Advances in gene delivery systems

Pharmaceutical Medicine

Volumn 25, Issue 5, 2011, Pages 293-306

  Kamimura, Kenya ^a   Suda, Takeshi ^a   Zhang, Guisheng ^b   Liu, Dexi ^b  

^a NIIGATA UNIVERSITY GRADUATE SCHOOL OF MEDICAL AND DENTAL SCIENCES   (Japan)

^b UNIVERSITY OF GEORGIA   (United States)

Author keywords

Biotechnology; Drug delivery systems; Gene delivery systems; Gene therapies; Liposome encapsulation; Research and development; Viral gene therapy

Indexed keywords

ADENOVIRUS VECTOR; CHITOSAN; DENDRIMER; DNA POLYMER COMPLEX; FOAMY VIRUS VECTOR; HERPES SIMPLEX VIRUS VECTOR; HISTONE; LENTIVIRUS VECTOR; LIPOSOME; NATURAL PRODUCT; ONCORETROVIRUS VECTOR; PARVOVIRUS VECTOR; PEPTIDE DERIVATIVE; POLYALLYLAMINE; POLYAMIDOAMINE; POLYETHYLENEIMINE; POLYLYSINE; POLYMER; POLYPEPTIDE; PROTAMINE; RETROVIRUS VECTOR; UNCLASSIFIED DRUG; VELIMOGENE ALIPLASMID; VIRUS VECTOR;

BALLISTIC DNA INJECTION; CLINICAL TRIAL (TOPIC); DNA TRANSFER; ELECTRIC SHOCK; ELECTROPORATION; GENE DELIVERY SYSTEM; GENE EXPRESSION REGULATION; HUMAN; HYDRODYNAMICS BASED TRANSFECTION; HYDROPORATION; IN VITRO STUDY; IN VIVO STUDY; LIPOSOMAL GENE DELIVERY SYSTEM; MAGNETOFECTION; MELANOMA; MOLECULAR CLONING; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PHOTOPORATION; PRIORITY JOURNAL; REVIEW; SONOPORATION; TECHNIQUE; VIRAL GENE DELIVERY SYSTEM;

EID: 80052882155     PISSN: 11782595     EISSN: 11791993     Source Type: Journal    
DOI: 10.2165/11594020-000000000-00000     Document Type: Review

References (190)

1. Blaese RM, Culver KW, Miller AD, et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 1995; 270: 475-80
2. Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 2003; 4: 346-58 (Pubitemid 36538359)
3. Kay MA, Glorioso JC, Naldini L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med 2001; 7: 33-40 (Pubitemid 32095639)
4. Barquinero J, Eixarch H, Perez-Melgosa M. Retroviral vectors: new applications for an old tool. Gene Ther 2004; 11 (Suppl. 1): S3-9 (Pubitemid 39359441)
5. Daniel R, Smith JA. Integration site selection by retroviral vectors: molecular mechanism and clinical consequences. Hum Gene Ther 2008; 19: 557-68 (Pubitemid 351913525)
6. St George JA.Gene therapy progress and prospects: adenoviral vectors. Gene Ther 2003; 10: 1135-41 (Pubitemid 36896588)
7. Hacein-Bey-Abina S, Le Deist F, Carlier F, et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002; 346: 1185-93 (Pubitemid 34984579)
8. Aiuti A, Cattaneo F, Galimberti S, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009; 360: 447-58
9. Hacein-Bey-Abina S, Hauer J, Lim A, et al. Efficacy of gene therapy for Xlinked severe combined immunodeficiency. N Engl JMed 2010; 363: 355-64
10. Danos O, Mulligan RC. Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc Natl Acad Sci U S A 1988; 85: 6460-4
11. Sinn PL, Sauter SL, McCray PBJ. Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors - design, biosafety, and production. Gene Ther 2005; 12: 1089-98 (Pubitemid 41030614)
12. Pear WS, Nolan GP, Scott ML, et al. Production of high-titer helper-free retroviruses by transient transfection. Proc Natl Acad Sci U S A 1993; 90: 8392-6 (Pubitemid 23277674)
13. Roe T, Reynolds TC, Yu G, et al. Integration of murine leukemia virus DNA depends on mitosis. EMBO J 1993; 12: 2099-108 (Pubitemid 23157725)
14. Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. 20 years of gene therapy for SCID. Nat Immunol 2010; 11: 457-60
15. Hacein-Bey-Abina S, VonKalle C, Schmidt M, et al.LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302: 415-9 (Pubitemid 37296260)
16. Howe SJ, Mansour MR, Schwarzwaelder K, et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 2008; 118: 3143-50
17. Lombardo A,Genovese P, Beausejour CM, et al. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol 2007; 25: 1298-306 (Pubitemid 350076515)
18. Frankel AD, Young JA. HIV-1: fifteen proteins and an RNA. Annu Rev Biochem 1998; 67: 1-25 (Pubitemid 28411121)
19. Lewis PF, Emerman M. Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus. J Virol 1994; 68: 510-6
20. Naldini L, Blomer U, Gallay P, et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272: 263-7 (Pubitemid 26119138)
21. Vigna E, Naldini L. Lentiviral vectors: excellent tools for experimental gene transfer and promising candidates for gene therapy. J Gene Med 2000; 2: 308-16
22. Má trai J, Chuah MK, VandenDriessche T. Recent advances in lentiviral vector development and applications. Mol Ther 2010; 18: 477-90
23. Xu K, Ma H, McCown TJ, et al. Generation of a stable cell line producing high-titer self-inactivating lentiviral vectors. Mol Ther 2001; 3: 97-104 (Pubitemid 32509810)
24. Thomas S, Stauss HJ, Morris EC. Molecular immunology lessons from therapeutic T-cell receptor gene transfer. Immunology 2010; 129: 170-7
25. Qasim W, Mackey T, Sinclair J, et al. Lentiviral vectors for T-cell suicide gene therapy: preservation of T-cell effector function after cytokine-mediated transduction. Mol Ther 2007; 15: 355-60 (Pubitemid 46111886)
26. Cavalieri S, Cazzaniga S, Geuna M, et al. Human T lymphocytes transduced by lentiviral vectors in the absence of TCR activation maintain an intact immune competence. Blood 2003; 102: 497-505 (Pubitemid 36841966)
27. Frecha C, Levy C, Cosset FL, et al. Advances in the field of lentivector-based transduction of T and B lymphocytes for gene therapy. Mol Ther 2010; 18: 1748-57
28. Kaiser J. Gene therapy: beta-thalassemia treatment succeeds, with a caveat. Science 2009; 326: 1468-9
29. Cartier N, Hacein-Bey-Abina S, Bartholomae CC, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009; 326: 818-23
30. Mergia A, Heinkelein M. Foamy virus vectors. Curr Top Microbiol Immunol 2003; 277: 131-59 (Pubitemid 36612744)
31. Trobridge G, Josephson N, Vassilopoulos G, et al. Improved foamy virus vectors with minimal viral sequences. Mol Ther 2002; 6: 321-8 (Pubitemid 36305333)
32. Erlwein O, McClure MO. Progress and prospects: foamy virus vectors enter a new age. Gene Ther 2010; 17: 1423-9
33. Trobridge GD. Foamy virus vectors for gene transfer. Expert Opin Biol Ther 2009; 9: 1427-36
34. Bauer Jr TR, Allen JM,Hai M, et al. Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors. Nat Med 2008; 14: 93-7
35. Si Y, Pulliam AC, Linka Y, et al. Overnight transduction with foamyviral vectors restores the long-term repopulating activity of Fancc-/- stem cells. Blood 2008; 112: 4458-65
36. Rowe WP, Huebner RJ, Gilmore LK, et al. Isolation of a cytopathogenic agent from human adenoids undergoing spontaneous degeneration in tissue culture. Proc Soc Exp Biol Med 1953; 84: 570-3
37. Graham FL, Smiley J, Russell WC, et al. Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J Gen Virol 1977; 36: 59-74
38. Arnberg N. Adenovirus receptors: implications for tropism, treatment and targeting. Rev Med Virol 2009; 19: 165-78
39. Sharma A, Li X, Bangari DS, et al. Adenovirus receptors and their implications in gene delivery. Virus Res 2009; 143: 184-94
40. Flint J, Shenk T. Viral transactivating proteins. Ann Rev Genet 1997; 31: 177-212 (Pubitemid 28023706)
41. Danthinne X, Imperiale MJ. Production of first generation adenovirus vectors: a review. Gene Ther 2000; 7: 1707-14
42. Nevins JR. Mechanism of activation of early viral transcription by the adenovirus E1A gene product. Cell 1981; 26: 213-20 (Pubitemid 12244499)
43. Bett AJ, Haddara W, Prevec L, et al. An efficient and flexible system for construction of adenovirus vectors with insertions or deletions in early regions 1 and 3. Proc Natl Acad Sci U S A 1994; 91: 8802-6
44. Bergelson JM, Cunningham JA, Droguett G, et al. Isolation of a common receptor for Coxsackie B viruses and adenoviruses 2 and 5. Science 1997; 275: 1320-3 (Pubitemid 27114163)
45. Leopold PL, Kreitzer G, Miyazawa N, et al. Dynein- and microtubulemediated translocation of adenovirus serotype 5 occurs after endosomal lysis. Hum Gene Ther 2000; 11: 151-65 (Pubitemid 30044048)
46. He TC, Zhou S, da Costa LT, et al. A simplified system for generating recombinant adenoviruses. Proc Natl Acad Sci U S A 1998; 95: 2509-14 (Pubitemid 28145972)
47. Spergel JM, Chen Kiang S. Interleukin 6 enhances a cellular activity that functionally substitutes for E1A protein in transactivation. Proc Natl Acad Sci U S A 1991; 88: 6472-6 (Pubitemid 21914968)
48. Yang Y, Nunes FA, Berencsi K, et al. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci U S A 1994; 91: 4407-11 (Pubitemid 24143442)
49. Liu Q, Muruve DA. Molecular basis of the inflammatory response to adenovirus vectors. Gene Ther 2003; 10: 935-40 (Pubitemid 36722427)
50. Kochanek S, Schiedner G, Volpers C. High-capacity 'gutless' adenoviral vectors. Curr Opin Mol Ther 2001; 3: 454-63
51. Palmer DJ, Ng P. Helper-dependent adenoviral vectors for gene therapy. Hum Gene Ther 2005; 16: 1-16 (Pubitemid 40250327)
52. Kim IH, Jozkowicz A, Piedra PA, et al. Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector. Proc Natl Acad Sci U S A 2001; 98: 13282-7 (Pubitemid 33051354)
53. Brunetti Pierri N, Palmer DJ, Beaudet AL, et al. Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates. Hum Gene Ther 2004; 15: 35-46 (Pubitemid 38147187)
54. Yu W, Fang H. Clinical trials with oncolytic adenovirus in China. Curr Cancer Drug Targets 2007; 7: 141-8 (Pubitemid 46348628)
55. Nemunaitis J, Khuri F, Ganly I, et al. Phase II trial of intratumoral administration of ONYX-015, a replication-selective adenovirus, in patients with refractory head and neck cancer. J Clin Oncol 2001; 19: 289-98 (Pubitemid 32112839)
56. Atchison RW, Casto BC, Hammon WM. Adenovirus-associated defective virus particles. Science 1965; 149: 754-6
57. Hoggan MD, Blacklow NR, Rowe WP. Studies of small DNA viruses found in various adenovirus preparations: physical, biological, and immunological characteristics. Proc Natl Acad Sci U S A 1966; 55: 1467-74
58. Daya S, Berns KI. Gene therapy using adeno-associated virus vectors. Clin Microbiol Rev 2008; 21: 583-93
59. Hermonat PL, Muzyczka N. Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells. Proc Natl Acad Sci U S A 1984; 81: 6466-70 (Pubitemid 15221855)
60. Smith RH. Adeno-associated virus integration: virus versus vector. Gene Ther 2008; 15: 817-22 (Pubitemid 351712614)
61. Samulski RJ, Chang LS, Shenk T. Helper-free stocks of recombinant adenoassociated viruses: normal integration does not require viral gene expression. J Virol 1989; 63: 3822-8 (Pubitemid 19205454)
62. Grimm D, Kern A, Rittner K, et al. Novel tools for production and purification of recombinant adenoassociated virus vectors. Hum Gene Ther 1998; 9: 2745-60 (Pubitemid 29027018)
63. Xiao X, Li J, Samulski RJ. Production of high-titer recombinant adenoassociated virus vectors in the absence of helper adenovirus. J Virol 1998; 72: 2224-32 (Pubitemid 28100780)
64. Kay MA, Manno CS, Ragni MV, et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 2000; 24: 257-61 (Pubitemid 30132193)
65. Manno CS, Chew AJ, Hutchison S, et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 2003; 101: 2963-72 (Pubitemid 36857981)
66. Manno CS, Pierce GF, Arruda VR, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006; 12: 342-7 (Pubitemid 43355084)
67. Epstein AL. Progress and prospects: biological properties and technological advances of herpes simplex virus type 1-based amplicon vectors. Gene Ther 2009; 16: 709-15
68. Manservigi R, Argnani R, Marconi P. HSV recombinant vectors for gene therapy. Open Virol J 2010; 4: 123-56
69. Burton EA, Bai Q, Goins WF, et al. Replication-defective genomic herpes simplex vectors: design and production. Curr Opin Biotechnol 2002; 13: 424-8 (Pubitemid 35379926)
70. Friedman GK, Pressey JG, Reddy AT, et al. Herpes simplex virus oncolytic therapy for pediatric malignancies. Mol Ther 2009; 17: 1125-35
71. Shah AC, Benos D, Gillespie GY, et al. Oncolytic viruses: clinical applications as vectors for the treatment of malignant gliomas. J Neurooncol 2003; 65: 203-26 (Pubitemid 37526814)
72. Glorioso JC, Fink DJ. Herpes vector-mediated gene transfer in the treatment of chronic pain. Mol Ther 2009; 17: 13-8
73. Goss JR, Gold MS, Glorioso JC. HSV vector-mediated modification of primary nociceptor afferents: an approach to inhibit chronic pain. Gene Ther 2009; 16: 493-501
74. Lieber A, Steinwaerder DS, Carlson CA, et al. Integrating adenovirus-adenoassociated virus hybrid vectors devoid of all viral genes. J Virol 1999; 73: 9314-24 (Pubitemid 29487087)
75. Recchia A, Parks RJ, Lamartina S, et al. Site-specific integration mediated by a hybrid adenovirus/adeno-associated virus vector. Proc Natl Acad Sci U S A 1999; 96: 2615-20 (Pubitemid 29148766)
76. Oehmig A, Fraefel C, Breakefield XO, et al. Herpes simplex virus type 1 amplicons and their hybrid virus partners, EBV, AAV, and retrovirus. Curr Gene Ther 2004; 4: 385-408 (Pubitemid 39534754)
77. Fisher KD, Stallwood Y, Green NK, et al. Polymer-coated adenovirus permits efficient retargeting and evades neutralising antibodies. Gene Ther 2001; 8: 341-8 (Pubitemid 32241933)
78. Avery OT, MacLeod CM, McCarty M. Studies on the chemical nature of the substance inducing transformation of pneumococcal types: inductions of transformation by a deoxyribonucleic acid fraction isolated from pneumococcus type III. J Exp Med 1979; 149: 297-326
79. Graham FL, van der Eb AJ. Transformation of rat cells by DNA of human adenovirus 5. Virology 1973; 54: 536-9
80. Pathak A, Patnaik S, Gupta Kailash C. Recent trends in non-viral vectormediated gene delivery. Biotechnol J 2009; 4: 1559-72
81. Mintzer MA, Simanek EE. Nonviral vectors for gene delivery. Chem Rev 2009; 109: 259-302
82. Bangham AD, Standish MM, Watkins JC. Diffusion of univalent ions across the lamellae of swollen phospholipids. J Mol Biol 1965; 13: 238-52
83. Felgner PL, Gadek TR, Holm M, et al. Lipofection: a highly efficient, lipidmediated DNA-transfection procedure. Proc Natl Acad Sci U S A 1987; 84: 7413-7
84. Simoes S, Filipe A, Faneca H, et al. Cationic liposomes for gene delivery. Expert Opin Drug Deliv 2005; 2: 237-54 (Pubitemid 41135785)
85. Liu D, Ren T, Gao X. Cationic transfection lipids. Curr Med Chem 2003; 10: 1307-15 (Pubitemid 36763938)
86. Montier T, Benvegnu T, Jaffres PA, et al. Progress in cationic lipid-mediated gene transfection: a series of bio-inspired lipids as an example. Curr Gene Ther 2008; 8: 296-312
87. Zhou X, Huang L. DNA transfection mediated by cationic liposomes containing lipopolylysine: characterization and mechanism of action. Biochim Biophys Acta 1994; 1189: 195-203 (Pubitemid 24042719)
88. Xu L, Huang CC, Huang W, et al. Systemic tumor-targeted gene delivery by anti-transferrin receptor scFv-immunoliposomes. Mol Cancer Ther 2002; 1: 337-46
89. Pardridge WM. Re-engineering biopharmaceuticals for delivery to brain with molecular Trojan horses. Bioconjug Chem 2008; 19: 1327-38 (Pubitemid 352032329)
90. Durcan N, Murphy C, Cryan SA. Inhalable siRNA: potential as a therapeutic agent in the lungs. Mol Pharm 2008; 5: 559-66
91. Stopeck AT, Hersh EM, Akporiaye ET, et al. Phase I study of direct gene transfer of an allogeneic histocompatibility antigen, HLA-B7, in patients with metastatic melanoma. J Clin Oncol 1997; 15: 341-9 (Pubitemid 27020592)
92. Stopeck AT, Jones A, Hersh EM, et al. Phase II study of direct intralesional gene transfer of allovectin-7, an HLA-B7/beta2-microglobulin DNAliposome complex, in patients with metastatic melanoma. Clin Cancer Res 2001; 7: 2285-91 (Pubitemid 32751626)
93. Boussif O, Lezoualc'h F, Zanta MA, et al. 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: 7297-301
94. Goula D, Remy JS, Erbacher P, et al. Size, diffusibility and transfection performance of linear PEI/DNA complexes in the mouse central nervoussystem. Gene Ther 1998; 5: 712-7 (Pubitemid 28256824)
95. Chemin I, Moradpour D, Wieland S, et al. Liver-directed gene transfer: a linear polyethlenimine derivative mediates highly efficient DNA delivery to primary hepatocytes in vitro and in vivo. J Viral Hepat 1998; 5: 369-75 (Pubitemid 28529876)
96. Haensler J, Szoka FC J. Polyamidoamine cascade polymers mediate efficient transfection of cells in culture. Bioconjug Chem 1993; 4: 372-9
97. Tang MX, Redemann CT, Szoka FC J. In vitro gene delivery by degraded polyamidoamine dendrimers. Bioconjug Chem 1996; 7: 703-14 (Pubitemid 27012712)
98. Rudolph C, Lausier J, Naundorf S, et al. In vivo gene delivery to the lung using polyethylenimine and fractured polyamidoamine dendrimers. J Gene Med 2000; 2: 269-78
99. Nimesh S, KumarR, Chandra R. Novel polyallylamine-dextran sulfate-DNA nanoplexes: highly efficient non-viral vector for gene delivery. Int J Pharm 2006; 320: 143-9 (Pubitemid 44128699)
100. Erbacher P, Zou S, Bettinger T, et al. Chitosan-based vector/DNA complexes for gene delivery: biophysical characteristics and transfection ability. Pharm Res 1998; 15: 1332-9 (Pubitemid 28427918)
101. Schatzlein AG, Zinselmeyer BH, Elouzi A, et al. Preferential liver gene expression with polypropylenimine dendrimers. J Control Release 2005; 101: 247-58 (Pubitemid 39600987)
102. Balicki D, Beutler E. Histone H2A significantly enhances in vitro DNA transfection. Mol Med 1997; 3: 782-7 (Pubitemid 28047421)
103. Balicki D, Putnam CD, Scaria PV, et al. Structure and function correlation in histoneH2A peptide-mediated gene transfer. Proc Natl Acad SciUSA2002; 99: 7467-71 (Pubitemid 34568714)
104. Sweeney P, Karashima T, Ishikura H, et al. Efficient therapeutic gene delivery after systemic administration of a novel polyethylenimine/DNA vector in an orthotopic bladder cancer model. Cancer Res 2003; 63: 4017-20\ (Pubitemid 36917921)
105. Konstan MW, Davis PB, Wagener JS, et al. Compacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete cystic fibrosis transmembrane regulator reconstitution. Hum Gene Ther 2004; 15: 1255-69 (Pubitemid 40052437)
106. Avrameas A, Ternynck T, Nato F, et al. Polyreactive anti-DNA monoclonal antibodies and a derived peptide as vectors for the intracytoplasmic and intranuclear translocation of macromolecules. Proc Natl Acad Sci U S A 1998; 95: 5601-6 (Pubitemid 28224141)
107. Lee RJ, Huang L. Folate-targeted, anionic liposome-entrapped polylysinecondensedDNAfor tumor cell-specific gene transfer. J Biol Chem 1996; 271: 8481-7 (Pubitemid 26108687)
108. Sorgi FL, Bhattacharya S, Huang L. Protamine sulfate enhances lipidmediated gene transfer. Gene Ther 1997; 4: 961-8 (Pubitemid 27437722)
109. Lee LK, Williams CL, Devore D, et al. Poly(propylacrylic acid) enhances cationic lipid-mediated delivery of antisense oligonucleotides. Biomacromolecules 2006; 7: 1502-8 (Pubitemid 43811206)
110. Murphy EA, Waring AJ, Haynes SM, et al. Compaction of DNA in an anionic micelle environment followed by assembly into phosphatidylcholine liposomes. Nucleic Acids Res 2000; 28: 2986-92 (Pubitemid 30599274)
111. Li S, Huang L. In vivo gene transfer via intravenous administration of cationic lipid-protamine-DNA (LPD) complexes. Gene Ther 1997; 4: 891-900 (Pubitemid 27437714)
112. Wolff JA, Malone RW, Williams P, et al. Direct gene transfer into mouse muscle in vivo. Science 1990; 247: 1465-8
113. Choate KA, Khavari PA. Direct cutaneous gene delivery in a human genetic skin disease. Hum Gene Ther 1997; 8: 1659-65
114. Park SW, Gwon HC, Jeong JO, et al. Intracardiac echocardiographic guidance and monitoring during percutaneous endomyocardial gene injection in porcine heart. Hum Gene Ther 2001; 2: 893-903
115. Hickman MA, Malone RW, Lehmann-Bruinsma K, et al. Gene expression following direct injection of DNA into liver. Hum Gene Ther 1994; 5: 1477-83
116. Habib NA, Ding SF, el-Masry R, et al. Preliminary report: the short-term effects of direct p53 DNA injection in primary hepatocellular carcinomas. Cancer Detect Prev 1996; 20: 103-7 (Pubitemid 3042342)
117. Prausnitz MR, Mikszta JA, Cormier M, et al. Microneedle-based vaccines. Curr Top Microbiol Immunol 2009; 333: 369-93
118. Hartikka J, SawdeyM, Cornefert Jensen F, et al. An improved plasmid DNA expression vector for direct injection into skeletal muscle. Hum Gene Ther 1996; 7: 1205-17 (Pubitemid 26245507)
119. Klein RM, Wolf ED, Wu R, et al. High-velocity microprojectiles for delivering nucleic acids into living cells: 1987. Biotechnology 1992; 24: 384-6
120. O'Brien J, Lummis SC. An improved method of preparing microcarriers for biolistic transfection. Brain Res Brain Res Protoc 2002; 10: 12-5
121. Yang NS, Burkholder J, Roberts B, et al. In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment. Proc Natl Acad Sci U S A 1990; 87: 9568-72 (Pubitemid 120016484)
122. Wang S, Joshi S, Lu S. Delivery of DNA to skin by particle bombardment. Methods Mol Biol 2004; 245: 185-96
123. Kim D, Hoory T, Monie A, et al. Enhancement of DNA vaccine potency through coadministration of CIITA DNA with DNA vaccines via gene gun. J Immunol 2008; 180: 7019-27
124. Roberts LK, Barr LJ, Fuller DH, et al. Clinical safety and efficacy of a powdered hepatitis B nucleic acid vaccine delivered to the epidermis by a commercial prototype device. Vaccine 2005; 23: 4867-78 (Pubitemid 41242881)
125. Drape RJ, Macklin MD, Barr LJ, et al. Epidermal DNA vaccine for influenza is immunogenic in humans. Vaccine 2006; 24: 4475-81 (Pubitemid 43776768)
126. Cassaday RD, Sondel PM, King DM, et al. A phase I study of immunization using particle-mediated epidermal delivery of genes for gp100 and GM-CSF into uninvolved skin of melanoma patients. Clin Cancer Res 2007; 13: 540-9 (Pubitemid 46225360)
127. Yager EJ,Dean HJ, Fuller DH. Prospects for developing an effective particlemediated DNA vaccine against influenza. Expert Rev Vaccines 2009; 8: 1205-20
128. Kaur T, Slavcev RA, Wettig SD. Addressing the challenge: current and future directions in ovarian cancer therapy. Curr Gene Ther 2009; 9: 434-58
129. Neumann E, Schaefer-Ridder M, Wang Y, et al. Gene transfer into mouse lyoma cells by electroporation in high electric fields. EMBO J 1982; 1: 841-5
130. Favard C, Dean DS, RolsM-P. Electrotransfer as a nonviral method of gene delivery. Curr Gene Ther 2007; 7: 67-77 (Pubitemid 46322289)
131. Heller LC, Ugen K, Heller R. Electroporation for targeted gene transfer. Expert Opin Drug Deliv 2005; 2: 255-68 (Pubitemid 41130881)
132. van Drunen Littel-van den Hurk S, Hannaman D. Electroporation for DNA immunization: clinical application. Expert Rev Vaccines 2010; 9: 503-17
133. Wells DJ. Gene therapy progress and prospects: electroporation and other physical methods. Gene Ther 2004; 11: 1363-9 (Pubitemid 39263889)
134. Heller R, Jaroszeski MJ, Glass LF, et al. Phase I/II trial for the treatment of cutaneous and subcutaneous tumors using electrochemotherapy. Cancer 1996; 77: 964-71 (Pubitemid 26061293)
135. Bodles-Brakhop AM, Heller R, Draghia-Akli R. Electroporation for the delivery of DNA-based vaccines and immunotherapeutics: current clinical developments. Mol Ther 2009; 17: 585-92
136. Kim HJ, Greenleaf JF, Kinnick RR, et al. Ultrasound-mediated transfection of mammalian cells. Hum Gene Ther 1996; 7: 1339-46 (Pubitemid 26318673)
137. Tata DB, Dunn F, Tindall DJ. Selective clinical ultrasound signals mediate differential gene transfer and expression in two human prostate cancer cell lines: LnCap and PC-3. Biochem Biophys Res Commun 1997; 234: 64-7 (Pubitemid 27267532)
138. Bao S, Thrall BD, Gies RA, et al. In vivo transfection of melanoma cells by lithotripter shock waves. Cancer Res 1998; 58: 219-21 (Pubitemid 28047057)
139. Huber PE, Jenne J,Debus J, et al.Acomparison of shockwave and sinusoidalfocused ultrasound-induced localized transfection of HeLa cells. Ultrasound Med Biol 1999; 25: 1451-7
140. Sonoda S, Tachibana K, Uchino E, et al. Gene transfer to corneal epithelium and keratocytes mediated by ultrasound with microbubbles. Invest Ophthalmol Vis Sci 2006; 47: 558-64 (Pubitemid 46768382)
141. Hynynen K, McDannold N, Martin H, et al. The threshold for brain damage in rabbits induced by bursts of ultrasound in the presence of an ultrasound contrast agent (Optison).Ultrasound Med Biol 2003; 29: 473-81 (Pubitemid 36434336)
142. Manome Y, Nakayama N, Nakayama K, et al. Insonation facilitates plasmid DNA transfection into the central nervous system and microbubbles enhance the effect. Ultrasound Med Biol 2005; 31: 693-702 (Pubitemid 40615189)
143. Shimamura M, Sato N, Taniyama Y, et al. Development of efficient plasmid DNA transfer into adult rat central nervous system using microbubbleenhanced ultrasound. Gene Ther 2004; 11: 1532-9 (Pubitemid 39359461)
144. Shimamura M, Sato N, Taniyama Y, et al. Gene transfer into adult rat spinal cord using naked plasmid DNA and ultrasound microbubbles. J Gene Med 2005; 7: 1468-74 (Pubitemid 41672405)
145. Sheyn D, Kimelman-Bleich N, Pelled G, et al. Ultrasound-based nonviral gene delivery induces bone formation in vivo. Gene Ther 2007; 15: 257-66 (Pubitemid 351223462)
146. Guo H, Leung JCK, Chan LYY, et al. Ultrasound-contrast agent mediated naked gene delivery in the peritoneal cavity of adult rat. Gene Ther 2007; 14: 1712-20 (Pubitemid 350238448)
147. Hou C-C, Wang W, Huang XR, et al. Ultrasound-microbubble-mediated gene transfer of inducible Smad7 blocks transforming growth factor-beta signaling and fibrosis in rat remnant kidney. Am J Pathol 2005; 166: 761-71 (Pubitemid 40315633)
148. Chen S, Ding J-H, Bekeredjian R, et al. Efficient gene delivery to pancreatic islets with ultrasonic microbubble destruction technology. Proc Natl Acad Sci U S A 2006; 103: 8469-74 (Pubitemid 43838479)
149. Shen ZP, Brayman AA, Chen L, et al. Ultrasound with microbubbles enhances gene expression of plasmid DNA in the liver via intraportal delivery. Gene Ther 2008; 15: 1147-55
150. Miao CH, Brayman AA, Loeb KR, et al. Ultrasound enhances gene delivery of human factor IX plasmid. Hum Gene Ther 2005; 16: 893-905 (Pubitemid 40982337)
151. Ohta S, Suzuki K, Tachibana K, et al. Microbubble-enhanced sonoporation: efficient gene transduction technique for chick embryos. Genesis 2003; 37: 91-101 (Pubitemid 37413695)
152. Nakashima M, Tachibana K, Iohara K, et al. Induction of reparative dentin formation by ultrasound-mediated gene delivery of growth/differentiation factor 11. Hum Gene Ther 2003; 14: 591-7 (Pubitemid 36438942)
153. Taniyama Y, Tachibana K, Hiraoka K, et al. Development of safe and efficient novel nonviral gene transfer using ultrasound: enhancement of transfection efficiency of naked plasmidDNAin skeletal muscle. Gene Ther 2002; 9: 372-80 (Pubitemid 34414035)
154. Lu QL, Liang H-D, Partridge T, et al. Microbubble ultrasound improves the efficiency of gene transduction in skeletal muscle in vivo with reduced tissue damage. Gene Ther 2003; 10: 396-405 (Pubitemid 36372447)
155. Bekeredjian R, Chen S, Frenkel PA, et al. Ultrasound-targeted microbubble destruction can repeatedly direct highly specific plasmid expression to the heart. Circulation 2003; 108: 1022-6 (Pubitemid 37048235)
156. Bekeredjian R, Grayburn PA, Shohet RV. Use of ultrasound contrast agents for gene or drug delivery in cardiovascular medicine. J Am Coll Cardiol 2005; 45: 329-35 (Pubitemid 40186780)
157. Suzuki R, Takizawa T, Negishi Y, et al. Tumor specific ultrasound enhanced gene transfer in vivo with novel liposomal bubbles. J Control Release 2008; 125: 137-44 (Pubitemid 350299602)
158. Hayashi S, Mizuno M, Yoshida J, et al. Effect of sonoporation on cationic liposome-mediated IFNbeta gene therapy for metastatic hepatic tumors of murine colon cancer. Cancer Gene Ther 2009; 16: 638-43
159. Suzuki R, Namai E, Oda Y, et al. Cancer gene therapy by IL-12 gene delivery using liposomal bubbles and tumoral ultrasound exposure. J Control Release 2010; 142: 245-50
160. Barreiro O, Aguilar Rio J, Tejera E, et al. Specific targeting of human inflamed endothelium and in situ vascular tissue transfection by the use of ultrasound contrast agents. JACC Cardiovasc Imaging 2009; 2: 997-1005
161. Kodama T, Aoi A, Watanabe Y, et al. Evaluation of transfection efficiency in skeletal muscle using nano/microbubbles and ultrasound. Ultrasound Med Biol 2010; 36: 1196-205
162. Zeira E, Manevitch A, Khatchatouriants A, et al. Femtosecond infrared laseran efficient and safe in vivo gene delivery system for prolonged expression. Mol Ther 2003; 8: 342-50 (Pubitemid 37062844)
163. Chakravarty P, Qian W, El-Sayed MA, et al. Delivery of molecules into cells using carbon nanoparticles activated by femtosecond laser pulses. Nat Nanotechnol 2010; 5: 607-11
164. Plank C, Anton M, Rudolph C, et al. Enhancing and targeting nucleic acid delivery by magnetic force. Expert Opin Biol Ther 2003; 3: 745-58 (Pubitemid 36934974)
165. Dobson J. Gene therapy progress and prospects:magnetic nanoparticle-based gene delivery. Gene Ther 2006; 13: 283-7 (Pubitemid 43223096)
166. Mykhaylyk O, Antequera YS, Vlaskou D, et al. Generation of magnetic nonviral gene transfer agents and magnetofection in vitro. Nat Protoc 2007; 2: 2391-411
167. Scherer F, Anton M, Schillinger U, et al. Magnetofection: enhancing and targeting gene delivery by magnetic force in vitro and in vivo. Gene Ther 2002; 9: 102-9 (Pubitemid 34189525)
168. Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by
169. Zhang G, Budker V, Wolff JA. High levels of foreign gene xpression in hepatocytes after tail vein injections of naked plasmid DNA. Hum Gene Ther 1999; 10: 1735-7 (Pubitemid 29328217)
170. Zhang G, Gao X, Song YK, et al. Hydroporation as the mechanism of hydrodynamic delivery. Gene Ther 2004; 11: 675-82 (Pubitemid 38519275)
171. Suda T, Liu D. Hydrodynamic gene delivery: its principles and applications. Mol Ther 2007; 15: 2063-9 (Pubitemid 350148920)
172. Brunetti-Pierri N, Stapleton GE, Palmer DJ, et al. Pseudo-hydrodynamic delivery of helper-dependent adenoviral vectors into non-human primates for liver-directed gene therapy. Mol Ther 2007; 15: 732-40 (Pubitemid 46431993)
173. Brunetti-Pierri N, Palmer DJ, Mane V, et al. Increased hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors. Mol Ther 2005; 12: 99-106 (Pubitemid 40862361)
174. Condiotti R, Curran MA, Nolan GP, et al. Prolonged liver-specific transgene expression by a non-primate lentiviral vector. Biochem Biophys Res Commun 2004; 320: 998-1006 (Pubitemid 38887242)
175. Arad U, Zeira E, El-Latif MA, et al. Liver-targeted gene therapy by SV40- based vectors using the hydrodynamic injection method. Hum Gene Ther 2005; 16: 361-71 (Pubitemid 40471896)
176. Herweijer H, Wolff JA. Gene therapy progress and prospects: hydrodynamic gene delivery. Gene Ther 2007; 14: 99-107 (Pubitemid 46043816)
177. Qiao C, Li J, Zheng H, et al. Hydrodynamic limb vein injection of adenoassociated virus serotype 8 vector carrying caninemyostatin propeptide gene into normal dogs enhances muscle growth. Hum Gene Ther 2009; 20: 1-10
178. Chen L, Zhu F, Li J, et al. The enhancing effects of the light chain on heavy chain secretion in split delivery of factor VIII gene. Mol Ther 2007; 15: 1856-62 (Pubitemid 47434194)
179. Doenecke A, Kromer A, Scherer MN, et al. AAV plasmid DNA simplifies liver-directed in vivo gene therapy: comparison of expression levels after plasmid DNA-, adeno-associated virus- and adenovirus-mediated liver transfection. J Gene Med 2010; 12: 810-7
180. Suda T, Kamimura K, Kubota T, et al. Progress toward liver-based gene therapy. Hepatol Res 2009; 39: 325-40
181. Eastman SJ, Baskin KM, Hodges BL, et al. Development of catheter-based procedures for transducing the isolated rabbit liver with plasmid DNA. Hum Gene Ther 2002; 13: 2065-77 (Pubitemid 35417351)
182. Yoshino H, Hashizume K, Kobayashi E. Naked plasmidDNA transfer to the porcine liver using rapid injection with large volume. Gene Ther 2006; 13: 1696-702 (Pubitemid 44849034)
183. Alino SF, Herrero MJ, Noguera I, et al. Pig liver gene therapy by noninvasive interventionist catheterism. Gene Ther 2007; 14: 334-43 (Pubitemid 46231927)
184. Fabre JW, Grehan A, Whitehorne M, et al. Hydrodynamic gene delivery to the pig liver via an isolated segment of the inferior vena cava. Gene Ther 2008; 15: 452-62 (Pubitemid 351331003)
185. Suda T, Suda K, Liu D. Computer-assisted hydrodynamic gene delivery. Mol Ther 2008; 16: 1098-104 (Pubitemid 351737075)
186. Kamimura K, Suda T, Xu W, et al. Image-guided, lobe-specific hydrodynamic gene delivery to swine liver. Mol Ther 2009; 17: 491-9
187. Kamimura K, Guisheng Z, Liu D. Image-guided, intravascular hydrodynamic gene delivery to skeletal muscle in pigs. Mol Ther 2010; 18: 93-100
188. The Journal of Gene Medicine. Gene therapy clinical trials worldwide [online]. Available from URL: http://www.wiley.com/legacy/wileychi/genmed/ clinical/ [Accessed 2011 Aug 2]
189. Marshall E. Gene therapy death prompts review of adenovirus vector. Science 1999; 286: 2244-5 (Pubitemid 30016842)
190. Check E. Second cancer case halts gene-therapy trials. Nature 2003; 421: 305 (Pubitemid 36157908)