RNA-interference effectors and their delivery

Critical Reviews in Therapeutic Drug Carrier Systems

Volumn 23, Issue 2, 2006, Pages 137-164

  Doody, A ^a   Putnam, D ^a  

^a Cornell University   (United States)

Author keywords

Delivery; Polycations; Polymers; RNA interference; RNAi; siRNA; Small interfering RNA; Transfection

Indexed keywords

CYCLODEXTRIN DERIVATIVE; DNA VACCINE; MACROGOL; MESSENGER RNA; PLASMID DNA; POLYMER; PROTAMINE; RETROVIRUS VECTOR; SMALL INTERFERING RNA;

CYTOSOL; DRUG DELIVERY SYSTEM; HYDROLYSIS; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NUCLEOTIDE SEQUENCE; PROTEIN EXPRESSION; REVIEW; RNA INTERFERENCE; SEQUENCE HOMOLOGY;

EID: 33748747613     PISSN: 07434863     EISSN: None     Source Type: Journal    
DOI: 10.1615/critrevtherdrugcarriersyst.v23.i2.30     Document Type: Review

References (141)

1. Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature. 1998;391: 806-811.
2. Guo S, Kemphues KJ. par-1, a gene required for establishing polarity in C. elegans embryos, encodes a putative Ser/Thr kinase that is asymmetrically distributed. Cell. 1995;81:611-620.
3. Hammond SM, Bernstein E, Beach D, Hannon GJ. An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells. Nature. 2000;404:293-296.
4. Ingelbrecht I, Van Houdt H, Van Montagu M, Depicker A. Posttranscriptional silencing of reporter transgenes in tobacco correlates with DNA methylation. Proc Natl Acad Sci U S A. 1994;91:10502-10506.
5. Bernstein E, Caudy AA, Hammond SM, Hannon GJ. Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature. 2001;409:363-366.
6. Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature. 2001;411:494-498.
7. Elbashir SM, Lendeckel W, Tuschl T. RNA interference is mediated by 21- and 22-nucleotide RNAs. Genes Dev. 2001;15:188-200.
8. Tuschl T, Zamore PD, Lehmann R, Bartel DP, Sharp PA. Targeted mRNA degradation by double-stranded RNA in vitro. Genes Dev. 1999;13:3191-3197.
9. Zamore PD, Tuschl T, Sharp PA, Bartel DP. RNAi: double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell. 2000;101:25-33.
10. Ma JB, Ye K, Patel DJ. Structural basis for overhang-specific small interfering RNA recognition by the PAZ domain. Nature. 2004;429:318-322.
11. Song JJ, Smith SK, Hannon GJ, Joshua-Tor L. Crystal structure of Argonaute and its implications for RISC slicer activity. Science. 2004;305:1434-1437.
12. Nykanen A, Haley B, Zamore PD. ATP requirements and small interfering RNA structure in the RNA interference pathway. Cell. 2001;107:309-321.
13. Schwarz DS, Hutvagner G, Haley B, Zamore PD. Evidence that siRNAs function as guides, not primers, in the Drosophila and human RNAi pathways. Mol Cell. 2002;10:537-548.
14. Martinez J, Patkaniowska A, Urlaub H, Luhrmann R, Tuschl T. Single-stranded antisense siRNAs guide target RNA cleavage in RNAi. Cell. 2002;110:563-574.
15. Baulcombe DC. Fast forward genetics based on virus-induced gene silencing. Curr Opin Plant Biol. 1999;2:109-113.
16. Cogoni C, Irelan JT, Schumacher M, Schmidhauser TJ, Selker EU, Macino G. Transgene silencing of the al-1 gene in vegetative cells of Neurospora is mediated by a cytoplasmic effector and does not depend on DNA-DNA interactions or DNA methylation. Embo J. 1996;15:3153-3163.
17. Cogoni C, Macino G. Posttranscriptional gene silencing in Neurospora by a RecQ DNA helicase. Science. 1999;286:2342-2344.
18. Cogoni C, Macino G. Gene silencing in Neurospora crassa requires a protein homologous to RNA-dependent RNA polymerase. Nature. 1999;399:166-169.
19. Kennerdell JR, Carthew RW. Use of dsRNA-mediated genetic interference to demonstrate that frizzled and frizzled 2 act in the wingless pathway. Cell. 1998;95:1017-1026.
20. Lohmann JU, Endl I, Bosch TC. Silencing of developmental genes in Hydra. Dev Biol. 1999;214:211-214.
21. Misquitta L, Paterson BM. Targeted disruption of gene function in Drosophila by RNA interference (RNA-i): a role for nautilus in embryonic somatic muscle formation. Proc Natl Acad Sci U S A. 1999;96:1451-1456.
22. Ngo H, Tschudi C, Gull K, Ullu E. Double-stranded RNA induces mRNA degradation in Trypanosoma brucei. Proc Natl Acad Sci U S A. 1998;95:14687-14692.
23. Sanchez Alvarado A, Newmark PA. Double-stranded RNA specifically disrupts gene expression during planarian regeneration. Proc Natl Acad Sci U S A. 1999;96:5049-5054.
24. Vaucheret H, Beclin C, Elmayan T, Feuerbach F, Godon C, Morel JB, Mourrain P, Palauqui JC, Vernhettes S. Transgene-induced gene silencing in plants. Plant J. 1998;16:651-659.
25. Wargelius A, Ellingsen S, Fjose A. Double-stranded RNA induces specific developmental defects in zebrafish embryos. Biochem Biophys Res Commun. 1999;263:156-161.
26. Waterhouse PM, Graham MW, Wang MB. Virus resistance and gene silencing in plants can be induced by simultaneous expression of sense and antisense RNA. Proc Natl Acad Sci U S A. 1998;95:13959-13964.
27. Waterhouse PM, Smith NA, Wang MB. Virus resistance and gene silencing: killing the messenger. Trends Plant Sci. 1999;4:452-457.
28. Wianny F, Zernicka-Goetz M. Specific interference with gene function by double-stranded RNA in early mouse development. Nat Cell Biol. 2000;2:70-75.
29. Minks MA, West DK, Benvin S, Baglioni C. Structural requirements of double-stranded RNA for the activation of 2',5'-oligo(A) polymerase and protein kinase of interferon-treated HeLa cells. J Biol Chem. 1979;254:10180-10183.
30. Pebernard S, Iggo RD. Determinants of interferon-stimulated gene induction by RNAi vectors. Differentiation. 2004;72:103-111.
31. Stark GR, Kerr IM, Williams BR, Silverman RH, Schreiber RD. How cells respond to interferons. Annu Rev Biochem. 1998;67:227-264.
32. Provost P, Dishart D, Doucet J, Frendewey D, Samuelsson B, Radmark O. Ribonuclease activity and RNA binding of recombinant human Dicer. Embo J. 2002;21:5864-5874.
33. Svoboda P, Stein P, Schultz RM. RNAi in mouse oocytes and preimplantation embryos: effectiveness of hairpin dsRNA. Biochem Biophys Res Commun. 2001;287:1099-1104.
34. Sandy P, Ventura A, Jacks T. Mammalian RNAi: a practical guide. Biotechniques. 2005;39:215-224.
35. Brummelkamp TR, Bernards R, Agami R. A system for stable expression of short interfering RNAs in mammalian cells. Science. 2002;296:550-553.
36. Lee NS, Dohjima T, Bauer G, Li H, Li MJ, Ehsani A, Salvaterra P, Rossi J. Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat Biotechnol. 2002;20:500-505.
37. Miyagishi M, Taira K. Strategies for generation of an siRNA expression library directed against the human genome. Oligonucleotides. 2003;13:325-333.
38. Patil SD, Rhodes DG, Burgess DJ. DNA-based therapeutics and DNA delivery systems: a comprehensive review. AAPS J. 2005;7:E61-E77.
39. Pouton CW, Seymour LW. Key issues in non-viral gene delivery. Adv Drug Del Rev. 2001;46:187-203.
40. Luo D and Saltzman WM. Synthetic DNA delivery systems. Nat Biotech. 2000;18:33-37.
41. Zhang S, Xu Y, Qiao W, Liu D, Li Z. Cationic compounds used in lipoplexes and polyplexes for gene delivery. J Control Release. 2004;100:165-180.
42. Merdan T, Kopecek J, Kissel T. Prospects for cationic polymers in gene and oligonucleotide therapy against cancer. Adv Drug Deliv Rev. 2002;54:715-758.
43. Amarzguioui M, Holen T, Babaie E, Prydz H. Tolerance for mutations and chemical modifications in a siRNA. Nucleic Acids Res. 2003;31:589-595.
44. Braasch DA, Jensen S, Liu Y, Kaur K, Arar K, White MA, Corey DR. RNA interference in mammalian cells by chemically-modified RNA. Biochemistry. 2003;42:7967-7975.
45. Chiu YL, Rana TM. siRNA function in RNAi: a chemical modification analysis. Rna. 2003;9:1034-1048.
46. Czauderna F, Fechtner M, Dames S, Aygun H, Klippel A, Pronk GJ, Giese K, Kaufmann J. Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells. Nucleic Acids Res. 2003;31:2705-2716.
47. Harborth J, Elbashir SM, Vandenburgh K, Manninga H, Scaringe SA, Weber K, Tuschl T. Sequence, chemical, and structural variation of small interfering RNAs and short hairpin RNAs and the effect on mammalian gene silencing. Antisense Nucleic Acid Drug Dev. 2003;13:83-105.
48. Layzer JM, McCaffrey AP, Tanner AK, Huang Z, Kay MA, Sullenger BA. In vivo activity of nuclease-resistant siRNAs. Rna. 2004;10:766-771.
49. Soutschek J, Akinc A, Bramlage B, Charisse K, Constien R, Donoghue M, Elbashir S, Geick A, Hadwiger P, Harborth J, John M, Kesavan V, Lavine G, Pandey RK, Racie T, Rajeev KG, Röhl I, Toudjarska I, Wang G, Wuschko S, Bumcrot D, Koteliansky V, Limmer S, Manoharan M, Vornlocher HP. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature. 2004;432:173-178.
50. Hoshika S, Minakawa N, Matsuda A. Synthesis and physical and physiological properties of 4'-thioRNA: application to post-modification of RNA aptamer toward NF-kappaB. Nucleic Acids Res. 2004;32:3815-3825.
51. Hall AH, Wan J, Shaughnessy EE, Ramsay Shaw B, Alexander KA. RNA interference using boranophosphate siRNAs: structure-activity relationships. Nucleic Acids Res. 2004;32:5991-6000.
52. Lorenz C, Hadwiger P, John M, Vornlocher HP, Unverzagt C. Steroid and lipid conjugates of siRNAs to enhance cellular uptake and gene silencing in liver cells. Bioorg Med Chem Lett. 2004;14:4975-4977.
53. Sui G, Soohoo C, Affar el B, Gay F, Shi Y, Forrester WC, Shi Y. A DNA vector-based RNAi technology to suppress gene expression in mammalian cells. Proc Natl Acad Sci U S A. 2002;99:5515-5520.
54. Xia H, Mao Q, Eliason SL, Harper SQ, Martins IH, Orr HT, et al. RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nat Med. 2004;10:816-820.
55. Dykxhoorn DM, Novina CD, Sharp PA. Killing the messenger: short RNAs that silence gene expression. Nat Rev Mol Cell Biol. 2003;4:457-467.
56. Kafri T, Blomer U, Peterson DA, Gage FH, Verma IM. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet. 1997;17:314-317.
57. Li MJ, Bauer G, Michienzi A, Yee JK, Lee NS, Kim J, Li S, Castanotto D, Zaia J, Rossi JJ. Inhibition of HIV-1 infection by lentiviral vectors expressing Pol III-promoted anti-HIV RNAs. Mol Ther. 2003;8:196-206.
58. Naldini L, Blomer U, Gallay P, Ory D, Mulligan R, Gage FH, Verma IM, Trono D. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science. 1996;272:263-267.
59. Qin XF, An DS, Chen IS, Baltimore D. Inhibiting HIV-1 infection in human T cells by lentiviral-mediated delivery of small interfering RNA against CCR5. Proc Natl Acad Sci U S A. 2003;100:183-188.
60. Scherr M, Battmer K, Ganser A, Eder M. Modulation of gene expression by lentiviral-mediated delivery of small interfering RNA. Cell Cycle. 2003;2:251-257.
61. Uchida N, Sutton RE, Friera AM, He D, Reitsma MJ, Chang WC, Veres G, Scollay R, Weissman IL. HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells. Proc Natl Acad Sci U S A. 1998;95:11939-11944.
62. Svoboda J, Hejnar J, Geryk J, Elleder D, Vernerova Z. Retroviruses in foreign species and the problem of provirus silencing. Gene. 2000;261:181-188.
63. Marshall E. Gene therapy. Second child in French trial is found to have leukemia. Science. 2003;299:320.
64. Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet. 2003;4:346-358.
65. Niidome T, Huang L. Gene therapy progress and prospects: nonviral vectors. Gene Ther. 2002;9:1647-1652.
66. Scherphof G, Van Leeuwen B, Wilschut J, Damen J. Exchange of phosphatidylcholine between small unilamellar liposomes and human plasma high-density lipoprotein involves exclusively the phospholipid in the outer monolayer of the liposomal membrane. Biochim Biophys Acta. 1983;732:595-599.
67. Hsu MJ, Juliano RL. Interactions of liposomes with the reticuloendothelial system. II: Nonspecific and receptor-mediated uptake of liposomes by mouse peritoneal macrophages. Biochim Biophys Acta. 1982;720:411-419.
68. Nishikawa M, Huang L. Nonviral vectors in the new millennium: delivery barriers in gene transfer. Hum Gene Ther. 2001;12:861-870.
69. Weaver JC. Electroporation: a general phenomenon for manipulating cells and tissues. J Cell Biochem. 1993;51:426-435.
70. Neumann E, Kakorin S, Toensing K. Fundamentals of electroporative delivery of drugs and genes. Bioelectrochem Bioenerg. 1999;48:3-16.
71. Krönke J, Kittler R, Buchholz F, Windisch MP, Pietschmann T, Bartenschlager R, Frese M. Alternative approaches for efficient inhibition of hepatitis C virus RNA replication by small interfering RNAs. J Virol. 2004;78:3436-3446.
72. Randall G, Grakoui A, Rice CM. Clearance of replicating hepatitis C virus replicon RNAs in cell culture by small interfering RNAs. Proc Natl Acad Sci U S A. 2003;100:235-240.
73. Sen A, Steele R, Ghosh AK, Basu A, Ray R, Ray RB. Inhibition of hepatitis C virus protein expression by RNA interference. Virus Res. 2003;96:27-35.
74. Takigawa Y, Nagano-Fujii M, Deng L, Hidajat R, Tanaka M, Mizuta H, Hotta H. Suppression of hepatitis C virus replicon by RNA interference directed against the NS3 and NS5B regions of the viral genome. Microbiol Immunol. 2004;48:591-598.
75. Wilson JA, Jayasena S, Khvorova A, Sabatinos S, Rodrigue-Gervais IG, Arya S, Sarangi F, Harris-Brandts M, Beaulieu S, Richardson CD. RNA interference blocks gene expression and RNA synthesis from hepatitis C replicons propagated in human liver cells. Proc Natl Acad Sci U S A. 2003;100:2783-2788.
76. Zhang J, Yamada O, Sakamoto T, Yoshida H, Iwai T, Matsushita Y, Shimamura H, Araki H, Shimotohno K. Down-regulation of viral replication by adenoviral-mediated expression of siRNA against cellular cofactors for hepatitis C virus. Virology. 2004;320:135-143.
77. Geiss BJ, Pierson TC, Diamond MS. Actively replicating West Nile virus is resistant to cytoplasmic delivery of siRNA. Virol J. 2005;2:53.
78. Ptasznik A, Nakata Y, Kalota A, Emerson SG, Gewirtz AM. Short interfering RNA (siRNA) targeting the Lyn kinase induces apoptosis in primary, and drug-resistant, BCR-ABL1(+) leukemia cells. Nat Med. 2004;10:1187-1189.
79. Boll A, Schrader M. Elongation of Peroxisomes as an Indicator for Efficient Dynamin-like Protein 1 Knock Down in Mammalian Cells. J Histochem Cytochem. 2005;53:1037-1040.
80. Schiffelers RM, Xu J, Storm G, Woodle MC, Scaria PV. Effects of treatment with small interfering RNA on joint inflammation in mice with collagen-induced arthritis. Arthritis Rheum. 2005;52:1314-1318.
81. Takabatake Y, Isaka Y, Mizui M, Kawachi H, Shimizu F, Ito T, Hori M, Imai E. Exploring RNA interference as a therapeutic strategy for renal disease. Gene Ther. 2005;12:965-973.
82. Akaneya Y, Jiang B, Tsumoto T. RNAi-induced gene silencing by local electroporation in targeting brain region. J Neurophysiol. 2005;93:594-602.
83. Davidson JM, Krieg T, Eming SA. Particle-mediated gene therapy of wounds. Wound Repair Regen. 2000;8:452-459.
84. Kuriyama S, Mitoro A, Tsujinoue H, Nakatani T, Yoshiji H, Tsujimoto T, Yamazaki M, Fukui H. Particle-mediated gene transfer into murine livers using a newly developed gene gun. Gene Ther. 2000;7:1132-1136.
85. Lin MT, Pulkkinen L, Uitto J, Yoon K. The gene gun: current applications in cutaneous gene therapy. Int J Dermatol. 2000;39:161-170.
86. Muangmoonchai R, Wong SC, Smirlis D, Phillips IR, Shephardl EA. Transfection of liver in vivo by biolistic particle delivery: its use in the investigation of cytochrome P450 gene regulation. Mol Biotechnol. 2002;20:145-151.
87. Yoshida S, Kashiwamura SI, Hosoya Y, Luo E, Matsuoka H, Ishii A, Fujimura A, Kobayashi E. Direct immunization of malaria DNA vaccine into the liver by gene gun protects against lethal challenge of Plasmodium berghei sporozoite. Biochem Biophys Res Commun. 2000;271:107-115.
88. Kim TW, Lee JH, He L, Boyd DA, Hardwick JM, Hung CF, Wu T. Modification of professional antigen-presenting cells with small interfering RNA in vivo to enhance cancer vaccine potency. Cancer Res. 2005;65:309-316.
89. Endoh M, Koibuchi N, Sato M, Morishita R, Kanzaki T, Murata Y, Kaneda Y. Fetal gene transfer by intrauterine injection with microbubble-enhanced ultrasound. Mol Ther. 2002;5:501-508.
90. Lawrie A, Brisken AF, Francis SE, Cumberland DC, Crossman DC, Newman CM. Microbubble-enhanced ultrasound for vascular gene delivery. Gene Ther. 2000;7:2023-2027.
91. Shohet RV, Chen S, Zhou YT, Wang Z, Meidell RS, Unger RH, Grayburn PA. Echocardiographic destruction of albumin microbubbles directs gene delivery to the myocardium. Circulation. 2000;101:2554-2556.
92. Song J, Chappell JC, Qi M, VanGieson EJ, Kaul S, Price RJ. Influence of injection site, microvascular pressure and ultrasound variables on microbubble-mediated delivery of microspheres to muscle. J Am Coll Cardiol. 2002;39:726-731.
93. Taniyama Y, Tachibana K, Hiraoka K, Namba T, Yamasaki K, Hashiya N, Aoki M, Ogihara T, Yasufumi K, Morishita R. Local delivery of plasmid DNA into rat carotid artery using ultrasound. Circulation. 2002;105:1233-1239.
94. Teupe C, Richter S, Fisslthaler B, Randriamboavonjy V, Ihling C, Fleming I, Busse R, Zeiher AM, Dimmeler S. Vascular gene transfer of phosphomimetic endothelial nitric oxide synthase (S1177D) using ultrasound-enhanced destruction of plasmid-loaded microbubbles improves vasoreactivity. Circulation. 2002;105:1104-1109.
95. Unger EC, Hersh E, Vannan M, McCreery T. Gene delivery using ultrasound contrast agents. Echocardiography. 2001;18:355-361.
96. Budker V, Zhang G, Danko I, Williams P, Wolff J. The efficient expression of intravascularly delivered DNA in rat muscle. Gene Ther. 1998;5:272-276.
97. Budker V, Zhang G, Knechtle S, Wolff JA. Naked DNA delivered intraportally expresses efficiently in hepatocytes. Gene Ther. 1996;3:593-598.
98. Zhang G, Budker V, Williams P, Subbotin V, Wolff JA. Efficient expression of naked DNA delivered intraarterially to limb muscles of nonhuman primates. Hum Gene Ther. 2001;12:427-438.
99. Zhang G, Vargo D, Budker V, Armstrong N, Knechtle S, Wolff JA. Expression of naked plasmid DNA injected into the afferent and efferent vessels of rodent and dog livers. Hum Gene Ther. 1997;8:1763-1772.
100. Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther. 1999;6:1258-1266.
101. Zhang G, Budker V, Wolff JA. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum Gene Ther. 1999;10:1735-1737.
102. Budker V, Budker T, Zhang G, Subbotin V, Loomis A, Wolff JA. Hypothesis: naked plasmid DNA is taken up by cells in vivo by a receptor-mediated process. J Gene Med. 2000;2:76-88.
103. Kobayashi N, Kuramoto T, Yamaoka K, Hashida M, Takakura Y. Hepatic uptake and gene expression mechanisms following intravenous administration of plasmid DNA by conventional and hydrodynamics-based procedures. J Pharmacol Exp Ther. 2001;297:853-860.
104. Felgner PL, Gadek TR, Holm M, Roman R, Chan HW, Wenz M, Northrop JP, Ringold GM, Danielsen M. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci U S A. 1987;84:7413-7417.
105. Golan R, Pietrasanta LI, Hsieh W, Hansma HG. DNA toroids: stages in condensation. Biochemistry. 1999;38:14069-71406.
106. Hansma HG, Golan R, Hsieh W, Lollo CP, Mullen-Ley P, Kwoh D. DNA condensation for gene therapy as monitored by atomic force microscopy. Nucleic Acids Res. 1998;26:2481-2487.
107. Keller M, Harbottle RP, Perouzel E, Colin M, Shah I, Rahim A, Vaysse L, Bergau A, Moritz S, Brahimi-Horn C, Coutelle C, Miller AD. Nuclear localisation sequence templated nonviral gene delivery vectors: investigation of intracellular trafficking events of LMD and LD vector systems. Chembiochem. 2003;4:286-298.
108. Lasic DD, Papahadjopoulos D. Liposomes revisited. Science. 1995;267:1275-1276.
109. Zelphati O, Nguyen C, Ferrari M, Felgner J, Tsai Y, Felgner PL. Stable and monodisperse lipoplex formulations for gene delivery. Gene Ther. 1998;5:1272-12782.
110. Bloomfield VA. DNA condensation. Curr Opin Struct Biol. 1996;6:334-341.
111. Boulikas T, Martin F. Histones, Protamine, and Polylysine but not Poly(E:K) Enhance Transfection Efficiency. Int J Oncol. 1997;10:317-322.
112. Keller M, Tagawa T, Preuss M, Miller AD. Biophysical characterization of the DNA binding and condensing properties of adenoviral core peptide mu. Biochemistry. 2002;41:652-659.
113. Stewart L, Manvell M, Hillery E, Etheridge C, Cooper R, Stark H, van-Heel M, Preuss M, Alton EWFW, Miller AD. Cationic lipids for gene therapy part 4 - Physico-chemical analysis of cationic liposome-DNA complexes (lipoplexes) with respect to in vitro and in vivo gene delivery efficiency. Journal of the Chemical Society, Perkin Transactions 2. 2001;4:624-632.
114. Chesnoy S, Huang L. DNA condensed by polycations and lipids for gene transfer. S.T.P. Pharma Sci. 1999;9:5-12.
115. Keller M, Jorgensen MR, Perouzel E, Miller AD. Thermodynamic aspects and biological profile of CDAN/DOPE and DC-Chol/DOPE lipoplexes. Biochemistry. 2003;42:6067-6077.
116. Kreiss P, Cameron B, Rangara R, Mailhe P, Aguerre-Charriol O, Airiau M, Scherman D, Crouzet J, Pitard B. Plasmid DNA size does not affect the physicochemical properties of lipoplexes but modulates gene transfer efficiency. Nucleic Acids Res. 1999;27:3792-3798.
117. Ross PC, Hui SW. Lipoplex size is a major determinant of in vitro lipofection efficiency. Gene Ther. 1999;6:651-659.
118. Simberg D, Danino D, Talmon Y, Minsky A, Ferrari ME, Wheeler CJ, Barenholz Y. Phase behavior, DNA ordering, and size instability of cationic lipoplexes. Relevance to optimal transfection activity. J Biol Chem. 2001;276:47453-47459.
119. Zuidam NJ, Barenholz Y. Characterization of DNA-lipid complexes commonly used for gene delivery. Int J Pharm. 1999;183:43-46.
120. Zuidam NJ, Hirsch-Lerner D, Margulies S, Barenholz Y. Lamellarity of cationic liposomes and mode of preparation of lipoplexes affect transfection efficiency. Biochim Biophys Acta. 1999;1419:207-220.
121. Schiffelers RM, Ansari A, Xu J, Zhou Q, Tang Q, Storm G, Molema G, Lu PY, Scaria PV, Woodle MC. Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle. Nucleic Acids Res. 2004;32:e149.
122. Spagnou S, Keller M, 122, M. Lipidic carriers of siRNA: differences in the formulation, cellular uptake, and delivery with plasmid DNA. Biochemistry. 2004;43:13348-13356.
123. Boussif O, Lezoualc'h F, Zanta MA, Mergny MD, Scherman D, Demeneix B, Behr JP. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci U S A. 1995;92:7297-7301.
124. Urban-Klein B, Werth S, Abuharbeid S, Czubayko F, Aigner A. RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo. Gene Ther. 2005;12:461-466.
125. Slamon DJ, Godolphin W, Jones LA, Holt JA. Wong SG, Keith DE, Levin WJ, Stuart SG, Udove J, Ullrich A, Press MF. Studies of the HER-2/neu proto-oncogene in human breast and ovarian cancer. Science. 1989;244:707-712.
126. Kim B, Tang Q, Biswas PS, Xu J, Schiffelers RM, Xie FY, Ansari A, Scaria PV, Woodle MC, Lu P, Rouse BT. Inhibition of ocular antiogenesis by siRNA targeting vascular endothelial growth factor pathway genes. Am. J. Pathology. 2004;165: 2177-2185.
127. Thomas M, Lu JJ, Ge Q, Zhang C, Chen J, Klibanov AM. Full deacylation of polyethylenimine dramatically boosts its gene delivery efficiency and specificity to mouse lung. Proc Natl Acad Sci U S A. 2005;102:5679-5684.
128. Read ML, Singh S, Ahmed Z, Stevenson M, Briggs SS, Oupicky D, Barrett LB, Spice R, Kendall M, Berry M, Preece JA, Logan A, Seymour LW. A versatile reducible polycation-based system for efficient delivery of a broad range of nucleic acids. Nucleic Acids Res. 2005;33:e86.
129. Putnam D, Gentry CA, Pack DW, Langer R. Polymer-based gene delivery with low cytotoxicity by a unique balance of side chain termini. Proc Natl Acad Sci USA. 2001;98,:1200-1205.
130. Midoux P and Monsigny M. Efficient gene transfer by histidylated polylysine/pDNA complexes. Bioconjug Chem. 1999;10:406-411.
131. Sandvig A, Berry M, Barrett LB, Butt A, Logan A. Meylin-, reactive glia-, and scarderived CNS axon growth inhibitors: expression, receptor signaling, and correlation with axon regeneration. Glia. 2004;46:225-251.
132. Itaka K, Kanayama N, Nishiyama N, Jang W-D, Yamasaki Y, Nakamura K, Kawaguchi H, Kataoka K. Supramolecular nanocarrier of siRNA from PEG-based block catiomer carrying diamine side chain with distinctive pKa directed to enhance intracellular gene silencing. J Am Chem Soc. 2004;126:13612-13613.
133. Oishi M, Nagasaki Y, Itaka K, Nishiyama N, Kataoka K. Lactosylated poly(ethylene glycol)-siRNA conjugate through acid-labile □-thiopropionate linkage to construct pH-sensitive polyion complex micelles achieving enhanced gene silencing in hepatoma cells. J Am Chem Soc. 2005;127:1624-1625.
134. Leng Q, Scaria P, Zhu J, Ambulos N, Campbell P, Mixon A.J. Highly branched HK peptides are effective carriers of siRNA. J Gene Medicine. 2005;7:977-986.
135. Chen QR, Zhang L, Stass SA, Mixson AJ. Branched co-polymers of histidine and lysine are efficient carriers of plasmids. Nucleic Acids Res. 2001;29:1334-1340.
136. Gonzolez H, Hwang SJ, Davis ME. New class of polymers for the delivery of macromolecular therapeutics. Bioconj Chem. 1999;10:1068-1074.
137. Pun SH, Davis ME. Development of a nonviral gene delivery vehicle for systemic application. Bioconj Chem. 2002;13:630-639.
138. Hu-Lieskovan S, Heidel JD, Bartlett DW, Davis ME, Triche TJ. Sequence-specific knockdown of EWS-FLI1 by targeted, nonviral delivery of small interfering RNA inhibits tumor growth in a murine model of metastatic Ewing's sarcoma. Cancer Res. 2005;65:8984-8992.
139. Sorgi FL, Bhattacharya S, Huang L. Protamine sulfate enhances lipid-mediated gene transfer. Gene Ther. 1997;4:961-968.
140. Park YJ, Liang JF, Ko KS, Kim SW, Yang VC. Low molecular weight protamine as an efficient and nontoxic gene carrier: in vitro study. J Gene Med. 2003;5:700-711.
141. Song E, Zhu P, Lee S-K, Chowdhury D, Kussman S, Dykxhoorn DM, Feng Y, Palliser D, Weiner DB, Shankar P, Marasco WA, Lieberman J. Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nature Biotech. 2005;23:709-717.