carrier. J Control Release 69(1):97-108
132. Park IK, Kim TH, Park YH et al (2001) Galactosylated chitosan-graft- poly(ethylene glycol) as hepatocyte-targeting DNA carrier. J Control Release 76:349-362
133. Park IK, Ihm JE, Park YH et al (2003) Galactosylated chitosan (GC)-graft-poly(vinyl pyrro-lidone) (PVP) as hepatocyte-targeting DNA carrier: In vivo transfection. Arch Pharm Res 27 (12):1284-1289
134. Hashimoto M, Morimoto M, Saimoto H et al (2006) Lactosylated chitosan for DNA delivery into hepatocytes: the effect of lactosylation on the physicochemical properties and intracel-lular trafficking of pDNA/chitosan complexes. Bioconjug Chem 17(2):309-316
135. Hashimoto M, Morimoto M, Saimoto H et al (2006) Gene transfer by DNA/mannosylated chitosan complexes into mouse peritoneal macrophages. Biotechnol Lett 28(11):815-821
136. Forrest ML, Gabrielson N, Pack DW (2005) Cyclodextrin-polyethylenimine conjugates for targeted in vitro gene delivery. Biotechnol Bioeng 89(4):416-423
137. Liu Y, Wenning L, Lynch M et al (2004) New poly(D-glucaramidoamine)s induce DNA nanoparticle formation and efficient gene delivery into mammalian cells. J Am Chem Soc 126(24):7422-7423
138. Reineke TM, Davis ME (2003) Structural effects of carbohydrate-containing polycations on gene delivery. 1. Carbohydrate size and its distance from charge centers. Bioconjug Chem 14 (1):247-254
139. Reineke TM, Davis ME (2003) Structural effects of carbohydrate-containing polycations on gene delivery. 2. Charge center type. Bioconjug Chem 14(1):255-261
140. Srinivasachari S, Liu Y, Zhang G et al (2006) Trehalose click polymers inhibit nanoparticle aggregation and promote pDNA delivery in serum. J Am Chem Soc 128(25):8176-8184
141. Hwang SJ, Bellocq NC, Davis ME (2001) Effects of structure of beta-cyclodextrin-contain-ing polymers on gene delivery. Bioconjug Chem 12(2):280-290
142. Boussif O, Lezoualc'h F, Zanta MA, Mergny MD, Scherman D, Demeneix B, Behr JP (1995) A versatile vector for gene and oligonuceotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci USA 92:7297-7301
143. Grosse S, Aron Y, Honore I, Thevenot G, Danel C, Roche A-C, Monsigny M, Fajac I (2004) Lactosylated polyethylenimine for gene transfer into airway epithelial cells: role of the sugar moiety in cell delivery and intracellular trafficking of the complexes. J Gene Med 2004 (6):345-356
144. Liu Y, Wenning L, Lynch M et al (2006) Gene delivery with novel poly(L-tartaramidoamine) s. In: Svenson S (ed) Polymeric drug delivery, volume I: particulate drug carriers. American Chemical Society, Washington DC, pp 217-227
145. Metzke M, O'Connor N, Maiti S et al (2005) Saccharide-peptide hybrid copolymers as biomaterials. Angew Chem Int Ed 44:6529-6533
146. Liu Y, Reineke TM (2006) Poly(glycoamidoamine)s for gene delivery: stability of polyplexes and efficacy with cardiomyoblast cells. Bioconjug Chem 17(1):101-108
147. Prevette LE, Kodger TE, Reineke TM et al (2007) Deciphering the role of hydrogen bonding in enhancing pDNA-polycation interactions. Langmuir 23(19):9773-9784
148. Lee C-C, Liu Y, Reineke TM (2008) General structure-activity relationship for poly(gly-coamidoamine)s: the effect of amine density on cytotoxicity and DNA delivery efficiency. Bioconjug Chem 19(2):428-440
149. Liu Y, Reineke TM (2007) Poly(glycoamidoamine)s for gene delivery. structural effects on cellular internalization, buffering capacity, and gene expression. Bioconjug Chem 18(1):19-30
150. Liu Y, Reineke TM (2010) Degradation of poly(glycoamidoamine) DNA delivery vehicles: polyamide hydrolysis at physiological conditions promotes DNA release. Biomacromole-cules 11(2):316-325
151. Taori VP, Liu Y, Reineke TM (2009) DNA delivery in vitro via surface release from multilayer assemblies with poly(glycoamidoamine)s. Acta Biomater 5(3):925-933
152. Paiva C, Panek A (1996) Biotechnological applications of the disaccharide trehalose. Biotechnol Annu Rev 2:293
153. Lins RD, Pereira CS, Hünenberger PH (2004) Trehalose-protein interaction in aqueous solution. Proteins 55(1):177-186
154. Wagner E (2004) Pharm Res 21:8-14
155. Srinivasachari S, Liu Y, Prevette LE et al (2007) Effects of trehalose click polymer length on pDNA complex stability and delivery efficacy. Biomaterials 28:2885-2898
156. Prevette LE, Lynch ML, Kizjakina K et al (2008) Correlation of amine number and pDNA binding mechanism for trehalose-based polycations. Langmuir 24(15):8090-8101
157. Ortiz-Mellet C, Benito JM, Garcia Fernandez JM et al (1998) Cyclodextrin-scaffolded glyco-clusters. Chem Eur J 4(12):2523-2531
158. Perez-Balderas F, Ortega-Munoz M, Morales-Sanfrutos J et al (2003) Multivalent neogly-coconjugates by regiospecific cycloaddition of alkynes and azides using organic-soluble copper catalysts. Org Lett 5(11):1951-1954
159. Garcia-Lopez JJ, Hernandez-Mateo F, Isac-Garcia J et al (1999) Synthesis of per-glycosy-lated beta-cyclodextrins having enhanced lectin binding affinity. J Org Chem 64(2):522-531
160. Gadella A, Defaye J (1991) Selective halogenation at primary postions of cyclomaltooligo-saccharides and a synthesis of per-3,6-anhydro cyclomaltooligosaccharides. Angew Chem Int Ed 30:78-80
161. Andre S, Kaltner H, Furuike T et al (2004) Persubstituted cyclodextrin-based glycoclusters as inhibitors of protein-carbohydrate recognition using purified plant and mammalian lectins and wild-type and lectin-gene-transfected tumor cells as targets. Bioconjug Chem 15(1):87-98
162. Bellocq NC, Pun SH, Jensen GS et al (2003) Transferrin-containing, cyclodextrin polymer-based particles for tumor-targeted gene delivery. Bioconjug Chem 14(6):1122-1132
163. Davis ME, Bellocq NC (2001) Cyclodextrin-containing polymers for gene delivery. J Incl Phenom Macro Chem 44:17-22
164. Croyle MA, Roessler BJ, Hsu C-P et al (1998) Beta-cyclodextrins enhance adenoviral-mediated gene delivery to the intestine. Pharm Res 15(9):1349-1355
165. Boger J, Corcoran RJ, Lehn J-M (1978) Cyclodextrin chemistry. Selective modification of all primary hydroxyl groups of alpha- and beta-cyclodextrins. Helv Chim Acta 61:2190-2218
166. Mocanu G, Vizitiu D, Carpov A (2001) Cyclodextrin polymers. J Bioact Compat Polym 16:315-328
167. Zhao Q, Temsamani J, Agrawal S (1995) Use of cyclodextrin and its derivatives as carriers for oligonucleotide delivery. Antisense Res Dev 5(3):185-192
168. Salem LB, Bosquillon C, Dailey LA et al (2009) Sparing methylation of [beta]-cyclodextrin mitigates cytotoxicity and permeability induction in respiratory epithelial cell layers in vitro. J Control Release 136(2):110-116
169. Yang C, Li H, Goh SH et al (2007) Cationic star polymers consisting of [alpha]-cyclodextrin core and oligoethylenimine arms as nonviral gene delivery vectors. Biomaterials 28 (21):3245-3254
170. Cryan S-A (2004) Cell transfection with polycationic cyclodextrin vectors. Eur J Pharm Sci 21(5):625-633
171. Gonzalez H, Hwang SJ, Davis ME (1999) New class of polymers for the delivery of macromolecular therapeutics. Bioconjug Chem 10(6):1068-1074
172. Srinivasachari S, Fichter KM, Reineke TM (2008) Polycationic b-cyclodextrin "click clusters": monodisperse and versatile scaffolds for nucleic acid delivery. J Am Chem Soc 130:4618-4627
173. Srinivasachari S, Reineke TM (2009) Versatile supramolecular pDNA vehicles via "click polymerization" of [beta]-cyclodextrin with oligoethyleneamines. Biomaterials 30 (5):928-938
174. Hu-Lieskovan S, Heidel JD, Bartlett DW, Davis ME, Triche TJ (2005) Sequence-specific knockdown of EWS-EFI1 by targeted, nonviral delivery of small interfering RNA inhibits tumor growth in a murine model of metastatic Ewing's sarcoma. Cancer Res 65:8984-8992
175. Pun SH, Davis ME (2002) Development of a nonviral gene delivery vehicle for systemic application. Bioconjug Chem 13:630-639
176. Pun SH, Tack F, Bellocq NC, Cheng J, Grubbs BH, Jensen GS, Davis ME, Brewster M, Janicot M, Janssens B, Floren W, Bakker A (2004) Targeted delivery of RNA-cleaving DNA enzyme (DNAzyme) to tumor tissue by transferrin-modifed, cyclodextrin-based particles. Cancer Biol Ther 3(7):641-650
177. Heidel JD, Yu ZP, Liu JYC et al (2007) Administration in non-human primates of escalating intravenous doses of targeted nanoparticles containing ribonucleotide reductase subunit M2 siRNA. Proc Natl Acad Sci USA 104(14):5715-5721
178. Davis ME (2009) The first targeted delivery of siRNA in humans via a self-assembling, cyclodextrin polymer-based nanoparticle: from concept to clinic. Mol Pharm 6(3):659-668
179. Zanta M-A, Boussif O, Adib A, Behr J-P (1997) In vitro gene delivery to hepatocytes with galactosylated polyethylenimine. Bioconjug Chem 8:839-844
180. Kunath K, von Harpe A, Fischer D et al (2003) Galactose-PEI-DNA complexes for targeted gene delivery: degree of substitution affects complex size and transfection efficiency. J Control Rel 88(1):159-172
181. Nishikawa M, Yamauchi M, Morimoto K et al (2000) Hepatocyte-targeted in vivo gene expression by intravenous injection of plasmid DNA complexed with synthetic multifunctional gene delivery system. Gene Ther 7(7):548-555
182. Chen C-p, Kim J-s, Liu D et al (2007) Synthetic PEGylated glycoproteins and their utility in gene delivery. Bioconjug Chem 18(2):371-378
183. Rozema DB, Lewis DL, Wakefield DH et al (2007) Dynamic polyconjugates for targeted in vivo delivery of siRNA to hepatocytes. Proc Natl Acad Sci USA 104(32):12982-12987
184. Weiss SI, Sieverling N, Niclasen M et al (2006) Uronic acids functionalized polyethylenei-mine (PEI)-polyethyleneglycol (PEG)-graft- copolymers as novel synthetic gene carriers. Biomaterials 27(10):2302-2312
185. Diebold SS, Kursa M, Wagner E, Cotten M, Zenke M (1999) Mannose polyethylenimine conjugates for targeted DNA delivery into dendritic cells. J Biol Chem 274:19087-19094
186. Reineke TM (2006) Poly(glycoamidoamine)s: cationic glycopolymers for DNA delivery. J Polym Sci A Polym Chem 44(24):6895-6908