The development of gene therapy: From monogenic recessive disorders to complex diseases such as cancer

Methods in Molecular Biology

Volumn 542, Issue , 2009, Pages 5-54

  Gillet, Jean Pierre ^a   MacAdangdang, Benjamin ^a   Fathke, Robert L ^b   Gottesman, Michael M ^a   Kimchi Sarfaty, Chava ^b  

^a NATIONAL CANCER INSTITUTE   (United States)

^b CENTER FOR BIOLOGICS EVALUATION AND RESEARCH   (United States)

Author keywords

Antiangiogenic therapy; chemoinducible gene therapy; gene delivery systems; gene silencing; history of gene therapy; immunotherapy; oncolytic viruses; suicide gene therapy; tumor suppressors

Indexed keywords

HUMAN IMMUNODEFICIENCY VIRUS;

GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; HISTORY; HUMAN; NEOPLASM; RECESSIVE GENE; REVIEW;

GENE THERAPY; GENE TRANSFER TECHNIQUES; GENES, RECESSIVE; GENETIC VECTORS; HISTORY, 20TH CENTURY; HISTORY, 21ST CENTURY; HUMANS; NEOPLASMS;

EID: 67650299973     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-59745-561-9_1     Document Type: Review

References (378)

1. Lederberg , J.1963) Biological future of man. In: Wolstenholme, G., ed. Man and His Future . London : Churchill , 265 .
2. Tatum , E.1967) Molecular biology, nucleic acids, and the future of medicine. In: Lyght, C.E., ed. Reflections on Research and the Future of Medicine . New York : McGraw-Hill , 31-49 .
3. Tatum , E. L.1966) Molecular biology, nucleic acids, and the future of medicine . Perspect. Biol. Med. 10 , 19-32
4. Lederberg , J.1966) Experimental genetics and human evolution . Am. Naturalist 100 , 519-531 .
5. Burnet , M.1971) Genes, Dreams and Reality . New York : Basic Books . 71 .
6. Culliton , B. J.1989) French Anderson's 20-year crusade . Science 246 , 748 .
7. Nirenberg , M. W.1967) Will society be prepared? Science 157 , 633 .
8. Sinsheimer , R.1969) The prospect for designed genetic change . Am. Sci. 57 , 134-142 .
9. Terheggen , H. , Schwenk , A. , Van Sande , M. , Lowenthal , A. , and Columbo , J.1969) Argininemia with arginase deficiency . Lancet 2 , 748-749 .
10. Rogers , S.1966) Shope papilloma virus: A passenger in man and its significance to the potential control of the host genome . Nature 212 , 1220-1222 .
11. Meselson , M. , and Yuan , R.1968) DNA restriction enzyme from E. coli . Nature 217 , 1110-1114 .
12. Kelly , T. J. , Jr. , and Smith , H. O.1970) A restriction enzyme from Hemophilus influenzae. II . J. Mol. Biol. 51 , 393-409 .
13. Smith , H. O. , and Wilcox , K. W.1970) A restriction enzyme from Hemophilus influenzae. I. Purification and general properties . J. Mol. Biol. 51 , 379-391 .
14. Danna , K. , and Nathans , D.1971) Specific cleavage of simian virus 40 DNA by restriction endonuclease of Hemophilus influenzae . Proc. Natl. Acad. Sci. USA 68 , 2913-2917 .
15. Wigler , M. , Silverstein , S. , Lee , L. S. , Pellicer , A. , Cheng , Y. , and Axel , R.1977) Transfer of purified herpes virus thymidine kinase gene to cultured mouse cells . Cell 11 , 223-232 .
16. Anderson , W. F. , Killos , L. , Sanders-Haigh , L. , Kretschmer , P. J. , and Diacumakos , E. G.1980) Replication and expression of thymidine kinase and human globin genes microinjected into mouse fibroblasts . Proc. Natl. Acad. Sci. USA 77 , 5399-5403 .
17. Anderson , W. F. , and Fletcher , J. C.1980) Sounding boards. Gene therapy in human beings: when is it ethical to begin? N. Engl. J. Med. 303 , 1293-1297 .
18. Miller , A. D. , Jolly , D. J. , Friedmann , T. , and Verma , I. M.1983) A transmissible retrovirus expressing human hypoxanthine phosphoribosyltransferase (HPRT): Gene transfer into cells obtained from humans deficient in HPRT . Proc. Natl. Acad. Sci. USA 80 , 4709-4713 .
19. Willis , R. C. , Jolly , D. J. , Miller , A. D. , Plent , M. M. , Esty , A. C. , Anderson , P. J. , Chang , H. C. , Jones , O. W. , Seegmiller , J. E. , and Friedmann , T.1984) Partial phenotypic correction of human Lesch-Nyhan (hypoxanthine- guanine phosphoribosyltransferasedeficient) lymphoblasts with a transmissible retroviral vector . J. Biol. Chem. 259 , 7842-7849 .
20. Joyner , A. , Keller , G. , Phillips , R. A. , and Bernstein , A.1983) Retrovirus transfer of a bacterial gene into mouse haematopoietic progenitor cells . Nature 305 , 556-558
21. Watanabe , S. , and Temin , H. M.1983) Construction of a helper cell line for avian reticuloendotheliosis virus cloning vectors . Mol. Cell. Biol. 3 , 2241-2249 .
22. Mann , R. , Mulligan , R. C. , and Baltimore , D.1983) Construction of a retrovirus packaging mutant and its use to produce helperfree defective retrovirus . Cell 33 , 153-159 .
23. Williams , D. A. , Lemischka , I. R. , Nathan , D. G. , and Mulligan , R. C.1984) Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse . Nature 310 , 476-480 .
24. Van Doren , K. , Hanahan , D. , and Gluzman , Y.1984) Infection of eukaryotic cells by helperindependent recombinant adenoviruses: Early region 1 is not obligatory for integration of viral DNA . J. Virol. 50 , 606-614 .
25. Hermonat , P. L. , and Muzyczka , N.1984) Use of adeno-associated virus as a mammalian DNA cloning vector: Transduction of neomycin resistance into mammalian tissue culture cells . Proc. Natl. Acad. Sci. USA 81 , 6466-6470 .
26. Tratschin , J. D. , West , M. H. , Sandbank , T. , and Carter , B. J.1984) A human parvovirus, adeno-associated virus, as a eukaryotic vector: Transient expression and encapsidation of the prokaryotic gene for chloramphenicol acetyltransferase . Mol. Cell. Biol. 4 , 2072-2081 .
27. Eglitis , M. A. , Kantoff , P. , Gilboa , E. , and Anderson , W. F.1985) Gene expression in mice after high efficiency retroviral-mediated gene transfer . Science 230 , 1395-1398 .
28. Rosenberg , S. A. , Aebersold , P. , Cornetta , K. , Kasid , A. , Morgan , R. A. , Moen , R. , Karson , E. M. , Lotze , M. T. , Yang , J. C. , Topalian , S. L. , et al.1990) Gene transfer into humans- -immunotherapy of patients with adVanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction . N. Engl. J. Med. 323 , 570-578 .
29. Blaese , R. M. , Culver , K. W. , Miller , A. D. , Carter , C. S. , Fleisher , T. , Clerici , M. , Shearer , G. , Chang , L. , Chiang , Y. , Tolstoshev , P. , Greenblatt , J. J. , Rosenberg , S. A. , Klein , H. , Berger , M. , Mullen , C. A. , Ramsey , W. J. , Muul , L. , Morgan , R. A. , and Anderson , W. F.1995) T lymphocyte-directed gene therapy for ADA- SCID: Initial trial results after 4 years . Science 270 , 475-480 .
30. Wilson , J. M. , Grossman , M. , Raper , S. E. , Baker , J. R. , Jr. , Newton , R. S. , and Thoene , J. G.1992) Ex vivo gene therapy of familial hypercholesterolemia . Hum. Gene Ther. 3 , 179-222 .
31. Grossman , M. , Raper , S. E. , Kozarsky , K. , Stein , E. A. , Engelhardt , J. F. , Muller , D. , Lupien , P. J. , and Wilson , J. M.1994) Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia . Nat. Genet. 6 , 335-341 .
32. Grossman , M. , Rader , D. J. , Muller , D. W. , Kolansky , D. M. , Kozarsky , K. , Clark , B. J. , 3rd , Stein , E. A. , Lupien , P. J. , Brewer , H. B. , Jr. , Raper , S. E. , et al.1995) A pilot studyof ex vivo gene therapy for homozygous familial hypercholesterolaemia . Nat. Med. 1 , 1148-1154 .
33. 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. , Bousso , P. , Deist , F. L. , and Fischer , A.2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease . Science 288 , 669-672 .
34. Hacein-Bey-Abina , S. , Von Kalle , C. , Schmidt , M. , McCormack , M. P. , Wulffraat , N. , Leboulch , P. , Lim , A. , Osborne , C. S. , Pawliuk , R. , Morillon , E. , Sorensen , R. , Forster , A. , Fraser , P. , Cohen , J. I. , de Saint Basile , G. , Alexander , I. , Wintergerst , U. , Frebourg , T. , Aurias , A. , Stoppa-Lyonnet , D. , Romana , S. , Radford- Weiss , I. , Gross , F. , Valensi , F. , Delabesse , E. , Macintyre , E. , Sigaux , F. , Soulier , J. , Leiva , L. E. , Wissler , M. , Prinz , C. , Rabbitts , T. H. , Le Deist , F. , Fischer , A. , and Cavazzana-Calvo , M.2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 . Science 302 , 415-419 .
35. Nienhuis , A. W. , Dunbar , C. E. , and Sorrentino , B. P.2006) Genotoxicity of retroviral integration in hematopoietic cells . Mol. Ther. 13 , 1031-1049 .
36. Bester , A. C. , Schwartz , M. , Schmidt , M. , Garrigue , A. , Hacein-Bey-Abina , S. , Cavazzana- Calvo , M. , Ben-Porat , N. , Von Kalle , C. , Fischer , A. , and Kerem , B.2006) Fragile sites are preferential targets for integrations of MLV vectors in gene therapy . Gene Ther. 13 , 1057-1059 .
37. Dave , U. P. , Jenkins , N. A. , and Copeland , N. G.2004) Gene therapy insertional mutagenesis insights . Science 303 , 333 .
38. Aiuti , A. , Slavin , S. , Aker , M. , Ficara , F. , Deola , S. , Mortellaro , A. , Morecki , S. , Andolfi , G. , Tabucchi , A. , Carlucci , F. , Marinello , E. , Cattaneo , F. , Vai , S. , Servida , P. , Miniero , R. , Roncarolo , M. G. , and Bordignon , C.2002) Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning . Science 296 , 2410-2413 .
39. Gaspar , H. B. , Bjorkegren , E. , Parsley , K. , Gilmour , K. C. , King , D. , Sinclair , J. , Zhang , F. , Giannakopoulos , A. , Adams , S. , Fairbanks , L. D. , Gaspar , J. , Henderson , L. , Xu-Bayford , J. H. , Davies , E. G. , Veys , P. A. , Kinnon , C. , and Thrasher , A. J.2006) Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEGADA and use of mild preconditioning . Mol. Ther. 14 , 505-513 .
40. Rainov , N. G.2000) 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. 11 , 2389-2401 .
41. Khuri , F. R. , Nemunaitis , J. , Ganly , I. , Arseneau , J. , Tannock , I. F. , Romel , L. , Gore , M. , Ironside , J. , MacDougall , R. H. , Heise , C. , Randlev , B. , Gillenwater , A. M. , Bruso , P. , Kaye , S. B. , Hong , W. K. , and Kirn , D. H.2000) 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 . Nat. Med. 6 , 879-885 .
42. Alton , E.2007) Progress and prospects: Gene therapy clinical trials (part 1) . Gene Ther. 14 , 1439-1447 .
43. Griesenbach , U.2007) Progress and prospects: Gene therapy clinical trials (part 2) . Gene Ther. 14 , 1555-1563 .
44. Edelstein , M. L. , Abedi , M. R. , and Wixon , J.2007) Gene therapy clinical trials worldwide to 2007-an update . J. Gene Med. 9 , 833-842 .
45. Berns , A.2004) Good news for gene therapy . N. Engl. J. Med. 350 , 1679-1680 .
46. Aghi , M. , Hochberg , F. , and Breakefield , X. O.2000) Prodrug activation enzymes in cancer gene therapy . J. Gene Med. 2 , 148-164 .
47. Rux , J. J. , and Burnett , R. M.2004) Adenovirus structure . Hum. Gene Ther. 15 , 1167-1176 .
48. Majhen , D. , and Ambriovic-Ristov , A.2006) Adenoviral vectors - how to use them in cancer gene therapy? Virus Res. 119 , 121 - 133.
49. Xia , D. , Henry , L. J. , Gerard , R. D. , and Deisenhofer , J.1994) Crystal structure of the receptor-binding domain of adenovirus type 5 fiber protein at 1.7 A resolution . Structure 2 , 1259-1270 .
50. Bergelson , J. M. , Cunningham , J. A. , Droguett , G. , Kurt-Jones , E. A. , Krithivas , A. , Hong , J. S. , Horwitz , M. S. , Crowell , R. L. , and Finberg , R. W.1997) Isolation of a common receptor for Coxsackie B viruses and adenoviruses 2 and 5 . Science 275 , 1320-1323 .
51. Li , J. , Lad , S. , Yang , G. , Luo , Y. , Iacobelli- Martinez , M. , Primus , F. J. , Reisfeld , R. A. , and Li , E.2006) Adenovirus fiber shaft contains a trimerization element that supports peptide fusion for targeted gene delivery . J. Virol. 80 , 12324-12331 .
52. Meier , O. , Boucke , K. , Hammer , S. V. , Keller , S. , Stidwill , R. P. , Hemmi , S. , and Greber , U. F.2002) Adenovirus triggers macropinocytosis and endosomal leakage together with its clathrinmediated uptake . J. Cell. Biol. 158 , 1119-1131 .
53. Young , L. S. , Searle , P. F. , Onion , D. , and Mautner , V.2006) Viral gene therapy strategies: From basic science to clinical application . J. Pathol. 208 , 299-318 .
54. Bischoff , J. R. , Kirn , D. H. , Williams , A. , Heise , C. , Horn , S. , Muna , M. , Ng , L. , Nye , J. A. , Sampson-Johannes , A. , Fattaey , A. , and McCormick , F.1996) An adenovirus mutant that replicates selectively in p53-deficient human tumor cells . Science 274 , 373-376 .
55. Heise , C. , Hermiston , T. , Johnson , L. , Brooks , G. , Sampson-Johannes , A. , Williams , A. , Hawkins , L. , and Kirn , D.2000) An adenovirus E1A mutant that demonstrates potent and selective systemic anti-tumoral efficacy . Nat. Med. 6 , 1134-1139 .
56. Fukuda , K. , Abei , M. , Ugai , H. , Seo , E. , Wakayama , M. , Murata , T. , Todoroki , T. , Tanaka , N. , Hamada , H. , and Yokoyama , K. K.2003) E1A, E1B double-restricted adenovirus for oncolytic gene therapy of gallbladder cancer . Cancer Res. 63 , 4434-4440 .
57. Balague , C. , Noya , F. , Alemany , R. , Chow , L. T. , and Curiel , D. T.2001) Human papillomavirus E6E7-mediated adenovirus cell killing: Selectivity of mutant adenovirus replication in organotypic cultures of human keratinocytes . J. Virol. 75 , 7602-7611 .
58. Ali , H. , LeRoy , G. , Bridge , G. , and Flint , S. J.2007) The adenovirus L4 33-kilodalton protein binds to intragenic sequences of the major late promoter required for late phasespecific stimulation of transcription . J. Virol. 81 , 1327-1338 .
59. Kaplan , J. M.2005) Adenovirus-based cancer gene therapy . Curr. Gene Ther. 5 , 595-605 .
60. Flint , J. , and Shenk , T.1997) Viral transactivating proteins . Annu. Rev. Genet. 31 , 177-212 .
61. Yew , P. R. , and Berk , A. J.1992) Inhibition of p53 transactivation required for transformation by adenovirus early 1B protein . Nature 357 , 82-85 .
62. Mizuguchi , H. , and Hayakawa , T.2004) Targeted adenovirus vectors . Hum. Gene Ther. 15 , 1034-1044 .
63. Raper , S. E. , Chirmule , N. , Lee , F. S. , Wivel , N. A. , Bagg , A. , Gao , G. P. , Wilson , J. M. , and Batshaw , M. L.2003) Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient followingadenoviral gene transfer . Mol. Genet. Metab. 80 , 148-158 .
64. Bett , A. J. , Haddara , W. , Prevec , L. , and Graham , F. L.1994) An efficient and flexible system for construction of adenovirus vectors with insertions or deletions in early regions 1 and 3 . Proc. Natl. Acad. Sci. USA 91 , 8802-8806 .
65. Schaack , J.2005) Adenovirus vectors deleted for genes essential for viral DNA replication . Front Biosci. 10 , 1146-1155 .
66. Yang , Y. , Nunes , F. A. , Berencsi , K. , Furth , E. E. , Gonczol , E. , and Wilson , J. M.1994) Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy . Proc. Natl. Acad. Sci. USA 91 , 4407-4411 .
67. Knowles , M. R. , Hohneker , K. W. , Zhou , Z. , Olsen , J. C. , Noah , T. L. , Hu , P. C. , Leigh , M. W. , Engelhardt , J. F. , Edwards , L. J. , Jones , K. R. , and et al.1995) A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis . N. Engl. J. Med. 333 , 823-831 .
68. Amalfitano , A. , Hauser , M. A. , Hu , H. , Serra , D. , Begy , C. R. , and Chamberlain , J. S.1998) Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted . J. Virol. 72 , 926-933 .
69. Gorziglia , M. I. , Lapcevich , C. , Roy , S. , Kang , Q. , Kadan , M. , Wu , V. , Pechan , P. , and Kaleko , M.1999) Generation of an adenovirus vector lacking E1, e2a, E3, and all of E4 except open reading frame 3 . J. Virol. 73 , 6048-6055 .
70. Ji , L. , Bouvet , M. , Price , R. E. , Roth , J. A. , and Fang , B.1999) Reduced toxicity, attenuated immunogenicity and efficient mediation of human p53 gene expression in vivo by an adenovirus vector with deleted E1-E3 and inactivated E4 by GAL4-TATA promoter replacement . Gene Ther. 6 , 393-402 .
71. Lieber , A. , Steinwaerder , D. S. , Carlson , C. A. , and Kay , M. A.1999) Integrating adenovirus- adeno-associated virus hybrid vectors devoid of all viral genes . J. Virol. 73 , 9314-9324 .
72. Steinwaerder , D. S. , Carlson , C. A. , and Lieber , A.1999) Generation of adenovirus vectors devoid of all viral genes by recombination between inverted repeats . J. Virol. 73 , 9303-9313 .
73. Schiedner , G. , Morral , N. , Parks , R. J. , Wu , Y. , Koopmans , S. C. , Langston , C. , Graham , F. L. , Beaudet , A. L. , and Kochanek , S.1998) Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity . Nat. Genet. 18 , 180-183 .
74. Gall , J. , Kass-Eisler , A. , Leinwand , L. , and Falck-Pedersen , E.1996) Adenovirus type 5 and 7 capsid chimera: Fiber replacement alters receptor tropism without affecting primary immune neutralization epitopes . J. Virol. 70 , 2116-2123 .
75. Chillon , M. , Bosch , A. , Zabner , J. , Law , L. , Armentano , D. , Welsh , M. J. , and Davidson , B. L.1999) Group D adenoviruses infect primary central nervous system cells more efficiently than those from group C . J. Virol. 73 , 2537-2540 .
76. Shayakhmetov , D. M. , Papayannopoulou , T. , Stamatoyannopoulos , G. , and Lieber , A.2000) Efficient gene transfer into human CD34(+) cells by a retargeted adenovirus vector . J. Virol. 74 , 2567-2583 .
77. Denby , L. , Work , L. M. , Graham , D. , Hsu , C. , von Seggern , D. J. , Nicklin , S. A. , and Baker , A. H.2004) Adenoviral serotype 5 vectors pseudotyped with fibers from subgroup D show modified tropism in vitro and in vivo . Hum. Gene Ther. 15 , 1054-1064 .
78. Suominen , E. , Toivonen , R. , Grenman , R. , and Savontaus , M.2006) Head and neck cancer cells are efficiently infected by Ad5/35 hybrid virus . J. Gene Med. 8 , 1223-1231 .
79. Denby , L. , Work , L. M. , Seggern , D. J. , Wu , E. , McVey , J. H. , Nicklin , S. A. , and Baker , A. H.2007) Development of renal-targeted vectors through combined in vivo phage display and capsid engineering of adenoviral fibers from serotype 19p . Mol. Ther. 15 , 1647-1654 .
80. Wickham , T. J. , Tzeng , E. , Shears , L. L. , 2nd , Roelvink , P. W. , Li , Y. , Lee , G. M. , Brough , D. E. , Lizonova , A. , and Kovesdi , I.1997) Increased in vitro and in vivo gene transfer by adenovirus vectors containing chimeric fiber proteins J. Virol. 71 , 8221-8229 .
81. Hemminki , A. , Kanerva , A. , Liu , B. , Wang , M. , Alvarez , R. D. , Siegal , G. P. , and Curiel , D. T.2003) Modulation of coxsackie-adenovirus receptor expression for increased adenoviral transgene expression . Cancer Res. 63 , 847-853 .
82. Vincent , T. , Pettersson , R. F. , Crystal , R. G. , and Leopold , P. L.2004) Cytokine-mediated downregulation of coxsackievirus-adenovirus receptor in endothelial cells . J. Virol. 78 , 8047-8058 .
83. Oh , I. K. , Mok , H. , and Park , T. G.2006) Folate immobilized and PEGylated adenovirus for retargeting to tumor cells . Bioconjug. Chem. 17 , 721-727 .
84. Leamon , C. P. , and Low , P. S.1991) Delivery of macromolecules into living cells: A method that exploits folate receptor endocytosis . Proc. Natl. Acad. Sci. USA 88 , 5572-5576 .
85. Paillart , J. C. , Shehu-Xhilaga , M. , Marquet , R. , and Mak , J.2004) Dimerization of retroviral RNA genomes: An inseparable pair . Nat. Rev. Microbiol. 2 , 461-472 .
86. Andrake , M. D. , and Skalka , A. M.1996) Retroviral integrase, putting the pieces together . J. Biol. Chem. 271 , 19633-19636 .
87. Khan , E. , Mack , J. P. , Katz , R. A. , Kulkosky , J. , and Skalka , A. M.1991) Retroviral integrase domains: DNA binding and the recognition of LTR sequences . Nucleic Acids Res. 19 , 851-860 .
88. Balvay , L. , Lopez Lastra , M. , Sargueil , B. , Darlix , J. L. , and Ohlmann , T.2007) Translational control of retroviruses . Nat. Rev. Microbiol. 5 , 128-140 .
89. Bender , W. , Chien , Y. H. , Chattopadhyay , S. , Vogt , P. K. , Gardner , M. B. , and Davidson , N.1978) High-molecular-weight RNAs of AKR, NZB, and wild mouse viruses and avian reticuloendotheliosis virus all have similar dimer structures . J. Virol. 25 , 888-896 .
90. Goff , S. P. , and Lobel , L. I.1987) Mutants of murine leukemia viruses and retroviral replication . Biochim. Biophys. Acta 907 , 93-123 .
91. Miller , A. D.1996) Cell-surface receptors for retroviruses and implications for gene transfer . Proc. Natl. Acad. Sci. USA 93 , 11407-11413 .
92. White , J. M.1992) Membrane fusion . Science 258 , 917-924 .
93. Thomas , C. E. , Ehrhardt , A. , and Kay , M. A.2003) Progress and problems with the use of viral vectors for gene therapy . Nat. Rev. Genet. 4 , 346-358 .
94. Yi , Y. , Hahm , S. H. , and Lee , K. H.2005) Retroviral gene therapy: Safety issues and possible solutions . Curr. Gene Ther. 5 , 25-35 .
95. Xu , L. , Mei , M. , Haskins , M. E. , Nichols , T. C. , O'Donnell , P. , Cullen , K. , Dillow , A. , Bellinger , D. , and Ponder , K. P.2007) Immune response after neonatal transfer of a human factor IX-expressing retroviral vector in dogs, cats, and mice . Thromb. Res. 120 , 269-280 .
96. Li , Z. , Kustikova , O. S. , Kamino , K. , Neumann , T. , Rhein , M. , Grassman , E. , Fehse , B. , and Baum , C.2007) Insertional mutagenesis by replication-deficient retroviral vectors encoding the large T oncogene . Ann. N Y Acad. Sci. 1106 , 95-113 .
97. Rodrigues , T. , Carvalho , A. , Carmo , M. , Carrondo , M. J. , Alves , P. M. , and Cruz , P. E.2007) Scaleable purification process for gene therapy retroviral vectors . J. Gene Med. 9 , 233-243 .
98. Weber , E. , Anderson , W. F. , and Kasahara , N.2001) Recent adVances in retrovirus vectormediated gene therapy: Teaching an old vector new tricks . Curr. Opin. Mol. Ther. 3 , 439-453 .
99. Sinn , P. L. , Sauter , S. L. , and McCray , P. B. , Jr.2005) Gene therapy progress and prospects: Development of improved lentiviral and retroviral vectors - design, biosafety, and production . Gene Ther. 12 , 1089-1098 .
100. Kaiser , J.2003) Gene therapy. Seeking the cause of induced leukemias in X-SCID trial . Science 299 , 495 .
101. Maxfield , L. F. , Fraize , C. D. , and Coffin , J. M.2005) Relationship between retroviral DNA-integration-site selection and host cell transcription . Proc. Natl. Acad. Sci. USA102 , 1436-1441 .
102. Tan , W. , Zhu , K. , Segal , D. J. , Barbas , C. F. , 3rd , and Chow , S. A.2004) Fusion proteins consisting of human immunodeficiency virus type 1 integrase and the designed polydactyl zinc finger protein E2C direct integration of viral DNA into specific sites . J. Virol. 78 , 1301-1313 .
103. Ellis , J. , and Yao , S.2005) Retrovirus silencing and vector design: ReleVance to normal and cancer stem cells? Curr. Gene Ther. 5 , 367-373 .
104. Wei , G. H. , Liu , D. P. , and Liang , C. C.2005) Chromatin domain boundaries: Insulators and beyond . Cell Res. 15 , 292-300 .
105. Dalba , C. , Klatzmann , D. , Logg , C. R. , and Kasahara , N.2005) Beyond oncolytic virotherapy: Replication-competent retrovirus vectors for selective and stable transduction of tumors . Curr. Gene Ther. 5 , 655-667 .
106. Wang , W. J. , Tai , C. K. , Kasahara , N. , and Chen , T. C.2003) Highly efficient and tumor-restricted gene transfer to malignant gliomas by replication-competent retroviral vectors . Hum. Gene Ther. 14 , 117-127 .
107. Wang , W. , Tai , C. K. , Kershaw , A. D. , Solly , S. K. , Klatzmann , D. , Kasahara , N. , and Chen , T. C.2006) Use of replication-competent retroviral vectors in an immunocompetent intracranial glioma model . Neurosurg. Focus 20 , E25 .
108. Solly , S. K. , Trajcevski , S. , Frisen , C. , Holzer , G. W. , Nelson , E. , Clerc , B. , Abordo- Adesida , E. , Castro , M. , Lowenstein , P. , and Klatzmann , D.2003) Replicative retroviral vectors for cancer gene therapy . Cancer Gene Ther. 10 , 30-39 .
109. Hiraoka , K. , Kimura , T. , Logg , C. R. , Tai , C. K. , Haga , K. , Lawson , G. W. , and Kasahara , N.2007) Therapeutic efficacy of replication- competent retrovirus vector-mediated suicide gene therapy in a multifocal colorectal cancer metastasis model . Cancer Res. 67 , 5345-5353 .
110. Li , Z. , Dullmann , J. , Schiedlmeier , B. , Schmidt , M. , von Kalle , C. , Meyer , J. , Forster , M. , Stocking , C. , Wahlers , A. , Frank , O. , Ostertag , W. , Kuhlcke , K. , Eckert , H. G. , Fehse , B. , and Baum , C.2002) Murine leukemia induced by retroviral gene marking . Science 296 , 497 .
111. Donahue , R. E. , Kessler , S. W. , Bodine , D. , McDonagh , K. , Dunbar , C. , Goodman , S. , Agricola , B. , Byrne , E. , Raffeld , M. , Moen , R. , and et al.1992) Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer . J. Exp. Med. 176 , 1125-1135 .
112. Mitrophanous , K. , Yoon , S. , Rohll , J. , Patil , D. , Wilkes , F. , Kim , V. , Kingsman , S. , Kingsman , A. , and Mazarakis , N.1999) Stable gene transfer to the nervous system using non-primate lentiviral vector . Gene Ther. 6 1808-1818 .
113. Poeschla , E. M. , Wong-Staal , F. , and Looney , D. J.1998) Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors . Nat. Med. 4 , 354-357 .
114. Wiznerowicz , M. , and Trono , D.2005) Harnessing HIV for therapy, basic research and biotechnology . Trends Biotechnol. 23 , 42-47 .
115. Naldini , L. , Blomer , U. , Gallay , P. , Ory , D. , Mulligan , R. , Gage , F. H. , Verma , I. M. , and Trono , D.1996) In vivo gene delivery and stable transduction of nondividing cells by lentiviral vector . Science 272 263-267 .
116. Lewis , P. , Hensel , M. , and Emerman , M.1992) Human immunodeficiency virus infection of cells arrested in the cell cycle . EMBO J. 11 , 3053-3058 .
117. Blomer , U. , Naldini , L. , Kafri , T. , Trono , D. , Verma , I. M. , and Gage , F. H.1997) Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector . J. Virol. 71 , 6641-6649 .
118. Miyoshi , H. , Takahashi , M. , Gage , F. H. , and Verma , I. M.1997) Stable and efficient gene transfer into the retina using an HIVbased lentiviral vector . Proc. Natl. Acad. Sci. USA 94 , 10319-10323 .
119. Kafri , T.2004) Gene delivery by lentivirus vectors an overview . Methods Mol. Biol. 246 , 367-390 .
120. Mazarakis , N. D. , Azzouz , M. , Rohll , J. B. , Ellard , F. M. , Wilkes , F. J. , Olsen , A. L. , Carter , E. E. , Barber , R. D. , Baban , D. F. , Kingsman , S. M. , Kingsman , A. J. , O'Malley , K. , and Mitrophanous , K. A.2001) Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery . Hum. Mol. Genet. 10 , 2109-2121 .
121. Wong , L. F. , Azzouz , M. , Walmsley , L. E. , Askham , Z. , Wilkes , F. J. , Mitrophanous , K. A. , Kingsman , S. M. , and Mazarakis , N. D.2004) Transduction patterns of pseudotyped lentiviral vectors in the nervous system . Mol. Ther. 9 , 101-111 .
122. Deglon , N. , Tseng , J. L. , Bensadoun , J. C. , Zurn , A. D. , Arsenijevic , Y. , Pereira de Almeida , L. , Zufferey , R. , Trono , D. , and Aebischer , P.2000) Self-inactivating lentiviral vectors with enhanced transgene expression as potential gene transfer system in Parkinson's disease . Hum. Gene Ther. 11 , 179-190 .
123. Han , J. J. , Mhatre , A. N. , Wareing , M. , Pettis , R. , Gao , W. Q. , Zufferey , R. N. , Trono , D. , and Lalwani , A. K.1999) Transgene expression in the guinea pig cochlea mediated by lentivirus-derived gene transfer vector . Hum. Gene Ther. 10 1867-1873 .
124. Miyoshi , H. , Blomer , U. , Takahashi , M. , Gage , F. H. , and Verma , I. M.1998) Development of a self-inactivating lentivirus vector . J. Virol. 72 , 8150-8157 .
125. Pan , D. , Gunther , R. , Duan , W. , Wendell , S. , Kaemmerer , W. , Kafri , T. , Verma , I. M. , and Whitley , C. B.2002) Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow . Mol. Ther. 6 , 19-29 .
126. Vigna , E. , and Naldini , L.2000) Lentiviral vectors: Excellent tools for experimental gene transfer and promising candidates for gene therapy . J. Gene Med. 2 , 308-316 .
127. Jakobsson , J. , and Lundberg , C.2006) Lentiviral vectors for use in the central nervous system . Mol. Ther. 13 , 484-493 .
128. Dull , T. , Zufferey , R. , Kelly , M. , Mandel , R. J. , Nguyen , M. , Trono , D. , and Naldini , L.1998) A third-generation lentivirus vector with a conditional packaging system . J. Virol. 72 , 8463-8471 .
129. Kim , V. N. , Mitrophanous , K. , Kingsman , S. M. , and Kingsman , A. J.1998) Minimal requirement for a lentivirus vector based on human immunodeficiency virus type 1 . J. Virol. 72 , 811-816 .
130. Kotsopoulou , E. , Kim , V. N. , Kingsman , A. J. , Kingsman , S. M. , and Mitrophanous , K. A.2000) A Rev-independent human immunodeficiency virus type 1 (HIV-1)-based vector that exploits a codon-optimized HIV-1 gag-pol gene . J. Virol. 74 , 4839-4852 .
131. Rohll , J. B. , Mitrophanous , K. A. , Martin- Rendon , E. , Ellard , F. M. , Radcliffe , P. A. , Mazarakis , N. D. , and Kingsman , S. M.2002) Design, production, safety, evaluation, and clinical applications of nonprimate lentiviral vectors . Methods Enzymol. 346 , 466-500 .
132. Sirin , O. , and Park , F.2003) Regulating gene expression using self-inactivating lentiviral vectors containing the mifepristoneinducible system . Gene 323 , 67-77 .
133. Reiser , J. , Lai , Z. , Zhang , X. Y. , and Brady , R. O.2000) Development of multigene and regulated lentivirus vectors . J. Virol. 74 , 10589-10599 .
134. Ralph , G. S. , Binley , K. , Wong , L. F. , Azzouz , M. , and Mazarakis , N. D.2006) Gene therapy for neurodegenerative and ocular diseases using lentiviral vectors . Clin. Sci.Lond) 110 , 37-46 .
135. Krisky , D. M. , Marconi , P. C. , Oligino , T. J. , Rouse , R. J. , Fink , D. J. , Cohen , J. B. , Watkins , S. C. , and Glorioso , J. C.1998) Development of herpes simplex virus replication- defective multigene vectors for combination gene therapy applications . Gene Ther. 5 , 1517-1530 .
136. Glorioso , J. C. , and Fink , D. J.2004) Herpes vector-mediated gene transfer in treatment of diseases of the nervous system . Annu. Rev. Microbiol. 58 , 253-271 .
137. Latchman , D. S.1999) Herpes simplex virus vectors for gene therapy in Parkinson' disease and other diseases of the nervous system . J. R. Soc. Med. 92 , 566-570 .
138. Kristensson , K. , Lycke , E. , and Sjostrand , J.1971) Spread of herpes simplex virus in peripheral nerves . Acta Neuropathol.Berl) 17 , 44-53 .
139. Berges , B. K. , Wolfe , J. H. , and Fraser , N. W.2007) Transduction of brain by herpes simplex virus vectors . Mol. Ther. 15 , 20-29 .
140. Burton , E. A. , and Glorioso , J. C.2001) Multi-modal combination gene therapy for malignant glioma using replication-defective HSV vectors . Drug Discov. Today 6 , 347-356 .
141. Yenari , M. A. , Fink , S. L. , Sun , G. H. , Chang , L. K. , Patel , M. K. , Kunis , D. M. , Onley , D. , Ho , D. Y. , Sapolsky , R. M. , and Steinberg , G. K.1998) Gene therapy with HSP72 is neuroprotective in rat models of stroke and epilepsy . Ann. Neurol. 44 , 584-591 .
142. Yamada , M. , Oligino , T. , Mata , M. , Goss , J. R. , Glorioso , J. C. , and Fink , D. J.1999) Herpes simplex virus vector-mediated expression of Bcl-2 prevents 6-hydroxydopamine-induced degeneration of neurons in the substantia nigra in vivo . Proc. Natl. Acad. Sci. USA 96 , 4078-4083 .
143. Martino , G. , Poliani , P. L. , Furlan , R. , Marconi , P. , Glorioso , J. C. , Adorini , L. , and Comi , G.2000) Cytokine therapy in immune-mediated demyelinating diseases of the central nervous system: A novel gene therapy approach . J. Neuroimmunol. 107 , 184-190 .
144. Sun , M. , Kong , L. , Wang , X. , Holmes , C. , Gao , Q. , Zhang , G. R. , Pfeilschifter , J. , Goldstein , D. S. , and Geller , A. I.2004) Coexpression of tyrosine hydroxylase, GTP cyclohydrolase I, aromatic amino acid decarboxylase, and vesicular monoamine transporter 2 from a helper virus-free herpes simplex virus type 1 vector supports highlevel, long-term biochemical and behavioral correction of a rat model of Parkinson's disease . Hum. Gene Ther. 15 , 1177-1196 .
145. Samaniego , L. A. , Neiderhiser , L. , and DeLuca , N. A.1998) Persistence and expression of the herpes simplex virus genome in the absence of immediate-early proteins . J. Virol. 72 , 3307-3320 .
146. Bell , J. C. , Lichty , B. , and Stojdl , D.2003) Getting oncolytic virus therapies off the ground . Cancer Cell 4 , 7-11 .
147. Kesari , S. , Randazzo , B. P. , Valyi-Nagy , T. , Huang , Q. S. , Brown , S. M. , MacLean , A. R. , Lee , V. M. , Trojanowski , J. Q. , and Fraser , N. W.1995) Therapy of experimental human brain tumors using a neuroattenuated herpes simplex virus mutant . Lab. Invest. 73 , 636-648 .
148. Yoon , S. S. , Nakamura , H. , Carroll , N. M. , Bode , B. P. , Chiocca , E. A. , and Tanabe , K. K.2000) An oncolytic herpes simplex virus type 1 selectively destroys diffuse liver metastases from colon carcinoma . FASEB J. 14 , 301-311 .
149. Varghese , S. , and Rabkin , S. D.2002) Oncolytic herpes simplex virus vectors for cancer virotherapy . Cancer Gene Ther. 9 , 967-978 .
150. Markert , J. M. , Medlock , M. D. , Rabkin , S. D. , Gillespie , G. Y. , Todo , T. , Hunter , W. D. , Palmer , C. A. , Feigenbaum , F. , Tornatore , C. , Tufaro , F. , and Martuza , R. L.2000) Conditionally replicating herpes simplex virus mutant, G207 for the treatment of malignant glioma: Results of a phase I trial . Gene Ther. 7 , 867-874 .
151. Rampling , R. , Cruickshank , G. , Papanastassiou , V. , Nicoll , J. , Hadley , D. , Brennan , D. , Petty , R. , MacLean , A. , Harland , J. , McKie , E. , Mabbs , R. , and Brown , M.2000) Toxicity evaluation of replication-competent herpes simplex virus (ICP 34.5 null mutant 1716) in patients with recurrent malignant glioma . Gene Ther. 7 , 859-866 .
152. Papanastassiou , V. , Rampling , R. , Fraser , M. , Petty , R. , Hadley , D. , Nicoll , J. , Harland , J. , Mabbs , R. , and Brown , M.2002) 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. 9 , 398-406 .
153. MacKie , R. M. , Stewart , B. , and Brown , S. M.2001) Intralesional injection of herpes simplex virus 1716 in metastatic melanoma . Lancet 357 , 525-526 .
154. Liddington , R. C. , Yan , Y. , Moulai , J. , Sahli , R. , Benjamin , T. L. , and Harrison , S. C.1991) Structure of simian virus 40 at 3.8-A resolution . Nature 354 , 278-284 .
155. Nakanishi , A. , Nakamura , A. , Liddington , R. , and Kasamatsu , H.2006) Identification of amino acid residues within simian virus 40 capsid proteins Vp1, Vp2, and Vp3 that are required for their interaction and for viral infection . J. Virol. 80 , 8891-8898 .
156. Kimchi-Sarfaty , C. , and Gottesman , M. M.2006) SV40 In vitro packaging:
157. Marple , A. H. , Gottesman , M. M. , and Kimchi-Sarfaty , C.2007) Efficient gene therapy delivery by SV40-derived vectors . In: Hefferon , K. , ed. Virus Expression Vectors . Woodbury, TriVandrum, India : Transworld Research Network .
158. Strayer , D. S.1999) Gene therapy using SV40-derived vectors: what does the future hold? J. Cell Physiol. 181 , 375-384 .
159. Kimchi-Sarfaty , C. , Garfield , S. , Nathan , A. S. , Saadia , A. , Carlos , C. , Dhanalakshmi , C. , and Gottesman , M. M.2004) The pathway of uptake of SV40 pseudovirions packaged in vitro: From MHC class I receptors to the nucleus . Ther. Mol. Biol. 8 , 439-450 .
160. Rund , D. , Dagan , M. , Dalyot-Herman , N. , Kimchi-Sarfaty , C. , Schoenlein , P. V. , Gottesman , M. M. , and Oppenheim , A.1998) Efficient transduction of human hematopoietic cells with the human multidrug resistance gene 1 via SV40 pseudovirions . Hum. Gene Ther. 9 , 649-657 .
161. Kimchi-Sarfaty , C. , Vieira , W. D. , Dodds , D. , Sherman , A. , Kreitman , R. J. , Shinar , S. , and Gottesman , M. M.2006) SV40 Pseudovirion gene delivery of a toxin to treat human adenocarcinomas in mice . Cancer Gene Ther. 13 , 648-657 .
162. Kodama , K. , Katayama , Y. , Shoji , Y. , and Nakashima , H.2006) The features and shortcomings for gene delivery of current non-viral carriers . Curr. Med. Chem. 13 , 2155-2161 .
163. Park , T. G. , Jeong , J. H. , and Kim , S. W.2006) Current status of polymeric gene delivery systems . Adv. Drug Deliv. Rev. 58 , 467-486 .
164. Gardlik , R. , Palffy , R. , Hodosy , J. , Lukacs , J. , Turna , J. , and Celec , P.2005) Vectors and delivery systems in gene therapy . Med. Sci. Monit. 11 , RA110 - 121 .
165. Smith , B. F. , Baker , H. J. , Curiel , D. T. , Jiang , W. , and Conry , R. M.1998) Humoral and cellular immune responses of dogs immunized with a nucleic acid vaccine encoding human carcinoembryonic antigen . Gene Ther. 5 , 865-868 .
166. Velaz-Faircloth , M. , Cobb , A. J. , Horstman , A. L. , Henry , S. C. , and Frothingham , R.1999) Protection against Mycobacterium avium by DNA vaccines expressing mycobacterial antigens as fusion proteins with green fluorescent protein . Infect. Immun. 67 , 4243-4250 .
167. Yu , W. H. , Kashani-Sabet , M. , Liggitt , D. , Moore , D. , Heath , T. D. , and Debs , R. J.1999) Topical gene delivery to murine skin . J. Invest. Dermatol. 112 , 370-375 .
168. Zhang , G. , Vargo , D. , Budker , V. , Armstrong , N. , Knechtle , S. , and Wolff , J. A.1997) Expression of naked plasmid DNA injected into the afferent and efferent vessels of rodent and dog livers . Hum. Gene Ther. 8 , 1763-1772 .
169. Yang , J. P. , and Huang , L.1996) Direct gene transfer to mouse melanoma by intratumor injection of free DNA . Gene Ther. 3 , 542-548 .
170. Tupin , E. , Poirier , B. , Bureau , M. F. , Khallou-Laschet , J. , Vranckx , R. , Caligiuri , G. , Gaston , A. T. , Duong Van Huyen , J. P. , Scherman , D. , Bariety , J. , Michel , J. B. , and Nicoletti , A.2003) Non-viral gene transfer of murine spleen cells achieved by in vivo electroporation . Gene Ther. 10 , 569-579 .
171. Gronevik , E. , Mathiesen , I. , and Lomo , T.2005) Early events of electroporationmediated intramuscular DNA vaccination potentiate Th1-directed immune responses . J. Gene Med. 7 , 1246-1254 .
172. Dileo , J. , Miller , T. E. , Jr. , Chesnoy , S. , and Huang , L.2003) Gene transfer to subdermal tissues via a new gene gun design . Hum. Gene Ther. 14 , 79-87 .
173. Matsuno , Y. , Iwata , H. , Umeda , Y. , Takagi , H. , Mori , Y. , Miyazaki , J. , Kosugi , A. , andHirose , H.2003) Nonviral gene gun mediated transfer into the beating heart . ASAIO J. 49 , 641-644 .
174. Yoshizawa , J. , Li , X. K. , Fujino , M. , Kimura , H. , Mizuno , R. , Hara , A. , Ashizuka , S. , Kanai , M. , Kuwashima , N. , Kurobe , M. , and Yamazaki , Y.2004) Successful in utero gene transfer using a gene gun in midgestational mouse fetuses . J. Pediatr. Surg. 39 , 81-84 .
175. Kodama , T. , Tan , P. H. , Offiah , I. , Partridge , T. , Cook , T. , George , A. J. , and Blomley , M. J.2005) Delivery of oligodeoxynucleotides into human saphenous veins and the adjunct effect of ultrasound and microbubbles . Ultrasound Med. Biol. 31 , 1683-1691 .
176. Haag , P. , Frauscher , F. , Gradl , J. , Seitz , A. , Schafer , G. , Lindner , J. R. , Klibanov , A. L. , Bartsch , G. , Klocker , H. , and Eder , I. E.2006) Microbubble-enhanced ultrasound to deliver an antisense oligodeoxynucleotide targeting the human androgen receptor into prostate tumours . J. Steroid Biochem. Mol. Biol. 102 , 103-113 .
177. Felgner , P. L. , Gadek , T. R. , Holm , M. , Roman , R. , Chan , H. W. , Wenz , M. , Northrop , J. P. , Ringold , G. M. , and Danielsen , M.1987) Lipofection: A highly efficient, lipidmediated DNA-transfection procedure . Proc. Natl. Acad. Sci. USA 84 , 7413-7417 .
178. Dass , C. R.2002) Biochemical and biophysical characteristics of lipoplexes pertinent to solid tumour gene therapy . Int. J. Pharm. 241 , 1-25 .
179. Zhdanov , R. I. , Podobed , O. V. , and Vlassov , V. V.2002) Cationic lipid-DNA complexeslipoplexes- for gene transfer and therapy . Bioelectrochemistry 58 , 53-64 .
180. Farhood , H. , Serbina , N. , and Huang , L.1995) The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer . Biochim. Biophys. Acta 1235 , 289-295 .
181. Hong , K. , Zheng , W. , Baker , A. , and Papahadjopoulos , D.1997) Stabilization of cationic liposome-plasmid DNA complexes by polyamines and poly(ethylene glycol)-phospholipid conjugates for efficient in vivo gene delivery . FEBS Lett. 400 , 233-237 .
182. Semple , S. C. , Chonn , A. , and Cullis , P. R.1996) Influence of cholesterol on the association of plasma proteins with liposomes . Biochemistry 35 , 2521-2525 .
183. Hattori , Y. , Kawakami , S. , Nakamura , K. , Yamashita , F. , and Hashida , M.2006) Efficient gene transfer into macrophages and dendritic cells by in vivo gene delivery with mannosylated lipoplex via the intraperitoneal route . J. Pharmacol. Exp. Ther. 318 , 828-834 .
184. Hattori , Y. , Suzuki , S. , Kawakami , S. , Yamashita , F. , and Hashida , M.2005) The role of dioleoylphosphatidylethanolamine (DOPE) in targeted gene delivery with mannosylated cationic liposomes via intravenous route . J. Control. Release 108 , 484-495 .
185. Kawakami , S. , Harada , A. , Sakanaka , K. , Nishida , K. , Nakamura , J. , Sakaeda , T. , Ichikawa , N. , Nakashima , M. , and Sasaki , H.2004) In vivo gene transfection via intravitreal injection of cationic liposome/plasmid DNA complexes in rabbits . Int. J. Pharm. 278 , 255-262 .
186. Monck , M. A. , Mori , A. , Lee , D. , Tam , P. , Wheeler , J. J. , Cullis , P. R. , and Scherrer , P.2000) Stabilized plasmid-lipid particles: Pharmacokinetics and plasmid delivery to distal tumors following intravenous injection . J. Drug. Target. 7 , 439-452 .
187. Ishiwata , H. , Suzuki , N. , Ando , S. , Kikuchi , H. , and Kitagawa , T.2000) Characteristics and biodistribution of cationic liposomes and their DNA complexes . J. Control. Rel. 69 , 139-148 .
188. Anwer , K. , Kao , G. , Proctor , B. , Anscombe , I. , Florack , V. , Earls , R. , Wilson , E. , McCreery , T. , Unger , E. , Rolland , A. , and SulliVan , S. M.2000) Ultrasound enhancement of cationic lipid-mediated gene transfer to primary tumors following systemic administration . Gene Ther. 7 , 1833-1839 .
189. Anwer , K. , Kao , G. , Proctor , B. , Rolland , A. , and SulliVan , S.2000) Optimization of cationic lipid/DNA complexes for systemic gene transfer to tumor lesions . J. Drug Target. 8 , 125-135 .
190. Wu , C. H. , Wilson , J. M. , and Wu , G. Y.1989) Targeting genes: Delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo . J. Biol. Chem. 264 , 16985-16987 .
191. Wagner , E. , Ogris , M. , and Zauner , W.1998) Polylysine-based transfection systems utilizing receptor-mediated delivery . Adv. Drug. Deliv. Rev. 30 , 97-113 .
192. Schaffer , D. V. , and Lauffenburger , D. A.1998) Optimization of cell surface binding enhances efficiency and specificity of molecular conjugate gene delivery . J. Biol. Chem. 273 , 28004-28009 .
193. Forrest , M. L. , and Pack , D. W.2002) On the kinetics of polyplex endocytic trafficking: Implications for gene delivery vector design . Mol. Ther. 6 , 57-66 .
194. Pouton , C. W. , Lucas , P. , Thomas , B. J. , Uduehi , A. N. , Milroy , D. A. , and Moss , S. H.1998) Polycation-DNA complexes for gene delivery: A comparison of the biopharmaceutical properties of cationic polypeptides and cationic lipids . J. Control Rel. 53 , 289-299 .
195. Kircheis , R. , Kichler , A. , Wallner , G. , Kursa , M. , Ogris , M. , Felzmann , T. , Buchberger , M. , and Wagner , E.1997) Coupling of cellbinding ligands to polyethylenimine for targeted gene delivery . Gene Ther. 4 , 409-418 .
196. Dash , P. R. , Read , M. L. , Barrett , L. B. , Wolfert , M. A. , and Seymour , L. W.1999) Factors affecting blood clearance and in vivo distribution of polyelectrolyte complexes for gene delivery . Gene Ther. 6 , 643-650 .
197. Nguyen , H. K. , Lemieux , P. , Vinogradov , S. V. , Gebhart , C. L. , Guerin , N. , Paradis , G. , Bronich , T. K. , Alakhov , V. Y. , and Kabanov , A. V.2000) Evaluation of polyether-polyethyleneimine graft copolymers as gene transfer agents . Gene Ther. 7 , 126-138 .
198. Hudde , T. , Rayner , S. A. , Comer , R. M. , Weber , M. , Isaacs , J. D. , Waldmann , H. , Larkin , D. F. , and George , A. J.1999) Activated polyamidoamine dendrimers, a nonviral vector for gene transfer to the corneal endothelium . Gene Ther. 6 , 939-943 .
199. Dennig , J. , and Duncan , E.2002) Gene transfer into eukaryotic cells using activated polyamidoamine dendrimers . J. Biotechnol. 90 , 339-347 .
200. Truong-Le , V. L. , August , J. T. , and Leong , K. W.1998) Controlled gene delivery by DNA-gelatin nanospheres . Hum. Gene Ther. 9 , 1709-1717 .
201. Van de Wetering , P. , Moret , E. E. , Schuurmans-Nieuwenbroek , N. M. , Van Steenbergen , M. J. , and Hennink , W. E.1999) Structure-activity relationships of water-soluble cationic methacrylate/methacrylamide polymers for nonviral gene delivery . Bioconjug. Chem. 10 , 589-597 .
202. Van der Woude , I. , Wagenaar , A. , Meekel , A. A. , ter Beest , M. B. , Ruiters , M. H. , Engberts , J. B. , and Hoekstra , D.1997) Novel pyridinium surfactants for efficient, nontoxic in vitro gene delivery . Proc. Natl. Acad. Sci. USA 94 , 1160-1165 .
203. Mansouri , S. , Lavigne , P. , Corsi , K. , Benderdour , M. , Beaumont , E. , and Fernandes , J. C.2004) Chitosan-DNA nanoparticles as non-viral vectors in gene therapy: Strategies to improve transfection efficacy . Eur. J. Pharm. Biopharm. 57 , 1-8 .
204. Chen , J. , Yang , W. L. , Li , G. , Qian , J. , Xue , J. L. , Fu , S. K. , and Lu , D. R.2004) Transfection of mEpo gene to intestinal epithelium in vivo mediated by oral delivery of chitosan- DNA nanoparticles . World J. Gastroenterol. 10 , 112-116 .
205. Roy , K. , Mao , H. Q. , Huang , S. K. , and Leong , K. W.1999) Oral gene delivery with chitosan - DNA nanoparticles generates immunologic protection in a murine model of peanut allergy . Nat. Med. 5 , 387-391 .
206. Gomez-Sebastian , S. , Gimenez-Cassina , A. , Diaz-Nido , J. , Lim , F. , and Wade-Martins , R.2007) Infectious delivery and expression of a 135 kb human FRDA genomic DNA locus complements Friedreich's ataxia deficiency in human cells . Mol. Ther. 15 , 248-254 .
207. Hibbitt , O. C. , Harbottle , R. P. , Waddington , S. N. , Bursill , C. A. , Coutelle , C. , Channon , K. M. , and Wade-Martins , R.2007) Delivery and long-term expression of a 135 kb LDLR genomic DNA locus in vivo by hydrodynamic tail vein injection . J. Gene Med. 9 , 488-497 .
208. Kanzaki , S. , Beyer , L. , Karolyi , I. J. , Dolan , D. F. , Fang , Q. , Probst , F. J. , Camper , S. A. , and Raphael , Y.2006) Transgene correction maintains normal cochlear structure and function in 6-month-old Myo15a mutant mice . Hear. Res. 214 , 37-44 .
209. Lim , F. , Palomo , G. M. , Mauritz , C. , Gimenez- Cassina , A. , Illana , B. , Wandosell , F. , and Diaz-Nido , J.2007) Functional recovery in a Friedreich's ataxia mouse model by frataxin gene transfer using an HSV-1 amplicon vector . Mol. Ther. 15 , 1072-1078 .
210. Suda , T. , Katoh , M. , Hiratsuka , M. , Takiguchi , M. , Kazuki , Y. , Inoue , T. , and Oshimura , M.2006) Heat-regulated production and secretion of insulin from a human artificial chromosome vector . Biochem. Biophys. Res. Commun. 340 , 1053-1061 .
211. Paglia , P. , Medina , E. , Arioli , I. , Guzman , C. A. , and Colombo , M. P.1998) Gene transfer in dendritic cells, induced by oral DNA vaccination with Salmonella typhimurium, results in protective immunity against a murine fibrosarcoma . Blood 92 , 3172-3176 .
212. Sizemore , D. R. , Branstrom , A. A. , and Sadoff , J. C.1995) Attenuated Shigella as a DNA delivery vehicle for DNA-mediated immunization . Science 270 , 299-302 .
213. Vassaux , G. , Nitcheu , J. , Jezzard , S. , and Lemoine , N. R.2006) Bacterial gene therapy strategies . J. Pathol. 208 , 290-298 .
214. Palffy , R. , Gardlik , R. , Hodosy , J. , Behuliak , M. , Resko , P. , RadVansky , J. , and Celec , P.2006) Bacteria in gene therapy: Bactofection versus alternative gene therapy . Gene Ther. 13 , 101-105 .
215. Al-Mariri , A. , Tibor , A. , Lestrate , P. , Mertens , P. , De Bolle , X. , and Letesson , J. J.2002) Yersinia enterocolitica as a vehicle for a nakedDNA vaccine encoding Brucella abortus bacterioferritin or P39 antigen . Infect. Immun. 70 , 1915-1923 .
216. Woo , P. C. , Wong , L. P. , Zheng , B. J. , and Yuen , K. Y.2001) Unique immunogenicity of hepatitis B virus DNA vaccine presented by live-attenuated Salmonella typhimurium . Vaccine 19 , 2945-2954 .
217. Flo , J. , Tisminetzky , S. , and Baralle , F.2001) Oral transgene vaccination mediated by attenuated Salmonellae is an effective method to prevent Herpes simplex virus-2 induced disease in mice . Vaccine 19 , 1772-1782 .
218. Niethammer , A. G. , Xiang , R. , Becker , J. C. , Wodrich , H. , Pertl , U. , Karsten , G. , Eliceiri , B. P. , and Reisfeld , R. A.2002) A DNA vaccine against VEGF receptor 2 prevents effective angiogenesis and inhibits tumor growth . Nat. Med. 8 , 1369-1375 .
219. Feng , K. K. , Zhao , H. Y. , Qiu , H. , Liu , J. X. , and Chen , J.2005) Combined therapy with flk1-based DNA vaccine and interleukin-12 results in enhanced antiangiogenic and antitumor effects . Cancer Lett. 221 , 41-47 .
220. Theys , J. , Landuyt , A. W. , Nuyts , S. , Van Mellaert , L. , Lambin , P. , and Anne , J.2001) Clostridium as a tumor-specific delivery system of therapeutic proteins . Cancer Detect. Prev. 25 , 548-557 .
221. Fujimori , M. , Amano , J. , and Taniguchi , S.2002) The genus Bifidobacterium for cancer gene therapy . Curr. Opin. Drug Discov. Dev. 5 , 200-203 .
222. Luo , X. , Li , Z. , Lin , S. , Le , T. , Ittensohn , M. , Bermudes , D. , Runyab , J. D. , Shen , S. Y. , Chen , J. , King , I. C. , and Zheng , L. M.2001) Antitumor effect of VNP20009, an attenuated Salmonella, in murine tumor models . Oncol. Res. 12 , 501-508 .
223. Nemunaitis , J. , Cunningham , C. , Senzer , N. , Kuhn , J. , Cramm , J. , Litz , C. , Cavagnolo , R. , Cahill , A. , Clairmont , C. , and Sznol , M.2003) Pilot trial of genetically modified, attenuated Salmonella expressing the E. coli cytosine deaminase gene in refractory cancer patients . Cancer Gene Ther. 10 , 737-744 .
224. Nuyts , S. , Van Mellaert , L. , Theys , J. , Landuyt , W. , Lambin , P. , and Anne , J.2001) The use of radiation-induced bacterial promoters in anaerobic conditions: A means to control gene expression in clostridium- mediated therapy for cancer . Radiat. Res. 155 , 716-723 .
225. Kumar , S. , Walia , V. , Ray , M. , and Elble , R. C.2007) p53 in breast cancer: mutation and countermeasures . Front Biosci. 12 , 4168-4178 .
226. Cai , D. W. , Mukhopadhyay , T. , Liu , Y. , Fujiwara , T. , and Roth , J. A.1993) Stable expression of the wild-type p53 gene in human lung cancer cells after retrovirusmediated gene transfer . Hum. Gene Ther. 4 , 617-624 .
227. Watts , G. S. , Oshiro , M. M. , Junk , D. J. , Wozniak , R. J. , Watterson , S. , Domann , F. E. , and Futscher , B. W.2004) The acetyltransferase p300/CBP-associated factor is a p53 target gene in breast tumor cells . Neoplasia 6 , 187-194 .
228. Eastham , J. A. , Grafton , W. , Martin , C. M. , and Williams , B. J.2000) Suppression of primary tumor growth and the progression to metastasis with p53 adenovirus in human prostate cancer . J. Urol. 164 , 814-819 .
229. Giuliano , M. , Catalano , A. , Strizzi , L. , Vianale , G. , Capogrossi , M. , and Procopio , A.2000) Adenovirus-mediated wild-type p53 overexpression reverts tumourigenicity of human mesothelioma cells . Int. J. Mol. Med. 5 , 591-596 .
230. Su , S. , Watanabe , A. , Yamamoto , M. , Nakajima , E. , Miyake , K. , and Shimada , T.2006) Mutations in p53 cDNA sequence introduced by retroviral vector . Biochem. Biophys. Res. Commun. 340 , 567-572 .
231. Guo , J. , and Xin , H.2006) Chinese gene therapy. Splicing out the West? Science 314 , 1232-1235 .
232. Gabrilovich , D. I.2006) INGN 201 (Advexin): Adenoviral p53 gene therapy for cancer . Expert Opin. Biol. Ther. 6 , 823-832 .
233. Pisters , L. L. , Pettaway , C. A. , Troncoso , P. , McDonnell , T. J. , Stephens , L. C. , Wood , C. G. , Do , K. A. , Brisbay , S. M. , Wang , X. , Hossan , E. A. , EVans , R. B. , Soto , C. , Jacobson , M. G. , Parker , K. , Merritt , J. A. , Steiner , M. S. , and Logothetis , C. J.2004) Evidence that transfer of functional p53 protein results in increased apoptosis in prostate cancer . Clin. Cancer Res. 10 , 2587-2593 .
234. Swisher , S. G. , Roth , J. A. , Komaki , R. , Gu , J. , Lee , J. J. , Hicks , M. , Ro , J. Y. , Hong , W. K. , Merritt , J. A. , Ahrar , K. , Atkinson , N. E. , Correa , A. M. , Dolormente , M. , Dreiling , L. , El-Naggar , A. K. , Fossella , F. , Francisco , R. , Glisson , B. , Grammer , S. , Herbst , R. , Huaringa , A. , Kemp , B. , Khuri , F. R. , Kurie , J. M. , Liao , Z. , McDonnell , T. J. , Morice , R. , Morello , F. , Munden , R. , Papadimitrakopoulou , V. , Pisters , K. M. , Putnam , J. B. , Jr. , Sarabia , A. J. , Shelton , T. , Stevens , C. , Shin , D. M. , Smythe , W. R. , Vaporciyan , A. A. , Walsh , G. L. , and Yin , M.2003) 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. 9 , 93-101 .
235. Lockley , M. , Fernandez , M. , Wang , Y. , Li , N. F. , Conroy , S. , Lemoine , N. , and McNeish , I.2006) Activity of the adenoviral E1A deletion mutant dl922-947 in ovarian cancer: Comparison with E1A wild-type viruses, bioluminescence monitoring, and intraperitoneal delivery in icodextrin . Cancer Res. 66 , 989-998 .
236. Zabarovsky , E. R. , Lerman , M. I. , and Minna , J. D.2002) Tumor suppressor genes on chromosome 3p involved in the pathogenesis of lung and other cancers . Oncogene 21 , 6915-6935 .
237. Ji , L. , Nishizaki , M. , Gao , B. , Burbee , D. , Kondo , M. , Kamibayashi , C. , Xu , K. , Yen , N. , Atkinson , E. N. , Fang , B. , Lerman , M. I. , Roth , J. A. , and Minna , J. D.2002) Expression of several genes in the human chromosome 3p21.3 homozygous deletion region by an adenovirus vector results in tumor suppressor activities in vitro and in vivo . Cancer Res. 62 , 2715-2720 .
238. Deng , W. G. , Nishizaki , M. , Fang , B. , Roth , J. A. , and Ji , L.2007) Induction of apoptosis by tumor suppressor FHIT via death receptor signaling pathway in human lung cancer cells . Biochem. Biophys. Res. Commun. 355 , 993-999 .
239. Guo , W. , Zhu , H. , Zhang , L. , Davis , J. , Teraishi , F. , Roth , J. A. , Stephens , C. , Fueyo , J. , Jiang , H. , Conrad , C. , and Fang , B.2006) Combination effect of oncolytic adenovirotherapy and TRAIL gene therapy in syngeneic murine breast cancer models . Cancer Gene Ther. 13 , 82-90 .
240. Fillat , C. , Carrio , M. , Cascante , A. , and Sangro , B.2003) Suicide gene therapy mediated by the Herpes Simplex virus thymidine kinase gene/Ganciclovir system: Fifteen years of application . Curr. Gene Ther. 3 , 13-26 .
241. Negroni , L. , Samson , M. , Guigonis , J. M. , Rossi , B. , Pierrefite-Carle , V. , and Baudoin , C.2007) Treatment of colon cancer cells using the cytosine deaminase/5-fluorocytosine suicide system induces apoptosis, modulation of the proteome, and Hsp90beta phosphorylation . Mol. Cancer Ther. 6 , 2747-2756 .
242. Braybrooke , J. P. , Slade , A. , Deplanque , G. , Harrop , R. , Madhusudan , S. , Forster , M. D. , Gibson , R. , Makris , A. , Talbot , D. C. , Steiner , J. , White , L. , Kan , O. , Naylor , S. , Carroll , M. W. , Kingsman , S. M. , and Harris , A. L.2005) Phase I study of MetXia-P450 gene therapy and oral cyclophosphamide for patients with adVanced breast cancer or melanoma . Clin. Cancer Res. 11 , 1512-1520 .
243. Dachs , G. U. , Tupper , J. , and Tozer , G. M.2005) From bench to bedside for genedirected enzyme prodrug therapy of cancer . Anticancer Drugs 16 , 349-359 .
244. Mathis , J. M. , Williams , B. J. , Sibley , D. A. , Carroll , J. L. , Li , J. , Odaka , Y. , Barlow , S. , Nathan , C. O. , Li , B. D. , and DeBenedetti , A.2006) Cancer-specific targeting of an adenovirus- delivered herpes simplex virus thymidine kinase suicide gene using translational control . J. Gene Med. 8 , 1105-1120 .
245. Kagaya , T. , Nakamoto , Y. , Sakai , Y. , Tsuchiyama , T. , Yagita , H. , Mukaida , N. , and Kaneko , S.2006) Monocyte chemoattractant protein-1 gene delivery enhances antitumor effects of herpes simplex virus thymidine kinase/ganciclovir system in a model of colon cancer . Cancer Gene Ther. 13 , 357-366 .
246. Zhan , J. , Gao , Y. , Wang , W. , Shen , A. , Aspelund , A. , Young , M. , Laquerre , S. , Post , L. , and Shen , Y.2005) Tumor-specific intravenous gene delivery using oncolytic adenoviruses . Cancer Gene Ther. 12 , 19-25 .
247. Liu , Y. , and Deisseroth , A.2006) Oncolytic adenoviral vector carrying the cytosine deaminase gene for melanoma gene therapy . Cancer Gene Ther. 13 , 845-855 .
248. Wang , J. , Lu , X. X. , Chen , D. Z. , Li , S. F. , and Zhang , L. S.2004) Herpes simplex virus thymidine kinase and ganciclovir suicide gene therapy for human pancreatic cancer . World J. Gastroenterol. 10 , 400-403 .
249. Fogar , P. , Greco , E. , Basso , D. , Habeler , W. , Navaglia , F. , Zambon , C. F. , Tormen , D. , Gallo , N. , Cecchetto , A. , Plebani , M. , and Pedrazzoli , S.2003) Suicide gene therapy with HSV-TK in pancreatic cancer has no effect in vivo in a mouse model . Eur. J. Surg. Oncol. 29 , 721-730 .
250. Shand , N. , Weber , F. , Mariani , L. , Bernstein , M. , Gianella-Borradori , A. , Long , Z. , Sorensen , A. G. , and Barbier , N.1999) 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. 10 , 2325-2335 .
251. Rainov , N. G.2000) 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. 11 , 2389-2401 .
252. Kikuchi , E. , Menendez , S. , Ozu , C. , Ohori , M. , Cordon-Cardo , C. , Logg , C. R. , Kasahara , N. , and Bochner , B. H.2007) Deliveryof replication-competent retrovirus expressing Escherichia coli purine nucleoside phosphorylase increases the metabolism of the prodrug, fludarabine phosphate and suppresses the growth of bladder tumor xenografts . Cancer Gene Ther. 14 , 279-286 .
253. Stover , M. L. , Wang , C. K. , McKinstry , M. B. , Kalajzic , I. , Gronowicz , G. , Clark , S. H. , Rowe , D. W. , and Lichtler , A. C.2001) Bone-directed expression of Col1a1 promoter- driven self-inactivating retroviral vector in bone marrow cells and transgenic mice . Mol. Ther. 3 , 543-550 .
254. Vile , R. G. , Diaz , R. M. , Miller , N. , Mitchell , S. , Tuszyanski , A. , and Russell , S. J.1995) Tissue-specific gene expression from Mo- MLV retroviral vectors with hybrid LTRs containing the murine tyrosinase enhancer/ promoter . Virology 214 , 307-313 .
255. Engels , B. , Cam , H. , Schuler , T. , Indraccolo , S. , Gladow , M. , Baum , C. , Blankenstein , T. , and Uckert , W.2003) Retroviral vectors for high-level transgene expression in T lymphocytes . Hum. Gene Ther. 14 , 1155-1168 .
256. Wang , J. M. , Hou , J. , Qiu , X. F. , Kurachi , K. , and Xue , J. L.2004) Hybrid retroviral vector with MCK enhancers inserted in LTR for stable and specific expression of human factor IX in skeletal muscle . Chin. Med. J. Engl ) 117 , 893-898 .
257. Woo , Y. , Adusumilli , P. S. , and Fong , Y.2006) AdVances in oncolytic viral therapy . Curr. Opin. Investig. Drugs 7 , 549-559 .
258. Crompton , A. M. , and Kirn , D. H.2007) From ONYX-015 to armed vaccinia viruses: The education and evolution of oncolytic virus development . Curr. Cancer Drug Targets 7 , 133-139 .
259. Barzon , L. , Zanusso , M. , Colombo , F. , and Palu , G.2006) Clinical trials of gene therapy, virotherapy, and immunotherapy for malignant gliomas . Cancer Gene Ther. 13 , 539-554 .
260. Hotte , S. J. , Lorence , R. M. , Hirte , H. W. , Polawski , S. R. , Bamat , M. K. , O'Neil , J. D. , Roberts , M. S. , Groene , W. S. , and Major , P. P.2007) An optimized clinical regimen for the oncolytic virus PV701 . Clin. Cancer Res. 13 , 977-985 .
261. Laurie , S. A. , Bell , J. C. , Atkins , H. L. , Roach , J. , Bamat , M. K. , O'Neil , J. D. , Roberts , M. S. , Groene , W. S. , and Lorence , R. M.2006) phase 1 clinical study of intravenous administration of PV701, an oncolytic virus, using two-step desensitization . Clin. Cancer Res. 12 , 2555-2562 .
262. Lorence , R. M. , Roberts , M. S. , O'Neil , J. D. , Groene , W. S. , Miller , J. A. , Mueller , S. N. , and Bamat , M. K.2007) Phase 1 clinical experience using intravenous administration of PV701, an oncolytic Newcastle disease virus . Curr. Cancer Drug Targets 7 , 157-167 .
263. Pecora , A. L. , Rizvi , N. , Cohen , G. I. , Meropol , N. J. , Sterman , D. , Marshall , J. L. , Goldberg , S. , Gross , P. , O'Neil , J. D. , Groene , W. S. , Roberts , M. S. , Rabin , H. , Bamat , M. K. , and Lorence , R. M.2002) Phase I trial of intravenous administration of PV701, an oncolytic virus, in patients with adVanced solid cancers . J. Clin. Oncol. 20 , 2251-2266 .
264. Marcato , P. , Shmulevitz , M. , Pan , D. , Stoltz , D. , and Lee , P. W.2007) Ras transformation mediates reovirus oncolysis by enhancing virus uncoating, particle infectivity, and apoptosis-dependent release . Mol. Ther. 15 , 1522-1530 .
265. Hallahan , D. E. , Beckett , M. A. , Kufe , D. , and Weichselbaum , R. R.1990) The interaction between recombinant human tumor necrosis factor and radiation in 13 human tumor cell lines . Int. J. Radiat. Oncol. Biol. Phys. 19 , 69-74 .
266. Hallahan , D. E. , Spriggs , D. R. , Beckett , M. A. , Kufe , D. W. , and Weichselbaum , R. R.1989) Increased tumor necrosis factor alpha mRNA after cellular exposure to ionizing radiation . Proc. Natl. Acad. Sci. USA 86 , 10104-10107 .
267. Datta , R. , Rubin , E. , Sukhatme , V. , Qureshi , S. , Hallahan , D. , Weichselbaum , R. R. , and Kufe , D. W.1992) Ionizing radiation activates transcription of the EGR1 gene via CArG elements . Proc. Natl. Acad. Sci. USA 89 , 10149-10153 .
268. Mezhir , J. J. , Smith , K. D. , Posner , M. C. , Senzer , N. , Yamini , B. , Kufe , D. W. , and Weichselbaum , R. R.2006) Ionizing radiation: A genetic switch for cancer therapy . Cancer Gene Ther. 13 , 1-6 .
269. Weichselbaum , R. R. , Hallahan , D. E. , Beckett , M. A. , Mauceri , H. J. , Lee , H. , Sukhatme , V. P. , and Kufe , D. W.1994) Gene therapy targeted by radiation preferentially radiosensitizes tumor cells . Cancer Res. 54 , 4266-4269 .
270. Seung , L. P. , Mauceri , H. J. , Beckett , M. A. , Hallahan , D. E. , Hellman , S. , and Weichselbaum , R. R.1995) Genetic radiotherapy overcomes tumor resistance to cytotoxic agents . Cancer Res. 55 , 5561-5565 .
271. Chung , T. D. , Mauceri , H. J. , Hallahan , D. E. , Yu , J. J. , Chung , S. , Grdina , W. L. , Yajnik , S. , Kufe , D. W. , and Weichselbaum , R. R.1998) Tumor necrosis factor-alpha-based gene therapy enhances radiation cytotoxicity inhuman prostate cancer . Cancer Gene Ther. 5 , 344-349 .
272. Staba , M. J. , Mauceri , H. J. , Kufe , D. W. , Hallahan , D. E. , and Weichselbaum , R. R.1998) Adenoviral TNF-alpha gene therapyand radiation damage tumor vasculature ina human malignant glioma xenograft . Gene Ther. 5 , 293-300 .
273. Senzer , N. , Mani , S. , Rosemurgy , A. , Nemunaitis , J. , Cunningham , C. , Guha , C. , Bayol , N. , Gillen , M. , Chu , K. , Rasmussen , C. ,Rasmussen , H. , Kufe , D. , Weichselbaum , R. , and Hanna , N.2004) TNFerade biologic, an adenovector with a radiation-inducible promoter, carrying the human tumor necrosis factor alpha gene: A phase I study in patients with solid tumors . J. Clin. Oncol. 22 , 592-601 .
274. Mundt , A. J. , Vijayakumar , S. , Nemunaitis , J. , Sandler , A. , Schwartz , H. , Hanna , N. , Peabody , T. , Senzer , N. , Chu , K. , Rasmussen , C. S. , Kessler , P. D. , Rasmussen , H. S. , Warso , M. , Kufe , D. W. , Gupta , T. D. , and Weichselbaum , R. R.2004) A Phase trial of TNFerade biologic in patients with soft tissue sarcoma in the extremities . Clin. Cancer Res. 10 , 5747-5753 .
275. Mezhir , J. J. , Schmidt , H. , Yamini , B. , Senzer , N. N. , Posner , M. C. , Kufe , D. W. , and Weichselbaum , R. R.2005) Chemo-inducible gene therapy . Anticancer Drugs 16 , 1053-1058 .
276. Lopez , C. A. , Kimchi , E. T. , Mauceri , H. J. ,Park , J. O. , Mehta , N. , Murphy , K. T. , Beckett , M. A. , Hellman , S. , Posner , M. C. , Kufe , D. W. , and Weichselbaum , R. R.2004) Chemoinducible gene therapy: A strategy toenhance doxorubicin antitumor activity . Mol. Cancer Ther. 3 , 1167-1175 .
277. Park , J. O. , Lopez , C. A. , Gupta , V. K. , Brown , C. K. , Mauceri , H. J. , Darga , T. E. , Manan , A. , Hellman , S. , Posner , M. C. , Kufe , D. W. , and Weichselbaum , R. R.2002) Transcriptional control of viral gene therapy by cisplatin . J. Clin. Invest. 110 , 403-410 .
278. Yamini , B. , Yu , X. , Gillespie , G. Y. , Kufe , D. W. , and Weichselbaum , R. R.2004) Transcriptional targeting of adenovirally delivered tumor necrosis factor alpha by temozolomide in experimental glioblastoma . Cancer Res. 64 , 6381-6384 .
279. Yamini , B. , Yu , X. , Pytel , P. , Galanopoulos , N. , Rawlani , V. , Veerapong , J. , Bickenbach , K. , and Weichselbaum , R. R.2007) Adenovirally Delivered Tumor Necrosis Factor- {alpha} Improves the Antiglioma Efficacy of Concomitant Radiation and Temozolomide Therapy . Clin. Cancer Res. 13 , 6217-6223 .
280. Walther , W. , Stein , U. , Fichtner , I. , Alexander , M. , Shoemaker , R. H. , and Schlag , P. M.2000) Mdr1 promoter-driven tumor necrosis factor-alpha expression for a chemotherapy- controllable combined in vivo gene therapy and chemotherapy of tumors . Cancer Gene Ther. 7 , 893-900 .
281. Walther , W. , Stein , U. , and Schlag , P. M.2002) Use of the human MDR1 promoter for heat-inducible expression of therapeutic genes . Int. J. Cancer 98 , 291-296 .
282. Walther , W. , Arlt , F. , Fichtner , I. , Aumann , J. , Stein , U. , and Schlag , P. M.2007) Heatinducible in vivo gene therapy of colon carcinoma by human mdr1 promoter-regulated tumor necrosis factor-alpha expression . Mol. Cancer Ther. 6 , 236-243 .
283. Devi , G. R.2006) siRNA-based approaches in cancer therapy . Cancer Gene Ther. 13 , 819-829 .
284. Karagiannis , T. C. , and El-Osta , A.2005) RNA interference and potential therapeutic applications of short interfering RNAs . Cancer Gene Ther. 12 , 787-795 .
285. O'Reilly , M. S. , Holmgren , L. , Shing , Y. , Chen , C. , Rosenthal , R. A. , Moses , M. , Lane , W. S. , Cao , Y. , Sage , E. H. , and Folkman , J.1994) Angiostatin: A novel angiogenesis inhibitor that mediates the suppression of metastases by a Lewis lung carcinoma . Cell 79 , 315-328 .
286. O'Reilly , M. S. , Boehm , T. , Shing , Y. , Fukai , N. , Vasios , G. , Lane , W. S. , Flynn , E. , Birkhead , J. R. , Olsen , B. R. , and Folkman , J.1997) Endostatin: An endogenous inhibitor of angiogenesis and tumor growth . Cell 88 , 277-285 .
287. Dawson , D. W. , Volpert , O. V. , Gillis , P. , Crawford , S. E. , Xu , H. , Benedict , W. , and Bouck , N. P.1999) Pigment epitheliumderived factor: A potent inhibitor of angiogenesis . Science 285 , 245-248 .
288. Millauer , B. , Longhi , M. P. , Plate , K. H. , Shawver , L. K. , Risau , W. , Ullrich , A. , and Strawn , L. M.1996) Dominant-negative inhibition of Flk-1 suppresses the growth of many tumor types in vivo . Cancer Res. 56 , 1615-1620 .
289. Mae , M. , O'Connor , T. P. , and Crystal , R. G.2005) Gene transfer of the vascular endothelial growth factor receptor flt-1 suppresses pulmonary metastasis associated with lung growth . Am. J. Respir. Cell. Mol. Biol. 33 , 629-635 .
290. Streck , C. J. , Zhou , J. , Ng , C. Y. , Zhang , Y. , Nathwani , A. C. , and Davidoff , A. M.2004) Longterm recombinant adeno-associated, virus-mediated, liver-generated expression of an angiogenesis inhibitor improves survival in mice with disseminated neuroblastoma . J. Am. Coll. Surg. 199 , 78-86 .
291. Ferrara , N. , Hillan , K. J. , Gerber , H. P. , and Novotny , W.2004) Discovery and development of bevacizumab, an anti-VEGF antibody for treating cancer . Nat. Rev. Drug Discov. 3 , 391-400 .
292. Drevs , J. , Hofmann , I. , Hugenschmidt , H. , Wittig , C. , Madjar , H. , Muller , M. , Wood , J. , Martiny-Baron , G. , Unger , C. , and Marme , D.2000) Effects of PTK787/ZK 222584, a specific inhibitor of vascular endothelial growth factor receptor tyrosine kinases, on primary tumor, metastasis, vessel density, and blood flow in a murine renal cell carcinoma model . Cancer Res. 60 , 4819-4824 .
293. Hurwitz , H. , Fehrenbacher , L. , Novotny , W. , Cartwright , T. , Hainsworth , J. , Heim , W. , Berlin , J. , Baron , A. , Griffing , S. , Holmgren , E. , Ferrara , N. , Fyfe , G. , Rogers , B. , Ross , R. , and Kabbinavar , F.2004) Bevacizumab plus irinotecan, fluorouracil, and leucovorin for metastatic colorectal cancer . N. Engl. J. Med. 350 , 2335-2342 .
294. Giantonio , B. J. , Catalano , P. J. , Meropol , N. J. , O'Dwyer , P. J. , Mitchell , E. P. , Alberts , S. R. , Schwartz , M. A. , and Benson , A. B. , 3rd (2007) Bevacizumab in combination with oxaliplatin, fluorouracil, and leucovorin (FOLFOX4) for previously treated metastatic colorectal cancer: Results from the Eastern Cooperative Oncology Group Study E3200 . J. Clin. Oncol. 25 , 1539-1544 .
295. Cohen , M. H. , Gootenberg , J. , Keegan , P. , and Pazdur , R.2007) FDA drug approval summary: Bevacizumab plus FOLFOX4 as second-line treatment of colorectal cancer . Oncologist 12 , 356-361 .
296. Cohen , M. H. , Gootenberg , J. , Keegan , P. , and Pazdur , R.2007) FDA drug approval summary: Bevacizumab (Avastin) plus Carboplatin and Paclitaxel as first-line treatment of adVanced/metastatic recurrent nonsquamous non-small cell lung cancer . Oncologist 12 , 713-718 .
297. O'Reilly , M. S. , Holmgren , L. , Chen , C. , and Folkman , J.1996) Angiostatin induces and sustains dormancy of human primary tumors in mice . Nat. Med. 2 , 689-692 .
298. Subramanian , I. V. , Ghebre , R. , and Ramakrishnan , S.2005) Adeno-associated virusmediated delivery of a mutant endostatin suppresses ovarian carcinoma growth in mice . Gene Ther. 12 , 30-38 .
299. Subramanian , I. V. , Bui Nguyen , T. M. , Truskinovsky , A. M. , Tolar , J. , Blazar , B. R. , and Ramakrishnan , S.2006) Adeno-associated virus-mediated delivery of a mutant endostatin in combination with carboplatin treatment inhibits orthotopic growth of ovarian cancer and improves long-term survival . Cancer Res. 66 , 4319-4328 .
300. Zhang , X. , Xu , J. , Lawler , J. , Terwilliger , E. , and Parangi , S.2007) Adeno-associated virus-mediated antiangiogenic gene therapy with thrombospondin-1 type 1 repeats and endostatin . Clin. Cancer Res. 13 , 3968-3976 .
301. Albini , A. , Marchisone , C. , Del Grosso , F. , Benelli , R. , Masiello , L. , Tacchetti , C. , Bono , M. , Ferrantini , M. , Rozera , C. , Truini , M. , Belardelli , F. , Santi , L. , and Noonan , D. M.2000) Inhibition of angiogenesis and vascular tumor growth by interferon-producing cells: A gene therapy approach . Am. J. Pathol. 156 , 1381-1393 .
302. Sanches , R. , Kuiper , M. , Penault-Llorca , F. , Aunoble , B. , D'Incan , C. , and Bignon , Y. J.2000) Antitumoral effect of interleukin- 12-secreting fibroblasts in a mouse model of ovarian cancer: Implications for the use of ovarian cancer biopsy-derived fibroblasts as a vehicle for regional gene therapy . Cancer Gene Ther. 7 , 707-720 .
303. Saleh , M. , Jonas , N. K. , Wiegmans , A. , and Stylli , S. S.2000) The treatment of established intracranial tumors by in situ retroviral IFNgamma transfer . Gene Ther. 7 , 1715-1724 .
304. Xue , S. A. , and Stauss , H. J.2007) Enhancing immune responses for cancer therapy . Cell. Mol. Immunol. 4 , 173-184 .
305. Sobol , R. E.2006) The rationale for prophylactic cancer vaccines and need for a paradigm shift . Cancer Gene Ther. 13 , 725-731 .
306. Thomas , S. , Hart , D. P. , Xue , S. A. , Cesco- Gaspere , M. , and Stauss , H. J.2007) T-cell receptor gene therapy for cancer: The progress to date and future objectives . Expert Opin. Biol. Ther. 7 , 1207-1218 .
307. Akbulut , H. , Tang , Y. , Akbulut , K. G. , Maynard , J. , Zhang , L. , and Deisseroth , A.2006) Antitumor immune response induced by i.t. injection of vector-activated dendritic cells and chemotherapy suppresses metastatic breast cancer . Mol. Cancer Ther. 5 , 1975-1985 .
308. Ribas , A. , Butterfield , L. H. , Glaspy , J. A. , and Economou , J. S.2003) Current developments in cancer vaccines and cellular immunotherapy . J. Clin. Oncol. 21 , 2415-2432 .
309. Vetter , K. M. , and Geller , S. E.2007) Moving forward: human papillomavirus vaccination and the prevention of cervical cancer . J. Womens Health ( Larchmt ) 16 , 1258-1268 .
310. Bosch , F. X. , Lorincz , A. , Munoz , N. , Meijer , C. J. , and Shah , K. V.2002) The causal relation between human papillomavirus and cervical cancer . J. Clin. Pathol. 55 , 244-265 .
311. Rosenberg , S. A. , and Dudley , M. E.2004) Cancer regression in patients with metastatic melanoma after the transfer of autologous antitumor lymphocytes . Proc. Natl. Acad. Sci. USA 101 (Suppl 2) , 14639-14645 .
312. Morgan , R. A. , Dudley , M. E. , Wunderlich , J. R. , Hughes , M. S. , Yang , J. C. , Sherry , R. M. , Royal , R. E. , Topalian , S. L. , Kammula , U. S. , Restifo , N. P. , Zheng , Z. , Nahvi , A. , de Vries , C. R. , Rogers-Freezer , L. J. , Mavroukakis , S. A. , and Rosenberg , S. A.2006) Cancer regression in patients after transfer of genetically engineered lymphocytes . Science 314 , 126-129 .
313. Gillet , J. P. , Kimchi-Sarfaty , C. , Shinar , S. , Licht , T. , Lee , C. , Hafkemeyer , P. , Hrycyna , C. , Pastan , I. , and Gottesman , M.2008) Selectable markers for gene therapy . In: Templeton , N. , ed. Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, 3rd edition . Boca Raton, FL : Taylor & Francis .
314. Flasshove , M. , Banerjee , D. , Leonard , J. P. , Mineishi , S. , Li , M. X. , Bertino , J. R. , and Moore , M. A.1998) Retroviral transduction of human CD34 + umbilical cord blood progenitor cells with a mutated dihydrofolate reductase cDNA . Hum. Gene Ther. 9 , 63-71 .
315. Wang , G. , Weiss , C. , Sheng , P. , and Bresnick , E.1996) Retrovirus-mediated transfer of the human O6-methylguanine-DNA methyltransferase gene into a murine hematopoietic stem cell line and resistance to the toxic effects of certain alkylating agents . Biochem. Pharmacol. 51 , 1221-1228 .
316. Letourneau , S. , Greenbaum , M. , and Cournoyer , D.1996) Retrovirus-mediated gene transfer of rat glutathione S-transferase Yc confers in vitro resistance to alkylating agents in human leukemia cells and in clonogenic mouse hematopoietic progenitor cells . Hum. Gene Ther. 7 , 831-840 .
317. Magni , M. , Shammah , S. , Schiro , R. , Mellado , W. , Dalla-Favera , R. , and Gianni , A. M.1996) Induction of cyclophosphamide-resistance by aldehyde-dehydrogenase gene transfer . Blood 87 , 1097-1103 .
318. Zaboikin , M. , Srinivasakumar , N. , and Schuening , F.2006) Gene therapy with drug resistance genes . Cancer Gene Ther. 13 , 335-345 .
319. Gottesman , M. M. , Hrycyna , C. A. , Schoenlein , P. V. , Germann , U. A. , and Pastan , I.1995) Genetic analysis of the multidrug transporter . Annu. Rev. Genet. 29 , 607-649 .
320. D'Hondt , V. , Caruso , M. , and Bank , A.1997) Retrovirus-mediated gene transfer of the multidrug resistance-associated protein (MRP) cDNA protects cells from chemotherapeutic agents . Hum. Gene Ther. 8 , 1745-1751 .
321. Ujhelly , O. , Ozvegy , C. , Varady , G. , Cervenak , J. , Homolya , L. , Grez , M. , Scheffer , G. , Roos , D. , Bates , S. E. , Varadi , A. , Sarkadi , B. , and Nemet , K.2003) Application of human multidrug transporter (ABCG2) variant as selectable marker in gene transfer to progenitor cells . Hum. Gene Ther. 14 403-412 .
322. Szakacs , G. , Paterson , J. K. , Ludwig , J. A. , Booth-Genthe , C. , and Gottesman , M. M.2006) Targeting multidrug resistance in cancer . Nat. Rev. Drug Discov. 5 , 219-234 .
323. Gillet , J. P. , Efferth , T. , and Remacle , J.2007) Chemotherapy-induced resistance by ATP-binding cassette transporter genes . Biochim. Biophys. Acta 1775 , 237-262 .
324. Hesdorffer , C. , Antman , K. , Bank , A. , Fetell , M. , Mears , G. , and Begg , M.1994) Human MDR gene transfer in patients with adVanced cancer . Hum. Gene Ther. 5 , 1151-1160 .
325. O'Shaughnessy , J. A. , Cowan , K. H. , Nienhuis , A. W. , McDonagh , K. T. , Sorrentino , B. P. , Dunbar , C. E. , Chiang , Y. , Wilson , W. , Goldspiel , B. , Kohler , D. , and et al.1994) Retroviral mediated transfer of the human multidrug resistance gene (MDR-1) into hematopoietic stem cells during autologous transplantation after intensive chemotherapy for metastatic breast cancer . Hum. Gene Ther. 5 , 891-911 .
326. Cowan , K. H. , Moscow , J. A. , Huang , H. , Zujewski , J. A. , O'Shaughnessy , J. , Sorrentino , B. , Hines , K. , Carter , C. , Schneider , E. , Cusack , G. , Noone , M. , Dunbar , C. , Steinberg , S. , Wilson , W. , Goldspiel , B. , Read , E. J. , Leitman , S. F. , McDonagh , K. , Chow , C. , Abati , A. , Chiang , Y. , Chang , Y. N. , Gottesman , M. M. , Pastan , I. , and Nienhuis , A.1999) Paclitaxel chemotherapy after autologous stem-cell transplantation and engraftment of hematopoietic cells transduced with a retrovirus containing the multidrug resistance complementary DNA (MDR1) in metastatic breast cancer patients . Clin. Cancer Res. 5 , 1619-1628 .
327. Hanania , E. G. , Giles , R. E. , KaVanagh , J. , Fu , S. Q. , Ellerson , D. , Zu , Z. , Wang , T. , Su , Y. , Kudelka , A. , Rahman , Z. , Holmes , F. , Hortobagyi , G. , Claxton , D. , Bachier , C. , Thall , P. , Cheng , S. , Hester , J. , Ostrove , J. M. , Bird , R. E. , Chang , A. , Korbling , M. , Seong , D. , Cote , R. , Holzmayer , T. , Deisseroth , A. B. , and et al.1996) Results of MDR-1 vector modification trial indicate that granulocyte/macrophage colony-forming unit cells do not contribute to posttransplant hematopoietic recovery following intensive systemic therapy . Proc. Natl. Acad. Sci. USA 93 , 15346-15351 .
328. Hesdorffer , C. , Ayello , J. , Ward , M. , Kaubisch , A. , Vahdat , L. , Balmaceda , C. , Garrett , T. , Fetell , M. , Reiss , R. , Bank , A. , and Antman , K.1998) Phase I trial of retroviral- mediated transfer of the human MDR1 gene as marrow chemoprotection in patients undergoing high-dose chemotherapy and autologous stem-cell transplantation . J. Clin. Oncol. 16 , 165-172 .
329. Gottesman , M. M.2003) Cancer gene therapy: An awkward adolescence . Cancer Gene Ther. 10 , 501-508 .
330. Parker , A. L. , Newman , C. , Briggs , S. , Seymour , L. , and Sheridan , P. J.2003) Nonviral gene delivery: Techniques and implications for molecular medicine . Expert Rev. Mol. Med. 5 , 1-15 .
331. Berk , A. J.1986) Adenovirus promoters and E1A transactivation . Annu. Rev. Genet. 20 , 45-79 .
332. Roth , J. , and Dobbelstein , M.2003) Interaction of p53 with the adenovirus E1B-55 kDa protein . Methods Mol. Biol. 234 , 135-149 .
333. Sundararajan , R. , Cuconati , A. , Nelson , D. , and White , E.2001) Tumor necrosis factoralpha induces Bax-Bak interaction and apoptosis, which is inhibited by adenovirus E1B 19K . J. Biol. Chem. 276 , 45120-45127 .
334. Kindsmuller , K. , Groitl , P. , Hartl , B. , Blanchette , P. , Hauber , J. , and Dobner , T.2007) Intranuclear targeting and nuclear export of the adenovirus E1B-55K protein are regulated by SUMO1 conjugation . Proc. Natl. Acad. Sci. USA 104 , 6684-6689 .
335. Wei , M. C. , Zong , W. X. , Cheng , E. H. , Lindsten , T. , Panoutsakopoulou , V. , Ross , A. J. , Roth , K. A. , MacGregor , G. R. , Thompson , C. B. , and Korsmeyer , S. J.2001) Proapoptotic BAX and BAK: A requisite gateway to mitochondrial dysfunction and death . Science 292 , 727-730 .
336. Han , J. , Goldstein , L. A. , Gastman , B. R. , Rabinovitz , A. , Wang , G. Q. , Fang , B. , and Rabinowich , H.2004) Differential involvement of Bax and Bak in TRAIL-mediated apoptosis of leukemic T cells . Leukemia 18 , 1671-1680 .
337. Swaminathan , S. , and Thimmapaya , B.1996) Transactivation of adenovirus E2-early promoter by E1A and E4 6/7 in the context of viral chromosome . J. Mol. Biol. 258 , 736-746 .
338. Horwitz , M. S.2001) Adenovirus immunoregulatory genes and their cellular targets . Virology 279 , 1-8 .
339. Horwitz , M. S.2004) Function of adenovirus E3 proteins and their interactions with immunoregulatory cell proteins . J. Gene Med. 6 (Suppl 1) , S172 - 183 .
340. Weitzman , M. D.2005) Functions of the adenovirus E4 proteins and their impact on viral vectors . Front Biosci. 10 , 1106-1117 .
341. Peng , K. W. , Morling , F. J. , Cosset , F. L. , Murphy , G. , and Russell , S. J.1997) A gene delivery system activatable by disease-associated matrix metalloproteinases . Hum. Gene Ther. 8 , 729-738 .
342. Buchholz , C. J. , Peng , K. W. , Morling , F. J. , Zhang , J. , Cosset , F. L. , and Russell , S. J.1998) In vivo selection of protease cleavage sites from retrovirus display libraries . Nat. Biotechnol. 16 , 951-954 .
343. Peng , K. W. , Vile , R. , Cosset , F. L. , and Russell , S.1999) Selective transduction of protease-rich tumors by matrix-metalloproteinase- targeted retroviral vectors . Gene Ther. 6 , 1552-1557 .
344. Maurice , M. , Mazur , S. , Bullough , F. J. , Salvetti , A. , Collins , M. K. , Russell , S. J. , and Cosset , F. L.1999) Efficient gene delivery to quiescent interleukin-2 (IL-2)-dependent cells by murine leukemia virus-derived vectors harboring IL-2 chimeric envelope glycoproteins . Blood 94 , 401-410 .
345. Cosset , F. L. , Morling , F. J. , Takeuchi , Y. , Weiss , R. A. , Collins , M. K. , and Russell , S. J.1995) Retroviral retargeting by envelopes expressing an N-terminal binding domain . J. Virol. 69 , 6314-6322 .
346. Fielding , A. K. , Maurice , M. , Morling , F. J. , Cosset , F. L. , and Russell , S. J.1998) Inverse targeting of retroviral vectors: Selective gene transfer in a mixed population of hematopoietic and nonhematopoietic cells . Blood 91 , 1802-1809 .
347. Gordon , E. M. , Liu , P. X. , Chen , Z. H. , Liu , L. , Whitley , M. D. , Gee , C. , Groshen , S. , Hinton , D. R. , Beart , R. W. , and Hall , F. L.2000) Inhibition of metastatic tumor growth in nude mice by portal vein infusions of matrix-targeted retroviral vectors bearing a cytocidal cyclin G1 construct . Cancer Res. 60 , 3343-3347 .
348. Hall , F. L. , Liu , L. , Zhu , N. L. , Stapfer , M. , Anderson , W. F. , Beart , R. W. , and Gordon , E. M.2000) Molecular engineering of matrix-targeted retroviral vectors incorporating a surveillance function inherent in von Willebrand factor . Hum. Gene Ther. 11 , 983-993 .
349. Lorimer , I. A. , and Lavictoire , S. J.2000) Targeting retrovirus to cancer cells expressing a mutant EGF receptor by insertion of a single chain antibody variable domain in the envelope glycoprotein receptor binding lobe . J. Immunol. Methods 237 , 147-157 .
350. Martin , F. , Chowdhury , S. , Neil , S. , Phillipps , N. , and Collins , M. K.2002) Envelope- targeted retrovirus vectors transducer melanoma xenografts but not spleen or liver . Mol. Ther. 5 , 269-274 .
351. Engelstadter , M. , Bobkova , M. , Baier , M. , Stitz , J. , Holtkamp , N. , Chu , T. H. , Kurth , R. , Dornburg , R. , Buchholz , C. J. , and Cichutek , K.2000) Targeting human T cells by retroviral vectors displaying antibody domains selected from a phage display library . Hum. Gene Ther. 11 , 293-303 .
352. Khare , P. D. , Shao-Xi , L. , Kuroki , M. , Hirose , Y. , Arakawa , F. , Nakamura , K. , Tomita , Y. , and Kuroki , M.2001) Specifically targeted killing of carcinoembryonic antigen (CEA)-expressing cells by a retroviral vector displaying single-chain variable fragmented antibody to CEA and carrying the gene for inducible nitric oxide synthase . Cancer Res. 61 , 370-375 .
353. Somia , N. V. , Miyoshi , H. , Schmitt , M. J. , and Verma , I. M.2000) Retroviral vector targeting to human immunodeficiency virus type 1-infected cells by receptor pseudotyping . J. Virol. 74 , 4420-4424 .
354. Bittner , A. , Mitnacht-Kraus , R. , and Schnierle , B. S.2002) Specific transduction of HIV-1 envelope expressing cells by retroviral vectors pseudotyped with hybrid CD4/ CXCR4 receptors . J. Virol. Methods 104 , 83-92 .
355. Kimchi-Sarfaty , C. , Alexander , N. S. , Brittain , S. , Ali , S. , and Gottesman , M. M.2004) Transduction of multiple cell types using improved conditions for gene delivery and expression of SV40 pseudovirions packaged in vitro . Biotechniques 37 , 270-275 .
356. Oppenheim , A. , Peleg , A. , Fibach , E. , and Rachmilewitz , E. A.1986) Efficient introduction of plasmid DNA into human hemopoietic cells by encapsidation in simian virus 40 pseudovirions . Proc. Natl. Acad. Sci. USA 83 , 6925-6929 .
357. Kimchi-Sarfaty , C. , Brittain , S. , Garfield , S. , Caplen , N. J. , Tang , Q. , and Gottesman , M. M.2005) Efficient delivery of RNA interference effectors via in vitro-packaged SV40 pseudovirions . Hum. Gene Ther. 16 , 1110-1115 .
358. Kimchi-Sarfaty , C. , Arora , M. , Sandalon , Z. , Oppenheim , A. , and Gottesman , M. M.2003) High cloning capacity of in vitro packaged SV40 vectors with no SV40 virus sequences . Hum. Gene Ther. 14 , 167-177 .
359. Vera , M. , Prieto , J. , Strayer , D. S. , and Fortes , P.2004) Factors influencing the production of recombinant SV40 vectors . Mol. Ther. 10 , 780-791 .
360. Strayer , D. S. , Lamothe , M. , Wei , D. , Milano , J. , and Kondo , R.2001) Generation of recombinant SV40 vectors for gene transfer . Methods Mol. Biol. 165 , 103-117 .
361. Vera , M. , and Fortes , P.2004) Simian virus- 40 as a gene therapy vector . DNA Cell Biol. 23 , 271-282 .
362. Grzmil , M. , Thelen , P. , Hemmerlein , B. , Schweyer , S. , Voigt , S. , Mury , D. , and Burfeind , P.2003) Bax inhibitor-1 is overexpressed in prostate cancer and its specific down-regulation by RNA interference leads to cell death in human prostate carcinoma cells . Am. J. Pathol. 163 , 543-552
363. Yin , J. Q. , Gao , J. , Shao , R. , Tian , W. N. , Wang , J. , and Wan , Y.2003) siRNA agents inhibit oncogene expression and attenuate human tumor cell growth . J. Exp. Ther. Oncol. 3 , 194-204 .
364. Duxbury , M. S. , Ito , H. , Benoit , E. , Zinner , M. J. , Ashley , S. W. , and Whang , E. E.2003) RNA interference targeting focal adhesion kinase enhances pancreatic adenocarcinoma gemcitabine chemosensitivity . Biochem. Biophys. Res. Commun. 311 , 786-792 .
365. Sanceau , J. , Truchet , S. , and Bauvois , B.2003) Matrix metalloproteinase-9 silencing by RNA interference triggers the migratoryadhesive switch in Ewing's sarcoma cells . J. Biol. Chem. 278 , 36537-36546 .
366. Zhang , L. , Yang , N. , Mohamed-Hadley , A. , Rubin , S. C. , and Coukos , G.2003) Vectorbased RNAi, a novel tool for isoform-specific knock-down of VEGF and anti-angiogenesis gene therapy of cancer . Biochem. Biophys. Res. Commun. 303 , 1169-1178 .
367. De Schrijver , E. , Brusselmans , K. , Heyns , W. , Verhoeven , G. , and Swinnen , J. V.2003) RNA interference-mediated silencing of the fatty acid synthase gene attenuates growth and induces morphological changes and apoptosis of LNCaP prostate cancer cells . Cancer Res. 63 , 3799-3804 .
368. Nieth , C. , Priebsch , A. , Stege , A. , and Lage , H.2003) Modulation of the classical multidrug resistance (MDR) phenotype by RNA interference (RNAi) . FEBS Lett. 545 , 144-150 .
369. Lois , C. , Refaeli , Y. , Qin , X. F. , and Van Parijs , L.2001) Retroviruses as tools to studythe immune system . urr. Opin. Immunol. 13 , 496-504 .
370. Spankuch-Schmitt , B. , Bereiter-Hahn , J. , Kaufmann , M. , and Strebhardt , K.2002) Effect of RNA silencing of polo-like kinase-1 (PLK1) on apoptosis and spindle formation in human cancer cells . J. Natl. Cancer Inst. 94 , 1863-1877 .
371. Konnikova , L. , Kotecki , M. , Kruger , M. M. , and Cochran , B. H.2003) Knockdown of STAT3 expression by RNAi induces apoptosis in astrocytoma cells . BMC Cancer 3 , 23 .
372. Nagy , P. , Arndt-Jovin , D. J. , and Jovin , T. M.2003) Small interfering RNAs suppress the expression of endogenous and GFP-fused epidermal growth factor receptor (erbB1) and induce apoptosis in erbB1-overexpressing cells . Exp Cell Res. 285 , 39-49 .
373. Zhang , M. , Zhang , X. , Bai , C. X. , Chen , J. , and Wei , M. Q.2004) Inhibition of epidermal growth factor receptor expression by RNA interference in A549 cells . Acta Pharmacol. Sin. 25 , 61-67 .
374. Butz , K. , Ristriani , T. , Hengstermann , A. , Denk , C. , Scheffner , M. , and Hoppe-Seyler , F.2003) siRNA targeting of the viral E6 oncogene efficiently kills human papillomavirus- positive cancer cells . Oncogene 22 , 5938-5945 .
375. Li , X. P. , Li , G. , Peng , Y. , Kung , H. F. , and Lin , M. C.2004) Suppression of Epstein- Barr virus-encoded latent membrane protein- 1 by RNA interference inhibits the metastatic potential of nasopharyngeal carcinoma cells . Biochem. Biophys. Res. Commun. 315 , 212-218 .
376. Wilda , M. , Fuchs , U. , Wossmann , W. , and Borkhardt , A.2002) Killing of leukemic cells with a BCR/ABL fusion gene by RNA interference (RNAi) . Oncogene 21 , 5716-5724 .
377. Wohlbold , L. , Van der Kuip , H. , Miething , C. , Vornlocher , H. P. , Knabbe , C. , Duyster , J. , and Aulitzky , W. E.2003) Inhibition of bcrabl gene expression by small interfering RNA sensitizes for imatinib mesylate (STI571) . Blood 102 , 2236-2239 .
378. Kosciolek , B. A. , Kalantidis , K. , Tabler , M. , and Rowley , P. T.2003) Inhibition of telomerase activity in human cancer cells by RNA interference . Mol. Cancer Ther. 2 , 209-216.