DNA as therapeutics; An update

Indian Journal of Pharmaceutical Sciences

Volumn 71, Issue 5, 2009, Pages 488-498

  Saraswat, P ^a   Soni, R R ^b   Bhandari, A ^c   Nagori, B P ^d  

^a GANDHI MEDICAL COLLEGE   (India)

^b Jaipur Fertility and Microsurgery Research Center   (India)

^c Department of Pharmacy   (India)

^d L M College of Science and Technology (Pharmacy Wing)   (India)

Author keywords

Antisense; DNA delivery systems; Gene therapy; Gene therapy trials; Gene transfer technology; Liposomes; Nonviral vectors; Nucleic acid therapeutics; Viral vectors

Indexed keywords

ADENOVIRUS VECTOR; ANTIGEN; ANTISENSE OLIGONUCLEOTIDE; APTAMER; BETA GALACTOSIDASE; CGP 69846A; CHITOSAN; DAUNORUBICIN; DENDRIMER; DNA; DNA VACCINE; DOXORUBICIN; FOMIVIRSEN; GENDICINE; GLYCYRRHIZIC ACID; LIPOFECTAMINE; LIPOSOME; MG 98; NAKED DNA; OLIGONUCLEOTIDE; PHOSPHATIDYLCHOLINE; PLASMID DNA; POLYETHYLENEIMINE; POLYLYSINE; RETROVIRUS VECTOR;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; DNA VECTOR; GENE TARGETING; GENE THERAPY; GENE TRANSFER; HEAD AND NECK CARCINOMA; HUMAN; MALARIA; MEDICAL RESEARCH; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PLASMID; RETINITIS; REVIEW; RNA INTERFERENCE; SEVERE COMBINED IMMUNODEFICIENCY; VIRAL GENE DELIVERY SYSTEM;

EID: 72749125839     PISSN: 0250474X     EISSN: 19983743     Source Type: Journal    
DOI: 10.4103/0250-474X.58169     Document Type: Review

References (181)

1. Anderson WF. Human gene therapy. Science 1992;256:808-813
2. Baltimore DP. Intracellular immunization. Nature 1988;335:395-397
3. Mizutani TN, Kato M, Hirota K, Sugiyama A, Murakami J, Shimotohno K. Inhibition of hepatitis C virus replication by antisense oligonucleotids in culture cells. Biochem Biophys Res Commun 1995;212:906-911
4. SoRelle R. Who owns your DNA? Who will own it? Circulation 2000;101:e67-8.
5. Report of the United Kingdom Health Minister's Gene Therapy Advisory Committee. Guidance on making proposals to conduct gene therapy research on human subjects. Hum Gene Ther 1995;6:335-346
6. Rosenberg SA, Blaese RM, Branner MK, Deisseroth AB, Ledley FD, Lotze MT, et al. Human gene marker/therapy protocols. Hum Gene Ther 2000;11:919-979
7. Lehrman S. Virus treatment questioned after gene therapy death. Nature 1999;401:517-518
8. Anon S. The increasing opacity of gene therapy. Nature 1999;402:107.
9. Crooke ST. An overview of progress in antisense therapeutics. Antisense Nucleic Acid Drug Dev 1998;8:115-122
10. Stull RA, Szoka FC Jr. Antigene, ribozyme and aptamer nucleic acid drugs: Progress and prospects. Pharm Res 1995;12:463-465
11. Patil SD, Burgess DJ. DNA-based pharmaceuticals: Therapeutics for the 21st century. AAPS Newsmagazine 2003;6:27.
12. Baker BF. The role of antisense oligonucleotides in the wave of genomic information. Nucleosides Nucleotides Nucleic Acids 2001;20:397-399
13. Van Ommen GJ, Bakker E, Den Dunnen JT. The human genome project and the future of diagnostics, treatment, and prevention. Lancet 1999;354:5-10.
14. Uherek C, Wels W. DNA-carrier proteins for targeted gene delivery. Adv Drug Deliv Rev 2000;44:153-166
15. Johnston SA, Talaat AM, McGuire MG. Genetic Immunization What's in a name? Arch Med Res 2002;33:325-329
16. Anderson WF. Human gene therapy. Nature (London) 1998;392:25-30.
17. Vorburger SA, Hunt KK. Adenoviral gene therapy. Oncologist 2002;7:46-59.
18. Otsu M, Candotti F. Gene therapy in infants with severe combined immunodeficiency. BioDugs 2002;16:229-239
19. Zhaohui P. China OKs Gene Therapy Drug. Genetic Engineering News 2003;6. [http://www. p53therapy.com/news/(November 19, 2003)].
20. Galanis E, Russell S. Cancer gene therapy clinical trials: Lessons for the future. Br J Cancer 2001;85:1432-1436
21. Mulherkar R. Gene therapy for cancer. Curr Sci 2001;81:555-560
22. Baekelandt V, De Strooper B, Nuttin B, Debysre Z. Gene therapeutic strategies for neurodegenerative diseases. Curr Opin Mol 2000;2:540-554
23. Bunnel BA, Morgan RA. Gene therapy for HIV infection. Dugs Today 1996;32:209-224
24. Horner AA, Van Uden JH, Jubeldia JM, Broide D, Raz E. DNA-based immunothrapeutics for the treatment of allergic disease. Immunol Rev 2001;179:102-118
25. Crooke ST. Molecular mechanisms of antisense drugs. Biochem Biophys Acta 1999;1489:31-43.
26. Crooke ST. Molecular mechanisms of antisense drugs: Human RNaseH. Antisense Nucleic Acid Drug Dev 1999;9:377-379
27. Speedie M. Antisense therapeutic agents. In: Williams DA, Lemke TL, editors. Foye's Principles of Medicinal Chemistry. 5th ed. Philadelphia: Lippincott, Williams & Wilkins; 2005. p. 1053-1054
28. Akhtar S, Hughes MD, Khan A. The delivery of antisense therapeutics. Adv Drug Deliv Rev 2000;44:3-21.
29. Crooke ST. Vitravene another piece in the mosaic. Antisense Nucleic acid Drug Dev 1998;8:vii-v3.
30. Mardan T, Kopecek J, Kissel T. Prospects for cationic polymers in gene and oligonucleotide therapy against cancer. Adv Drug Deliv Rev 2002;54:715-758
31. Agarawal S, Tang JY. GEM-91-an antisense oligonucleotide phosphorothioate as a therapeutic agent for AIDS. Antisense Res Dev 1992;2:261-266
32. Alt M, Ring R, Hofschneider PH, Paumgartner G, Caselmann WH. Specific inhibition of hepatitis C viral gene expression by antisense phosphorothioate oligodeoxynucleotides. Hepatology 1995;22:707-717
33. Azad RF, Driver VB, Tanaka K, Cooke RM, Anderson KP. Antiviral activity of a phosphorothioate oligonucleotide complementary to RNA of the human cytomegalovirus major immediate-early region. Antimicrob Agents Chemother 1993;37:1945-1954
34. Bordier B, Helena C, Barr PJ, Litvak S, Sarih-Cottin L. In vitro effects of antisense oligonucleotides on human immunodeficiency virus type 1 reverse transcription. Nucleic Acids Res 1992;20:1999-2006.
35. Cowsert LM, Fox MC, Zon G, Mirabelli MK. In vitro evaluation of phosphorothioate oligonucleotides targeted to the E2 mRNA of papillomavirus: Potential treatment for the genital warts. Antimicrob Agents Chemother 1993;37:171-177
36. Gerviax A, Li X, Kraus G, Wong-Staal F. Multigene antivirus vectors inhibit divers human immunodeficiency virus type 1 clades. J Virol 1997;71:3048-3053
37. Hanecak R, Brown-Driver V, Fox MC, Azad RF, Furusako S, Nozaki C, et al. Antisense oligonucleotide inhibition of hepatitis C virus gene expression in transformed hepatocytes. J Virol 1996;70:5203-5212
38. Kinchington D, Galpin S, Jeroszewski J, Ghosh K, Subashinghe C, Cohen JS. A comparision of gag, pol, and rev antisense oligodeoxynucleotides as inhibitors of HIV-1. Antiviral Res 1992;17:53-62.
39. Lisziewicz J, Sun D, Klotman M, Agarwal S, Zamencik P, Gallo RC. Specific inhibition of human immunodeficiency virus type 1 replication by antisense oligonuclotides: An in vitro model for treatment. Proc Natl Acad Sci USA 1992;89:11209-11213
40. Lisziewicz J, Sun D, Metelev V, Zamencik P, Gallo RC, Agarwal S. Long term treatment of human immunodeficiency virus infected cells with antisense oligonucleotide phosphorothioates. Proc Natl Acad Sci USA 1993;90:3860-3864
41. Mirabelli CK, Bennet CF, Anderson K, Crooke ST. In vitro and in vivo pharmacologic activities of antisense oligonucleotides. AntiCancer Drug Design 1991;6:647-661
42. MorVan F, Porumb H, Degols G, Lefebvre I, Pampon A, Sproat BS, et al. Comparative evaluation of seven oligonucleotide analogues as potential antisense agents. J Med Chem 1993;36:280-287
43. Tonkinson JL, Stein CA. Antisense nucleic acids-prospects for antiviral intervention. Antiviral Chem Chemother 1993;4:193-199
44. Jayasena SD. Aptamers: An emerging class of molecules that rival antibodies in diagnostics. Clin Chem 1999;45:1628-1650
45. de Saultrait VR, Lozach PY, Altmeyer R, Tarrago-Litvak L, Litvak S, Andreola ML. DNA aptamers derived from HIV-1 RNaseH inhibitors are strong anti-integrase agents. J Mol Biol 2002;324:195-203.
46. Zhang L, Gasper WA, Stass SA, Ioffe OB, Davis MA, Mixson AJ. Angiogenic inhibition mediated by a DNAzyme that target vascular endothelial growth factor receptor 2. Cancer Res 2002;62:5463-5469
47. McAlister DV, Allen MG, Prausnitz MR. Microfabricated microneedles for gene and drug delivery. Annu Rev Biomed Eng 2000;2:289-313.
48. Luo D, Saltzman WM. Synthetic DNA delivery systems. Nat Biotechnol 2000;18:33-37
49. Regnier V, Tahiri A, Andre N, Lemaitre M, Le Doan T, Preat V. Electroporation-mediated delivery of 3′-protected phosphodiester oligodeoxynucleotides to the skin. J Control Release 2000;67:337-346
50. Huang L, Viroonchatapan E. Introduction. In: Huang L, Huang M-C, Wagner E, editors. Nonviral vectors for gene therapy. San Diego, CA: Academic Press; 1999. p. 3-22.
51. Kamiya H, Tsuchiya H, Yamazaki J, Harashima H. Intracellular trafficking and transgene expression of viral and non-viral gene vectors. Adv Drug Deliv Rev 2001;52:153-164
52. Mah C, Byrne BJ, Flotte TR. Virus-based gene delivery systems. Clin Pharmacokinet 2002;41:901-911
53. Lotze MT, Kost TA. Viruses as gene delivery vectors: Application to gene function, target validation, and assay development. Cancer Gene Ther 2002;9:692-699
54. McTaggart S, Al-Rubeai M. Retroviral vectors for human gene delivery. Biotechnol Adv 2002;20:1-31.
55. Martin KR, Klein RL, Quigley HA. Gene delivery to the eye using adeno-associated viral vectors. Methods 2002;28:267-275
56. Lien Y-HH, Lie L-W. Gene therapy for renal disorders: What are the benefits for the elderly? Drugs Aging 2002;19:553-560
57. Chamberlain JS. Gene therapy of muscular dystrophy. Hum Mol Genet 2002;11:2355-2362
58. Wolf JK, Jenkins AD. Gene therapy for ovarian cancer. Int J Oncol 2002;21:461-468
59. Walther W, Stein U. Viral vectors for gene transfer: A review of their use in the treatment of human diseases. Drugs 2000;60:249-271
60. Lever AML. Gene therapy in the fight against AIDS. Expert Opin Ther Patents 1996;6:161-167
61. Zhao W, Kobayashi M, Hosokawa M, Seth P. Adenoviral vectors for cancer gene therapy. Curr Genomics 2002;3:163-180
62. Hale SJ, Green NK. Viral approaches to cancer gene therapy. Expert Opin Ther Patents 2002;12:369-378
63. Roe T, Reynolds TC, Yu G, Brown PO. Integration of murine leukemia virus DNA depends on mitosis. EMBO J 1993;12:2099-2108
64. Lewis PF, Emerman M. Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus. J Virol 1994;68:510-516
65. Naldini L, Blomer U, Gally P. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996;272:263-267
66. Mann R, Mulligan RC, Baltimore D. Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus. Cell 1983;33:153-159
67. Cone RD, Mulligan RC. High-efficiency gene transfer into mammalian cells: Generation of helper-free recombinant retrovirus with broad mammalian host range. Proc Natl Acad Sci USA 1984;81:6349-6353
68. Kim SH, Yu SS, Park JS, Robbins PD, An CS, Kim S. Construction of retroviral vectors with improved safety, gene expression, and versatility. J Virol 1998;72:994-1004.
69. Sheridan PL, Bodner M, Lynn A. Generation of retroviral packaging and producer cell lines for large-scale vector production and clinical application: Improved safety and high titer. Mol Ther 2000;2:262-275
70. Fujji S, Huang S, Fong TC. Induction of melanoma-associated antigen systemic immunity upon intratumoral delivery of interferon-gamma retroviral vector in melanoma patients. Cancer Gene Ther 2000;7:1220-1230
71. Tait DL, Obermiller PS, Hatmaker AR. Ovarian cancer BRCA1 gene therapy: Phase I and II trial differences in immune response and vector stability. Clin Cancer Res 1999;5:1708-1714
72. Bordignon C, Notarangilo LD, Nobili N. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 1995;270:470-475
73. Onodera M, Ariga T, Kawamura N. Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immunodeficiency caused by adenosine deaminase deficiency. Blood 1998;91:30-36
74. Dunbar CE, Kohn DB, Shiffman R. Retroviral transfer of the glucoceribrosidase gene into CD34+ cells from patients with Goucher disease: In vivo detection of transduced cells without myeloblation. Hum Gen Ther 1998;9:2629-2640
75. Garton KJ, Ferri N, Raines EW. Efficient expression of exogenous genes in primary vascular cells using IRES-based retroviral vectors. Biotechniques 2002;32:830-843
76. Stewart AK, Lassam NJ, Quirt IC. Adenovector-mediated gene delivery of interleukin-2 in metastatic breast cancer and melanoma: Results of a phase 1 clinical trial. Gene Ther 1999;6:350-363
77. Alvarez RD, Barns MN, Gomez-Navarro J. A cancer gene therapy approach utilizing an anti-erbB-2 single-chain antibody-encoding adenovirus (AD21): A phase I trial. Clin Cancer Res 2000;6:3081-3087
78. Alvarez RD, Gomez-Navarro J, Wang M. Adenoviral-mediated suicide gene therapy for ovarian cancer. Mol Ther 2000;2:524-530
79. Byrnes AP, Rusby JE, Wood MJ. Adenovirus gene transfer causes inflammation in the brain. Neuroscience 1995;66:1015-1024
80. Yang Y, Jooss KU, Su Q. Immune responses to viral antigens versus transgene product in the elimination of recombinant adenovirus-infected hepatocytes in vivo. Gene Ther 1996;3:137-144
81. Yang Y, Su Q, Wilson JM. Role of viral antigens in destructive cellular immune responses to adenovirus vector-transduced cells in mouse lungs. J Virol 1996;70:7209-7212
82. Van Ginkel FW, McGhee JR, Liu C. Adenoviral gene delivery elicits distinct pulmonary-associated T helper cell responses to the vector and to its transgene. J Immunol 1997;159:685-693
83. Michou AI, Santoro L, Christ M. Adenovirus-mediated gene transfer: Influence of transgene, mouse strain and type of immune response on persistence of transgene expression. Gene Ther 1997;4:473-482
84. Bett AJ, Prevec L, Graham FL. Packaging capacity, and stability of human adenovirus type 5 vectors. J Virol 1993;67:5911-5921
85. Clemens PR, Kochanek S, Sunada Y. In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes. Gene Ther 1996;3:965-972
86. Kochanek S, Clemens PR, Mitani K. A new adenoviral vector: Replacement of viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and betagalactosidase. Proc Natl Acad Sci USA 1996;93:5731-5736
87. Parks RJ, Grahm FL. A helper-dependent system for adenovirus vector production helps define a lower limit for efficient DNA packaging. J Virol 1997;71:3293-3298
88. Morsy MA, Gu M, Motzel S. An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene. Proc Natl Acad Sci USA 1998;95:7866-7871
89. Schiedner G, Morral N, Parks RJ. Genomic DNA transfer with a highcapacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat Genet 1998;18:180-183
90. Heider H, Verca HB, Rusconi S, Asmis R. Comparision of lipidmediated, and adenoviral gene transfer in human monocyte-derived macrophages and COS-7 cells. Biotechniques 2000;28:260-270
91. Fisher KJ, Jooss K, Alston J. Recombinant adeno-associated virus for muscle directed gene therapy. Nat Med 1997;3:306-312
92. Jooss K, Yang Y, Fisher KJ. Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. J Virol 1998;72:4212-4223
93. Rose JA, Berns KI, Hoggan MD. Evidence for a single stranded adenovirus-associated virus genome: Formation of a DNA density hybrid on release of viral DNA. Proc Natl Acad Sci USA 1969;64: 863-869
94. Kotin RM, Siniscalco M, Samulski RJ. Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA 1990;87:2211-2215
95. Samulski RJ, Zhu X, Xiao X. Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J 1991;10:3941-3950
96. Samulski RJ, Chang LS, Shenk T. Helper-stocks of recombinant adenoassociated viruses: Normal integration does not require viral gene expression. J Virol 1989;63:3822-3828
97. Dong JY, Fang PD, Frizzel RA. Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum Gene Ther 1996;7:2101-2112
98. Hermonat PL, Quirk JG, Bishop BM. The packaging capacity of adenoassociated virus (AAV) and the potential for wild-type-plus AAV gene therapy vectors. FEBS Lett 1997;407:78-84.
99. Owens RA. Second generation adeno-associated virus type 2-based gene therapy systems with the potential for preferential integration into AAVS 1. Curr Gene Ther 2002;2:145-159
100. Nyberg-Hoffman C, Aguilar-Cardova E. Instability of adenoviral vectors during transport and its implication for clinical studies. Nat Med 1999;5:955-957
101. Carpenter DE, Stevens JG. Long term expression of a foreign gene from a unique position in the latent herpes simplex virus genome. Hum Gene Ther 1996;7:1447-1454
102. Lowenstein PR, Morrison EE, Bain D. Use of recombinant vectors derived from herpes simplex virus 1 mutant tsK for short-tem expression of transgenes encoding cytoplasmic and membrane anchored proteins in post mitotic-polarized cortical neurons and glial cells in vitro. Neuroscience 1994;60:1059-1077
103. Fattal E, Dellattre J, Dubernet C, Couvrur P. Liposomes for the delivery of nucleotides and oligonucleotides. S.T.P. Pharma Sciences 1999;9: 383-390
104. Fattal E, Dubernet C, Couvrur P. Liposome-based formulations for the delivery of oligonucleotids. S.T.P. Pharma Sciences 2001;11:31-44.
105. Pedroso de Lima MC, Simoes S, Pires P, Faneca H, Duzguns N. Cationic lipid-DNA complexes in gene delivery: From biophysics to biological applications. Adv Drug Deliv Rev 2001;47:277-294
106. Nakamura M, Devila-Zavala P, Tokuda H. Uptake and gene expression of naked plasmid DNA in cultured brain microvessel endothelial cells. Biochem Biophys Res Commun 1998;245:235-239
107. Mann MJ, Gibbons GH, Hutchinson H. Pressure-mediated oligonucleotide transfection of rat and human cardiovascular tissues. Proc Natl Acad Sci USA 1999;96:6411-6416
108. Wolff JA, Malone RW, Williams P. Direct gene transfer into mouse muscle in vivo. Science 1990;247:1465-1468
109. Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther 1999;6:1258-1266
110. Fynan EF, Webster RG, Fuller DH. DNA vaccines: Protective immunizations by parenteral, mucosal, and gene-gun inoculations. Proc Natl Acad Sci USA 1993;90:11478-11482
111. Wong TK, Neumann E. Electric field mediated gene transfer. Biochem Biophys Res Commun 1982;107:584-587
112. Neumann E, Schaefer-Ridder M, Wang Y. Gene transfer into lyoma by electroporation in high electric fields. EMBO J 1982;1:841-845
113. Rols MP, Delteil C, Golzio M. In vivo electrically mediated protein and gene transfer in murine melanoma. Nat Biotechnol 1998;16:168-171
114. Rizzuto G, Cappelletti M, Maione D. Efficient and regulated erythropoietin production by naked DNA injection and muscle electroporation. Proc Natl Acad Sci USA 1999;96:6417-6422
115. Hwang SJ, Davis ME. Cationic polymers for gene delivery: Designs for overcoming barriers to systemic administration. Curr Opin Mol Ther 2001;3:183-191
116. Lemkine GF, Demeneix BA. Polyethylenimins for in vivo gene delivery. Curr Opin Mol Ther 2001;3:178-182
117. Lollo CP, Banaszczyk MG, Mullen PM. Poly-L-lysine based gene delivery systems: Synthesis, purification, and application. Methods Mol Med 2002;69:1-13.
118. Borchard G. Chitosans for gene delivery. Adv Drug Deliv Rev 2001;52:145-150
119. Boussif O, Lezoualc'h F, Zanta MA. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: Polyethylenimin. Proc Natl Acad Sci USA 1995;92:7297-7301
120. Wadhwa MS, Knoell DL, Young AP. Targeted gene delivery with a low molecular weight glycopeptide carrier. Bioconjug Chem 1995;6:283-291
121. Kukowska-Latello JF, Bielinska AU, Johnson J. Efficient transfer of genetic material into mammalian cells using Starbust polyamidoamine dendrimers. Proc Natl Acad Sci USA 1996;93:4897-4902
122. Tang MX, Szoka FC. The influence of polymer structure on the interactions of cationic polymers with DNA and morphology of the resulting complexes. Gene Ther 1997;4:823-832
123. Mumper RJ, Wang J, Klakamp SL. Protective interactive noncondensing (PINC) polymers for enhanced plasmid distribution and expression in rat skeletal muscle. J Control Release 1998;52:191-203.
124. Naughton BA, Dai Y, Sibanda B. Long-term expression of a retrovirally introduced beta-galactosidas gene in rodent cells implanted in vivo using biodegradable polymer meshes. Somat Cell Mol Genet 1992;18:451-462
125. Singh M, Briones M, Ott G. Cationic microparticles: A potent delivery system for DNA vaccines. Proc Natl Acad Sci USA 2000;97:811-816
126. Jenkins RG, Herrick SE, Meng QH. An integrin-targeted non-viral vector for pulmonary gene therapy. Gene Ther 2000;7:393-400.
127. LeHoux JG, Grondin F. Some effects of chitosan on liver function in the rat. Endocrinology 1993;132:1078-1084
128. Godbey WT, Mikos AG. Recent progress in gene delivery using nonviral transfer complexes. J Control Release 2001;72:115-125
129. Tejada-Berges T, Granai CO, Gordinier M, Gajewski W. Caelyx/Doxil for the treatment of metastatic ovarian and breast cancer. Expert Rev Anticancer Ther 2002;2:143-150
130. Marshal J, Yew NS, Eastman SJ, Jiang C, Sheule RK, Cheng SH. Cationic lipid-mediated gene delivery to the airways. In: Wagner E, editors. Nonviral vectors for gene therapy. San Diego, CA: Academic Press; 1999. p. 39-68.
131. Felgner PL, Gadek TR, Holm M. Lipofection: A highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 1987;84:7413-7417
132. Caplen NJ, Alton EW, Middleton PG. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat Med 1995;1:39-46.
133. Hyde SC, Southern KW, Gileadi U. Repeate administration of DNA/ liposome to the nasal epithelium of patients with cystic fibrosis. Gene Ther 2000;7:1156-1165
134. Noone PG, Hohnker KW, Zhou Z. Safety and biological efficacy of a lipid-CFTR complex for gene transfer in the nasal epithelium of adult patients with cystic fibrosis. Mol Ther 2000;1:105-114
135. Nabel GJ, Nabel EG, Yang ZY. Direct gene transfer with DNAliposome complexes in melanoma: Expression, biological activity, and lack of toxicity in humans. Proc Natl Acad Sci USA 1993;90:11307-11311
136. Nabel GJ, Gordon D, Bishop DK. Immune response in human melanoma after transfer of an allogenic class I major histocompatibility complex gene with DNA-liposome complexes. Proc Natl Acad Sci USA 1996;93:15388-15393
137. Felgner JH, Kumar R, Sridhar CN. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J Biol Chem 1994;269:2550-2561
138. Hofland HEJ, Shephard L, SulliVan SM. Formation of stable cationic lipid/DNA complexes for gene transfer. Proc Natl Acad Sci USA 1996;93:7305-7309
139. Kang S-H, Zirbes EL, Kole R. Delivery of antisense oligonucleotides and plasmid DNA with various carrier agents. Antisense Nucleic Acid Drug Dev 1999;9:497-505. (Pubitemid 30040672)
140. Ellison KE, Bisphoric NH, Webster KA. Fusigenic liposome-mediated DNA transfer into cardiac myocytes. J Mol Cell Cardiol 1996;28:1385-1399
141. Simoes S, Slepushkin V, Gaspar R. Gene delivery by negatively charged ternary complexes of DNA, cationic liposomes and transferin or fusigenic peptides. Gene Ther 1998;5:955-964
142. Shinmura K, Morishita R, Aoki M. Catheter-delivered in vivo gene transfer into rat myocardium using the fusigenic liposomal mediated method. Jpn Heart J 2000;41:633-647
143. Compton SH, Mecklenbeck S, Mejia JE. Stable integration of large (>100kb) PAC constructs in HaCa T keratinocytes using an integrintargeting peptide delivery system. Gene Ther 2000;7:1600-1607
144. Lappalainen K, Jaaskelainen I, Syrjanen K, Urtti A, Syrjanen S. Comparison of cell proliferation and toxicity assays using two cationic liposomes. Pharm Res 1994;11:1127-1131
145. Patil SD, Rhodes DG, Burgess DJ. Anionic liposomal delivery system for DNA transfection. The AAPS Journal 2004;6:E29.
146. Dokka S, Toledo D, Shi X, Castranova V, Rojanasakul Y. Oxygen radical-mediated pulmonary toxicity induced by some cationic liposomes. Pharm Res 2000;17:521-525
147. Filion MC, Philips NC. Toxicity and immunomodulatory activity of liposomal vectors formulated with cationic lipids toward immune effector cells. Biochim Biophys Acta 1997;1329:345-356
148. Freimark BD, Blzinger HP, Florack VJ. Cationic lipids enhance cytokine and cell influx levels in the lung following administration of plasmid: Cationic lipid complexes. J Immunol 1998;160:4580-4586
149. Lee H, Williams SK, Allison SD, Anchordoquy TJ. Analysis of selfassembled cationic lipid-DNA gene carrier complexes using flow field-flow fractionation and light scattering. Anal Chem 2001;73:837-843
150. Audouy S, Molema G, De Leij L, Hoekstra D. Serum as a modulator of lipoplex-mediated gene transfection: Dependence of amphiphile cell type and complex stability. J Gene Med 2000;2:465-476
151. Liu F, Qi H, Huang L, Liu D. Factors controlling the efficiency of cationic lipid-mediated transfection in vivo via intravenous administration. Gene Ther 1997;4:517-523
152. Wheeler CJ, Felgner PL, Tsai YJ. A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. Proc Natl Acad Sci USA 1996;93:11454-11459
153. Patil SD, Rhodes DG. Influence of divalent cations on the conformation of phosphorothioate oligodeoxynucleotides: A circular dichroism study. Nucleic Acids Res 2000;28:2439-2445
154. Fillion P, Desjardins A, Sayasith K, Lagace J. encapsulation of DNA in negatively charged liposomes and inhibition of bacterial gene expression with fluid-liposome encapsulated antisense oligonucleotides. Biochim Biophys Acta 2001;1515:44-54.
155. Lakkaraju A, Dubinsky JM, Low WC, Rehman YE. Neurons are protected from excitotoxic death by p53 antisense oligonuclotides delivered in anionic liposomes. J Biol Chem 2001;276:32000-32007
156. Patil SD, Rhodes DG. Conformation of oligodeoxynucleotides associated with anionic liposomes. Nucleic Acids Res 2000;28:4125-4129
157. Patil SD, Rhodes DG, Burgess DJ. Biophysical characterization of anionic lipoplexes. Biochim Biophys Acta - Biomembranes 2005;7:E61-77.
158. Lee RJ, Huang L. Lipidic vector systems for gene transfer. Crit Rev Ther Drug Carrier Syst 1997;14:173-206.
159. Guo W, Gosselin MA, Lee RJ. Characterization of a novel dioleinbased LPD II vector for gene delivery. J Control Release 2002;83:121-132
160. Perrie Y, Gregoriadis G. Liposome-entrapped plasmid DNA: Characterization studies. Biochim Biophys Acta 2000;1475:125-132
161. Venugopalan P, Jain S, Sankar S, Singh P, Rawat A, Vyas SP. pHsensitive liposomes: Mechanism of triggered release to drug and gene delivery prospects. Pharmazai 2002;57:659-671
162. Maclean AL, Symonds G, Ward R. Immunoliposomes as targeted delivery vehicles for cancer therapeutics. Int J Oncol 1997;11:325-332
163. Legendre JY, Szoka FC Jr. Delivery of plasmid DNA into mammalian cell lines using pH-sensitive liposomes: Comparision with cationic liposomes. Pharm Res 1992;9:1235-1242
164. Reddy JA, Low PS. Enhanced folate receptor mediated gene therapy using a novel pH-sensitive lipid formulation. J Control Release 2000;64:27-37.
165. Sviridov YV, Zhadanov RI, Podobed OV, Tsvetkova TA, Konstantinov I, Bogdanenko EV. The LacZ gene transfer into L929 cells and [14C]-DNA tissue distribution following intraperitoneal administration of new pH-sensitive lipoplexes in mice. Cytobios 2001;106:7-14.
166. Xu L, Huang CC, Huang W. Systemic tumor-targeted gene delivery by anti-transferrin receptor scFv-immunoliposomes. Mol Cancer Ther 2002;1:337-346
167. Shi N, Zhang Y, Zhu C, Boado RJ, Pardridge WM. Brain-specific expression of an exogenous gene after IV administration. Proc Natl Acad Sci USA 2001;98:12754-12759
168. Khaw BA, da Silva J, Vural I, Narula J, Torchilin VP. Intracytoplasmic gene delivery for in vitro transfection with cytoskeleton-specific immunoliposomes. J Control Release 2001;75:199-210.
169. Krauss WC, Park JW, Kirpotin DB, Hong K, Benz CC. Emerging antibody-based HER2 (ErbB-2/new) therapeutics. Breast Disease 2000;11:113-124
170. Schatzlein A. Nano-treatment to torpedo cancer. Available from: http:// news.bbc.co.uk/2/hi/health/7935592.stm. [cited in 2009 Mar 10].
171. James WB, Alexander JS, Susie SB, Scott R, Robert H, Kamaljit B, et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 2008;358:2231-2239
172. Albert MM, Francesca S, Aric AP, Edward NP, Federico M, Jeannette B, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008;358:2240-2248
173. Ben H. Doctors test gene therapy to treat blindness. Available from: http://www.reuters.com/article/ scienceNews/idUSL016653620070501? pageNumber=1/. [cited in 2007 May 1].
174. Scott M. Dual gene therapy suppresses lung cancer in preclinical test. Available from: http://www.newswise.com/p/articles/view/526526/.[cited in 2007 Jan 11].
175. Morgan RA, Dudley ME, Wunderlich JR, Hughes MS, Yang JC, Sherry RM, et al. Cancer regression in patients mediated by transfer of genetically engineered lymphocytes. Available from: http://www.cancer. gov/newscenter/pressreleases/ MelanomaGeneTherapy/. [cited in 2006 Aug 31].
176. Anil A. Undercover genes slip into the brain. Available from: http:// www.newscientist.com/section/science-news/. [cited in 2003 Mar 22].
177. Bob H. Gene therapy may switch off Huntington's. Available from: http://www.newscientist.com/section/ science-news/. [cited in 2003 Mar 13].
178. Danny P. Subtle gene therapy tackles blood disorder. Available from: http://www.newscientist.com/section/ science-news/. [cited in 2002 Oct 11].
179. Emma Y. 'Miracle' gene therapy trial halted. Available from: http:// www.newscientist.com/section/science-news/. [cited in 2002 Oct 3].
180. Sylvia PW. DNA nanoballs boost gene therapy. Available from: http:// www.newscientist.com/section/science-news/. [cited in 2002 May 12].
181. Jennifer FW. Murine Gene Therapy Corrects Symptoms of Sickle Cell Disease. The Scientist 2002;16:36. Available from: http://www.thescientist. com. [cited in 2002 Mar 18].