Pulmonary gene therapy realistic hope for the future, or false dawn in the promised land?

Monaldi Archives for Chest Disease - Pulmonary Series

Volumn 59, Issue 1, 2003, Pages 17-24

  Jenkins, R G ^a   McAnulty, R J ^a   Hart, S L ^b   Laurent, G J ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; ANTIBODY; ASIALOOROSOMUCOID; CLATHRIN; EPIDERMAL GROWTH FACTOR; FIBROBLAST GROWTH FACTOR; IMMUNOSUPPRESSIVE AGENT; INTEGRIN; LIGAND; LIPOSOME; LUCIFERASE; POLYLYSINE; RECEPTOR; TRANSFERRIN; TRANSMEMBRANE CONDUCTANCE REGULATOR; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENOVIRUS; CARDIOVASCULAR DISEASE; CLINICAL TRIAL; COMBINED IMMUNODEFICIENCY; CYSTIC FIBROSIS; DRUG TOLERABILITY; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSFECTION; HEAD AND NECK CANCER; HEMOPHILIA; HUMAN; IMMUNOGENICITY; IN VITRO STUDY; IN VIVO STUDY; INFLAMMATION; INJURY; INTRON; LUNG CANCER; LUNG DISEASE; LUNG FIBROSIS; LUNG NON SMALL CELL CANCER; LUNG TOXICITY; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; RESPIRATORY DISTRESS SYNDROME; REVIEW; SENDAI VIRUS; SIDE EFFECT; VIRAL GENE DELIVERY SYSTEM;

EID: 0141571068     PISSN: 11220643     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (85)

1. Crystal, R.G., Mcelvaney, N.G., Rosenfeld, M.A., Chu, C.S., Mastrangeli, A., Hay, J.G., Brody, S.L., Jaffe, H.A., Eissa, N.T. and 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.
2. Bellon, G., Michel-Calemard, L., Thouvenot, D., Jagneaux, V., Poitevin, F., Malcus, C., Accart, N., Layani, M.P., 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., Schatz, C. and Pavirani, A. Aerosol administration of a recombinant adenovirus expressing CFTR to cystic fibrosis patients: A phase I clinical trial. Hum. Gene Ther. 1997; 8, 15-25.
3. Alton, E.W., Stern, M., Farley, R., Jaffe, A., Chadwick, S.L., Phillips, J., Davies, J., Smith, S.N., Browning, J., Davis, M.G., Hodson, M.E., Durham, S.R., Li, D., Jeffery, P.K., Scallan, M., Balfour, R., Eastman, S.J., Cheng, S.H., Smith, A.E., Meeker, D. and Geddes, D.M. 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.
4. Hyde, S.C., Southern, K.W., Gileadi, U., Fitzjohn, E.M., Mofford, K.A., Waddell, B.E., Gooi, H.C., Goddard, C.A., Hannavy, K., Smyth, S.E., Egan, J.J., Sorgi, F.L., Huang, L., Cuthbert, A.W., Evans, M.J., Colledge, W.H., Higgins, C.F., Webb, A.K. and Gill, D.R. Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis. Gene Ther. 2000; 7, 1156-1165.
5. Weill, D., Mack, M., Roth, J., Swisher, S., Proksch, S., Merritt, J. and Nemunaitis, J. Adenoviral-mediated p53 gene transfer to non-small cell lung cancer through endobronchial injection. Chest 2000; 118, 966-970.
6. Graham, F.L. Adenovirus vectors for high-efficiency gene transfer into mammalian cells. Immunol. Today. 2000; 21, 426-8.
7. Yonemitsu, Y., Kitson, C., Ferrari, S., Farley, R., Griesenbach, U., Judd, D., Steel, R., Scheid, P., Zhu, J., Jeffery, P.K., Kato, A., Hasan, M.K., Nagai, Y., Masaki, I., Fukumura, M., Hasegawa, M., Geddes, D.M. and Alton, E.W.F.W. Efficient gene transfer to airway epithelium using recombinant Sendai virus. Nat. Biotechnol. 2000; 18, 970-3.
8. Monahan, P.E. and Samulski, R.J. AAV vectors: Is clinical success on the horizon? Gene Ther. 2000; 7, 24-30.
9. Lehn, P., Fabrega, S., Oudrhiri, N. and Navarro, J. Gene delivery systems: Bridging the gap between recombinant viruses and artificial vectors. Adv. Drug Deliv. Rev. 1998; 39, 5-11.
10. Robbins, P.D. and Ghivizzani, S.C. Viral vectors for gene therapy. Pharmacol. Ther. 1998; 80, 35-47.
11. Yang, Y., Li, Q., Ertl, H.C. and Wilson, J.M. Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J. Virol. 1995; 69, 2004-2015.
12. Otake, K., Ennist, D.L., Harrod, K. and Trapnell, B.C. Nonspecific inflammation inhibits adenovirus-mediated pulmonary gene transfer and expression independent of specific acquired immune responses. Hum. Gene Ther. 1998; 9, 2207-2222.
13. Yei, S., Mittereder, N., Tang, K., O'Sullivan, C. and Trapnell, B.C. Adenovirus-mediated gene transfer for cystic fibrosis: Quantitative evaluation of repeated in vivo vector administration to the lung. Gene Ther. 1994; 1, 192-200.
14. Flotte, T.R., Afione, S.A., Conrad, C., McGrath, S.A., Solor, R., Oka, H., Zeitlin, P.L., Guggino, W.B. and Carter, B.J. Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc. Natl. Acad. Sci. U. S. A. 1993; 90, 10613-10617.
15. Beck, S.E., Jones, L.A., Chesnut, K., Walsh, S.M., Reynolds, T.C., Carter, B.J., Askin, F.B., Flotte, T.R., Guggino, W.B. Repeated delivery of adeno-associated virus vectors to the rabbit airway. J. Virol. 1999; 73, 9446-9455
16. Dong, J.Y., Fan, P.D. and Frizzell, R.A. Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum. Gene Ther. 1996a; 7, 2101-2112.
17. Nakai, H., Storm, T.A. and Kay, M.A. Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nat. Biotechnol. 2000; 18, 497-8.
18. Smith, J.G., Walzem, R.L. and German, J.B. Liposomes as agents of DNA transfer. Biochim. Biophys. Acta 1993; 1154, 327-40.
19. Niven, R., Zhang, Y. and Smith, J. Toward development of a non-viral gene therapeutic. Adv. Drug Deliv. Rev. 1997; 26, 135-150.
20. Cullis, P.R. and Chonn, A. Recent advances in liposome technologies and their applications for systemic gene delivery. Adv. Drug Deliv. Rev. 1997; 30, 73-83.
21. Alton, E. and Geddes, D. Cystic fibrosis clinical trials. Adv. Drug Deliv. Rev. 1998; 30, 205-217.
22. Scheule, R.K. and Cheng, S.H. Airway delivery of cationic lipid: DNA complexes for cystic fibrosis. Adv. Drug Deliv. Rev. 1998; 30, 173-184.
23. Ferrari, S., Moro, E., Pettenazzo, A., Behr, J.P., Zacchello, F. and Scarpa, M. ExGen 500 is an efficient vector for gene delivery to lung epithelial cells in vitro and in vivo. Gene Ther. 1997; 4, 1100-1106.
24. Zabner, J., Cheng, S.H., Meeker, D., Launspach, J., Balfour, R., Perricone, M.A., Morris, J.E., Marshall, J., Fasbender, A., Smith, A.E. and Welsh, M.J. Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo. J. Clin. Invest. 1997; 100, 1529-1537.
25. Matsui, H., Johnson, L.G., Randell, S.H. and Boucher, R.C. Loss of binding and entry of liposome-DNA complexes decreases transfection efficiency in differentiated airway epithelial cells. J. Biol. Chem. 1997; 272, 1117-1126.
26. Cotten, M., Wagner, E., Zatloukal, K., Phillips, S., Curiel, D.T. and Birnstiel, M.L. High-efficiency receptor-mediated delivery of small and large (48 kilobase gene constructs using the endosome-disruption activity of defective or chemically inactivated adenovirus particles. Proc. Natl. Acad. Sci. U. S. A. 1992; 89, 6094-6098.
27. Wagner, E., Zenke, M., Cotten, M., Beug, H. and Birnstiel, M.L. Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc. Natl. Acad. Sci. U. S. A. 1990; 87, 3410-4.
28. Cristiano, R.J. and Roth, J.A. Epidermal growth factor mediated DNA delivery into lung cancer cells via the epidermal growth factor receptor. Cancer Gene Ther. 1996; 3, 4-10.
29. Sosnowski, B.A., Gonzalez, A.M., Chandler, L.A., Buechler, Y.J., Pierce, G.F. and Baird, A. Targeting DNA to cells with basic fibroblast growth factor (FGF2). J. Biol. Chem. 1996; 271, 33647-33653.
30. Wu, G.Y. and Wu, C.H. Receptor-mediated in vitro gene transformation by a soluble DNA carrier system. J. Biol. Chem. 1987; 262, 4429-32.
31. Wu, G.Y. and Wu, C.H. Receptor-mediated gene delivery and expression in vivo. J. Biol. Chem. 1988; 263, 14621-4.
32. Wu, G.Y., Wilson, J.M., Shalaby, F., Grossman, M., Shafritz, D.A. and Wu, C.H. Receptor-mediated gene delivery in vivo. Partial correction of genetic analbuminemia in Nagase rats. J. Biol. Chem. 1991; 266, 14338-42.
33. Perales, J.C., Ferkol, T., Beegen, H., Ratnoff, O.D. and Hanson, R.W. Gene transfer in vivo: Sustained expression and regulation of genes introduced into the liver by receptor-targeted uptake. Proc. Natl. Acad. Sci. U. S. A. 1994; 91, 4086-4090.
34. Ferkol, T., Perales, J.C., Mularo, F. and Hanson, R.W. Receptor-mediated gene transfer into macrophages. Proc. Natl. Acad. Sci. U. S. A. 1996; 93, 101-5.
35. Ferkol, T., Perales, J.C., Eckman, E., Kaetzel, C.S., Hanson, R.W. and Davis, P.B. Gene transfer into the airway epithelium of animals by targeting the polymeric immunoglobulin receptor. J. Clin. Invest. 1995; 95, 493-502.
36. Wagner, E., Ogris, M. and Zauner, W. Polylysine-based transfection systems utilizing receptor-mediated delivery. Adv. Drug Deliv. Rev. 1998; 30, 97-113.
37. Isberg, R.R. Discrimination between intracellular uptake and surface adhesion of bacterial pathogens. Science 1991; 252, 934-8.
38. Newham, P. and Humphries, M.J. Integrin adhesion receptors: Structure, function and implications for biomedicine. Mol. Med. Today 1996; 2, 304-313.
39. Rankin, S., Isberg, R.R. and Leong, J.M. The integrin-binding domain of invasin is sufficient to allow bacterial entry into mammalian cells. Infect. Immun. 1992; 60, 3909-3912.
40. Bai, M., Campisi, L. and Freimuth, P. Vitronectin receptor antibodies inhibit infection of HeLa and A549 cells by adenovirus type 12 but not by adenovirus type 2. J. Virol. 1994; 68, 5925-5932.
41. Bergelson, J.M., Shepley, M.P., Chan, B.M., Hemler, M.E. and Finberg, R.W. Identification of the integrin VLA-2 as a receptor for echovirus 1. Science 1992; 255, 1718-1720.
42. Hart, S.L., Knight, A.M., Harbottle, R.P., Mistry, A., Hunger, H.D., Cutler, D.F., Williamson, R. and Coutelle, C. Cell binding and internalization by filamentous phage displaying a cyclic Arg-Gly-Asp-containing peptide. J. Biol. Chem. 1994; 269, 12468-12474.
43. Hart, S.L., Harbottle, R.P., Cooper, R., Miller, A., Williamson, R. and Coutelle, C. Gene delivery and expression mediated by an integrin-binding peptide. Gene Ther. 1995; 2, 552-554.
44. Jenkins, R.G., Herrick, S.E., Meng, Q.H., Kinnon, C., Laurent, G.J., Mcanulty, R.J. and Hart, S.L. An integrin-targeted non-viral vector for pulmonary gene therapy. Gene Ther. 2000; 7, 393-400
45. Hart, S.L., Arancibia, Carcamo, C.V., Wolfert, M.A., Mailhos, C., O'Reilly, N.J., Ali, R.R., Coutelle, C., George, A.J., Harbottle, R.P., Knight, A.M., Larkin, D.F., Levinsky, R.J., Seymour, L.W., Thrasher, A.J. and Kinnon, C. Lipid-mediated enhancement of transfection by a nonviral integrin-targeting vector. Hum. Gene Ther. 1998; 9, 575-585.
46. Lee, E.R., Marshall, J., Siegel, C.S., Jiang, C., Yew, N.S., Nichols, M.R., Nietupski, J.B., Ziegler, R.J., Lane, M.B., Wang, K.X., Wan, N.C., Scheule, R.K., Harris, D.J., Smith, A.E. and Cheng, S.H. Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lung. Hum. Gene Ther. 1996; 7, 1701-1717.
47. Dong, J.Y., Wang, D., Van Ginkel, F.W., Pascual, D.W. and Frizell, R.A. Systematic analysis of repeated gene delivery into animal lungs with a recombinant adenovirus vector. Hum. Gene Ther. 1996b; 7, 319-331.
48. Sawa, T., Miyazaki, H., Pittet, J.F., Widdicombe, J.H., Gropper, M.A., Hashimoto, S., Conrad, D.J., Folkesson, H.G., Debs, R., Forsayeth, J.R., Fox, B. and Wiener Kronish, J.P. Intraluminal water increases expression of plasmid DNA in rat lung. Hum. Gene Ther. 1996; 7, 933-941.
49. Tsan, M.F., White, J.E. and Shepard, B. Lung-specific direct in vivo gene transfer with recombinant plasmid DNA. Am. J. Physiol. 1995; 268, L1052-6.
50. Yew, N.S., Wysokenski, D.M., Wang, K.X., Ziegler, R.J., Marshall, J., McNeilly, D., Cherry, M., Osburn, W. and Cheng, S.H. Optimization of plasmid vectors for high-level expression in lung epithelial cells. Hum. Gene Ther. 1997; 8, 575-584.
51. Rosenfeld, M.A., Yoshimura, K., Trapnell, B.C., Yoneyama, K., Rosenthal, E.R., Dalemans, W., Fukayama, M., Bargon, J., Stier, L.E., Stratford Perricaudet, L. et al. In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 1992; 68, 143-155.
52. Thompson, J.F., Hayes, L.S. and Lloyd, D.B. Modulation of firefly luciferase stability and impact on studies of gene regulation. Gene 1991; 103, 171-177.
53. Smith, R.L., Geller, A.I., Escudero, K.W. and Wilcox, C.L. Long-term expression in sensory neurons in tissue culture from herpes simplex virus type 1 (HSV-1) promoters in an HSV-1-derived vector. J. Virol. 1995; 69, 4593-4599.
54. Engelhardt, J.F., Litzky, L. and Wilson, J.M. Prolonged transgene expression in cotton rat lung with recombinant adenoviruses defective in E2a. Hum. Gene Ther. 1994; 5, 1217-1229.
55. Vilquin, J.T., Guerette, B., Kinoshita, I., Roy, B., Goulet, M., Gravel, C., Roy, R. and Tremblay, J.P. FK506 immunosuppression to control the immune reactions triggered by first-generation adenovirus-mediated gene transfer. Hum. Gene Ther. 1995; 6, 1391-1401.
56. Jooss, K., Yang, Y. and Wilson, J.M. Cyclophosphamide diminishes inflammation and prolongs transgene expression following delivery of adenoviral vectors to mouse liver and lung. Hum Gene Ther 1996; 7, 1555-1566.
57. Smith, T.A., White, B.D., Gardner, J.M., Kaleko, M. and McClelland, A. Transient immunosuppression permits successful repetitive intravenous administration of an adenovirus vector. Gene Ther. 1996; 3, 496-502.
58. Caplen, N.J., Alton, E.W.F.W., Middleton, P.G., Dorin, J.R., Stevenson, B.J., Gao, X., Durham, S.R., Jeffery, P.K., Hodson, M.E., Coutelle, C., Huang, L., Porteous, D.J., Williamson, R. and Geddes, D.M. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat. Med. 1995; 1, 39-46.
59. Stankovics, J., Crane, A.M., Andrews, E., Wu, C.H., Wu, G.Y. and Ledley, F.D. Overexpression of human methylmalonyl CoA mutase in mice after in vivo gene transfer with asialoglycoprotein/polylysine/DNA complexes. Hum. Gene Ther. 1994; 5, 1095-104.
60. Scheule, R.K., St George, J.A., Bagley, R.G., Marshall, J., Kaplan, J.M., Akita, G.Y., Wang, K.X., Lee, E.R., Harris, D.J., Jiang, C., Yew, N.S., Smith, A.E. and Cheng, S.H. Basis of pulmonary toxicity associated with cationic lipid-mediated gene transfer to the mammalian lung. Hum. Gene Ther. 1997; 8, 689-707.
61. Krieg, A.M. Lymphocyte activation by CpG dinucleotide motifs in prokaryotic DNA. Trends. Microbiol. 1996; 4, 73-76.
62. Plank, C., Mechtler, K., Szoka, F.C. Jr. and Wagner, E. Activation of the complement system by synthetic DNA complexes: A potential barrier for intravenous gene delivery. Hum. Gene Ther. 1996; 7, 1437-46.
63. Jenkins, R.G., Meng, Q-H., Hodges, R.J., Kim, L.K., Laurent, G.J., Willis, D., Ayazi Shamlou, P., McAnulty, R.J., and Hart, S.L. Formation of LID vector complexes in water alters physico-chemical properties and enhances pulmonary gene expression in vivo. Gene Ther 2003. In Press.
64. Freimark, B.D., Blezinger, H.P., Florack, V.J., Nordstrom, J.L., Long, S.D., Deshpande, D.S., Nochumson, S. and Petrak, K.L. Cationic lipids enhance cytokine and cell influx levels in the lung following administration of plasmid: Cationic lipid complexes. J. Immunol. 1998; 160, 4580-4586.
65. Qin, L., Ding, Y., Pahud, D.R., Chang, E., Imperiale, M.J. and Bromberg, J.S. Promoter attenuation in gene therapy: Interferon-γ and tumor necrosis factor-α inhibit transgene expression. Hum. Gene Ther. 1997; 8, 2019-2029.
66. Scheule, R.K. The role of CpG motifs in immunostimulation and gene therapy. Adv. Drug Deliv. Rev. 2000. 44, 119-34.
67. Li, S., Rizzo, M.A., Bhattacharya, S. and Huang, L. Characterization of cationic lipid-protamine-DNA (LPD) complexes for intravenous gene delivery. Gene Ther. 1998; 5, 930-937.
68. Shillitoe, E.J. and Noonan, S. Strength and specificity of different gene promoters in oral cancer cells. Oral Oncol. 2000; 36, 214-20.
69. Cara, A., Lucarelli, E. and Cornaglia-Ferraris, P. Engineering viral promoters for gene transfer to human neuroblasts. Cell Mol. Neurobiol. 2000; 20, 409-15.
70. Urtti, A., Polansky, J., Lui, G.M. and Szoka, F.C. Gene delivery and expression in human retinal pigment epithelial cells: Effects of synthetic carriers, serum, extracellular matrix and viral promoters. J. Drug Target. 2000; 7, 413-21.
71. Kerner, M., Meyuhas, O., Hirsch-Lerner, D., Rosen, L.J., Min Z. and Barenholz, Y. Interplay in lipoplexes between type of pDNA promoter and lipid composition determines transfection efficiency of human growth hormone in NIH3T3 cells in culture. Biochim. Biophys. Acta 2001; 1532, 128-36.
72. Xu, Z., Mizuguchi, H., Ishii-Watabe, A., Uchida, E., Mayumi, T. and Hayakawa, T. Optimization of transcriptional regulatory elements for constructing plasmid vectors. Gene. 2001; 272, 149-56.
73. Carswell, S. and Alwine, J.C. Efficiency of utilization of the simian virus 40 late polyadenylation site: Effects of upstream sequences. Mol. Cell Biol. 1989; 9, 4248-4258.
74. Palmiter, R.D., Sandgren, E.P., Avarbock, M.R., Allen, D.D. and Brinster, R.L. Heterologous introns can enhance expression of transgenes in mice. Proc. Natl. Acad. Sci. U. S. A. 1991; 88, 478-482.
75. Choi, T., Huang, M., Gorman, C. and Jaenisch, R. A generic intron increases gene expression in transgenic mice. Mol. Cell Biol. 1991; 11, 3070-3074.
76. Armentano, D., Zabner, J., Sacks, C., Sookdeo, C.C., Smith, M.P., St. George, J.A., Wadsworth, S.C., Smith, A.E. and Gregory, R.J. Effect of the E4 region on the persistence of transgene expression from adenovirus vectors. J. Virol. 1997; 71, 2408-2416.
77. Lusky, M., Grave, L., Dieterle, A., Dreyer, D., Christ, M., Ziller, C., Furstenberger, P., Kintz, J., Hadji, D.A., Pavirani, A. and Mehtali, M. Regulation of adenovirus-mediated transgene expression by the viral E4 gene products: Requirement for E4 ORF3. J. Virol. 1999; 73, 8308-8319.
78. Armentano, D., Smith, M.P., Sookdeo, C.C., Zabner, J., Perricone, M.A., St George, J.A., Wadsworth, S.C. and Gregory, R.J. E4ORF3 requirement for achieving long-term transgene expression from the cytomegalovirus promoter in adenovirus vectors. J. Virol. 1999; 73, 7031-7034.
79. Yew, N.S., Marshall, J., Przybylska, M., Wysokenski, D.M., Ziegler, R.J., Rafter, P.W., Li, C., Armentano, D. and Cheng, S.H. Increased duration of transgene expression in the lung with plasmid DNA vectors harboring adenovirus E4 open reading frame 3. Hum. Gene Ther. 1999; 10, 1833-1843.
80. Gill, D.R., Smythe, S.E., Goddard, C.A., Pringle, I.A., Higgins, C.F., Colledge, W.H. and Hyde, S.C. Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor1α promoter. Gene Ther. 2001; 8, 1539-1546.
81. Harvey, B.H., Leopold, P.H., Hackett, N.R., Grasso, T.M., Williams, P.M., Tucker, A.Y., Kaner, R.J., Ferris, B., Gonda, I., Sweeney, T.D., Ramalingam, R., Kovesdi, I., Shak, S. and Crystal, R.G. Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus. J. Clin. Invest. 1999; 102, 1245-1255
82. Kay, M.A., Manno, C.S., Ragni, M.V., Larson, P.J., Couto, L.B., McClelland, A., Glader, B., Chew, A.J., Tau, S.J., Herzog, R.W., Arruda, V., Johnson, F., Scallan, C., Skarsgard, E., Flake, A.W. and High, K.A. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat. Genet. 2000; 24, 257-261.
83. Yla-Herttuala, S. and Martin, J.F. Cardiovascular gene therapy. Lancet 2000; 355, 213-222
84. Khuri, F.R., Nemunaitis, J., Ganly, I., Arseneau, J., Tannock, I.F., Heise, C., Randlev, B., Gillenwater, A.M., Bruso, P., Kaye, S.B., Hong, W.K. and Kirn, D.H. A controlled trial of intratumoral ONYX-O15, a selectively-replicating adenovirus, in combination with cisplatin and 5-fluorouracil in patients with recurrent head and neck cancer. Nat. Med. 2000; 6, 879-885.
85. Cavazzana-Calvo, M., Hacein-Bey, S., De Saint Basile, G., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J.L., Boussos, P., Deist, F.L., Fischer, A. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 2000; 288, 627-629.