Gene therapy

Tissue Engineering

Volumn , Issue , 2007, Pages 213-228

  Munson, J M ^a   Godbey, W T ^a  

^a Tulane University   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 85035212713     PISSN: None     EISSN: None     Source Type: Book    
DOI: None     Document Type: Chapter

References (124)

1. Wolff, J. and Lederberg, J., An early history of gene transfer and therapy, Hum. Gene Ther., 5, 469, 1994.
2. Little E., et al., The glucose-regulated proteins (GRP78 and GRP94): functions, gene regulation, and applications, Crit. Rev. Eukaryot. Gene Expr., 4, 1-18, 1994.
3. Haider, M., Megeed, Z., and Ghandehari, H., Genetically engineered polymers: status and prospects for controlled release, J. Control. Release, 95, 1, 2004.
4. Sun, X., Wu, Z., and Hu, J., Suicide gene therapy of hepatocellular carcinoma and delivery procedure and route of therapeutic gene in vivo, HPBD Int., 1, 373, 2003.
5. Nakaya, T. et al., Decoy approach usingRNA-DNA chimera oligonucleotides to inhibit the regulatory function of humanimmunodeficiency virus type 1 Rev protein, Antimicrob. Agents Chemother., 41, 319, 1997.
6. Browning, C. et al., Potent inhibition of human immunodeficiency virus type 1 (HIV-1) gene expression and virus production by an HIV-2 Tat activation-response RNA decoy, J. Virol., 73, 5191, 1999.
7. Pace, B.S., Qian, X., and Ofori-Acquah, S.F., Selective inhibition of beta-globin RNA transcripts by antisense RNA molecules, Cell. Mol. Biol., 50, 43, 2004.
8. Li, M.J. et al., Specific killing of Ph+ chronicmyeloid leukemia cells by a lentiviral vector-delivered anti-bcr/abl small hairpin RNA, Oligonucleotides, 13, 401, 2003.
9. Anderson, J. et al., Potent suppression of HIV type 1 infection by a short hairpin anti-CXCR4 siRNA, AIDS Res. Hum. Retroviruses, 19, 699, 2003.
10. Elbashir, S.M., Lendeckel, W., and Tuschl, T., RNA interference is mediated by 21- and 22- nucleotide RNAs, Genes Dev., 15, 188, 2001.
11. Elbashir, S.M. et al., Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells, Nature, 411, 494, 2001.
12. Miller, V.M. et al., Allele-specific silencing of dominant disease genes, Proc. Natl Acad. Sci. USA, 100, 7195, 2003.
13. Miller, V. et al., Allele-specific silencing of dominant disease genes, Proc. Natl Acad. Sci. USA, 100, 7195, 2003.
14. Douglas, J.T., Adenovirus-mediated gene delivery: an overview, Meth. Mol. Biol., 246, 3, 2004.
15. Huser, A. and Hofmann, C., Baculovirus vectors: novel mammalian cell gene-delivery vehicles and their applications, Am. J. Pharmacogenom., 3, 53, 2003.
16. Boldogkoi, Z. and Nogradi, A., Gene and cancer therapy - pseudorabies virus: a novel research and therapeutic tool? Curr. Gene Ther., 3, 155, 2003.
17. Goins, W.F. et al., Delivery using herpes simplex virus: an overview, Meth. Mol. Biol., 246, 257, 2004.
18. Lu, Y., Recombinant adeno-associated virus as delivery vector for gene therapy - a review, Stem Cells Dev., 13, 133 2004.
19. Gafni, Y. et al., Gene therapy platform for bone regeneration using an exogenously regulated, AAV-2-based gene expression system, Mol. Ther., 9, 587, 2004.
20. Hodge, J.W. et al., Modified vaccinia virus ankara recombinants are as potent as vaccinia recombinants in diversified prime and boost vaccine regimens to elicit therapeutic antitumor responses, Cancer Res., 63, 7942, 2003.
21. Hu, Y. et al., Yaba-like disease virus: an alternative replicating poxvirus vector for cancer gene therapy. J. Virol., 75, 10300, 2001.
22. Cockrell, A.S. and Kafri, T., HIV-1 vectors: fulfillment of expectations, further advancements, and still a way to go, Curr. HIV Res., 1, 419, 2003.
23. Cheng, L. et al., Human immunodeficiency virus type 2 (HIV-2) vector-mediated in vivo gene transfer into adult rabbit retina, Curr. Eye Res., 24, 196, 2002.
24. Kafri, T., Gene delivery by lentivirus vectors an overview, Meth. Mol. Biol., 246, 367, 2004.
25. Wolkowicz, R., Nolan, G.P., and Curran, M.A., Lentiviral vectors for the delivery of DNA into mammalian cells, Meth. Mol. Biol., 246, 391, 2004.
26. Rainov, N.G. and Ren, H., Clinical trials with retrovirus mediated gene therapy - what have we learned? J. Neuro-oncol., 65, 227, 2003.
27. Lilley, C. et al., Multiple immediate-early gene-deficient herpes simplex virus vectors allowing efficient gene delivery to neurons in culture and widespread gene delivery to the central nervous system in vivo, J. Virol., 75, 4343, 2001.
28. Cao, B. and Huard, J., Gene transfer to skeletal muscle using herpes simplex virus-based vectors, Meth. Mol. Biol., 246, 301, 2004.
29. Billelo, J. et al., Transient disruption of intercellular junctions enables baculovirus entry into nondividing hepatocytes, J. Virol., 75, 9857, 2001.
30. Hu, Y. et al., Yaba-like disease virus: an alternative replicating poxvirus vector for cancer gene therapy, J. Virol., 75, 10500, 2001.
31. Garrett, D. et al., In utero recombinant adeno-associated virus gene transfer in mice, rats, and primates, BMC Biotech., 3, 16, 2003.
32. Ahmed, B. et al., Efficient delivery of Cre-recombinase to neurons in vivo and stable transduction of neurons using adeno-associated and lentiviral vectors, BMC Neurosci., 5, 4, 2004.
33. Mastakov, M. et al., Immunological aspects of recombinant adeno-associated virus delivery to the mammalian brain, J. Virol., 76, 8446, 2002.
34. Zufferey, R., Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery, J. Virol., 72, 9873, 1998.
35. Curran, M. et al., Efficient transduction of nondividing cells by optimized felineimmunodeficiency virus vectors, Mol. Ther., 1, 31, 2000.
36. Reiser, J. et al., Development ofmultigene and regulated lentivirus vectors, J.Virol., 74, 10589, 2000.
37. Marschall, P., Malik, N., and Larin, Z., Transfer of YACs up to 2.3 Mb intact into human cells with polyethylenimine, Gene Ther., 6, 1634, 1999.
38. Vanderbyl, S., MacDonald, N., and de Jong, G., A flow cytometry technique for measuring chromosome-mediated gene transfer, Cytometry, 44, 100, 2001.
39. Rossenberg, S. van. et al., Stable polyplexes based on arginine-containing oligopeptides for in vivo gene delivery, Nature, 11, 457, 2004.
40. Chen, Q. et al., Branched co-polymers of histidine and lysine are efficient carriers of plasmids, Nucleic Acids Res., 29, 1334, 2001.
41. Toncheva V. et al., Novel vectors for gene delivery formed by self-assembly of DNA with poly(- lysine) grafted with hydrophilic polymers, Biochim. Biophys. Acta, 1380, 354, 1998.
42. Godbey, W., Wu, K., and Mikos A., Size matters: molecular weight affects the efficiency of poly(ethylenimine) as a gene delivery vehicle, J. Biomed. Mater. Res., 45, 268, 1999.
43. Fischer, D. et al., A novel non-viral vector for DNA delivery based on low molecular weight, branched polyethylenimine: effect of molecular weight on transfection efficiency and cytotoxicity, Pharm. Res., 16, 1273, 1999.
44. Tang, G. et al., Polyethylene glycol modified polyethylenimine for improved CNS gene transfer:effects of PEGylation extent, Biomaterials, 24, 2351, 2004.
45. Boussif, O. et al., A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine, Proc. Natl Acad. Sci. USA, 92, 7297, 1995.
46. Manuel, W., Zheng, J., and Hornsby, P., Transfection by polyethyleneimine-coated microspheres, J. Drug Target., 9, 15, 2001.
47. Kuskowska-Latallo, J. et al., Efficient transfer of genetic material into mammalian cells using Starburst polyamidoamine dendrimers, Proc. Natl Acad. Sci. USA, 93, 4897, 1996.
48. Maruyama-Tabata, H. et al., Effective suicide gene therapy in vivo by EBV-based plasmid vector coupled with polyamidoamine dendrimer, Gene Ther., 7, 53, 2000.
49. Mao, H.Q. et al., Chitosan-DNA nanoparticles as gene carriers: synthesis, characterization and transfection efficiency, J. Control. Release, 70, 399, 2001.
50. Mansouri, S. et al., Chitosan-DNA nanoparticles as non-viral vectors in gene therapy: strategies to improve transfection efficacy, Eur. J. Pharm. Biopharm., 57, 1, 2004.
51. Ishii, T., Okahata, Y., and Sato, T., Mechanism of cell transfection with plasmid/chitosan complexes, Biochim. Biophys. Acta, 1514, 51, 2001.
52. Padmanabhan, K. and Smith, T.A., Preliminary investigation of modified alginates as a matrix for gene transfection in a HeLa cell model, Pharm. Dev. Technol., 7, 97, 2002.
53. Gonzalez Ferreiro M. et al., Characterization of alginate/poly-l-lysine particles as antisense oligonucleotide carriers, Int. J. Pharm., 239, 47 2002.
54. Li, Z. et al., Controlled gene delivery system based on thermosensitive biodegradable hydrogel, Pharm. Res., 20, 884, 2003.
55. Girao Da Cruz, M., Simoes, S., and Pedroso De Lima, M., Improving lipoplex-mediated gene transfer into C6 glioma cells and primary neurons, Exp. Neurol., 187, 65, 2004.
56. Liu, F., Huang, L., and Liu, D., Factors controlling the efficiency of cationic lipid-mediated transfection in vivo via intravenous administration, Gene Ther., 4, 517, 1997.
57. Farhood, H., Serbina, N., and Huang, L., The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer, Biochim. Biophys. Acta, 1235, 289, 1995.
58. King, R., Gene delivery to mammalian cells by microinjection, Meth. Mol. Biol., 245, 167, 2004.
59. Wilmut, I. et al., Viable offspring derived from fetal and adult mammalian cells, Nature, 385, 810, 1997.
60. Hosaka, K. et al., Cloned mice derived from somatic cell nuclei, Hum. Cell, 13, 237, 2000.
61. Neumann, E. et al., Gene transfer into mouse myeloma cells by electroporation in high electric fields, EMBO J., 1, 841, 1982.
62. Golzio, M., Teissié, J., and Rols, M., Direct visualization at the single-cell level of electrically mediated gene delivery, Proc. Natl Acad. Sci. USA, 99, 1292, 2002.
63. Mir, L. et al., High-efficiency gene transfer into skeletal muscle mediated by electric pulses, Proc. Natl Acad. Sci USA, 96, 4262, 1999.
64. Wang, Y. et al., Combination of electroporation and DNA/dendrimer complexes enhances gene transfer into murine cardiac transplants, Am. J. Transplant., 1, 334, 2001.
65. Schakowski, F. et al., Novel non-viral method for transfection of primary leukemia cells and cell lines, Genet. Vaccines Ther., 2, 1, 2004.
66. Sun, W. et al., In vivo cytokine gene transfer by gene gun reduces tumor growth in mice, Proc. Natl Acad. Sci. USA, 92, 2889, 1995.
67. Matsuno Y. et al., Nonviral gene gun mediated transfer into the beating heart, ASAIO J., 49, 641, 2003.
68. Jeon, E., Kim, H.D., and Kim, J.S., Pluronic-grafted poly-(l)-lysine as a new synthetic gene carrier, J. Biomed. Mater. Res., 66A, 854, 2003.
69. Joubert, D. et al., A note on poly-l-lysine-mediated gene transfer in HeLa cells, Drug Deliv., 10, 209, 2003.
70. Tan, P.H. et al., Transferrin receptor-mediated gene transfer to the corneal endothelium, Transplantation, 71, 552, 2001.
71. Van Rossenberg S.M. et al., Targeted lysosome disruptive elements for improvement of parenchymal liver cell-specific gene delivery, J. Biol. Chem., 277, 45803, 2002.
72. Godbey, W., Wu, K., and Mikos, A., Tracking the intracellular path of poly(ethylenimine)/DNA complexes for gene delivery, Proc. Natl Acad. Sci. USA, 96, 5177, 1999.
73. Godbey, W.T. et al., Poly(ethylenimine)-mediated transfection: a new paradigm for gene delivery, J. Biomed. Mater. Res., 51, 321, 2000.
74. Suh, J., Wirtz, D., and Hanes, J., Efficient active transport of gene nanocarriers to the cell nucleus, Proc. Natl Acad. Sci. USA, 100, 3878, 2003.
75. Brunner, S. et al., Cell cycle dependence of gene transfer by lipoplex, polyplex and recombinant adenovirus, Gene Ther., 7, 7401, 2000.
76. Escriou, V. et al., Critical assessment of the nuclear import of plasmid during cationic lipidmediated gene transfer, J. Gene Med., 3, 179, 2001.
77. Zanta, M. et al., Gene delivery: a single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus, Proc. Natl Acad. Sci. USA, 96, 91, 1999.
78. Aronsohn, A.I. and Hughes, J.A., Nuclear localization signal peptides enhance cationic liposomemediated gene therapy, J. Drug Target., 5, 163, 1998.
79. Bremner, K. et al., Factors influencing the ability of nuclear localization sequence peptides to enhance nonviral gene delivery, Bioconjug. Chem., 15, 152, 2004.
80. Parveen, Z. et al., Cell-type-specific gene delivery into neuronal cells in vitro and in vivo, Virology, 314, 74, 2003.
81. White, S.J. et al., Identification of peptides that target the endothelial cell-specific LOX-1 receptor, Hypertension, 37, 449, 2001.
82. Argnani, R. et al., Specific targeted binding of herpes simplex virus type 1 to hepatocytes via the human hepatitis B virus preS1 peptide, Gene Ther., 2004.
83. Voinea, M., Binding and uptake of transferrin-bound liposomes targeted to transferrin receptors of endothelial cells, Vascul. Pharmacol., 39, 13, 2002.
84. Zhao, X.B. and Lee, R.J., Tumor-selective targeted delivery of genes and antisense oligodeoxyribonucleotides via the folate receptor, Adv. Drug Deliv. Rev., 56, 1193, 2004.
85. Yang, Q. et al., Development of novel cell surface CD34-targeted recombinant adenoassociated virus vectors for gene therapy, Hum. Gene Ther., 9, 1929, 1998.
86. Kunath, K. et al., Galactose-PEI-DNA complexes for targeted gene delivery: degree of substitution affects complex size and transfection efficiency, J. Control. Release, 88, 159, 2003.
87. Wilcox, D.A. et al., Integrin alphaIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus-transduced human hematopoietic cells, Proc. Natl Acad. Sci. USA, 96, 9654, 1999.
88. Park, C. et al., Targeting of therapeutic gene expression to the liver by using liver-type pyruvate kinase proximal promoter and the SV40 viral enhancer active in multiple cell types, Biochem. Biophys. Res. Commun., 314, 131, 2004.
89. Godbey, W.T. and Atala, A., Directed apoptosis in Cox-2-overexpressing cancer cells through expression-targeted gene delivery, Gene Ther., 10, 1519, 2003.
90. Ikegami, S. et al., Treatment efficiency of a suicide gene therapy using prostate-specific membrane antigen promoter/enhancer in a castrated mouse model of prostate cancer, Cancer Sci., 95, 367, 2004.
91. Itakura, K. et al., Expression in Escherichia coli of a chemically synthesized gene for the hormone somatostatin, Science, 198, 1056, 1977.
92. Lee, J. et al., In vitro model for liposome-mediated adenomatous polyposis coli gene transfer in a duodenal model, Dis. Col. Rec., 47, 219, 2004.
93. Krosl, J., In vitro expansion of hematopoietic stemcells by recombinant TAT-HOXB4 protein, Nat. Med., 9, 1428, 2003.
94. Li, R.H. et al., Dose control with cell lines used for encapsulated cell therapy, Tissue Eng., 5, 453, 1999.
95. Huang, Y.C. et al., Fabrication and in vitro testing of polymeric delivery system for condensed DNA, J. Biomed. Mat. Res., 67A, 1384, 2003.
96. Tomita, M. et al., Transgenic silkworms produce recombinant human type III procollagen in cocoons, Nat. Biotechnol., 21, 52, 2003.
97. Martínez-Serrano, A. et al., Ex vivo nerve growth factor gene transfer to the basal forebrain in presymptomatic middle-aged rats prevents the development of cholinergic neuron atrophy and cognitive impairment during aging, Proc. Natl Acad. Sci. USA, 95, 1858, 1998.
98. Magin-Lachmann, C. et al., In vitro and in vivo delivery of intact BAC DNA - comparison of different methods, J. Gene Med., 6, 195, 2004.
99. Brown, C.B., Use of the peritoneal cavity for therapeutic delivery, Perit. Dial. Int., 19, 512, 1999.
100. Shariat, S.F., Adenovirus-mediated transfer of inducible caspases: a novel “death switch” gene therapeutic approach to prostate cancer, Cancer Res., 61, 2562, 2001.
101. Xia, C.F. et al., Kallikrein gene transfer protects against ischemic stroke by promoting glial cell migration and inhibiting apoptosis, Hypertension, 43, 452, 2004.
102. Shi, N. et al., Brain-specific expression of an exogenous gene after i.v. administration, Proc. Natl Acad. Sci. USA, 98, 12754, 2001.
103. Wang, S. et al., Transgene expression in the brain stem effected by intramuscular injection of polyethylenimine/DNA complexes, Mol. Ther., 3, 658, 2001.
104. Shi, N. and Pardridge, W., Noninvasive gene targeting to the brain, Proc. Natl Acad. Sci. USA, 97, 7567, 2000.
105. Olivares, E.C. et al., Site-specific genomic integration produces therapeutic factor IX levels in mice, Nat. Biotechnol., 20, 1124, 2002.
106. Stribling, R. et al., Aerosol gene delivery in vivo, Proc. Natl Acad. Sci. USA, 89, 11277, 1992.
107. Auricchio, A. et al., Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins, J. Clin. Invest., 110, 499, 2002.
108. Koshkina, N.V. et al., Biodistribution and pharmacokinetics of aerosol and intravenously administered DNA-polyethyleneimine complexes: optimization of pulmonary delivery and retention, Mol. Ther., 8, 249, 2003.
109. Chen, J. et al., Transfection of mEpo gene to intestinal epothelium in vivo mediated by oral delivery of chitosan-DNA nanoparticles, World J. Gastroenterol., 10, 112, 2004.
110. Keller, E.T. et al., In vivo particle-mediated cytokine gene transfer into canine oral mucosa and epidermis, Cancer Gene Ther., 3, 186, 1996.
111. Weld, K.J. et al., Transrectal gene therapy of the prostate in the canine model, Cancer Gene Ther., 9, 189, 2002.
112. Journal of Gene Medicine Website, http://www.wiley.co.uk/genmed/clinical, Accessed 10/18/2005.
113. Cavazzana-Calvo, M. et al., Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease, Science, 288, 627, 2000.
114. Wong-Staal, F., Poeschla, E.M., and Looney, D.J., A controlled, phase 1 clinical trial to evaluate the safety and effects in HIV-1 infected humans of autologous lymphocytes transduced with a ribozyme that cleaves HIV-1 RNA, Hum. Gene Ther., 9, 2407, 1998.
115. Twins Study of Gene Therapy for HIV Infection, National Human Genome Research Institute (NHGRI).
116. Senger, D.R. et al., Tumor cells secrete a vascular permeability factor that promotes accumulation of ascites fluid, Science, 219, 983, 1983.
117. Losordo, D. et al., Gene therapy for myocardial angiogenesis: initial clinical results with direct myocardial injection of phVEGF165 as sole therapy for myocardial ischemia, Circulation, 98, 2800, 1998
118. Rosengart, T.K. et al., Six-month assessment of a phase I trial of angiogenic gene therapy for the treatment of coronary artery disease using direct intramyocardial administration of an adenovirus vector expressing the VEGF121 cDNA, Ann. Surg., 230, 466, 1999.
119. Sorscher, E., Study chair, Phase I Study of Liposome-Mediated Gene Transfer in Patients with Cystic Fibrosis, University of Alabama.
120. Moss, R.B. et al., Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial, Chest, 125, 509, 2004.
121. Tuszynski, M., Prin. invest., Gene therapy for Alzheimer’s Disease Clinical Trial, University of California, San Diego.
122. Tuszynski, M.H. and Blesch, A., Nerve growth factor: from animal models of cholinergic neuronal degeneration to gene therapy in Alzheimer’s disease, Prog. Brain Res., 146, 441, 2004.
123. deHaan, Hans, Prin. invest., Gene Therapy in Treating Patients with RecurrentMalignant Glioma, University of Alabama at Birmingham Comprehensive Cancer Center, Birmingham, AL.
124. Link, C., Prin. invest., Gene Therapy in Treating Women With Refractory or Relapsed Ovarian Epithelial Cancer, Fallopian Tube Cancer, or Peritoneal Cancer, Human Gene Therapy Research Institute, Des Moines, Iowa.