Pulmonary gene therapy

Pharmaceutical Gene Delivery Systems

Volumn , Issue , 2003, Pages 363-396

  Davies, Jane C ^a   Geddes, Duncan M ^b   Alton, Eric W F W ^a  

^a ROYAL BROMPTON HOSPITAL   (United Kingdom)

^b NATIONAL HEART AND LUNG INSTITUTE   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 3242741774     PISSN: None     EISSN: None     Source Type: Book    
DOI: None     Document Type: Chapter

References (160)

1. Ganderton D. Targeted delivery of inhaled drugs: current challenges and future goals. J Aerosol Med 1999; 12(Suppl)1:S3-8.
2. Tran ND, Porada CD, Almeida-Porada G, Glimp HA, Anderson WF, Zanjani ED. Induction of stable prenatal tolerance to beta-galactosidase by in utero gene transfer into preimmune sheep fetuses. Blood 2001; 97:3417-3423.
3. Larson JE, Morrow SL, Happel L, Sharp JF, Cohen JC. Reversal of cystic fibrosis phenotype in mice by gene therapy in utero. Lancet 1997; 349:619-620.
4. Boyle MP, Enke RA, Adams RJ, Guggino WB, Zeitlin PL. In utero aav-mediated gene transfer to rabbit pulmonary epithelium. Mol Ther 2001; 4:115-121.
5. Muller DW, Gordon D, San H, Yang Z, Pompili VJ, Nabel GJ, Nabel EG. Catheter-mediated pulmonary vascular gene transfer and expression. Circ Res 1994; 75: 1039-1049.
6. Cavazzana-Calvo M, Hacein-Bey S, Yates F, de Villartay JP, Le Deist F, Fischer A. Gene therapy of severe combined immunodeficiencies. J Gene Med 2001; 3: 201-206.
7. Gussoni E, Soneoka Y, Strickland CD, Buzney EA, Khan MK, Flint AF, Kunkel LM, Mulligan RC. Dystrophin expression in the mdx mouse restored by stem cell transplantation. Nature 1999; 401:390-394.
8. Ono K, Takii T, Onozaki K, Ikawa M, Okabe M, Sawada M. Migration of exogenous immature hematopoietic cells into adult mouse brain parenchyma under GFP-expressing bone marrow chimera. Biochem Biophys Res Comm 1999; 262:610-614.
9. Alison MR, Poulsom R, Jeffery R, Dhillon AP, Quaglia A, Jacob J, Novelli M, Prentice G, Williamson J, Wright NA. Hepatocytes from non-hepatic adult stem cells. Nature 2000; 406:257.
10. Jackson KA, Majka SM, Wang H, Pocius J, Hartley CJ, Majesky MW, Entman ML, Michael LH, Hirschi KK, Goodell MA. Regeneration of ischemic cardiac muscle and vascular endothelium by adult stem cells. J Clin Invest 2001; 107:1395-1402.
11. Krause DS, Thiese ND, Collector MI, Henegariu O, Hwang S, Gardner R, Neutzel S, Sharkis SJ. Multi-organ, multi-lineage engraftment by a single bone marrow-derived stem cell. Cell 2001; 105:369-377.
12. Kitson C Angel B, Judd D, Rothery S, Severs NJ, Dewar A, Huang L, Wadsworth SC, Cheng SH, Geddes DM, Alton EW. The extra- and intracellular barriers to lipid and adenovirus-mediated pulmonary gene transfer in native sheep airway epithelium. Gene Ther 1999; 6:534-546.
13. Lethem MI, James SL, Marriott C, Burke JF. The origin of DNA associated with mucus glycoproteins in cystic fibrosis sputum. Eur Respir J 1990; 3:19-23.
14. Ranasinha C, Assoufi B, Shak S, Christiansen D, Fuchs H, Empey D, Geddes D, Hodson M. Efficacy and safety of short-term administration of aerosolised recombinant human DNase I in adults with stable stage cystic fibrosis. Lancet 1993; 342: 199-202.
15. Stern M, Caplen NJ, Browning JE, Griesenbach U, Sorgi F, Huang L, Gruenert DC, Marriot C, Crystal RG, Geddes DM, Alton EW. The effects of mucolytic agents on gene transfer across a CF sputum barrier in vitro. Gene Ther 1998; 5:91-98.
16. van Heekeren A, Ferkol T, Tosi M. Effects of bronchopulmonary inflammation induced by pseudomonas aeruginosa on gene transfer to airway epithelial cells in mice. Gene Ther 1998; 5:345-351.
17. Virella-Lowell I, Poirier A, Chesnut KA, Brantly M, Flotte TR. Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients. Gene Ther 2000; 7:1783-1789.
18. Pickles RJ, Fahrner JA, Petrella JM, Boucher RC, Bergelson JM. Retargeting the coxsackievirus and adenovirus receptor to the apical surface of polarized epithelial cells reveals the glycocalyx as a barrier to adenovirus-mediated gene transfer. J Virol 2000; 74:6050-6057.
19. Drapkin PT, O’Riordan CR, Yi SM, Chiorini JA, Cardella J, Zabner J, Welsh MJ. Targeting the urokinase plasminogen activator receptor enhances gene transfer to human airway epithelia. J Clin Invest 2000: 105:589-596.
20. Walters RW, Grunst T, Bergelson JM, Finberg RW, Welsh MJ, Zabner J. Basolat-eral localization of fiber receptors limits adenovirus infection from the apical surface of airway epithelia. J Biol Chem 1999; 274:10219-10226.
21. Parsons DW, Grubb BR, Johnson LG, Boucher RC. Enhanced in vivo airway gene transfer via transient modification of host barrier properties with a surface-active agent. Hum Gene Ther 1998; 9:2661-2672.
22. Schneeberger EE, Lynch RD. Structure, function, and regulation of cellular tight junctions. Am J Physiol 1992; 262:L647-661.
23. Walters RW, Yi SM, Keshavjee S, Brown KE, Welsh MJ, Chiorini JA, Zabner J. Binding of adeno-associated virus type 5 to 2,3-linked sialic acid is required for gene transfer. J Biol Chem 2001; 276:20610-20616.
24. Zabner J, Fasbender AJ, Moninger T, Poellinger KA, Welsh MJ. Cellular and molecular barriers to gene transfer by a cationic lipid. J Biol Chem 1995; 270:18997-19007.
25. Duan D, Yue Y, Yan Z, Yang J, Engelhardt JF. Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J Clin Invest 2000; 105:1573-1587.
26. Lim DW, Yeom YI, Park TG. Poly(DMAEMA-NVP)-b-PEG-galactose as gene delivery vector for hepatocytes. Bioconjug Chem 2000; 11:688-695.
27. Lukacs GL, Haggie P, Seksek O, Lechardeur D, Freedman N, Verkman AS. Size-dependent DNA mobility in cytoplasm and nucleus. J Biol Chem 2000; 275:1625-1629.
28. Lechardeur D, Sohn KJ, Haardt M, Joshi PB, Monck M, Graham RW, Beatty B, Squire J, O’Brodovich H, Lukacs GL. Metabolic instability of plasmid DNA in the cytosol: a potential barrier to gene transfer. Gene Ther 1999; 6:482-497.
29. Wilson GL, Dean BS, Wang G, Dean DA. Nuclear import of plasmid DNA in digitonin-permeabilized cells requires both cytoplasmic factors and specific DNA sequences. J Biol Chem 1999; 274:22025-22032.
30. Bremner KH, Seymour LW, Pouton CW. Harnessing nuclear localization pathways for transgene delivery. Curr Opin Mol Ther 2001; 3:170-177.
31. Yonemitsu Y, Kitson C, Ferrari S, Farley R, Griesenbach U, Judd D, Steel R, Scheid P, Zhu J, Jeffery PK, Kato A, Hasan MK, Nagai Y, Masaki I, Fukumura M, Hase-gawa M, Geddes DM, Alton EW. Efficient gene transfer to airway epithelium using recombinant Sendai virus. Nat Biotech 2000; 18:970-973.
32. Crystal RG, McElvaney NG, Rosenfeld MA, Chu CS, Mastrangeli A, Hay JG, Brody SL, Jaffe HA, Eissa NT, Danel C. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat Genet 1994; 8:42-51.
33. Schwarzt 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 1997; 100:68-73.
34. Zabner J, Ramsay BW, Meeker DP, Aitken ML, Balfour RP, Gibson RL, Launs-pach J, Moscicki RA, Richards SM, Standaert TA, et al. Repeat administration of an adenovirus vector encoding cystic fibrosis transmembrane conductance regulator to the nasal epithelium of patients with cystic fibrosis. J Clin Invest 1996; 97:1504-1511.
35. Hyde SC, Southern KW, Gileadi U, Fitzjohn EM, Mofford KA, Waddell BE, Gooi HC, Goddard CA, Hannavy K, Smyth SE, Egan JJ, Sorgi FL, Huang L, Cuthbert AW, Evans MJ, Colledge WH, Higgins CF, Webb AK, Gill DR. Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis. Gene Ther 2000; 7:1156-1165.
36. CF Foundation 1995. Patient Registry 1994 Annual Data Report, CF Foundation, Bethesda, MD.
37. Rommens JM, Iannuzzi MC, Kerem B-S, Drumm ML, Melmer G, Dean M, Roz-mahel R, Cole JL, Kennedy D, Hidaka N, et al. Identification of the cystic fibrosis gene: chromosome walking and jumping. Science 1989; 245:1059-1065.
38. Welsh MJ, Smith AE. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis. Cell 1993; 73:1251-1254.
39. Koch C, Hoiby N. Pathogenesis of cystic fibrosis. Lancet 1993; 341:1065-1069.
40. Ismailov II, Awayda MS, Jovov B, Berdiev BK, Fuller CM, Dedman JR, Kaetzel M, Benos DJ. Regulation of epithelial sodium channels by the cystic fibrosis transmembrane conductance regulator. J Biol Chem 1996; 271:4725-4732.
41. Gabriel SE, Clarke LL, Boucher RC, Stutts MJ. CFTR and outward rectifying chloride channels are distinct proteins with a regulatory relationship. Nature 1993; 363: 263-268.
42. Mall M, Kunzelmann K, Hipper A, Busch AE, Greger R. cAMP stimulation of CFTR-expressing Xenopus oocytes activates a chromanol-inhibitable K+ conductance. Pflugers Arch 1996; 432:516-522.
43. Schreiber R, Greger R, Nitschke R, Kunzelmann K. Cystic fibrosis transmembrane conductance regulator activates water conductance in Xenopus oocytes. Pflugers Arch 1997; 434:841-847.
44. Cheng P-W, Boat TF, Cranfill K, Yankaskas JR, Boucher RC. Increased sulfation of glycoconjugates by cultured nasal epithelial cells from patients with cystic fibrosis. J Clin Invest 1989; 84:68-72.
45. Dosanjh A, Lencer W, Brown D, Ausiello DA, Stow JL. Heterologous expression of AF508 CFTR results in decreased sialylation of membrane glycoconjugates. Am J Physiol 1994; 266:C360-C366.
46. Saiman L, Prince A. Pseudomonas aeruginosa pili bind to asialoGM1 which is increased on the surface of cystic fibrosis epithelial cells. J Clin Invest 1993; 92: 1875-1880.
47. Govan JR, Nelson JW. Microbiology of cystic fibrosis lung infections: themes and issues. J R Soc Med 1993; 86 S20:11-18.
48. Oppenheimer EH, Esterly JR. Pathology of cystic fibrosis; review of the literature and comparison with 146 autopsied cases. Perspect Pediatr Pathol 1975; 2:241-278.
49. Chow CW, Landau LI, Taussig LM. Bronchial mucous glands in the newborn with cystic fibrosis. Eur J Pediatr 1982; 139:240-243.
50. Larson JE, Delcarpio JB, Farberman MM, Morrow SL, Cohen JC. CFTR modulates lung secretory cell proliferation and differentiation. Am J Physiol Lung Cell Mol Physiol 2000; 279:L333-341.
51. Khan TZ, Wagener JS, Bost T, Martinez J, Accurso FJ, Riches DW. Early pulmonary inflammation in infants with cystic fibrosis. Am J Respir Crit Care Med 1995; 151:1075-1082.
52. Abman SH, Ogle JW, Harbeck RJ, Butler-Simon N, Hammond KB, Accurso FJ. Early bactériologie, immunologic, and clinical courses of young infants with cystic fibrosis identified by neonatal screening. J Pediatr 1991; 119:211-217.
53. Tirouvanziam R, de Bentzmann S, Hubeau C, Hinnrasky J, Jacquot J, Peault B, Puchelle E. Inflammation and infection in naive human cystic fibrosis airway grafts. Am J Respir Cell Mol Biol 2000; 23:121-127.
54. Armstrong DS, Grimwood K, Carzino R, Carlin JB, Olinsky A, Phelan PD. Lower respiratory infection and inflammation in infants with newly diagnosed cystic fibrosis. BMJ 1995; 310:1571-1572.
55. Matsui H, Grubb BR, Tarran R, Randell SH, Gatzy JT, Davis CW, Boucher RC. Evidence for periciliary liquid layer depletion, not abnormal ion composition, in the pathogenesis of cystic fibrosis airways disease. Cell 1998; 95:1005-1015.
56. Smith JJ, Travis SM, Greenberg EP, Welsh MJ. Cystic fibrosis airway epithelia fail to kill bacteria because of abnormal airway surface fluid. Cell 1996; 85:229-236.
57. Knowles MR, Robinson JM, Wood RE, Pue CA, Mentz WM, Wager GC, Gatzy JT, Boucher RC. Ion composition of airway surface liquid of patients with cystic fibrosis as compared with normal and disease-control subjects. J Clin Invest 1997; 100:2588-2595.
58. Pier GB, Grout M, Zaidi TS, Olsen JC, Johnson LG, Yankaskas JR, Goldberg JB. Role of mutant CFTR in hypersusceptibility of cystic fibrosis patients to lung infections. Science 1996; 271:64-67.
59. Engelhardt JF, Zepeda M, Cohn JA, Yankaskas JR, Wilson JM. Expression of the cystic fibrosis gene in adult human lung. J Clin Invest 1994; 93:737-749.
60. Engelhardt JF, Yankaskas JR, Ernst SA, Yang Y, Marino CR, Boucher RC, Cohn JA, Wilson JM. Submucosal glands are the predominant site of CFTR expression in the human bronchus. Nat Genet 1992; 2:240-248.
61. Hong KU, Reynolds SD, Giangreco A, Hurley CM, Stripp BR. Clara cell secretory protein-expressing cells of the airway neuroepithelial body microenvironment include a label-retaining subset and are critical for epithelial renewal after progenitor cell depletion. Am J Respir Cell Mol Biol 2001; 24:671-681.
62. Warburton D, Wuenschell C, Flores-Delgado G, Anderson K. Commitment and differentiation of lung cell lineages. Biochem Cell Biol 1998; 76:971-995.
63. Wilson PD. Cystic fibrosis transmembrane conductance regulator in the kidney: clues to its role? Exp Nephrol 1999; 7:284-289.
64. Johnson LG, Olsen JC, Sarkadi B, Moore KL, Swanstrom R, Boucher RC. Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat Genet 1992; 2:21-25.
65. Dorin JR, Farley R, Webb S, Smith SN, Farini E, Delaney SJ, Wainwright BJ, Alton EW, Porteous DJ. A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction. Gene Ther 1996; 3:797-801.
66. Johnson LG, Boyles SE, Wilson J, Boucher RC. Normalization of raised sodium absorption and raised calcium-mediated chloride secretion by adenovirus-mediated expression of cystic fibrosis transmembrane conductance regulator in primary human cystic fibrosis airway epithelial cells. J Clin Invest 1995; 95:1377-1382.
67. Zhang Y, Jiang Q, Dudus L, Yankaskas JR, Engelhardt JF. Vector-specific profiles of two independent primary defects in cystic fibrosis airways. Hum Gene Ther 1998; 20:635-648.
68. Gan KH, Veeze HJ, van den Ouweland AM, Halley DJ, Scheffer H, van der Hout A, Overbeek SE, de Jongste JC, Bakker W, Heijerman HG. A cystic fibrosis mutation associated with mild lung disease. N Engl J Med 1995; 333:95-99.
69. Knowles MR, Paradiso AM, Boucher RC. In vivo nasal potential difference: techniques and protocols for assessing efficacy of gene transfer in cystic fibrosis. Hum Gene Ther 1995; 6:445-455.
70. Middleton PG, Geddes DM, Alton EWFW. Protocols for in vivo measurement of the ion transport defects in cystic fibrosis nasal epithelium. Eur Respir J 1994; 7: 2050-2056.
71. Wilschanski M, Famini C, Blau H, Rivlin J, Augarten A, Avital A, Kerem B, Kerem E. A pilot trial of the effect of gentamicin on nasal potential difference measurements in cystic fibrosis patients carrying stop mutations. Am J Respir Crit Care Med 2000; 161:860-865.
72. Alton EWFW, Stern M, Farley R, Jaffe A, Chadwick SL, Phillips J, Davies J, Smith SN, Browning J, Davies MG, Hodson ME, Durham SR, Li D, Jeffery PK, Scallan M, Balfour R, Eastman SJ, Cheng SH, Smith AE, Meeker D, Geddes DM. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. Lancet 1999; 353:947-954.
73. Davies JC, Davies MG, Jaffe A, Bush A, Scallon M, Geddes DM, Alton EWFW. Confirmation of abnormal chloride ion secretion in the lower airway of children with cystic fibrosis. Ped Pulmonol 2000; S20:293.
74. Welsh MJ An apical-membrane chloride channel in human tracheal epithelium. Science 1986; 232:1648-1650.
75. Alton EW, Rogers DF, Logan-Sinclair R, Yacoub M, Barnes PJ, Geddes DM. Bioelectric properties of cystic fibrosis airways obtained at heart-lung transplantation. Thorax 1992; 47:1010-1014.
76. Stern M, Munkonge FM, Caplen NJ, Sorgi F, Huang L, Geddes DM, Alton EW. Quantitative fluorescence measurements of chloride secretion in native airway epithelium from CF and non-CF subjects. Gene Ther 1995; 2:766-774.
77. Davies JC, Stern M, Dewar A, Caplen NJ, Munkonge FM, Pitt T, Sorgi F, Huang L, Bush A, Geddes DM, Alton EW. CFTR gene transfer reduces the binding of Pseudomonas aeruginosa to cystic fibrosis respiratory epithelium. Am J Respir Cell Mol Biol 1997; 16:657-663.
78. Drumm ML, Pope HA, Cliff WH, Rommens JM, Marvin SA, Tsui LC, Collins FS, Frizzell RA, Wilson JM. Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer. Cell 1990; 62:1227-1233.
79. Rich DP, Anderson MP, Gregory RJ, Cheng SH, Paul S, Jefferson DM, McCann JD, Klinger KW, Smith AE, Welsh MJ. Expression of cystic fibrosis transmembrane conductance regulator corrects defective chloride channel regulation in cystic fibrosis airway epithelial cells. Nature 1990; 347:358-363.
80. Dorin JR, Dickinson P, Alton EW, Smith SN, Geddes DM, Stevenson BJ, Kimber WL, Fleming S, Clarke AR, Hooper ML, et al. Cystic fibrosis in the mouse by targeted insertional mutagenesis. Nature 1992; 359:211-216.
81. Snouwaert JN, Brigman KK, Latour AM, Iraj E, Schwab U, Gilmour MI, Koller BH. A murine model of cystic fibrosis. Am J Respir Crit Care Med 1995; 151: S59-64.
82. Delaney SJ, Alton EWFW, Smith S, Lunn DP, Farley R, Lovelock PK, Thomson SA, Hume DA, Lamb D, Porteous DJ, Dorin JR, Wainwright BJ. Cystic fibrosis carrying the missense mutation G551D replicated human genotype-phenotype correlations. EMBO J 1996; 15:955-963.
83. Hyde SC, Gill DR, Higgins CF, Trezise AE, MacVinish LJ, Cuthbert AW, Ratcliff R, Evans MJ, Colledge WH. Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy. Nature 1993; 362:250-255.
84. Alton EWFW, Middleton PG, Caplen NJ, Smith SN, Steel DM, Munkonge FM, Jeffery PK, Geddes DM, Hart SL, Williamson R, et al. Non-invasive liposomemediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nat Genet 1993; 5:135-142.
85. Grubb BR, Pickles RJ, Ye H, Yankaskas JR, Vick RN, Engelhardt JF, Wilson JM, Johnson LG, Boucher RC. Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humans. Nature 1994; 371:802-806.
86. Pickles RJ, Barker PM, Ye H, Boucher RC. Efficient adenovirus-mediated gene transfer to basal but not columnar cells of cartilaginous airway epithelia. Hum Gene Ther 1996; 7:921-931.
87. Scaria A St George JA, Jiang C, Kaplan JM, Wadsworth SC, Gregory RJ. Adenovirus-mediated persistent cystic fibrosis transmembrane conductance regulator expression in mouse airway epithelium. J Virol 1998; 72:7302-7309.
88. Flotte TR, Afione SA, Conrad C, McGrath SA, Solow R, Oka H, Zeitlin PL, Gug-gino WB, Carter BJ. Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci USA 1993; 90:10613-10617.
89. 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 1993; 75:207-216.
90. Boucher RC, Knowles MR, Johnson LG, Olsen JC, Pickles R, Wilson JM, Engelhardt J, Yang Y, Grossman M. Gene therapy for cystic fibrosis using El-deleted adenovirus: a phase I trial in the nasal cavity. The University of North Carolina at Chapel Hill. Hum Gene Ther 1994; 5:615-639.
91. Zuckerman JB, Robinson CB, McCoy KS, Shell R, Sferra TJ, Chirmule N, Magosin SA, Propert KJ, Brown-Parr EC, Hughes JV, Tazelaar J, Baker C, Goldman MJ, Wilson JM. A phase I study of adenovirus-mediated transfer of the human cystic fibrosis transmembrane regulator gene to a lung segment of individuals with cystic fibrosis. Hum Gene Ther 1999; 10:2973-2985.
92. Bellon G, Michel-Calemard L, Thouvenot D, Jagneaux V, Poitevin F, Malcus C, Accart N, Layani MP, Aymard M, Bernon H, Bienvenu J, Courtney M, Doring G, Gilly B, Gilly R, Lamy D, Levrey H, Morel Y, Paulin C, Perraud F, Rodillon L, Sene C, So S, Touraine-Moulin F, Pavirani A, et al. Aerosol administration of a recombinant adenovirus expressing CFTR to cystic fibrosis patients: a phase I clinical trial. Hum Gene Ther 1997; 8:15-25.
93. Harvey B-G, Leopold PL, Hackett NR, Grasso TM, Williams PM, Tucker AL, Kaner RJ, Ferris B, Gonda I, Sweeney TD, Ramalingam R, Kovesdi I, Shak S, Crystal RG. Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus. J Clin Invest 1999; 104:1245-1255.
94. Harvey BG, Hackett NR, El-Sawy T, Rosengart TK, Hirschowitz EA, Lieberman MD, Lesser ML, Crystal RG: Variability of human systemic humoral immune responses to adenovirus gene transfer vectors administered to different organs. J Virol 1999; 73:6729-6742.
95. Wagner JA, Reynolds T, Moran ML, Moss RB, Wine JJ, Flotte TR, Gardner P. Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus. Lancet 1998; 351:1702-1703.
96. Caplen NJ, Alton EWFW, Middleton PG, Dorin JR, Stevenson BJ, Gao X, Durham SR, Jeffery PK, Hodson ME, Coutelle C, et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat Med 1995; 1: 39-46.
97. Gill DR, Southern KW, Mofford KA, Seddon T, Huang L, Sorgi F, Thomson A, MacVinish LJ, Ratcliff R, Bilton D, Lane DJ, Littlewood JM, Webb AK, Middleton PG, Colledge WH, Cuthbert AW, Evans MJ, Higgins CF, Hyde SC. A placebo controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther 1997; 4:199-209.
98. Porteous DJ, Dorin JR, McLachlan G, Davidson-Smith H, Davidson H, Stevenson BJ, Carothers AD, Wallace WA, Moralee S, Hoenes C, Kallmeyer G, Michaelis U, Naujoks K, Ho LP, Samways JM, Imrie M, Greening AP, Innes JA. Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to nasal epithelium of patients with cystic fibrosis. Gene Ther 1997; 4:210-218.
99. Noone PG, Hohneker KW, Zhou Z, Johnson LG, Foy C, Gipson C, Jones K, Noah TL, Leigh MW, Schwartzbach C, Efthimiou J, Pearlman R, Boucher RC, Knowles MR. 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.
100. 5000 formulation to the lungs of normal volunteers. Gene Ther 1997; 4:937-942.
101. Coakley RJ, Taggart C, O’Neill S, McElvaney NG. Alphal-antitrypsin deficiency: biological answers to clinical questions. Am J Med Sci 2001; 321:33-41.
102. Pierce JA. Alphal-antitrypsin augmentation therapy. Chest 1997; 112:872-874.
103. Kay MA, Graham F, Leland F, Woo SL. Therapeutic serum concentrations of human alpha-l-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes. Hepatology 1995; 21:815-819.
104. Song S, Morgan M, Ellis T, Poirier A, Chesnut K, Wang J, Brantly M, Muzyczka N, Byrne BJ, Atkinson M, Flotte TR. Sustained secretion of human alpha-1-anti-trypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci USA 1998; 95:14384-14388.
105. Bou-Gharios G, Wells DJ, Lu QL, Morgan JE, Partridge T. Differential expression and secretion of alphal anti-trypsin between direct DNA injection and implantation of transfected myoblast. Gene Ther 1999; 6:1021-1029.
106. 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 1994; 10:24-29.
107. Brigham KL, Lane KB, Meyrick B, Stecenko AA, Strack S, Cannon DR, Caudill M, Canonico AE. Transfection of nasal mucosa with a normal a1-antitrypsin gene in a1-antitrypsin-deficient subjects: comparison with protein therapy. Hum Gene Ther 2000; 11:1023-1032.
108. Canonico AE, Parker RE, Meyrick BO, Gao X, Lane K, Cannon D, Wilson T, Brigham KL. Alpha-1 antitrypsin (AAT) gene therapy is superior to protein therapy in a piglet in situ lung model. Am J Respir Lung Mol Biol 1997; 14:385-390.
109. Rom WN, Hay JG, Lee TC, Jiang Y, Tchou-Wong K-M. Molecular and genetic aspects of lung cancer. Am J Respir Crit Care Med 2000; 161:1355-1367.
110. Roth JA, Nguyen D, Lawrence DD, Kemp BL, Carrasco CH, Ferson DZ, Hong WK, Komaki R, Lee JJ, Nesbitt JC, Pisters KM, Putnam JB, Schea R, Shin DM, Walsh GL, Dolormente MM, Han CI, Martin FD, Yen N, Xu K, Stephens LC, McDonnell TJ, Mukhopadhyay T, Cai D. Retrovirus-mediated wild-type p53 gene transfer to tumors of patients with lung cancer. Nat Med 1996; 2:985-991.
111. Schuler M, Rochlitz C, Horowitz JA, Schlegel J, Perruchoud AP, Kommoss F, Bolliger CT, Kauczor HU, Dalquen P, Fritz MA, Swanson S, Herrmann R, Huber C. A phase I study of adenovirus-mediated wild-type p53 gene transfer in patients with advanced non-small cell lung cancer. Hum Gene Ther 1998; 9:2075-2082.
112. Swisher SG, Roth JA, Nemunaitis J, Lawrence DD, Kemp BL, Carrasco CH, Connors DG, El-Naggar AK, Fossella F, Glisson BS, Hong WK, Khuri FR, Kurie JM, Lee JJ, Lee JS, Mack M, Merritt JA, Nguyen DM, Nesbitt JC, Perez-Soler R, Pisters KM, Putnam JB Jr, Richli WR, Savin M, Waugh MK, et al. Adenovirus-mediated p53 gene transfer in advanced non-small cell lung cancer. J Natl Cancer Inst 1999; 91:763-771.
113. Nemunaitis J, Swisher SG, Timmons T, Connors D, Mack M, Doerksen L, Weill D, Wait J, Lawrence DD, Kemp BL, Fossella F, Glisson BS, Hong WK, Khuri FR, Kurie JM, Lee JJ, Lee JS, Nguyen DM, Nesbitt JC, Perez-Soler R, Pisters KM, Putnam JB, Richli WR, Shin DM, Walsh GL, et al. Adenovirus-mediated p53 gene transfer in sequence with cisplatin to tumors of patients with non-small cell lung cancer. J Clin Oncol 2000; 18:609-622.
114. Yen N, Ioannides CG, Xu K, Swisher SG, Lawrence DD, Kemp BL, El-Naggar AK, Cristiano RJ, Fang B, Glisson BS, Hong WK, Khuri FR, Kurie JM, Lee JJ, Lee JS, Merritt JA, Mukhopadhyay T, Nesbitt JC, Nguyen D, Perez-Soler R, Pisters KM, Putnam JB Jr, Schrump DS, Shin DM, Walsh GL, Roth JA. Cellular and humoral immune responses to adenovirus and p53 protein antigens in patients following intratumoral injection of an adenovirus vector expressing wild-type P53 (Ad-p53). Cancer Gene Ther 2000; 7:530-536.
115. Smythe WR. Prodrug/drug sensitivity gene therapy: current status. Curr Oncol Rep 2000; 2:17-22.
116. Sterman DH, Treat J, Litzky LA, Amin KM, Coonrod L, Molnar-Kimber K, Recio A, Knox L, Wilson JM, Albelda SM, Kaiser LR. Adenovirus-mediated herpes simplex virus thymidine kinase/ganciclovir gene therapy in patients with localised malignancy: results of a phase I clinical trial in malignant mesothelioma. Hum Gene Ther 1998; 9:1083-1092.
117. Tursz T, Cesne AL, Baldeyrou P, Gautier E, Opolon P, Schatz C, Pavirani A, Courtney M, Lamy D, Ragot T, Saulnier P, Andremont A, Monier R, Perricaudet M, Le Chevalier T. Phase I study of a recombinant adenovirus-mediated gene transfer in lung cancer patients. J Natl Cancer Inst 1996; 88:1857-1863.
118. Gahery-Segard H, Molinier-Frenkel V, Le Boulaire C, Saulnier P, Opolon P, Lengagne R, Gautier E, Le Cesne A, Zitvogel L, Venet A, Schatz C, Courtney M, Le Chevalier T, Tursz T, Guillet JG, Farace F. Phase I trial of recombinant adenovirus gene transfer in lung cancer. Longitudinal study of the immune responses to transgene and viral products. J Clin Invest 1997; 100:2218-2226.
119. Mukherjee S, Haenel T, Himbeck R, Scott B, Ramshaw I, Lake RA, Harnett G, Phillips P, Morey S, Smith D, Davidson JA, Musk AW, Robinson B. Replication-restricted vaccinia as a cytokine gene therapy vector in cancer: persistent transgene expression despite antibody generation. Cancer Gene Ther 2000; 7:663-670.
120. McCormick F. Interactions between adenovirus proteins and the p53 pathway: the development of ONYX-015. Semin Cancer Biol 2000; 10:453-459.
121. Bischoff JR, Kim DH, Williams A, Heise C, Horn S, Muna M, Ng L, Nye JA, Sampson-Johannes A, Fattaey A, McCormick F. An adenovirus mutant that replicates selectively in p53-deficient human tumor cells. Science 1996; 274:373-376.
122. Heise C, Sampson-Johannes A, Williams A, McCormick F, Von Hoff DD, Kim DH. ONYX-015, an E1B gene-attenuated adenovirus, causes tumor-specific cytoly-sis and antitumoral efficacy that can be augmented by standard chemotherapeutic agents. Nat Med 1997; 3:639-645.
123. Nemunaitis J, Cunningham C, Buchanan A, Blackburn A, Edelman G, Maples P, Netto G, Tong A, Randlev B, Olson S, Kim D. Intravenous infusion of a replication-selective adenovirus (ONYX-015) in cancer patients: safety, feasibility and biological activity. Gene Ther 2001; 8:746-759.
124. Weinacker AB, Vaszar LT. Acute respiratory distress syndrome: physiology and new management strategies. Annu Rev Med 2001; 52:221-237.
125. Dennehy KC, Bigatello LM. Pathophysiology of the acute respiratory distress syndrome. Int Anesthesiol Clin 1999; 37:1-13.
126. Rogy MA, Auffenberg T, Espat NJ, Philip R, Remick D, Wollenberg GK, Copeland EM 3rd, Moldawer LL. 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 1995; 181:2289-2293.
127. 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 1997; 4:140-149.
128. Conary JT, Parker RE, Christman BW, Faulks RD, King GA, Meyrick BO, Brigham KL. Protection of rabbit lungs from endotoxin injury by in vivo hyperexpression of the prostaglandin G/H synthase gene. J Clin Invest 1994; 93:1834-1840.
129. von der Leyen HE, Gibbons GH, Morishita R, Lewis NP, Zhang L, Nakajima M, Kaneda Y, Cooke JP, Dzau VJ. Gene therapy inhibiting neointimal vascular lesion: in vivo transfer of endothelial cell nitric oxide synthase gene. Proc Natl Acad Sci USA 1995; 92:1137-1141.
130. Kumasaka T, Quinlan WM, Doyle NA, Condon TP, Sligh J, Takei F, Beaudet Al, Bennett CF, Doerschuk CM. Role of the intercellular adhesion molecule-1 (ICAM-1) in endotoxin-induced pneumonia evaluated using ICAM-1 antisense oligonucleotides, anti-ICAM-1 monoclonal antibodies, and ICAM-1 mutant mice. J Clin Invest 1996; 97:2362-2369.
131. Lee JC, Laydon JT, McDonnell PC, Gallagher TF, Kumar S, Green D, McNulty D, Blumenthal MJ, Heys JR, Landvatter SW, et al. A protein kinase involved in the regulation of inflammatory cytokine biosynthesis. Nature 1994; 372:739-746.
132. Inoue S, Suzuki M, Nagashima Y, Suzuki S, Hashiba T, Tsuburai T, Ikehara K, Matsuse T, Ishigatsubo Y. Transfer of Heme Oxygenase 1 cDNA by a replication-deficient adenovirus enhances interleukin 10 production from alveolar macrophages that attenuates lipopolysaccharide-induced acute lung injury in mice. Hum Gene Ther 2001; 12:967-979.
133. Factor P. Role and regulation of lung Na, K-ATPase. Cell Mol Biol 2001; 47:347-361.
134. Factor P, Dumasius V, Saldias F, Brown LA, Sznajder JI. Adenovirus-mediated transfer of an Na+/K +-ATPase betal subunit gene improves alveolar fluid clearance and survival in hyperoxic rats. Hum Gene Ther 2000; 11:2231-2242.
135. Stern M, Ulrich K, Robinson C, Copeland J, Griesenbach U, Masse C, Cheng S, Munkonge F, Geddes D, Berthiaume Y, Alton E. Pretreatment with cationic lipidmediated transfer of the Na+K +-ATPase pump in a mouse model in vivo augments resolution of high permeability pulmonary oedema. Gene Ther 2000; 7:960-966.
136. Suissa S, Ernst P. Inhaled corticosteroids: impact on asthma morbidity and mortality. J Allergy Clin Immunol 2001; 107:937-944.
137. Lee NA, Gelfand EW, Lee JJ. Pulmonary T cells and eosinophils: coconspirators or independent triggers of allergic respiratory pathology? J Allergy Clin Immunol 2001; 107:945-957.
138. Lack G, Renz H, Saloga J, Bradley KL, Loader J, Leung DY, Larsen G, Gelfand EW. Nebulized but not parenteral IFN-gamma decreases IgE production and normalizes airways function in a murine model of allergen sensitization. J Immunol 1994; 152:2546-2554.
139. Schwarze J, Hamelmann E, Cieslewicz G, Tomkinson A, Joetham A, Bradley K, Gelfand EW. Local treatment with IL-12 is an effective inhibitor of airway hyperresponsiveness and lung eosinophilia after airway challenge in sensitized mice. J Allergy Clin Immunol 1998; 102:86-93.
140. Dow SW, Schwarze J, Heath TD, Potter TA, Gelfand EW. Systemic and local interferon gamma gene delivery to the lungs for treatment of allergen-induced airway hyperresponsiveness in mice. Hum Gene Ther 1999; 10:1905-1914.
141. Hogan SP, Foster PS, Tan X, Ramsay AJ. Mucosal IL-12 gene delivery inhibits allergic airways disease and restores local antiviral immunity. Eur J Immunol 1998; 28:413-423.
142. Mathieu M, Gougat C, Jaffuel D, Danielsen M, Godard P, Bousquet J, Demoly P. The glucocorticoid receptor gene as a candidate for gene therapy in asthma. Gene Ther 1999; 6:245-252.
143. Metzger WJ, Nyce JW. Oligonucleotide therapy of allergic asthma. J Allergy Clin Immunol 1999; 104:260-266.
144. Alton EW, Griesenbach U, Geddes DM. Gene therapy for asthma: inspired research or unnecessary effort? Gene Ther 1999; 6:155-156.
145. Fonseca C, Abraham D, Black CM. Lung fibrosis. Springer Semin Immunopathol 1999; 21:453-474.
146. Sime PJ, O’Reilly KM. Fibrosis of the lung and other tissues: new concepts in pathogenesis and treatment. Clin Immunol 2001; 99:308-319.
147. Nakao A, Fujii M, Matsumura R, Kumano K, Saito Y, Miyazono K, Iwamoto I. Transient gene transfer and expression of Smad7 prevents bleomycin-induced lung fibrosis in mice. J Clin Invest 1999; 104:5-11.
148. Kolb M, Margetts PJ, Galt T, Sime PJ, Xing Z, Schmidt M, Gauldie J. Transient transgene expression of decorin in the lung reduces the fibrotic response to bleomycin. Am J Respir Crit Care Med 2001; 163:770-777.
149. Sisson TH, Hattori N, Xu Y, Simon RH. Treatment of bleomycin-induced pulmonary fibrosis by transfer of urokinase-type plasminogen activator genes. Hum Gene Ther 1999; 10:2315-2323.
150. Epperly M, Bray J, Kraeger S, Zwacka R, Engelhardt J, Travis E, Greenberger J. Prevention of late effects of irradiation lung damage by manganese superoxide dismutase gene therapy. Gene Ther 1998; 5:196-208.
151. Ziesche R, Hofbauer E, Wittmann K, Petkov V, Block LH. A preliminary study of long-term treatment with interferon gamma-lb and low-dose prednisolone in patients with idiopathic pulmonary fibrosis. N Engl J Med 1999; 341:1264-1269.
152. Kuwano K, Hagimoto N, Kawasaki M, Yatomi T, Nakamura N, Nagata S, Suda T, Kunitake R, Maeyama T, Miyazaki H, Hara N. Essential roles of the Fas-Fas ligand pathway in the development of pulmonary fibrosis. J Clin Invest 1999; 104: 13-19.
153. Mal H, Dehoux M, Sleiman C, Boczkowski J, Leseche G, Pariente R, Fourier M. Early release of proinflammatory cytokines after lung transplantation. Chest 1998; 113:645-651.
154. Ward S, Muller NL. Pulmonary complications following lung transplantation. Clin Radiol 2000; 55:332-339.
155. Cassivi SD, Cardella JA, Fischer S, Liu M, Slutsky AS, Keshavjee S. Transtracheal gene transfection of donor lungs prior to organ procurement increases transgene levels at reperfusion and following transplantation. J Heart Lung Transplant 1999; 18:1181-1188.
156. Boasquevisque CH, Mora BN, Boglione M, Ritter JK, Scheule RK, Yew N, De-bruyne L, Qin L, Bromberg JS, Patterson GA. Liposome-mediated gene transfer in rat lung transplantation: A comparison between the in vivo and ex vivo approaches. J Thorac Cardiovasc Surg 1999; 117:8-14.
157. Itano H, Zhang W, Ritter JH, McCarthy TJ, Mohanakumar T, Patterson GA. Adenovirus-mediated gene transfer of human interleukin 10 ameliorates reperfusion injury of rat lung isografts. J Thorac Cardiovasc Surg 2000; 120:947-956.
158. Schmid RA, Stammberger U, Hillinger S, Gaspert A, Boasquevisque CH, Mali-piero U, Fontana A, Weder W. Fas ligand gene transfer combined with low dose cyclosporine A reduces acute lung allograft rejection. Transpl Int 2000; 13(Suppl 1):S324-328.
159. Mora BN, Boasquevisque CH, Boglione M, Ritter JM, Scheule RK, Yew NS, De-bruyne L, Qin L, Bromberg JS, Patterson GA. Transforming growth factor-betal gene transfer ameliorates acute lung allograft rejection. J Thorac Cardiovasc Surg 2000; 119:913-920.
160. Suda T, D’Ovidio F, Daddi N, Ritter JH, Mohanakumar T, Patterson GA. Recipient intramuscular gene transfer of active transforming growth factor-betal attenuates acute lung rejection. Ann Thorac Surg 2001; 71:1651-1656.