Application of gene therapy to acute inflammatory diseases

Shock

Volumn 12, Issue 2, 1999, Pages 83-101

  Moldawer, Lyle L ^a   Edwards, Paul D ^a   Josephs, Michael ^a   Minter, Rebecca M ^a   Copeland III, Edward M ^a   MacKay, Sally L D ^a  

^a UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

Author keywords

AAV; Adenovirus; Cytokines; Liposomes; Review; Ribozymes

Indexed keywords

CYTOKINE; LIPOSOME;

ACUTE DISEASE; ADENOVIRUS; ANIMAL; DRUG DELIVERY SYSTEM; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; HUMAN; INFLAMMATION; METHODOLOGY; PLASMID; REVIEW;

ACUTE DISEASE; ADENOVIRIDAE; ANIMALS; CYTOKINES; DRUG DELIVERY SYSTEMS; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; INFLAMMATION; LIPOSOMES; PLASMIDS;

EID: 0033175665     PISSN: 10732322     EISSN: None     Source Type: Journal    
DOI: 10.1097/00024382-199908000-00001     Document Type: Review

References (184)

1. Anderson WF: Human gene therapy. Nature 392:25-30, 1998 .
2. Crystal RG: Transfer of genes to humans: early lessons and obstacles to success. Science 270:404-10, 1995.
3. Favrot MC: Gene therapy: a review 6 years after the first trials in man. Round Table No 6 at Giens XIII. Therapie 53:381-4,1998.
4. Knoell DL, Yiu IM: Human gene therapy for hereditary diseases: a review of trials. Am J Health Syst Pharm 55:899-904, 1998.
5. Friedmann T: Overcoming the obstacles to gene therapy. Sci Am 276: 96-101, 1997.
6. Friedmann T: The road toward human gene therapy: a 25-year perspective. Ann Med 29:575-7, 1997.
7. Morgan RA, Anderson WF: Human gene therapy. Annu Rev Biochem 62:191-217, 1993.
8. Blaese RM: Gene therapy for cancer. Sci Am 276:111-5, 1997.
9. Crystal RG: The gene as the drug. Nat Med 1:15-7, 1995.
10. Gewirtz AM, Sokol DL, Ratajczak MZ: Nucleic acid therapeutics: state of the art and future prospects. Blood 92:712-36, 1998.
11. Lavrovsky Y, Chen S, Roy AK: Therapeutic potential and mechanism of action of oligonucleotides and ribozymes. Biochem Mol Med 62:11-22, 1997.
12. Brigham KL, Canonico AE, Meyrick BO, Schreier H, Stecenko AA, Conary JT: Gene therapy for inflammatory diseases. Prog Clin Biol Res 388:361-5, 1994.
13. Canonico AE, Brigham KL, Carmichael LC, et al: Plasmid-liposome transfer of the alpha 1 antitrypsin gene to cystic fibrosis bronchial epithelial cells prevents elastase-induced cell detachment and cytokine release. Am J Respir Cell Mol Biol 14:348-55, 1996.
14. Conary JT, Parker RE, Christman BW, et al: Protection of rabbit lungs from endotoxin injury by in vivo hyperexpression of the prostaglandin G/H synthase gene. J Clin Invest 93:1834-40, 1994.
15. Brigham KL, Stecenko AA: Gene therapy in acute critical illness. New Horiz 3:321-9, 1995.
16. Lan N, Howrey RP, Lee SW, Smith CA, Sullenger BA: Ribozyme-mediated repair of sickle beta-globin mRNAs in erythrocyte precursors. Science 280:1593-6, 1998.
17. Jones JT, Lee SW, Sullenger BA: Tagging ribozyme reaction sites to follow trans-splicing in mammalian cells. Nat Med 2:643-8, 1996.
18. Canonico AE, Conary JT, Meyrick BO, Brigham KL: Aerosol and intravenous transfection of human alpha 1-antitrypsin gene to lungs of rabbits. Am J Respir Cell Mol Biol 10:24-9, 1994.
19. Canonico AE, Plitman JD, Conary JT, Meyrick BO, Brigham KL: No lung toxicity after repeated aerosol or intravenous delivery of plasmidcationic liposome complexes. J Appl Physiol 77:415-9, 1994.
20. Brody SL, Metzger M, Danel C, Rosenfeld MA, Crystal RG: Acute responses of non-human primates to airway delivery of an adenovirus vector containing the human cystic fibrosis transmembrane conductance regulator cDNA. Hum Gene Ther 5:821-36, 1994.
21. Crystal RG, McElvaney NG, Rosenfeld MA, et al: Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat Genet 8:42-51, 1994.
22. Peeters MJ, Patijn GA, Lieber A, Meuse L, Kay MA: Adenovirus-mediated hepatic gene transfer in mice : comparison of intravascular and biliary administration. Hum Gene Ther 7:1693-9, 1996.
23. Vickers SM, Phillips JO, Kerby JD, Bynon JSJ, Thompson JA, Curiel DT: In vivo gene transfer to the human biliary tract. Gene Ther 3:825-8, 1996.
24. Kaplitt MG, Xiao X, Samulski RJ, et al: Long-term gene transfer in porcine myocardium after coronary infusion of an adeno-associated virus vector. Ann Thorac Surg 62:1669-76, 1996.
25. Prentice H, Kloner RA, Li Y, Newman L, Kedes L: Ischemic/reperfused myocardium can express recombinant protein following direct DNA or retroviral injection. J Mol Cell Cardiol 28:133-40, 1996.
26. Carducci MA, Simons JW: Renal, bladder, and prostate cancers: gene therapy. Cancer Treat Res 88:219-34, 1996.
27. Huard J, Lochmuller H, Acsadi G, Jani A, Massie B, Karpati G: The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants. Gene Ther 2:107-15, 1995.
28. 99Tc-labeled adenovirus knob (serotype 5). Gene Ther 5:798-808, 1998.
29. Nielsen LL, Gurnani M, Syed J, et al: Recombinant El-deleted adenovirus-mediated gene therapy for cancer: efficacy studies with p53 tumor suppressor gene and liver histology in tumor xenograft models. Hum Gene Ther 9:681-94, 1998.
30. Koeberl DD, Alexander IE, Halbert CL, Russell DW, Miller AD: Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc Natl Acad Sci USA 94:1426-31, 1997.
31. Kozarsky KF, Jooss K, Donahee M, Strauss JF, Wilson JM: Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene [published erratum appears in Nat Genet 13(3):374, 1996]. Nat Genet 13:54-62, 1996.
32. Kozarsky KF, Jooss K, Donahee M, Strauss JF, Wilson JM: Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene [published erratum appears in Nat Genet 13(3):374, 1996]. Nat Genet 13:54-62, 1996.
33. Peel AL, Zolotukhin S, Schrimsher GW, Muzyczka N, Reier PJ: Efficient transduction of green fluorescent protein in spinal cord neurons using adeno-associated virus vectors containing cell type-specific promoters. Gene Ther 4:16-24, 1997.
34. DeMatteo RP, McClane SJ, Fisher K, et al: Engineering tissue-specific expression of a recombinant adenovirus: selective transgene transcription in the pancreas using the amylase promoter. J Surg Res 72:155-61, 1997.
35. Klein RL, Meyer EM, Peel AL, et al: Neuron-specific transduction in the rat septohippocampal or nigrostriatal pathway by recombinant adeno-associated virus vectors. Exp Neurol 150:183-94, 1998.
36. Massie B, Couture F, Lamoureux L, et al: Inducible overexpression of a toxic protein by an adenovirus vector with a tetracycline-regulatable expression cassette. J Virol 72:2289-96, 1998.
37. Dhawan J, Rando TA, Elson SL, Bujard H, Blau HM: Tetracycline-regulated gene expression following direct gene transfer into mouse skeletal muscle. Somat Cell Mol Genet 21:233-40, 1995.
38. Fishman GI, Kaplan ML, Buttrick PM: Tetracycline-regulated cardiac gene expression in vivo. J Clin Invest 93:1864-8, 1994.
39. No D, Yao TP, Evans RM: Ecdysone-inducible gene expression in mammalian cells and transgenic mice. Proc Natl Acad Sci USA 93:3346-51, 1996.
40. Dusetti NJ, Vasseur S, Ortiz EM, et al: The pancreatitis-associated protein I promoter allows targeting to the pancreas of a foreign gene, whose expression is up-regulated during pancreatic inflammation. J Biol Chem 272:5800-804, 1997.
41. Knowles MR, Noone PG, Hohneker K, et al.: A double-blind, placebo controlled, dose ranging study to evaluate the safety and biological efficacy of the lipid-DNA complex GR213487B in the nasal epithelium of adult patients with cystic fibrosis. Hum Gene Ther 9:249-69, 1998.
42. Zhang HG, Zhou T, Yang P, Edwards CK, Curiel DT, Mountz JD: Inhibition of tumor necrosis factor alpha decreases inflammation and prolongs adenovirus gene expression in lung and liver. Hum Gene Ther 9:1875-84, 1998.
43. Denham W, Denham D, Yang J, et al: Transient human gene therapy: a novel cytokine regulatory strategy for experimental pancreatitis. Ann Surg 227:812-20, 1998.
44. Flotte TR, Afione SA, Conrad C, et al: Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci USA 90:10613-7, 1993.
45. Porada CD, Tran N, Eglitis M, et al: In utero gene therapy: transfer and long-term expression of the bacterial neo(r) gene in sheep after direct injection of retroviral vectors into preimmune fetuses. Hum Gene Ther 9:1571-85, 1998.
46. Patijn GA, Lieber A, Meuse L, Winther B, Kay MA: High-efficiency retrovirus-mediated gene transfer into the livers of mice. Hum Gene Ther 9:1449-56, 1998.
47. 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 17:314-7, 1997.
48. Jobin C, Panja A, Hellerbr C, et al: Inhibition of proinflammatory molecule production by adenovirus-mediated expression of a nuclear factor kappaB super-repressor in human intestinal epithelial cells. J Immunol 160:410-8, 1998.
49. Van Antwerp DJ, Seamus JM, Kafr i T, Green D, Verma IM: Suppression of TNF-alpha-induced apoptosis by NF-kB. Science 274:787-9, 1996.
50. Soares MP, Muniappan A, Kaczmarek E, et al: Adenovirus-mediated expression of a dominant negative mutant of p65/RelA inhibits proinflammatory gene expression in endothelial cells without sensitizing to apoptosis. J Immunol 161:4572-82, 1998.
51. Wrighton CJ, Hofer-Warbinek R, Moll T, Eytner R, Bach FH, de MR: Inhibition of endothelial cell activation by adenovirus-mediated expression of I kappa B alpha, an inhibitor of the transcription factor NF-kappa B. J Exp Med 183:1013-22, 1996.
52. Anrather J, Csizmadia V, Brostjan C, Soares MP, Bach FH, Winkler H: Inhibition of bovine endothelial cell activation in vitro by regulated expression of a transdominant inhibitor of NF-kappa B. J Clin Invest 99: 763-72, 1997.
53. Bohrer H, Qiu F, Zimmermann T, et al: Role of NFkappaB in the mortality of sepsis. J Clin Invest 100:972-85, 1997.
54. Marr RA, Addison CL, Snider D, Muller WJ, Gauldie J, Graham FL: Tumour immunotherapy using an adenoviral vector expressing a membrane-bound mutant of murine TNF alpha. Gene Ther 4:1181-8, 1997.
55. Ceneviva GD, Tzeng E, Hoyt DG, et al: Nitric oxide inhibits lipopolysaccharide-induced apoptosis in pulmonary artery endothelial cells. Am J Physiol 275:L717-28, 1998.
56. Tzeng E, Billiar TR, Williams DL, et al: Adenovirus-mediated inducible nitric oxide synthase gene transfer inhibits hepatocyte apoptosis. Surgery 124:278-83, 1998.
57. Moreno MB, Memon SA, Zacharchuk CM: Apoptosis signaling pathways in normal T cells: differential activity of Bcl-2 and IL-1beta-converting enzyme family protease inhibitors on glucocorticoid- and Fas-mediated cytotoxicity. J Immunol 157:3845-9, 1996.
58. Kirshenbaum LA, de MD: The bcl-2 gene product prevents programmed cell death of ventricular myocytes. Circulation 96:1580-5, 1997.
59. Huang DC, Cory S, Strasser A: Bcl-2, Bel-XL and adenovirus protein E1B19kD are functionally equivalent in their ability to inhibit cell death. Oncogene 14:405-14, 1997.
60. Clarke MF, Apel U, Benedict MA, et al: A recombinant Bcl-xs adenovirus selectively induces apoptosis in cancer cells but not in normal bone marrow cells. Proc Natl Acad Sci USA 92:11024-8, 1995.
61. Ksontini R, MacKay SL, Moldawer LL: Revisiting the role of tumor necrosis factor alpha and the response to surgical injury and inflammation. Arch Surg 133:558-67, 1998.
62. Pruitt JH, Copeland EM3, Moldawer LL: Interleukin-1 and interleukin-1 antagonism in sepsis, systemic inflammatory response syndrome, and septic shock. Shock 3:235-51, 1995.
63. Doughty LA, Patrene KD, Evans CH, Boggs SS, Robbins PD: Constitutive systemic expression of IL-1Ra or soluble TNF receptor by genetically modified hematopoietic cells suppresses LPS induction of IL-6 and IL-10. Gene Ther 4:252-7, 1997.
64. Kolls J, Peppel K, Silva M, Beutler B: Prolonged and effective blockade of tumor necrosis factor activity through adenovirus-mediated gene transfer. Proc Natl Acad Sci USA 91:215-9, 1994.
65. Kolls JK, Lei D, Nelson S, Summer WR, Greenberg S, Beutler B: Adenovirus-mediated blockade of tumor necrosis factor in mice protects against endotoxic shock yet impairs pulmonary host defense. J Infect Dis 171:570-5, 1995.
66. Rogy MA, Auffenberg T, Espat NJ,et al: Human tumor necrosis factor receptor (p55) and interleukin 10 gene transfer in the mouse reduces mortality to lethal endotoxemia and also attenuates local inflammatory responses. J Exp Med 181:2289-93, 1995.
67. Pelletier JP, Caron JP, Evans C, et al: In vivo suppression of early experimental osteoarthritis by interleukin-1 receptor antagonist using gene therapy. Arthritis Rheum 40:1012-9, 1997.
68. Bakker AC, Joosten LA, Arntz OJ, et al: Prevention of murine collagen-induced arthritis in the knee and ipsilateral paw by local expression of human interleukin-1 receptor antagonist protein in the knee. Arthritis Rheum 40:893-900, 1997.
69. Evans CH, Robbins PD, Ghivizzani SC, et al: Clinical trial to assess the safety, feasibility, and efficacy of transferring a potentially anti-arthritic cytokine gene to human joints with rheumatoid arthritis. Hum Gene Ther 7:1261-80, 1996.
70. Makarov SS, Olsen JC, Johnston WN, et al: Suppression of experimental arthritis by gene transfer of interleukin 1 receptor antagonist cDNA. Proc Natl Acad Sci USA 93:402-6, 1996.
71. Ghivizzani SC, Lechman ER, Kang R, et al: Direct adenovirus-mediated gene transfer of interleukin 1 and tumor necrosis factor alpha soluble receptors to rabbit knees with experimental arthritis has local and distal anti-arthritic effects. Proc Natl Acad Sci USA 95:4613-8, 1998.
72. Le CH, Nicolson AG, Morales A, Sewell KL: Suppression of collagen-induced arthritis through adenovirus-mediated transfer of a modified tumor necrosis factor alpha receptor gene. Arthritis Rheum 40:1662-9, 1997.
73. Chernajovsky Y, Adams G, Podhajcer OL, Mueller GM, Robbins PD, Feldmann M: Inhibition of transfer of collagen-induced arthritis into SCID mice by ex vivo infection of spleen cells with retroviruses expressing soluble tumor necrosis factor receptor. Gene Ther 2:731-5, 1995.
74. Yang GY, Liu XH, Kadoya C, et al: Attenuation of ischemic inflammatory response in mouse brain using an adenoviral vector to induce overexpression of interleukin-1 receptor antagonist. J Cereb Blood Flow Metab 18:840-7, 1998.
75. Betz AL, Yang GY, Davidson BL: Attenuation of stroke size in rats using an adenoviral vector to induce overexpression of interleukin-1 receptor antagonist in brain. J Cereb Blood Flow Metab 15:547-51, 1995.
76. Croxford JL, Triantaphyllopoulos K, Podhajcer OL, Feldmann M, Baker D, Chernajovsky Y: Cytokine gene therapy in experimental allergic encephalomyelitis by injection of plasmid DNA-cationic liposome complex into the central nervous system. J Immunol 160:5181-7, 1998.
77. Rosenfeld MA, Siegfried W, Yoshimura K, et al: Adenovirus-mediated transfer of a recombinant alpha 1-antitrypsin gene to the lung epithelium in vivo. Science 252:431-4, 1991.
78. Danel C, Erzuram SC, Prayssac P, et al: Gene therapy for oxidant injury-related diseases: adenovirus-mediated transfer of superoxide dismutase and catalase cDNAs protects against hyperoxia but not against ischemia-reperfusion lung injury. Hum. Gene Ther 9:1487-96, 1998.
79. Ahonen M, Baker AH, Kahari VM: Adenovirus-mediated gene delivery of tissue inhibitor of metalloproteinases-3 inhibits invasion and induces apoptosis in melanoma cells. Cancer Res 58:2310-5, 1998.
80. George SJ, Johnson JL, Angelini GD, Newby AC, Baker AH: Adenovirus-mediated gene transfer of the human TIMP-1 gene inhibits smooth muscle cell migration and neointimal formation in human saphenous vein. Hum Gene Ther 9:867-77, 1998.
81. Chang MW, Leiden JM: Gene therapy for vascular proliferative disorders. Semin Interv Cardiol 1:185-93, 1996.
82. Sioud M: Ribozyme modulation of lipopolysaccharide-induced tumor necrosis factor-alpha production by peritoneal cells in vitro and in vivo. Eur J Immunol 26:1026-31, 1996.
83. Kisich KO, Freedland SJ, Erickson KL: Factors altering ribozyme-mediated cleavage of tumor necrosis factor-alpha mRNA in vitro. Biochem Biophys Res Commun 236:205-11, 1997.
84. Durko M, Navab R, Shibata HR, Brodt P: Suppression of basement membrane type IV collagen degradation and cell invasion in human melanoma cells expressing an antisense RNA for MMP-1. Biochim Biophys Acta 1356:271-80, 1997.
85. Turck J, Pollock AS, Lee LK, Marti HP, Lovett DH: Matrix metalloproteinase 2 (gelatinase A) regulates glomerular mesangial cell proliferation and differentiation. J Biol Chem 271:15074-83, 1996.
86. Yang NS, Sun WH: Gene gun and other non-viral approaches for cancer gene therapy. Nat Med 1:481-3, 1995.
87. Sun WH, Burkholder JK, Sun J, et al: In vivo cytokine gene transfer by gene gun reduces tumor growth in mice. Proc Natl Acad Sci USA 92: 2889-93, 1995.
88. Gunzburg WH, Salmons B: Virus vector design in gene therapy. Mol Med Today 1:410-7, 1995.
89. Salmons B, Gunzburg WH: Targeting of retroviral vectors for gene therapy. Hum Gene Ther 4:129-41, 1993.
90. Mochizuki H, Schwartz JP, Tanaka K, Brady RO, Reiser J: High-titer human immunodeficiency virus type 1-based vector systems for gene delivery into nondividing cells. J Virol 72:8873-83, 1998.
91. Flotte TR, Carter BJ: Adeno-associated virus vectors for gene therapy. Gene Ther 2:357-62,1995.
92. Summerford C, Samulski RJ: Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 72:1438-45, 1998.
93. Summerford C, Bartlett JS, Samulski RJ: AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infection. Nat Med 5:78-82, 1999.
94. Song S, Morgan M, Ellis T, et al: Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci USA 95:14384-8, 1998.
95. Flotte TR, Carter BJ: Adeno-associated virus vectors for gene therapy. Gene Ther 2:357-62, 1995.
96. Mulligan RC: The basic science of gene therapy. Science 260:926-32, 1993.
97. Feigner PL, Gadek TR, Holm M, et al: Lipofectin: A highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 84:7413-7, 1987.
98. Crook K, Stevenson BJ, Dubouchet M, Porteous DJ: Inclusion of cholesterol in DOTAP transfection complexes increases the delivery of DNA to cells in vitro in the presence of serum. Gene Ther 5:137-43, 1998.
99. Yang JP, Huang L: Overcoming the inhibitory effect of serum on lipofection by increasing the charge ratio of cationic liposome to DNA. Gene Ther 4:950-60, 1997.
100. Felgner JH, Kumar R, Sridhar CN, et al: Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J Biol Chem 269:2550-61, 1994.
101. Gao X, Huang L: Cationic liposome mediated gene transfer. Gene Therapy 2:710-22, 1995.
102. Liu Y, Liggitt D, Zhong W, Tu G, Gaensler K, Debs R: Cationic liposome-mediated intravenous gene delivery. J Biol Chem 270:24864-70, 1995.
103. Felgner PL: Nonviral strategies for gene therapy. Sci Am 276:102-6, 1997.
104. Liu Y, Mounkes LC, Liggitt HD, et al: Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery. Nat Biotechnol 15:167-73, 1997.
105. Philip R, Liggitt D, Philip M, Dazin P, Debs R: In vivo gene delivery. Efficient transfection of T lymphocytes in adult mice. J Biol Chem 268: 16087-90, 1993.
106. Rose JK, Debs RA, Philip R, Ruis NM, Valone FH: Selective activation of human monocytes by the platelet-activating factor analog 1-O-hexadecyl-2-O-methyl-sn-glycero-3-phosphorylcholine. J Immunol 144: 3513-7, 1990.
107. Kaneda Y, Kunimitsu I, Uchida T: Increased Expression of DNA cointroduced with nuclear protein in adult rat liver. Science 243:375-8, 1989.
108. Kasama T, et.al: Regulation of neutrophil derived chemokine expression by IL-10. J Immunol 152:3559-69, 1994.
109. Wheeler CJ, Feigner PL, Tsai YJ, et al: A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. Proc Natl Acad Sci USA 93:11454-9, 1996.
110. Zanta MA, Belguise-Valladier P, Behr JP: Gene delivery: A single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus. Proc Natl Acad Sci USA 96:91-6, 1999.
111. Lebkowski JS, Okarma TB, Philip R: The challenges of recombinant adeno-associated virus manufacturing: alternative use of adeno-associated virus plasmid/liposome complexes for gene therapy applications. Curr Top Microbiol Immunol 218:51-9, 1996.
112. Stribling R, Brunette E, Liggitt D, Gaensler K, Debs R: Aerosol gene delivery in vivo. Proc Natl Acad Sci USA 89:11277-81, 1992.
113. Thierry AR, Lunardi Iskandar Y, Bryant JL, Rabinovich P, Gallo RC, Mahan LC: Systemic gene therapy: biodistribution and long-term expression of a transgene in mice. Proc Natl Acad Sci USA 92:9742-6, 1995.
114. Schmidt E, Christoph G, Zeller R, Leder P: Cytomegalovirus enhancer: a pan-active control element in transgenic mice. Mol Cell Biol 10:4406-11, 1990.
115. Philip R, Brunette E, Kilinski L, et al: Efficient and sustained gene expression in primary T lymphocytes and primary and cultured tumor cells mediated by adeno-associated virus plasmid DNA complexed to cationic liposomes. Mol Cell Biol 14:2411-8, 1994.
116. Wolff JA, Malone RW, Williams P, et al: Direct gene transfer into mouse muscle in vivo. Science 247:1465-8, 1990.
117. Debs R, Pian M, Gaensler K, Clements J, Friend DS, Dobbs L: Prolonged transgene expression in rodent lung cells. Am J Respir Cell Mol Biol 7:406-13, 1992.
118. Zhu N, Liggitt D, Liu Y, Debs R: Systemic gene expression after intravenous DNA delivery into adult mice. Science 261:209-11, 1993.
119. Wicks IP, Howell ML, Hancock T, Kohsaka H, Olee T, Carson DA: Bacterial lipopolysaccharide copurifies with plasmid DNA: implications for animal models and human gene therapy. Hum Gene Ther 6:317-23, 1995.
120. Denham W, Abouhamze A, Moldawer LL, Norman J: Cationic liposome-mediated gene transfer during acute pancreatitis: tissue specificity, duration, and effects of acute inflammation. J Gastrointest Surg 2:95-101, 1998.
121. Thornton FJ, Schaffer MR, Witte MB, et al: Enhanced collagen accumulation following direct transfection of the inducible nitric oxide synthase gene in cutaneous wounds. Biochem Biophys Res Commun 246: 654-9, 1998.
122. Szarka RJ, Wang N, Gordon L, Nation PN, Smith RH: A murine model of pulmonary damage induced by lipopolysaccharide via intranasal instillation. J Immunol Methods 202:49-57, 1997.
123. Strieter RM, Lynch JP, Basha MA, Standiford TJ, Kasahara K, Kunkel SL: Host responses in mediating sepsis and adult respiratory distress syndrome Semin Respir Infect 5:233-47, 1990.
124. Ginsberg HS, Moldawer LL, Sehgal PB, et al: A mouse model for investigating the molecular pathogenesis of adenovirus pneumonia. Proc Natl Acad Sci USA 88:1651-5, 1991.
125. Schwartz DA, Quinn TJ, Thorne PS, Sayeed S, Yi AK, Krieg AM: CpG motifs in bacterial DNA cause inflammation in the lower respiratory tract. J Clin Invest 100:68-73, 1997.
126. Krieg AM: An innate immune defense mechanism based on the recognition of CpG motifs in microbial DNA. J Lab Clin Med 128:128-33, 1996.
127. Klinman DM, Yi AK, Beaucage SL, Conover J, Krieg AM: CpG motifs present in bacteria DNA rapidly induce lymphocytes to secrete interleukin 6, interleukin 12, and interferon gamma. Proc Natl Acad Sci USA 93:2879-83, 1996.
128. Sparwasser T, Miethke T, Lipford G, et al: Macrophages sense pathogens via DNA motifs: induction of tumor necrosis factor-alpha-mediated shock. Eur J Immunol 27:1671-9, 1997.
129. McDonald RJ, Liggitt HD, Roche L, et al: Aerosol delivery of lipid:DNA complexes to lungs of rhesus monkeys. Pharm Res 15:671-9, 1998.
130. Scheule RK, St GJ, Bagley RG, et al: Basis of pulmonary toxicity associated with cationic lipid-mediated gene transfer to the mammalian lung. Hum Gene Ther 8:689-707, 1997.
131. Engelhardt JF, Simon RH, Yang Y, et al: Adenovirus-mediated transfer of the CFTR gene to lung of nonhuman primates: biological efficacy study. Hum Gene Ther 4:759-69, 1993.
132. Brody SL, Crystal RG: Adenovirus-mediated in vivo gene transfer. Ann N Y Acad Sci 716:90-101, 1994.
133. Zabner J, Couture LA, Gregory RJ, Graham SM, Smith AE, Welsh MJ: Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 75:207-16, 1993.
134. Knowles MR, Hohneker KW, Zhou Z, et al: A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. N Engl J Med 333:823-31, 1995.
135. Prince GA, Porter DD, Jenson AB, Horswood RL, Chanock RM, Ginsberg HS: Pathogenesis of adenovirus type 5 pneumonia in cotton rats (Sigmodon hispidus). J Virol 67:101-11, 1993.
136. Otake K, Ennist DL, Harrod K, Trapnell BC: Nonspecific inflammation inhibits adenovirus-mediated pulmonary gene transfer and expression independent of specific acquired immune responses. Hum Gene Ther 9:2207-22, 1998.
137. Zsengeller ZK, Wert SE, Hull WM, Hu X, Yei S, Trapnell BC, Whitsett JA: Persistence of replication-deficient adenovirus-mediated gene transfer in lungs of immune-deficient (nu/nu) mice. Hum Gene Ther 6:457-67, 1995.
138. Yang Y, Trinchieri G, Wilson JM: Recombinant IL-12 prevents formation of blocking IgA antibodies to recombinant adenovirus and allows repeated gene therapy to mouse lung. Nat Med 1:890-3, 1995.
139. Benihoud K, Saggio I, Opolon P, et al: Efficient, repeated adenovirus-mediated gene transfer in mice lacking both tumor necrosis factor alpha and lymphotoxin alpha. J Virol 72:9514-25, 1998.
140. McCoy RD, Davidson BL, Roessler BJ, Huffnagle GB, Simon RH: Expression of human interleukin-1 receptor antagonist in mouse lungs using a recombinant adenovirus: effects on vector-induced inflammation. Gene Ther 2:437-42, 1995.
141. Lieber A, He CY, Meuse L, et al: The role of Kupffer cell activation and viral gene expression in early liver toxicity after infusion of recombinant adenovirus vectors. J Virol 71:8798-807, 1997.
142. Elkon KB, Liu CC, Gall JG, et al: Tumor necrosis factor alpha plays a central role in immune-mediated clearance of adenoviral vectors. Proc Natl Acad Sci USA 94:9814-9, 1997.
143. Lieber A, He CY, Meuse L, Himeda C, Wilson C, Kay MA: Inhibition of NF-kappaB activation in combination with bcl-2 expression allows for persistence of first-generation adenovirus vectors in the mouse liver. J Virol 72:9267-77, 1998.
144. Hardy S, Kitamura M, Harris-Stansil T, Dai Y, Phipps ML: Construction of adenovirus vectors through Cre-lox recombination. J Virol 71:1842-9, 1997.
145. Baue AE: Multiple organ failure, multiple organ dysfunction syndrome, and systemic inflammatory response syndrome. Why no magic bullets? Arch Surg 132:703-7, 1997.
146. Baumhofer JM, Beinhauer BG, Wang JE, et al: Gene transfer with IL-4 and IL-13 improves survival in lethal endotoxemia in the mouse and ameliorates peritoneal macrophages immune competence. Eur J Immunol 28:610-5, 1998.
147. Xing Z, Ohkawara Y, Jordana M, Graham FL, Gauldie J: Adenoviral vector-mediated interleukin-10 expression in vivo: intramuscular gene transfer inhibits cytokine responses in endotoxemia. Gene Ther 4:140-9, 1997.
148. Wispe JR, Warner BB, Clark JC, et al: Human Mn-superoxide dismutase in pulmonary epithelial cells of transgenic mice confers protection from oxygen injury. J Biol Chem 267:23937-41, 1992.
149. Yasuda H, Nagata M, Arisawa K, et al: Local expression of immunoregulatory IL-12p40 gene prolonged syngeneic islet graft survival in diabetic NOD mice. J Clin Invest 102:1807-14, 1998.
150. Moritani M, Yoshimoto K, Ii S, et al: Prevention of adoptively transferred diabetes in nonobese diabetic mice with IL-10-transduced islet-specific Th1 lymphocytes. A gene therapy model for autoimmune diabetes. J Clin Invest 98:1851-9, 1996.
151. Taub PJ, Marmur JD, Zhang WX, et al: Effect of time on the viability of ischemic skin flaps treated with vascular endothelial growth factor (VEGF) cDNA. J Reconstr Microsurg 14:387-90, 1998.
152. Sun L, Xu L, Chang H, et al: Transfection with aFGF cDNA improves wound healing. J Invest Dermatol 108:313-8, 1997.
153. Yamasaki K, Edington HD, McClosky C, et al: Reversal of impaired wound repair in iNOS-deficient mice by topical adenoviral-mediated iNOS gene transfer. J Clin Invest 101:967-71, 1998.
154. Flotte TR, Carter BJ: Adeno-associated virus vectors for gene therapy of cystic fibrosis. Methods Enzymol 292:717-32, 1998.
155. Yang L, Lochmuller H, Luo J, et al: Adenovirus-mediated dystrophin minigene transfer improves muscle strength in adult dystrophic (MDX) mice. Gene Ther 5:369-79, 1998.
156. Yuasa K, Miyagoe Y, Yamamoto K, Nabeshima Y, Dickson G, Takeda S: Effective restoration of dystrophin-associated proteins in vivo by adenovirus-mediated transfer of truncated dystrophin cDNAs. FEBS Lett 425:329-36, 1998.
157. Parkman R, Gelfand EW: Severe combined immunodeficiency disease, adenosine deaminase deficiency and gene therapy. Curr Opin Immunol 3:547-51, 1991.
158. Crystal RG: The alpha 1-antitrypsin gene and its deficiency states. Trends Genet 5:411-7, 1989.
159. Grossman M, Wilson JM: Frontiers in gene therapy: LDL receptor replacement for hypercholesterolemia. J Lab Clin Med 119:457-60, 1992.
160. Wilson JM, Chowdhury JR: Prospects for gene therapy of familial hypercholesterolemia. Mol Biol Med 7:223-32, 1990.
161. Raper SE, Wilson JM, Yudkoff M, Robinson MB, Ye X, Batshaw ML: Developing adenoviral-mediated in vivo gene therapy for ornithine transcarbamylase deficiency. J Inherit Metab Dis 21 Suppl 1:119-37:119-37, 1998.
162. Pauly DF, Johns DC, Matelis LA, Lawrence JH, Byrne BJ, Kessler PD: Complete correction of acid alpha-glucosidase deficiency in Pompe disease fibroblasts in vitro, and lysosomally targeted expression in neonatal rat cardiac and skeletal muscle. Gene Ther 5:473-80, 1998.
163. Bank A, Markowitz D, Lerner N: Gene transfer. A potential approach to gene therapy for sickle cell disease. Ann N Y Acad Sci 565:37-43, 1989.
164. Lozier JN, Brinkhous KM: Gene therapy and the hemophilias. JAMA 271:47-51, 1994.
165. Chuah MK, Collen D, VandenDriessche T: Gene therapy for hemophilia: hopes and hurdles. Crit Rev Oncol Hematol 28:153-71, 1998.
166. Lewin AS, Drenser KA, Hauswirth WW, et al: Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa. Nat Med 4:967-71, 1998.
167. Russell JE, Liebhaber SA: Reversal of lethal alpha- and beta-thalassemias in mice by expression of human embryonic globins. Blood 92:3057-63, 1998.
168. Gale RP: Prospects for correction of thalassemia by genetic engineering. Prog Clin Biol Res 309:141-59, 1989.
169. Liu JM: Gene transfer for the eventual treatment of Fanconi's anemia. Semin Hematol 35:168-79, 1998.
170. Ohashi T, Watabe K, Sato Y, Saito I, Barranger JA, Eto Y: Successful transduction of oligodendrocytes and restoration of arylsulfatase A deficiency in metachromatic leukodystrophy fibroblasts using an adenovirus vector. Gene Ther 2:443-9, 1995.
171. Gilboa E, Smith C: Gene therapy for infectious diseases: the AIDS model. Trends Genet 10:139-44, 1994
172. Tung FT, Bowen SW: Targeted inhibition of hepatitis B virus gene expression: a gene therapy approach. Front Biosci 3:11-15 1998.
173. Nguyen JT, Wu P, Clouse ME, Hlatky L, Terwilliger EF: Adeno-associated virus-mediated delivery of antiangiogenic factors as an antitumor strategy. Cancer Res 58:5673-7, 1998
174. Oku T, Tjuvajev JG, Miyagawa T, et al: Tumor growth modulation by sense and antisense vascular endothelial growth factor gene expression: effects on angiogenesis, vascular permeability, blood volume, blood flow, fluorodeoxyglucose uptake, and proliferation of human melanoma intracerebral xenografts. Cancer Res 58:4185-92, 1998.
175. Ellis LM, Liu W, Wilson M: Down-regulation of vascular endothelial growth factor in human colon carcinoma cell lines by antisense transfection decreases endothelial cell proliferation. Surgery 120:871-8, 1996.
176. Tanaka T, Cao Y, Folkman J, Fine HA: Viral vector-targeted antiangiogenic gene therapy utilizing an angiostatin complementary DNA. Cancer Res 58:3362-9, 1998.
177. Singhal S, Kaiser LR: Cancer chemotherapy using suicide genes. Surg Oncol Clin N Am 7:505-36, 1998.
178. Mackensen A, Lindemann A, Mertelsmann R: Immunostimulatory cytokines in somatic cells and gene therapy of cancer. Cytokine Growth Factor Rev 8:119-28, 1997.
179. Searle PF, Young LS: Immunotherapy II: Antigens, receptors and co-stimulation. Cancer Metastasis Rev 15:329-49, 1996.
180. Favrot M, Coll JL, Louis N, Negoescu A: Cell death and cancer:
181. Macdonald TT: Viral vectors expressing immunoregulatory cytokines to treat inflammatory bowel disease. Gut 42:460-1, 1998.
182. Jorgensen C, Gay S: Gene therapy in osteoarticular diseases: where are we? Immunol Today 19:387-91, 1998.
183. Liu M, Slutsky AS: Anti-inflammatory therapies: application of molecular biology techniques in intensive care medicine. Intensive Care Med 23:718-31, 1997.
184. Eming SA, Morgan JR, Berger A: Gene therapy for tissue repair: approaches and prospects. Br J Plast Surg 50:491-500, 1997.