Viral gene therapy strategies: From basic science to clinical application

Journal of Pathology

Volumn 208, Issue 2, 2006, Pages 299-318

  Young, Lawrence S ^a   Searle, Peter F ^a   Onion, David ^a   Mautner, Vivien ^a  

^a UNIVERSITY DEPARTMENT OF MEDICINE   (United Kingdom)

Author keywords

Cancer; Gene therapy; Oncolytic; Virus

Indexed keywords

5 (1 AZIRIDINYL) 2,4 DINITROBENZAMIDE; ADENOSINE DEAMINASE; ADENOVIRUS VECTOR; ANGIOSTATIN; CISPLATIN; CYCLOPHOSPHAMIDE; CYTOCHROME P450; CYTOSINE DEAMINASE; CYTOTOXIC AGENT; ENDOSTATIN; ENZYME; FLUCYTOSINE; FLUOROURACIL; GANCICLOVIR; GENDICINE; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; HSV 1716; INTERLEUKIN 12; MACROGOL; MUTANT PROTEIN; NITROREDUCTASE; ONYX 015; PARVOVIRUS VECTOR; POLY[N (2 HYDROXYPROPYL)METHACRYLAMIDE]; PRODRUG; PROTEIN P53; RETROVIRUS VECTOR; RIBONUCLEOTIDE REDUCTASE; THYMIDINE KINASE; TUMOR NECROSIS FACTOR; TUMOR NECROSIS FACTOR RELATED APOPTOSIS INDUCING LIGAND; UNCLASSIFIED DRUG; UNINDEXED DRUG; VASCULOTROPIN; VIRUS VECTOR;

CANCER THERAPY; CARDIOVASCULAR DISEASE; CHINA; CLINICAL TRIAL; DRUG MEGADOSE; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE TARGETING; HEAD AND NECK CANCER; HUMAN; LOW DRUG DOSE; MONOGENIC DISORDER; NONHUMAN; PRIORITY JOURNAL; REVIEW; TARGET CELL; TRANSGENE; VACCINIA VIRUS; VIRAL GENE THERAPY; VIRUS; VIRUS ATTENUATION; VIRUS CELL INTERACTION; VIRUS MUTANT; VIRUS REPLICATION;

ADENOVIRIDAE; GENE THERAPY; GENETIC VECTORS; HERPESVIRUS 1, HUMAN; HUMANS; LENTIVIRUS; MODELS, GENETIC; NEOPLASMS; ONCOLYTIC VIROTHERAPY; RECOMBINATION, GENETIC; RETROVIRIDAE; SEVERE COMBINED IMMUNODEFICIENCY; VIRUS REPLICATION; VIRUSES;

EID: 31144475398     PISSN: 00223417     EISSN: 10969896     Source Type: Journal    
DOI: 10.1002/path.1896     Document Type: Review

References (260)

1. Cavazzana-Calvo M, Hacein-Bey-Abina S, De Saint Basile G, Gross F, Yvon E, Nusbaum P, et al. Gene therapy of human severe combined immunodeficiency (SLID)-X1 disease. Science 2000;288:669-672.
2. Cavazzana-Calvo M, Lagresle D, Hacein-Bey-Abina S, Fischer A. Gene therapy for severe combined immunodeficiency. Annu Rev Med 2005;56:585-602.
3. Hacein-Bey-Abina S, Le Deist F, Carlier F, Bouneaud C, Hue C, De Villartay JP, et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002;346:1185-1193.
4. Hacein-Bey-Abina S, von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SLID-X1. Science 2003;302:415-419.
5. Imren S, Fabry ME, Westerman KA, Pawliuk R, Tang P, Rosten PM, et al. High-level beta-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells. J Clin Invest 2004;114:953-962.
6. Graw J, Brackmann H-H, Oldenburg J, Schneppenheim R, Spannag M, Schwaab R. Haemophilia A: from mutation analysis to new therapies. Nature Rev Genet 2005;6:488-501.
7. Gregory CD, Dive C, Henderson S, Smith CA, Williams GT, Gordon J, et al. Activation of Epstein-Barr virus latent genes protects human B cells from death by apoptosis. Nature 1991;349:612-614.
8. Lee TWR, Matthews DA, Blair GE. Novel molecular approaches to cystic fibrosis gene therapy. Biochem J 2005;387:1-15.
9. Ostedgaard LS, Rokhlina T, Karp PH, Lashmit P, Afione S, Schmidt M, et al. A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia. Proc Natl Acad Sci U S A 2005;102:2952-2957.
10. Cossu G, Sampaolesi M. New therapies for muscular dystrophy: cautious optimism. Trends Mol Med 2004;10:516-520.
11. Nowak KJ, Davies KE. Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment. EMBO Rep 2004;5:872-876.
12. Chakkalakal JV, Thompson J, Parks RJ, Jasmin BJ. Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies. FASEB J 2005;19:880-891.
13. Li S, Kimura E, Fall BM, Reyes M, Angello JC, Welikson R, et al. Stable transduction of myogenic cells with lentiviral vectors expressing a minidystrophin. Gene Ther 2005;12:1099-1108.
14. Yla-Herttuala S, Alitalo K. Gene transfer as a tool to induce therapeutic vascular growth. Nature Med 2003;9:694-701.
15. Melo LG, Pachori AS, Gnecchi M, Dzau VJ. Genetic therapies for cardiovascular diseases. Trends Mol Med 2005;11:240-250.
16. Miller WH, Brosnan MJ, Graham D, Nicol CG, Morecroft I, Channon KM, et al. Targeting endothelial cells with adenovirus expressing nitric oxide synthase prevents elevation of blood pressure in stroke-prone spontaneously hypertensive rats. Mol Ther 2005;12:321-327.
17. Ram Z, Culver KW, Oshiro EM, Viola JJ, DeVroom HL, Otto E, et al. Therapy of malignant brain tumors by intratumoral implantation of retroviral vector-producing cells. Nature Med 1997;3:1354-1361.
18. Herman JR, Adler HL, Aguilar-Cordova E, Rojas-Martinez A, Woo S, Timme TL, et al. In situ gene therapy for adenocarcinoma of the prostate: a phase I clinical trial. Hum Gene Ther 1999;10:1239-1249.
19. Pandha HS, Martin LA, Rigg A, Hurst HC, Stamp GWH, Sikora K, et al. Genetic prodrug activation therapy for breast cancer: a phase I clinical trial of erbB-2-directed suicide gene expression. J Clin Oncol 1999;17:2180-2189.
20. McCormick F. Cancer gene therapy: fringe or cutting edge? Nature Rev Cancer 2001;1:130-141.
21. Djeha AH, Thomson TA, Leung H, Searle PF, Young LS, Kerr DJ, et al. Combined adenovirus-mediated nitroreductase gene delivery and CB1954 treatment: a well-tolerated therapy for established solid tumours. Mol Ther 2000;3:233-240.
22. Palmer DH, Mautner V, Mirza D, Oliff S, Gerritsen W, van der Sijp JRM, et al. Virus-directed enzyme prodrug therapy: intratumoral administration of a replication-deficient adenovirus encoding nitroreductase to patients with resectable liver cancer. J Clin Oncol 2004;22:1546-1552.
23. Szlosarek PW, Balkwill FR. Tumour necrosis factor α: a potential target for the therapy of solid tumours. Lancet 2003;4:565-573.
24. Ashkenazi A. Targeting death and decoy receptors of the tumour-necrosis factor superfamily. Nature Rev Cancer 2002;2:420-430.
25. Jacob D, Davis J, Zhu H, Zhang L, Teraishi F, Wu S, et al. Suppressing orthotopic pancreatic tumour growth with a fiber-modified adenovector expressing the TRAIL gene from the human telomerase reverse transcriptase promoter. Clin Cancer Res 2004;10:3335-3541.
26. Ma H, Liu Y, Liu S, Kung HF, Sun X, Zheng D, et al. Recombinant adeno-associated virus-mediated TRAIL gene therapy suppresses liver metastatic tumours. Int J Cancer 2005;116:314-321.
27. Indraccolo S. Undermining tumour angiogenesis by gene therapy: an emerging field. Curr Gene Ther 2004;4:297-308.
28. Ponnazhagan S, Mahendra G, Kumar S, Shaw DR, Stockard CR, Grizzle WE, et al. Adeno-associated virus 2-mediated antiangiogenic cancer gene therapy: long-term efficacy of a vector encoding angiostatin and endostatin over vectors encoding a single factor. Cancer Res 2004;64:1781-1787.
29. Pardoll DM. Spinning molecular immunology into successful immunotherapy. Nature Rev Immunol 2002;2:227-238.
30. Banchereau J, Palucka AK. Dendritic cells as therapeutic vaccines against cancer. Nature Rev Immunol 2005;5:296-306.
31. Sangro B, Mazzolini G, Ruiz J, Herraiz M, Quiroga J, Herrero I, et al. Phase I trial of intratumoral injection of an adenovirus encoding interleukin-12 for advanced digestive tumours. J Clin Oncol 2004;22:1389-1397.
32. Soiffer R, Lynch T, Mihm M, Jung K, Rhuda C, Schmollinger JC, et al. Vaccination with irradiated autologous melanoma cells engineered to secrete human granulocyte-macrophage colony-stimulating factor generates potent anti-tumour immunity in patients with metastatic melanoma. Proc Natl Acad Sci U S A 1998;95:13141-13146.
33. Chiocca EA. Oncolytic viruses. Nature Rev Cancer 2002;2:938-950.
34. Ries S, Brandts CH. Oncolytic viruses for the treatment of cancer: current strategies and clinical trials. Drug Dis Today 2004;9:759-767.
35. Peng Z. Current status of gendicine in China: recombinant human Ad-p53 agent for treatment of cancers. Hum Gene Ther 2005;16:1013-1024.
36. Diaz RM, Eisen T, Hart IR, Vile RG. Exchange of viral promoter/enhancer elements with heterologous regulatory sequences generates targeted hybrid long terminal repeat vectors for gene therapy of melanoma. J Virol 1998;72:789-795.
37. Jager U, Zhao Y, Porter CD. Endothelial cell-specific transcriptional targeting from a hybrid long terminal repeat retrovirus vector containing human prepro-endothelin-1 promoter sequences. J Virol 1999;73:9702-9709.
38. Cosset FL, Takeuchi Y, Battini JL, Weiss RA, Collins MK. High-titer packaging cells producing recombinant retroviruses resistant to human serum. J Virol 1995;69:7430-7436.
39. Yee JK, Moores JC, Jolly DJ, Wolff JA, Respess JG, Friedmann T. Gene expression from transcriptionally disabled retroviral vectors. Proc Natl Acad Sci U S A 1987;84:5197-201.
40. Yu SF, van Ruden T, Kantoff PW, Garber C, Seiberg M, Ruther U, et al. Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc Natl Acad Sci U S A 1986;83:3194-3198.
41. Miller DG, Adam MA, Miller AD. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 1990;10:4239-4242.
42. Puumalainen AM, Vapalahti M, Agrawal RS, Kossila M, Laukkanen J, Lehtolainen P, et al. Beta-galactosidase gene transfer to human malignant glioma in vivo using replication-deficient retroviruses and adenoviruses. Hum Gene Ther 1998;9:1769-1774.
43. Klatzmann D, Valery CA, Bensimon G, Marro B, Boyer O, Mokhtari K, et al. A phase I/II study of herpes simplex virus type 1 thymidine kinase 'suicide' gene therapy for recurrent glioblastoma. Study Group on Gene Therapy for Glioblastoma. Hum Gene Ther 1998;9:2595-2604.
44. Shand N, Weber F, Mariani L, Bernstein M, Gianella-Borradori A, Long Z, et al. A phase 1-2 clinical trial of gene therapy for recurrent glioblastoma multiforme by tumor transduction with the herpes simplex thymidine kinase gene followed by ganciclovir. GLI328 European-Canadian Study Group. Hum Gene Ther 1999;10:2325-2335.
45. Rainov NG. A phase III clinical evaluation of herpes simplex virus type 1 thymidine kinase and ganciclovir gene therapy as an adjuvant to surgical resection and radiation in adults with previously untreated glioblastoma multiforme. Hum Gene Ther 2000;11:2389-2401.
46. Sandmair AM, Loimas S, Puranen P, Immonen A, Kossila M, Puranen M, et al. Thymidine kinase gene therapy for human malignant glioma, using replication-deficient retroviruses or adenoviruses. Hum Gene Ther 2000;11:2197-2205.
47. Valery CA, Seithean D, Boyer O, Marro B, Hauw JJ, Kemeny JL, et al. Long-term survival after gene therapy for a recurrent glioblastoma. Neurology 2002;58:1109-1112.
48. Culver KW, Ram Z, Wallbridge S, Ishii H, Oldfield EH, Blaese RM. In vivo gene transfer with retroviral vector-producer cells for treatment of experimental brain tumors. Science 1992;256:1550-1552.
49. Ram Z, Culver KW, Wallbridge S, Blaese RM, Oldfield EH. In situ retroviral-mediated gene transfer for the treatment of brain tumors in rats. Cancer Res 1993;53:83-88.
50. Harsh GR, Deisboeck TS, Louis DN, Hilton J, Colvin M, Silver JS, et al. Thymidine kinase activation of ganciclovir in recurrent malignant gliomas: a gene-marking and neuropathological study. J Neurosurg 2000;92:804-811.
51. Takeuchi Y, Porter CD, Strahan KM, Preece AF, Gustafsson K, Cosset FL, et al. Sensitization of cells and retroviruses to human serum by (alpha 1-3) galactosyltransferase. Nature 1996;379:85-88.
52. Braybrooke JP, Slade A, Deplanque G, Harrop R, Madhusudan S, Forster MD, et al. Phase I study of MetXia-P450 gene therapy and oral cyclophosphamide for patients with advanced breast cancer or melanoma. Clin Cancer Res 2005;11:1512-1520.
53. Kan O, Kingsman S, Naylor S. Cytochrome P450-based cancer gene therapy: current status. Exp Opin Biol Ther 2002;2:857-868.
54. Blaese RM. Development of gene therapy for immunodeficiency: adenosine deaminase deficiency. Paediatr Res 1993;33(1 Suppl):S49-S53; discussion S53-S55.
55. Muul LM, Tuschong LM, Soenen SL, Jagadeesh GJ, Ramsey WJ, Long Z, et al. Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial. Blood 2003;101:2563-2569.
56. Kohn DB, Hershfield MS, Carbonaro D, Shigeoka A, Brooks J, Smogorzewska EM, et al. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nature Med 1998;4:775-780.
57. Aiuti A. Gene therapy for adenosine-deaminase-deficient severe combined immunodeficiency. Best Pract Res Clin Haematol 2004;17:505-516.
58. Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002;296:2410-2413.
59. Schmidt M, Hacein-Bey-Abina S, Wissler M, Carlier F, Lim A, Prinz C, et al. Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial. Blood 2005;105:2699-2706.
60. Hacein-Bey S, Gross F, Nusbaum P, Hue C, Hamel Y, Fischer A, et al. Optimization of retroviral gene transfer protocol to maintain the lymphoid potential of progenitor cells. Hum Gene Ther 2001;12:291-301.
61. Gaspar HB, Parsley KL, Howe S, King D, Gilmour KC, Sinclair J, et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 2004;364:2181-2187.
62. Thrasher AJ, Hacein-Bey-Abina S, Gaspar HB, Blanche S, Davies EG, Parsley K, et al. Failure of SCID-X1 gene therapy in older patients. Blood 2005;105:4255-4257.
63. Wu X, Li Y, Crise B, Burgess SM. Transcription start regions in the human genome are favored targets for MLV integration. Science 2003;300:1749-1751.
64. Mitchell RS, Beitzel BF, Schroder AR, Shinn P, Chen H, Berry CC, et al. Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol 2004;2:E234.
65. Fischer A, Cavazzana-Calvo M. Integration of retroviruses: a fine balance between efficiency and danger. PLoS Med 2005;2:e10.
66. Uchida N, Sutton RE, Friera AM, He D, Reitsma MJ, Chang WC, et al. HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells. Proc Natl Acad Sci U S A 1998;95:11939-11944.
67. Bukrinsky MI, Haggerty S, Dempsey MP, Sharova N, Adzhubel A, Spitz L, et al. A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells. Nature 1993;365:666-669.
68. Sirven A, Pflumio F, Zennou V, Titeux M, Vainchenker W, Coulombel L, et al. The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells. Blood 2000;96:4103-4110.
69. Klages N, Zufferey R, Trono D. A stable system for the high-titer production of multiply attenuated lentiviral vectors. Mol Ther 2000;2:170-176.
70. Kotsopoulou E, Kim VN, Kingsman AJ, Kingsman SM, Mitrophanous KA. A Rev-independent human immunodeficiency virus type 1 (HIV-1)-based vector that exploits a codon-optimized HIV-1 gag-pol gene. J Virol 2000;74:4839-4852.
71. Kim VN, Mitrophanous K, Kingsman SM, Kingsman AJ. Minimal requirement for a lentivirus vector based on human immunodeficiency virus type 1. J Virol 1998;72:811-816.
72. Gregory LG, Waddington SN, Holder MV, Mitrophanous KA, Buckley SM, Mosley KL, et al. Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy. Gene Ther 2004;11:1117-1125.
73. Siapati EK, Bigger BW, Miskin J, Chipchase D, Parsley KL, Mitrophanous K, et al. Comparison of HIV- and EIAV-based vectors on their efficiency in transducing murine and human hematopoietic repopulating cells. Mol Ther 2005;12:537-546.
74. Azzouz M, Martin-Rendon E, Barber RD, Mitrophanous KA, Carter EE, Rohll JB, et al. Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease. J Neurosci 2002;22:10302-10312.
75. Mitrophanous K, Yoon S, Rohll J, Patil D, Wilkes F, Kim V, et al. Stable gene transfer to the nervous system using a nonprimate lentiviral vector. Gene Ther 1999;6:1808-1818.
76. Wong LF, Azzouz M, Walmsley LE, Askham Z, Wilkes FJ, Mitrophanous KA, et al. Transduction patterns of pseudotyped lentiviral vectors in the nervous system. Mol Ther 2004;9:101-111.
77. Themis M, Waddington SN, Schmidt M, von Kalle C, Wang Y, Al-Allaf F, et al. Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol Ther 2005;12:763-771.
78. Rowe WP, Huebner RJ, Gilmore LK, Parrott RH, Ward TG. Isolation of cytopathogenic agent from human adenoids undergoing spontaneous degeneration in tissue culture. Proc Soc Exp Biol Med 1953;84:570-573.
79. Horwitz MS. Adenoviruses. In Fields Virology, Fields BN, Knipe DM (eds). Lippincott Williams & Wilkins: Philadelphia, PA, USA, 2001; 2301-2326.
80. Takafuji ET, Gaydos JC, Allen RG, Top FH. Simultaneous administration of live, enteric-coated adenovirus types-4, types-7, and types-21 vaccines - safety and immunogenicity. J Invest Dis 1979;140:48-53.
81. Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao GP, et al. Fatal systemic inflammatory response syndrome in an ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 2003;80:148-158.
82. Shenk TE. Adenoviridae: the viruses and their replication. In Fields Virology, Fields BN, Knipe DM (eds). Lippincott Williams & Wilkins: Philadelphia, PA, USA, 2001; 2265-2300.
83. Bergelson JM, Cunningham JA, Droguett G, KurtJones EA, Krithivas A, Hong JS, et al. Isolation of a common receptor for Coxsackie B viruses and adenoviruses 2 and 5. Science 1997;275:1320-1323.
84. Tomko RP, Xu RL, Philipson L. HCAR and MCAR: the human and mouse cellular receptors for subgroup C adenoviruses and group B Coxsackieviruses. Proc Natl Acad Sci U S A 1997;94:3352-3356.
85. Wickham TJ, Mathias P, Cheresh DA, Nemerow GR. Integrin-alpha-V-beta-3 and integrin-alpha-V-beta-5 promote adenovirus internalization but not virus attachment. Cell 1993;73:309-319.
86. Havenga MJE, Lemckert AAC, Ophorst O, van Meijer M, Germeraad WTV, Grimbergen J, et al. Exploiting the natural diversity in adenovirus tropism for therapy and prevention of disease. J Virol 2002;76:4612-4620.
87. Skog J, Edlund K, Widegren B, Salford LG, Wadell G, Mei YF. Efficient internalization into low-passage glioma cell lines using adenoviruses other than type 5: an approach for improvement of gene delivery to brain tumours. J Gen Virol 2004;85:2627-2638.
88. Stone D, Ni SH, Li ZY, Gaggar A, DiPaolo N, Feng QH, et al. Development and assessment of human adenovirus type 11 as a gene transfer vector. J Virol 2005;79:5090-5104.
89. Roy S, Gao GP, Lu Y, Zhou XY, Lock M, Calcedo R, et al. Characterization of a family of chimpanzee adenoviruses and development of molecular clones for gene transfer vectors. Hum Gene Ther 2004;15:519-530.
90. Both GW. Ovine atadenovirus: a review of its biology, biosafety profile and application as a gene delivery vector. Immunol Cell Biol 2004;82:189-195.
91. Hutchins B, Sajjadi N, Seaver S, Shepherd A, Bauer SR, Simek S, et al. Working toward an adenoviral vector testing standard. Mol Ther 2000;2:532-534.
92. Bett AJ, Haddara W, Prevec L, Graham FL. An efficient and flexible system for construction of adenovirus vectors with insertions or deletions in early region-1 and region-3. Proc Natl Acad Sci U S A 1994;91:8802-8806.
93. Graham FL, Smiley J, Russell WC, Nairn R. Characteristics of a human cell line transformed by DNA from human adenovirus type-5. J Gen Virol 1977;36:59-72.
94. Fallaux FJ, Kranenburg O, Cramer SJ, Houweling A, VanOrmondt H, Hoeben RC, et al. Characterization of 911: a new helper cell line for the titration and propagation of early region 1-deleted adenoviral vectors. Hum Gene Ther 1996;7:215-222.
95. Fallaux FJ, Bout A, Van der Velde I, Van den Wollenberg DJM, Hehir KM, Keegan J, et al. New helper cells and matched early region 1-deleted adenovirus vectors prevent generation of replication-competent adenoviruses. Hum Gene Ther 1998;9:1909-1917.
96. Jones N, Shenk T. Adenovirus type-5 early gene-function regulates expression of other early viral genes. Proc Natl Acad Sci U S A 1979;76:3665-3669.
97. Schaack J. Adenovirus vectors deleted for genes essential for viral DNA replication. Front Biosci 2005;10:1146-1155.
98. Yang Y, Nunes FA, Berencsi K, Furth EE, Gonczol E, Wilson JM. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci U S A 1994;91:4407-4411.
99. Yang Y, Wilson JM. Clearance of adenovirus-infected hepatocytes by MHC class I-restricted CD4+ CTLs in vivo. J Immunol 1995;155:2564-2570.
100. Wang Q, Finer MH. Second-generation adenovirus vectors. Nature Med 1996;2:714-716.
101. Amalfitano A, Hauser MA, Hu HM, Serra D, Begy CR, Chamberlain JS. Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted. J Virol 1998;72:926-933.
102. Mitani K, Graham FL, Caskey CT, Kochanek S. Rescue, propagation, and partial-purification of a helper virus-dependent adenovirus vector. Proc Natl Acad Sci U S A 1995;92:3854-3858.
103. Kochanek S. High-capacity adenoviral vectors for gene transfer and somatic gene therapy. Hum Gene Ther 1999;10:2451-2459.
104. Parks RJ, Chen L, Anton M, Sankar U, Rudnicki MA, Graham FL. A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal. Proc Natl Acad Sci U S A 1996;93:13565-13570.
105. Volpers C, Kochanek S. Adenoviral vectors for gene transfer and therapy. J Gene Med 2004;6:S164-S171.
106. Morral N, Parks RJ, Zhou HS, Langston C, Schiedner G, Quinones J, et al. High doses of a helper-dependent adenoviral vector yield supraphysiological levels of alpha(1)-antitrypsin with negligible toxicity. Hum Gene Ther 1998;9:2709-2716.
107. Morsy MA, Gu MC, Motzel S, Zhao J, Su Q, Allen H, et al. An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene. Proc Natl Acad Sci U S A 1998;95:7866-7871.
108. Schiedner G, Morral N, Parks RJ, Wu Y, Koopmans SC, Langston C, et al. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nature Genet 1998;18:180-183.
109. 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.
110. Crystal RG, McElvaney NG, Rosenfeld MA, Chu CS, Mastrangeli A, Hay JG, et al. Administration of an adenovirus containing the human CFTR cDNA to the respiratory-tract of individuals with cystic-fibrosis. Nature Genet 1994;8:42-51.
111. Welsh MJ, Smith AE, Zabner J, Rich DP, Graham SM, Gregory RJ. Cystic fibrosis gene therapy using an adenovirus vector: in vivo safety and efficacy in nasal epithelium. Hum Gene Ther 1994;5:209-219.
112. Knowles MR, Hohneker KW, Zhou ZQ, Olsen JC, Noah TL, Hu PC, et al. A controlled-study of adenoviral-vector-mediated gene-transfer in the nasal epithelium of patients with cystic-fibrosis. N Engl J Med 1995;333:823-831.
113. Harvey BG, Leopold PL, Hackett NR, Grasso TM, Williams PM, Tucker AL, et al. Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus. J Clin Invest 1999;104:1245-1255.
114. Zuckerman JB, Robinson CB, McCoy KS, Shell R, Sferra TJ, Chirmule N, et al. A phase I study of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator gene to a lung segment of individuals with cystic fibrosis. Hum Gene Ther 1999;10:2973-2985.
115. Walters RW, Grunst T, Bergelson JM, Finberg RW, Welsh MJ, Zabner J. Basolateral localization of fiber receptors limits adenovirus infection from the apical surface of airway epithelia. J Biol Chem 1999;274:10219-10226.
116. Stonebraker JR, Wagner D, Lefensty RW, Burns K, Gendler SJ, Bergelson JA, et al. Glycocalyx restricts adenoviral vector access to apical receptors expressed on respiratory epithelium in vitro and in vivo: role for tethered mucins as barriers to lumenal infection. J Virol 2004;78:13755-13768.
117. Raper SE, Yudkoff M, Chirmule N, Gao GP, Nunes F, Haskal ZJ, et al. A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency. Hum Gene Ther 2002;13:163-175.
118. Mian A, McCormack M, Mane V, Kleppe S, Ng P, Finegold M, et al. Long-term correction of ornithine transcarbamylase deficiency by WPRE-mediated overexpression using a helper-dependent adenovirus. Mol Ther 2004;10:492-499.
119. Rosengart TK, Lee LY, Patel SR, Kligfield PD, Okin PM, Hackett NR, 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 1999;230:466-470.
120. Rosengart TK, Lee LY, Patel SR, Sanborn TA, Parikh M, Bergman GW, et al. Angiogenesis gene therapy - phase I assessment of direct intramyocardial administration of an adenovirus vector expressing VEGF121 cDNA to individuals with clinically significant severe coronary artery disease. Circulation 1999;100:468-474.
121. Hedman M, Hartikainen J, Syvanne M, Stjernvall J, Hedman A, Kivela A, et al. Safety and feasibility of catheter-based local intracoronary vascular endothelial growth factor gene transfer in the prevention of postangioplasty and in-stent restenosis and in the treatment of chronic myocardial ischemia - phase II results of the Kuopio Angiogenesis Trial (KAT). Circulation 2003;107:2677-2683.
122. Rajagopalan S, Mohler ER, Lederman RJ, Mendelsohn FO, Saucedo JF, Goldman CK, et al. Regional angiogenesis with vascular endothelial growth factor in peripheral arterial disease - a phase II randomized, double-blind, controlled study of adenoviral delivery of vascular endothelial growth factor 121 in patients with disabling intermittent claudication. Circulation 2003;108:1933-1938.
123. Rajagopalan S, Shah M, Luciano A, Crystal R, Nabel EG. Adenovirus-mediated gene transfer of VEGF(121) improves lower-extremity endothelial function and flow reserve. Circulation 2001;104:753-755.
124. Makinen K, Manninen H, Hedman M, Matsi P, Mussalo H, Alhava E, et al. Increased vascularity detected by digital subtraction angiography after VEGF gene transfer to human lower limb artery: a randomized, placebo-controlled, double-blinded phase II study. Mol Ther 2002;6:127-133.
125. Grines CL, Watkins MW, Helmer G, Penny W, Brinker J, Marmur JD, et al. Angiogenic GENe Therapy (AGENT) trial in patients with stable angina pectoris. Circulation 2002;105:1291-1297.
126. Grines CL, Watkins MW, Mahmarian JJ, Iskandrian AE, Rade JJ, Marrott P, et al. A randomized, double-blind, placebo-controlled trial of Ad5FGF-4 gene therapy and its effect on myocardial perfusion in patients with stable angina. J Am Coll Cardiol 2003;42:1339-1347.
127. Penny WF, Hammond HK. Clinical use of intracoronary gene transfer of fibroblast growth factor for coronary artery disease. Curr Gene Ther 2004;4:225-230.
128. Baker AH. Designing gene delivery vectors for cardiovascular gene therapy. Prog Biophys Mol Biol 2004;84:279-299.
129. Lang FF, Bruner JM, Fuller GN, Aldape K, Prados MD, Chang S, et al. Phase I trial of adenovirus-mediated p53 gene therapy for recurrent glioma: biological and clinical results. J Clin Oncol 2003;21:2508-2518.
130. Swisher SG, Roth JA, Komaki R, Gu J, Lee JJ, Hicks M, et al. Induction of p53-regulated genes and tumor regression in lung cancer patients after intratumoral delivery of adenoviral p53 (INGN 201) and radiation therapy. Clin Cancer Res 2003;9:93-101.
131. Pagliaro LC, Keyhani A, Williams D, Woods D, Liu BS, Perrotte P, et al. Repeated intravesical instillations of an adenoviral vector in patients with locally advanced bladder cancer: a phase I study of p53 gene therapy. J Clin Oncol 2003;21:2247-2253.
132. Wolf JK, Bodurka DC, Gano JB, Deavers M, Ramondetta L, Ramirez PT, et al. A phase I study of Adp53 (INGN 201; ADVEXIN) for patients with platinum- and paclitaxel-resistant epithelial ovarian cancer. Gynecol. Oncol 2004;94:442-448.
133. Germano IM, Fable J, Gultekin SH, Silvers A. Adenovirus/herpes simplex-thymidine kinase/ganciclovir complex: preliminary results of a phase I trial in patients with recurrent malignant gliomas. J Neuro-Oncol 2003;65:279-289.
134. Immonen A, Vapalahti M, Tyynela K, Hurskainen H, Sandmair A, Vanninen R, et al. AdvHSV-tk gene therapy with intravenous ganciclovir improves survival in human malignant glioma: a randomised, controlled study. Mol Ther 2004;10:967-972.
135. Teh BS, Ayala G, Aguilar L, Mai WY, Timme TL, Vlachaki MT, et al. Phase I-II trial evaluating combined intensity-modulated radiotherapy and in situ gene therapy with or without hormonal therapy in treatment of prostate cancer - interim report on PSA response and biopsy data. Int J Radiat Oncol Biol Phys 2004;58:1520-1529.
136. Freytag SO, Stricker H, Pegg J, Paielli D, Pradhan DG, Peabody J, et al. Phase I study of replication-competent adenovirus-mediated double-suicide gene therapy in combination with conventional-dose three-dimensional conformal radiation therapy for the treatment of newly diagnosed, intermediate- to high-risk prostate cancer. Cancer Res 2003;63:7497-7506.
137. Kotin RM, Menninger JC, Ward DC, Berns KI. Mapping and direct visualization of a region-specific viral-DNA integration site on chromosome-19q13-Qter. Genomics 1991;10:831-834.
138. Samulski RJ, Chang LS, Shenk T. Helper-free stocks of recombinant adeno-associated viruses - normal integration does not require viral gene-expression. J Virol 1989;63:3822-3828.
139. Miller DG, Petek LM, Russell DW. Adeno-associated virus vectors integrate at chromosome breakage sites. Nature Genet 2004;36:767-773.
140. McCarty DM, Young SM, Samulski RJ. Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet 2004;38:819-845.
141. Summerford C, Samulski RJ. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 1998;72:1438-1445.
142. Qing K, Mah C, Hansen J, Zhou SZ, Dwarki V, Srivastava A. Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nature Med 1999;5:71-77.
143. Summerford C, Bartlett JS, Samulski RJ. Alpha V beta 5 integrin: a co-receptor for adeno-associated virus type 2 infection. Nature Med 1999;5:78-82.
144. Rabinowitz JE, Rolling F, Li CW, Conrath H, Xiao WD, Xiao X, et al. Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J Virol 2002;76:791-801.
145. Zabner J, Seiler M, Walters R, Kotin RM, Fulgeras W, Davidson BL, et al. Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J Virol 2000;74:3852-3858.
146. Blankinship MJ, Gregorevic P, Allen JM, Harper SQ, Harper H, Halbert CL, et al. Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol Ther 2004;10:671-678.
147. Davidoff AM, Gray JT, Ng CYC, Zhang YB, Zhou JF, Spence Y, et al. Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models. Mol Ther 2005;11:875-888.
148. Nathwani AC, McIntosh J, Davidoff AM. An update on gene therapy for hemophilia. Curr Haematol Rep 2005;4:287-293.
149. Duan D, Yue Y, Yan Z, Engelhardt JF. A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation. Nature Med 2000;6:595-598.
150. Sun L, Li J, Xiao X. Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization. Nature Med 2000;6:599-602.
151. Chirmule N, Propert KJ, Magosin SA, Qian Y, Qian R, Wilson JM. Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther 1999;6:1574-1583.
152. Beck SE, Jones LA, Chesnut K, Walsh SM, Reynolds TC, Carter BJ, et al. Repeated delivery of adeno-associated virus vectors to the rabbit airway. J Virol 1999;73:9446-9455.
153. Chirmule N, Xiao WD, Truneh A, Schnell MA, Hughes JV, Zoltick P, et al. Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nonhuman primate muscle. J Virol 2000;74:2420-2425.
154. Kok MR, Voutetakis A, Yamano S, Wang JH, Cotrim A, Katano H, et al. Immune responses following salivary gland administration of recombinant adeno-associated virus serotype 2 vectors. J Gene Med 2005;7:432-441.
155. Wagner JA, Nepomuceno IB, Messner AH, Moran ML, Batson EP, Dimiceli S, et al. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus
156. Moss RB, Rochman D, Spencer LT, Aitken ML, Zeitlhi PL, Waltz D, 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 2004;125:509-521.
157. Kaiser J. Gene therapy - side effects sideline hemophilia trial. Science 2004;304:1423-1425.
158. Endter C, Dobner T. Cell transformation by human adenoviruses. Curr Top Microbiol Immunol 2004;273:163-214.
159. O'Shea CC, Johnson L, Bagus B, Choi S, Nicholas C, Shen A, et al. Late viral RNA export, rather than p53 inactivation, determines ONYX-015 tumour selectivity. Cancer Cell 2004;6:611-623.
160. Khuri FR, Nemunaitis J, Ganly I, Arseneau J, Tannock IF, Romel L, et al. A controlled trial of intratumoral ONYX-015, a selectively-replicating adenovirus, in combination with cisplatin and 5-fluorouracil in patients with recurrent head and neck cancer. Nature Med 2000;6:879-885.
161. Hecht JR, Bedford R, Abbruzzese JL, Lahoti S, Reid TR, Soetikno RM, et al. A phase I/II trial of intratumoral endoscopic ultrasound injection of ONYX-015 with intravenous gemcitabine in unresectable pancreatic carcinoma. Clin Cancer Res 2003;9:555-561.
162. Mulvihlll S, Warren R, Venook A, Adler A, Randlev B, Heise C, et al. Safety and feasibility of injection with an E1B-55 kDa gene-deleted, replication-selective adenovirus (ONYX-015) into primary carcinomas of the pancreas: a phase I trial. Gene Ther 2001;8:308-315.
163. Vasey PA, Shulman LN, Campos S, Davis J, Gore M, Johnston S, et al. Phase I trial of intraperitoneal injection of the E1B-55-kD-gene-deleted adenovirus ONYX-015 (dl1520) given on days 1 through 5 every 3 weeks in patients with recurrent/refractory epithelial ovarian cancer. J Clin Oncol 2002;20:1562-1569.
164. Reid T, Galanis E, Abbruzzese J, Sze D, Andrews J, Romel L, et al. Intra-arterial administration of a replication-selective adenovirus (dl1520) in patients with colorectal carcinoma metastatic to the liver: a phase I trial. Gene Ther 2001;8:1618-1626.
165. Reid T, Galanis E, Abbruzzese J, Sze D, Wein LM, Andrews J, et al. Hepatic arterial infusion of a replication-selective oncolytic adenovirus (dl1520): phase II viral, immunologic, and clinical endpoints. Cancer Res 2002;62:6070-6079.
166. Reid TR, Freeman S, Post L, McCormick F, Sze DY. Effects of Onyx-015 among metastatic colorectal cancer patients that have failed prior treatment with 5-FU/leucovorin. Cancer Gene Ther 2005;12:673-681.
167. Rudin CM, Cohen EEW, Papadimitrakopoulou VA, Silverman S, Recant W, El-Naggar AK, et al. An attenuated adenovirus, ONYX-015, as mouthwash therapy for premalignant oral dysplasia. J Clin Oncol 2003;21:4546-4552.
168. Hamid O, Varterasian ML, Wadler S, Hecht JR, Benson A, Galanis E, et al. Phase II trial of intravenous CI-1042 in patients with metastatic colorectal cancer. J Clin Oncol 2003;21:1498-1504.
169. Nemunaitis J, Cunningham C, Buchanan A, Blackburn A, Edelman G, Maples P, et al. Intravenous infusion of a replication-selective adenovirus (ONYX-015) in cancer patients: safety, feasibility and biological activity. Gene Ther 2001;8:746-759.
170. Heise C, Hermiston T, Johnson L, Brooks G, Sampson-Johannes A, Williams A, et al. An adenovirus E1A mutant that demonstrates potent and selective systemic anti-tumoral efficacy. Nature Med 2000;6:1134-1139.
171. Fueyo J, Gomez-Manzano C, Alemany R, Lee PSY, McDonnell TJ, Mitlianga P, et al. A mutant oncolytic adenovirus targeting the Rb pathway produces anti-glioma effect in vivo. Oncogene 2000;19:2-12.
172. Hallden G, Hill R, Wang Y, Anand A, Liu T-C, Lemoine N, et al. Novel immunocompetent murine tumour models for the assessment of replication-competent oncolytic adenovirus efficacy. Mol Ther 2003;8:412-424.
173. Liu T-C, Hallden G, Wang Y, Brooks G, Francis J, Lemoine N, et al. An E1B-19 kDa gene deletion mutant adenovirus demonstrates tumour necrosis factor-enhanced cancer selectivity and enhanced oncolytic potency. Mol Ther 2004;9:786-803.
174. Wang Y, Hallden G, Hill R, Anand A, Liu T-C, Francis J, et al. E3 gene manipulations affect oncolytic adenovirus activity in immunocompetent tumour models. Nature Biotech 2003;21:1328-1335.
175. Chen Y, DeWeese T, Dilley J, Zhang YW, Li YH, Ramesh N, et al. CV706, a prostate cancer-specific adenovirus variant, in combination with radiotherapy produces synergistic antitumor efficacy without increasing toxicity. Cancer Res 2001;61:5453-5460.
176. Yu DC, Chen Y, Seng M, Dilley J, Henderson DR. The addition of adenovirus type 5 region E3 enables calydon virus 787 to eliminate distant prostate tumor xenografts. Cancer Res 1999;59:4200-4203.
177. DeWeese TL, van der Pool H, Li SD, Mikhak B, Drew R, Goemann M, et al. A phase I trial of CV706, a replication-competent, PSA selective oncolytic adenovirus, for the treatment of locally recurrent prostate cancer following radiation therapy. Cancer Res 2001;61:7464-7472.
178. Huang TG, Savontaus MJ, Shinozaki K, Sauter BV, Woo SLC. Telomerase-dependent oncolytic adenovirus for cancer treatment. Gene Ther 2003;10:1241-1247.
179. Post DE, Van Meir EG. A novel hypoxia-inducible factor (HIF) activated oncolytic adenovirus for cancer therapy. Oncogene 2003;22:2065-2072.
180. Li YH, Yu DC, Chen Y, Amin P, Zhang H, Nguyen N, et al. A hepatocellular carcinoma-specific adenovirus variant, CV890, eliminates distant human liver tumors in combination with doxorubicin. Cancer Res 2001;61:6428-6436.
181. Kurihara T, Brough DE, Kovesdi I, Kufe DW. Selectivity of a replication-competent adenovirus for human breast carcinoma cells expressing the MUC1 antigen. J Clin Invest 2000;106:763-771.
182. Hernandez-Alcoceba R, Pihalja M, Qian DL, Clarke MF. New oncolytic adenoviruses with hypoxia- and estrogen receptor-regulated replication. Hum Gene Ther 2002;13:1737-1750.
183. Fuerer C, Iggo R. Adenoviruses with Tcf binding sites in multiple early promoters show enhanced selectivity for tumour cells with constitutive activation of the wnt signalling pathway. Gene Ther 2002;9:270-281.
184. Hermiston TW, Kuhn I. Armed therapeutic viruses: strategies and challenges to arming oncolytic viruses with therapeutic genes. Cancer Gene Ther 2002;9:1022-1035.
185. Wildner O, Blaese RM, Morris JC. Therapy of colon cancer with oncolytic adenovirus is enhanced by the addition of herpes simplex virus thymidine kinase. Cancer Res 1999;59:410-413.
186. Chen MJ, Green NK, Reynolds GM, Flavell JR, Mautner V, Kerr DJ, et al. Enhanced efficacy of Escherichia coli nitroreductase/CB1954 prodrug activation gene therapy using an E1B-55K-deleted oncolytic adenovirus vector. Gene Ther 2004;11:1126-1136.
187. Freytag SO, Khil M, Stricker H, Peabody J, Menon M, DePeralta-Venturina M, et al. Phase I study of replication-competent adenovirus-mediated double suicide gene therapy for the treatment of locally recurrent prostate cancer. Cancer Res 2002;62:4968-4976.
188. Ren X-W, Liang M, Meng X, Ye X, Ma H, Zhao Y, et al. A tumour-specific conditionally replicative adenovirus vector expressing TRAIL for gene therapy of hepatocellular carcinoma. Cancer Gene Ther (in press).
189. Bernt KM, Ni SH, Tieu AT, Lieber A. Assessment of a combined, adenovirus-mediated oncolytic and immunostimulatory tumor therapy. Cancer Res 2005;65:4343-4352.
190. Chase M, Chung RY, Chiocca EA. An oncolytic viral mutant that delivers the CYP2B1 transgene and augments cyclophosphamide chemotherapy. Nature Biotech 1998;16:444-448.
191. Yoon SS, Nakamura H, Carroll NM, Bode BP, Chiocca EA, Tanabe KK. An oncolytic herpes simplex virus type 1 selectively destroys diffuse liver metastases from colon carcinoma. FASEB J 2000;14:301-311.
192. He B, Gross M, Roizman B. The gamma(1)34.5 protein of herpes simplex virus I complexes with protein phosphatase 1 alpha to dephosphorylate the alpha subunit of the eukaryotic translation initiation factor 2 and preclude the shutoff of protein synthesis by double-stranded RNA-activated protein kinase. Proc Natl Acad Sci U S A 1997;94:843-848.
193. Farassati F, Yang AD, Lee PWK. Oncogenes in Ras signalling pathway dictate host-cell permissiveness to herpes simplex virus 1. Nature Cell Biol 2001;3:745-750.
194. Leib DA, Machalek MA, Williams BRG, Silverman RH, Virgin HW. Specific phenotypic restoration of an attenuated virus by knockout of a host resistance gene. Proc Natl Acad Sci U S A 2000;97:6097-6101.
195. Rampling R, Cruickshank G, Papanastassiou V, Nicoll J, Hadley D, Brennan D, et al. Toxicity evaluation of replication-competent herpes simplex virus (ICP 34.5 null mutant 1716) in patients with recurrent malignant glioma. Gene Ther 2000;7:859-866.
196. Papanastassiou V, Rampling R, Fraser M, Petty R, Hadley D, Nicoll J, et al. The potential for efficacy of the modified (ICP 34.5(-)) herpes simplex virus HSV1716 following intratumoural injection into human malignant glioma: a proof of principle study. Gene Ther 2002;9:398-406.
197. Brown M. Killer into cure - oncolytic viruses. Microbiol Today 2005;32:128-131.
198. MacKie RM, Stewart B, Brown SM. Intralesional injection a herpes simplex virus 1716 in metastatic melanoma. Lancet 2001;357:525-526.
199. Markert JM, Medlock MD, Rabkin SD, Gillespie GY, Todo T, Hunter WD, et al. Conditionally replicating herpes simplex virus mutant, G207, for the treatment of malignant glioma: results of a phase I trial. Gene Ther 2000;7:867-874.
200. Liu P, Speck SH. Synergistic autoactivation of the Epstein-Barr virus immediate-early BRLF1 promoter by Rta and Zta. Virology 2003;10:199-206.
201. Russell SJ. RNA viruses as virotherapy agents. Cancer Gene Ther 2002;9:961-966.
202. Stojdl DF, Lichty BD, tenOever BR, Paterson JM, Power AT, Knowles S, et al. VSV strains with defects in their ability to shutdown innate immunity are potent systemic anti-cancer agents. Cancer Cell 2003;4:263-275.
203. Pecora AL, Rizvi N, Cohen GI, Meropol NJ, Sterman D, Marshall JL, et al. Phase I trial of intravenous administration of PV701, an oncolytic virus, in patients with advanced solid cancers. J Clin Oncol 2002;20:2251-2266.
204. Csatary LK, Gosztonyi G, Szeberenyi J, Fabian Z, Liszka V, Bodey B, et al. MTH-68/H oncolytic viral treatment in human high-grade gliomas. J Neuro-Oncol 2004;67:83-93.
205. Strong JE, Coffey MC, Tang D, Sabinin P, Lee PWK. The molecular basis of viral oncolysis: usurpation of the Ras signaling pathway by reovirus. EMBO J 1998;17:3351-3362.
206. Norman KL, Lee PWK. Not all viruses are bad guys: the case for reovirus in cancer therapy. Drug Dis Today 2005;10:847-855.
207. Zeh HJ, Bartlett DL. Development of a replication-selective, oncolytic poxvirus for the treatment of human cancers. Cancer Gene Ther 2002;9:1001-1012.
208. Gromeier M, Lachmann S, Rosenfeld MR, Gutin PH, Wimmer E. Intergeneric poliovirus recombinants for the treatment of malignant glioma. Proc Natl Acad Sci U S A 2000;97:6803-6808.
209. Myers R, Greiner S, Harvey M, Soeffker D, Frenzke M, Abraham K, et al. Oncolytic activities of approved mumps and measles vaccines for therapy of ovarian cancer. Cancer Gene Ther 2005;12:593-599.
210. Miller AD, Garcia JV, von Suhr N, Lynch CM, Wilson C, Eiden MV. Construction and properties of retrovirus packaging cells based on gibbon ape leukemia virus. J Virol 1991;65:2220-2224.
211. Markowitz D, Goff S, Bank A. Construction and use of a safe and efficient amphotropic packaging cell line. Virology 1988;167:400-406.
212. Sandrin V, Boson B, Salmon P, Gay W, Negre D, Le Grand R, et al. Lentiviral vectors pseudotyped with a modified RD114 envelope glycoprotein show increased stability in sera and augmented transduction of primary lymphocytes and CD34+ cells derived from human and nonhuman primates. Blood 2002;100:823-832.
213. Mazarakis ND, Azzouz M, Rohll JB, Ellard FM, Wilkes FJ, Olsen AL, et al. Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery. Hum Mol Genet 2001;10:2109-2121.
214. Kobinger GP, Weiner DJ, Yu QC, Wilson JM. Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo. Nature Biotech 2001;19:225-230.
215. Medina MF, Kobinger GP, Rux J, Gasmi M, Looney DJ, Bates P, et al. Lentiviral vectors pseudotyped with minimal filovirus envelopes increased gene transfer in murine lung. Mol Ther 2003;8:777-789.
216. Kasahara N, Dozy AM, Kan YW. Tissue-specific targeting of retroviral vectors through ligand-receptor interactions. Science 1994;266:1373-1376.
217. Han X, Kasahara N, Kan YW. Ligand-directed retroviral targeting of human breast cancer cells. Proc Natl Acad Sci U S A 1995;92:9747-9751.
218. Cosset FL, Morling FJ, Takeuchi Y, Weiss RA, Collins MK, Russell SJ. Retroviral retargeting by envelopes expressing an N-terminal binding domain. J Virol 1995;69:6314-6322.
219. Marin M, Noel D, Valsesia-Wittman S, Brockly F, Etienne-Julan M, Russell S, et al. Targeted infection of human cells via major histocompatibility complex class I molecules by Moloney murine leukemia virus-derived viruses displaying single-chain antibody fragment-envelope fusion proteins. J Virol 1996;70:2957-62.
220. Fielding AK, Maurice M, Morling FJ, Cosset FL, Russell SJ. Inverse targeting of retroviral vectors: selective gene transfer in a mixed population of hematopoietic and nonhematopoietic cells. Blood 1998;91:1802-1809.
221. Peng KW, Morling FJ, Cosset FL, Murphy G, Russell SJ. A gene delivery system activatable by disease-associated matrix metalloproteinases. Hum Gene Ther 1997;8:729-738.
222. Chandrashekran A, Gordon MY, Darling D, Farzaneh F, Casimir C. Growth factor displayed on the surface of retroviral particles without manipulation of envelope proteins is biologically active and can enhance transduction. J Gene Med 2004;6:1189-1196.
223. Chester J, Ruchatz A, Gough M, Crittenden M, Chong H, Loic-Cosset F, et al. Tumor antigen-specific induction of transcriptionally targeted retroviral vectors from chimeric immune receptor-modified T cells. Nature Biotech 2002;20:256-263.
224. Crittenden M, Gough M, Chester J, Kottke T, Thompson J, Ruchatz A, et al. Pharmacologically regulated production of targeted retrovirus from T cells for systemic antitumor gene therapy. Cancer Res 2003;63:3173-3180.
225. Jee YS, Lee SG, Lee JC, Kim MJ, Lee JJ, Kim DY, et al. Reduced expression of Coxsackievirus and adenovirus receptor (CAR) in tumor tissue compared to normal epithelium in head and neck squamous cell carcinoma patients. Anticancer Res 2002;22:2629-2634.
226. Nicklin SA, Wu E, Nemerow GR, Baker AH. The influence of adenovirus fiber structure and function on vector development for gene therapy. Mol Ther 2005;12:384-393.
227. Law LK, Davidson BL. What does it take to bind CAR? Mol Ther 2005;12:599-609.
228. Xia D, Henry LJ, Gerard RD, Deisenhofer J. Crystal-structure of the receptor-binding domain of adenovirus type-5 fiber protein at 1.7-angstrom resolution. Structure 1994;2:1259-1270.
229. Kirby I, Davison E, Beavil AJ, Soh CPC, Wickham TJ, Roelvink PW, et al. Identification of contact residues and definition of the CAR-binding site of adenovirus type 5 fiber protein. J Virol 2000;74:2804-2813.
230. Michael SI, Hong JS, Curiel DT, Engler JA. Addition of a short peptide ligand to the adenovirus fiber protein. Gene Ther 1995;2:660-668.
231. Wickham TJ, Roelvink PW, Brough DE, Kovesdi I. Adenovirus targeted to heparan-containing receptors increases its gene delivery efficiency to multiple cell types. Nature Biotech 1996;14:1570-1573.
232. Dmitriev I, Krasnykh V, Miller CR, Wang MH, Kashentseva E, Mikheeva G, et al. An adenovirus vector with genetically modified fibers demonstrates expanded tropism via utilization of a Coxsackievirus and adenovirus receptor-independent cell entry mechanism. J Virol 1998;72:9706-9713.
233. Vigne E, Mahfouz I, Dedieu JF, Brie A, Perricaudet M, Yeh P. RGD inclusion in the hexon monomer provides adenovirus type 5-based vectors with a fiber knob-independent pathway for infection. J Virol 1999;73:5156-5161.
234. Dmitriev IP, Kashentseva EA, Curiel DT. Engineering of adenovirus vectors containing heterologous peptide sequences in the C terminus of capsid protein IX. J Virol 2002;76:6893-6899.
235. Dechecchi MC, Tamanini A, Bonizzato A, Cabrini G. Heparan sulfate glycosaminoglycans are involved in adenovirus type 5 and 2-host cell interactions. Virology 2000;268:382-390.
236. Dechecchi MC, Melotti P, Bonizzato A, Santacatterina M, Chilosi M, Cabrini G. Heparan sulfate glycosaminoglycans are receptors sufficient to mediate the initial binding of adenovirus types 2 and 5. J Virol 2001;75:8772-8780.
237. Gall J, Kass Eisler A, Leinwand L, Falck Pedersen E. Adenovirus type 5 and 7 capsid chimera: fiber replacement alters receptor tropism without affecting primary immune neutralization epitopes. J Virol 1996;70:2116-2123.
238. Krasnykh VN, Mikheeva GV, Douglas JT, Curiel DT. Generation of recombinant adenovirus vectors with modified fibers for altering viral tropism. J Virol 1996;70:6839-6846.
239. Stevenson SC, Rollence M, White B, Weaver L, McClelland A. Human adenovirus serotype-3 and serotype-5 bind to 2 different cellular receptors via the fiber head domain. J Virol 1995;69:2850-2857.
240. Stevenson SC, Rollence M, Marshall Neff J, McClelland A. Selective targeting of human cells by a chimeric adenovirus vector containing a modified fiber protein. J Virol 1997;71:4782-4790.
241. Zabner J, Chillon M, Grunst T, Moninger TO, Davidson BL, Gregory R, et al. A chimeric type 2 adenovirus vector with a type 17 fiber enhances gene transfer to human airway epithelia. J Virol 1999;73:8689-8695.
242. Shayakhmetov DM, Papayannopoulou T, Stamatoyannopoulos G, Lieber A. Efficient gene transfer into human CD34(+) cells by a retargeted adenovirus vector. J Virol 2000;74:2567-2583.
243. Saban SD, Nepomuceno RR, Gritton LD, Nemerow GR, Stewart PL. CryoEM structure at 9 angstrom resolution of an adenovirus vector targeted to hematopoietic cells. J Mol Biol 2005;349:526-537.
244. Havenga MJE, Lemckert AAC, Grimbergen JM, Vogels R, Huisman LGM, Valerio D, et al. Improved adenovirus vectors for infection of cardiovascular tissues. J Virol 2001;75:3335-3342.
245. Nicklin SA, White SJ, Watkins SJ, Hawkins RE, Baker AH. Selective targeting of gene transfer to vascular endothelial cells by use of peptides isolated by phage display. Circulation 2000;102:231-237.
246. Xia HB, Anderson B, Mao QW, Davidson BL. Recombinant human adenovirus: targeting to the human transferrin receptor improves gene transfer to brain microcapillary endothelium. J Virol 2000;74:11359-11366.
247. Fontana L, Nuzzo M, Urbanelli L, Monaci P. General strategy for broadening adenovirus tropism. J Virol 2003;77:11094-11104.
248. Pereboev A, Pereboeva L, Curiel DT. Phage display of adenovirus type 5 fiber knob as a tool for specific ligand selection and validation. J Virol 2001;75:7107-7113.
249. Krasnykh V, Belousova N, Korokhov N, Mikheeva G, Curiel DT. Genetic targeting of an adenovirus vector via replacement of the fiber protein with the phage T4 fibritin. J Virol 2001;75:4176-4183.
250. Hong SS, Magnusson MK, Henning P, Lindholm L, Boulanger PA. Adenovirus stripping: a versatile method to generate adenovirus vectors with new cell target specificity. Mol Ther 2003;7:692-699.
251. Wickham TJ, Segal DM, Roelvink PW, Carrion ME, Lizonova A, Lee GM, et al. Targeted adenovirus gene transfer to endothelial and smooth muscle cells by using bispecific antibodies. J Virol 1996;70:6831-6838.
252. Douglas JT, Rogers BE, Rosenfeld ME, Michael SI, Feng MZ, Curiel DT. Targeted gene delivery by tropism-modified adenoviral vectors. Nature Biotech 1996;14:1574-1578.
253. Rogers BE, Douglas JT, Ahlem C, Buchsbaum DJ, Frincke J, Curiel DT. Use of a novel cross-linking method to modify adenovirus tropism. Gene Ther 1997;4:1387-1392.
254. Miller CR, Buchsbaum DJ, Reynolds PN, Douglas JT, Gillespie GY, Mayo MS, et al. Differential susceptibility of primary and established human glioma cells to adenovirus infection: targeting via the epidermal growth factor receptor achieves fiber receptor-independent gene transfer. Cancer Res 1998;58:5738-5748.
255. O'Riordan CR, Lachapelle A, Delgado C, Parkes V, Wadsworth SC, Smith AE, et al. PEGylation of adenovirus with retention of infectivity and protection from neutralizing antibody in vitro and in vivo. Hum Gene Ther 1999;10:1349-1358.
256. Romanczuk H, Galer CE, Zabner J, Barsomian G, Wadsworth SC, O'Riordan CR. Modification of an adenoviral vector with biologically selected peptides: a novel strategy for gene delivery to cells of choice. Hum Gene Ther 1999;10:2615-2626.
257. Croyle MA, Yu QC, Wilson JM. Development of a rapid method for the PEGylation of adenoviruses with enhanced transduction and improved stability under harsh storage conditions. Hum Gene Ther 2000;11:1713-1722.
258. Smith JS, Keller JR, Lohrey NC, McCauslin CS, Ortiz M, Cowan K, et al. Redirected infection of directly biotinylated recombinant adenovirus vectors through cell surface receptors and antigens. Proc Natl Acad Sci U S A 1999;96:8855-8860.
259. Green NK, Herbert CW, Hale SJ, Hale AB, Mautner V, Harkins R, et al. Extended plasma circulation time and decreased toxicity of polymer-coated adenovirus. Gene Ther 2004;11:1256-1263.
260. Fisher KD, Stallwood Y, Green NK, Ulbrich K, Mautner V, Seymour LW. Polymer-coated adenovirus permits efficient retargeting and evades neutralising antibodies. Gene Ther 2001;8:341-348.