• Ulrich R. Hengge


Die Gentherapie ist eine junge Wissenschaft, die Nukleinsäuren zur Therapie einsetzt (Hengge u. Bardenheuer 2004). Die somatische Gentherapie befasst sich mit der Behandlung von somatischen (Körper-)Zellen (⧁ Tab. 4.1.1), wobei das therapeutische Gen ein im Organismus benötigtes Protein kodiert.


  1. Alba R, Bosch A, Chillon M. Gutless adenovirus: last-generation adenovirus for gene therapy. Gene Ther 2005; 12: S18–27PubMedCrossRefGoogle Scholar
  2. An DS, Xie Y, Mao SH, Morizono K, Kung SK, Chen IS. Efficient lentiviral vectors for short hairpin RNA delivery into human cells. Hum GeneTher 2003; 14:1207–1212Google Scholar
  3. Arruda VR, Schuettrumpf J, Herzog RW, Nichols TC, Robinson N, Lotfi Y, Mingozzi F, Xiao W, Couto LB, High KA. Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1. Blood 2004; 103:85–92PubMedCrossRefGoogle Scholar
  4. Basner-Tschakarjan E, Mirmohammdsadegh A, Hengge UR. Uptake and trafficking of DNA in keratinocytes: Evidence for DNA-binding proteins. GeneTher 2004; 11:765–774Google Scholar
  5. Baum C, Dullmann J, Li Z, Fehse B, Meyer J, Williams DA, von Kalle C. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood 2003; 101: 2099–2114PubMedCrossRefGoogle Scholar
  6. Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev P, Greenblatt JJ, Rosenberg SA, Klein H, Berger M, Mullen CA, Ramsey WJ, Muul L, Morgan RA, Anderson WF. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 1995; 270:475–480PubMedCrossRefGoogle Scholar
  7. BouHamdan M, Strayer DS, Wei D, Mukhtar M, Duan LX, Hoxie J, Pomerantz RJ. Inhibition of HIV-1 infection by down-regulation of the CXCR4 co-receptor using an intracellular single chain variable fragment against CXCR4. Gene Ther 2001; 8: 408–418PubMedCrossRefGoogle Scholar
  8. Brenner MK, Heslop H, Krance R, Horowitz M, Strother D, Nüchtern J, Grilley B, Martingano E, Cooper K. Phase I study of chemokine and cytokine gene-modified autologous neuroblastoma cells for treatment of relapsed/refractory neuroblastoma using an adenoviral vector. Hum Gene Ther 2000; 11:1477–1488PubMedCrossRefGoogle Scholar
  9. Browning MT, Schmidt RD, Lew KA, Rizvi TA. Primate and feline lentivirus vector RNA packaging and propagation by heterologous lentivirus virions. J Virol 2001; 75:5129–5140PubMedCrossRefGoogle Scholar
  10. Büning H, Braun-Falco M, Hallek M. Progress in the use of adenoassociated viral vectors for gene therapy. Cells Tissues Organs 2004; 177:139–150PubMedCrossRefGoogle Scholar
  11. Cebere I, Dorrell L, McShane H, Simmons A, McCormack S, Schmidt C, Smith C, Brooks M, Roberts JE, Darwin SC, Fast PE, Conlon C, Rowland-Jones S, McMichael AJ, Hanke T. Phase I clinical trial safety of DNA-and modified virus Ankara-vectored human immunodeficiency virus type 1 (HIV-1) vaccines administered alone and in a prime-boost regime to healthy HIV-1-uninfected volunteers. Vaccine 2006; 24:417–425PubMedCrossRefGoogle Scholar
  12. Chen WC, Huang L. Non-viral vector as vaccine carrier. Adv Genet 2005; 54:315–337PubMedCrossRefGoogle Scholar
  13. Chernajovsky Y, Layward L, Lemoine N. Fighting cancer with oncolytic viruses. BMJ 2006; 332:170–172PubMedCrossRefGoogle Scholar
  14. Coburn GA, Cullen BR. Potent and specific inhibition of human immunodeficiency virus type 1 replication by RNA interference. J Virol 2002; 76:9225–9231PubMedCrossRefGoogle Scholar
  15. Costa SM, Freire MS, Alves AM. DNA vaccine against the nonstructural 1 protein (NS1) of dengue 2 virus. Vaccine 2006; 24: 4562–4564PubMedCrossRefGoogle Scholar
  16. Cunningham CC, Chada S, Merritt JA, Tong A, Senzer N, Zhang Y, Mhashilkar A, Parker K, Vukelja S, Richards D, Hood J, Coffee K, Nemunaitis J. Clinical and local biological effects of an intratumoral injection of mda-7 (IL24; INGN 241) in patients with advanced carcinoma: a phase I study. Mol Ther 2005; 11:149–159PubMedCrossRefGoogle Scholar
  17. Ding W, Zhang L, Yan Z, Engelhardt JF. Intracellular trafficking of adeno-associated viral vectors. Gene Ther 2005; 12:873–880PubMedCrossRefGoogle Scholar
  18. Edelstein ML, Abedi MR, Wixon J, Edelstein RM. Gene therapy clinical trials world wide 1989–2004-an overview. J Gene Med 2004; 6:597–602PubMedCrossRefGoogle Scholar
  19. Epstein AL, Marconi P, Argnani R, Manservigi R. HSV-1-derived recombinant and amplicon vectors for gene transfer and gene therapy. Curr Gene Ther 2005; 5:445–458PubMedCrossRefGoogle Scholar
  20. Erbacher P, Bettinger T, Brion E, Coll JL, Plank C, Behr JP, Remy JS. Genuine DNA/polyethylenimine (PEI) complexes improve transfection properties and cell survival. J Drug Target 2004; 12: 223–236PubMedCrossRefGoogle Scholar
  21. Fillat C, Carrio M, Cascante A, Sangro B. Suicide gene therapy mediated by the Herpes Simplex virus thymidine kinase gene/Ganciclovir system: fifteen years of application. Curr Gene Ther 2003; 3:13–26PubMedCrossRefGoogle Scholar
  22. Fisher PB, Gopalkrishnan RV, Chada S, Ramesh R, Grimm EA, Rosenfeld MR, Curiel DT, Dent P. mda-7/IL-24, a novel cancer selective apoptosis inducing cytokine gene: from the laboratory into the clinic. Cancer Biol Ther 2003; 2: S23–37PubMedGoogle Scholar
  23. Flotte TR. Adeno-associated virus-based gene therapy for inherited disorders. Pediatr Res 2005; 58:1143–1147PubMedCrossRefGoogle Scholar
  24. Galanis E, Okuno SH, Nascimento AG, Lewis BD, Lee RA, Oliveira AM, Sloan JA, Atherton P, Edmonson JH, Erlichman C, Randlev B, Wang Q, Freeman S, Rubin J. Phase I-II trial of ONYX-015 in combination with MAP chemotherapy in patients with advanced sarcomas. Gene Ther 2005; 12:437–445PubMedCrossRefGoogle Scholar
  25. Gilboa E, Vieweg J. Cancer immunotherapy with mRNA-transfected dendritic cells. Immunol Rev 2004; 199: 251–263PubMedCrossRefGoogle Scholar
  26. Glorioso JC, Fink DJ. Herpes vector-mediated gene transfer in treatment of diseases of the nervous system. Annu Rev Microbiol 2004; 58: 253–271PubMedCrossRefGoogle Scholar
  27. Goncalves J, Silva F, Freitas-Vieira A, Santa-Marta M, Malho R, Yang X, Gabuzda D, Barbas C 3rd. Functional neutralization of HIV-1 Vif protein by intracellular immunization inhibits reverse transcription and viral replication. J Biol Chem 2002; 277: 32036–32045PubMedCrossRefGoogle Scholar
  28. Goonetilleke N, Moore S, Dally L, Winstone N, Cebere I, Mahmoud A, Pinheiro S, Gillespie G, Brown D, Loach V, Roberts J, Guimaraes-Walker A, Hayes P, Loughran K, Smith C, De Bont J, Verlinde C, Vooijs D, Schmidt C, Boaz M, Gilmour J, Fast P, Dorrell L, Hanke T, McMichael AJ. Induction of multifunctional human immunodeficiency virus type 1 (HIV-1)-specificTcellscapableof proliferation in healthy subjects by using a prime-boost regimen of DNA-and modified vaccinia virus Ankara-vectored vaccines expressing HIV-1 Gag coupled to CD8+ T-cell epitopes. J Virol 2006; 80:4717–4728PubMedCrossRefGoogle Scholar
  29. Grimm D, Streetz KL, Jopling CL, Storm TA, Pandey K, Davis CR, Marion P, Salazar F, Kay MA. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways p537. Nature 2006; 441:537–541PubMedCrossRefGoogle Scholar
  30. Grimm D, Zhou S, Nakai H, Thomas CE, Storm TA, Fuess S, Matsushita T, Allen J, Surosky R, Lochrie M, Meuse L, McClelland A, Colosi P, Kay MA. Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood 2003; 102: 2412–2419PubMedCrossRefGoogle Scholar
  31. Grunebach F, Muller MR, Brossart P. New developments in dendritic cell-based vaccinations: RNA translated into clinics. Cancer Immunol Immunother 2005; 54: 517–525PubMedCrossRefGoogle Scholar
  32. Hacein-Bey-Abina S, von Kalle C, Schmidt M, Le Deist F, Wulffraat N, Mclntyre E, Radford I, Villeval JL, Fraser CC, Cavazzana-Calvo M, Fischer A. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003a; 348: 255–256PubMedCrossRefGoogle Scholar
  33. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, 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 LE, Wissler M, Prinz C, Rabbitts TH, Le Deist F, Fischer A, Cavazzana-Calvo M. LMO2-associated clonalT cell proliferation in two patients after gene therapy for SCID-X1. Science 2003b; 302: 415–419PubMedCrossRefGoogle Scholar
  34. Heinzerling L, Burg G, Dummer R, Maier T, Oberholzer PA, Schultz J, Elzaouk L, Pavlovic J, Moelling K. Intratumoral injection of DNA encoding human interleukin 12 into patients with metastatic melanoma: clinical efficacy. Hum Gene Ther 2005; 16: 35–48PubMedCrossRefGoogle Scholar
  35. Heller L, Merkler K, Westover J, Cruz Y, Coppola D, Benson K, Daud A, Heller R. Evaluation of toxicity following electrically mediated interleukin-12 gene delivery in a B16 mouse melanoma model. Clin Cancer Res 2006; 12: 3177–3183PubMedCrossRefGoogle Scholar
  36. Hengge UR, Bardenheuer W. Gene therapy and the skin. Am J Med Genet C Semin Med Genet 2004; 131C: 93–100PubMedCrossRefGoogle Scholar
  37. Hengge UR, Chan EF, Foster RA, Walker PS, Vogel JC. Cytokine gene expression in epidermis with biological effects following injection of naked DNA. Nat Genet 1995; 10: 161–166PubMedCrossRefGoogle Scholar
  38. Hengge UR, Dexling B, Mirmohammadsadegh A. Safety and pharmacokinetics of naked plasmid DNA in the skin: studies on dissemination and ectopic expression. J Invest Dermatol 2001; 116: 979–982PubMedCrossRefGoogle Scholar
  39. Hengge, A Mirmohammadsadegh. Adeno-associated virus (AAV) expresses transgenes long-term in epidermis and hair follicles. Mol Ther 2002; 2: 189–194Google Scholar
  40. Hengge UR, Schadendorf D. Modification of melanoma cells via ballistic gene delivery for vaccination. In: Lasic, Templeton (Hrsg.): GeneTherapy:Therapeutic mechanisms and strategies. Marcel Dekker, New York, 2000, 317–338Google Scholar
  41. Hengge UR, Walker PS, Vogel JC. Expression of naked DNA in human, pig, and mouse skin. J Clin Invest 1996; 97: 2911–2916PubMedCrossRefGoogle Scholar
  42. Hersey P, Menzies SW, Halliday GM, Nguyen T, Farrelly ML, DeSilva C, Lett M. Phase I/II study of treatment with dendritic cell vaccines in patients with disseminated melanoma. Cancer Immunol Immunother 2004; 53: 125–134PubMedCrossRefGoogle Scholar
  43. Jacque JM, Triques K, Stevenson M. Modulation of HIV-1 replication by RNA interference. Nature 2002; 418: 379–380CrossRefGoogle Scholar
  44. Karanikas V, Lurquin C, Colau D, van Baren N, De Smet C, Lethe B, Connerotte T, Corbiere V, Demoitie MA, Lienard D, Dreno B, Velu T, Boon T, Coulie PG. Monoclonal anti-MAGE-3 CTL responses in melanoma patients displaying tumor regression after vaccination with a recombinant canarypox virus. J Immunol 2003; 171: 4898–4904PubMedGoogle Scholar
  45. Kaufman HL, Deraffele G, Mitcham J, Moroziewicz D, Cohen SM, Hurst-Wicker KS, Cheung K, Lee DS, Divito J, Voulo M, Donovan J, Dolan K, Manson K, Panicali D, Wang E, Horig H, Marincola FM. Targeting the local tumor microenvironment with vaccinia virus expressing B7.1 for the treatment of melanoma. J Clin Invest 2005; 115: 1903–1912PubMedCrossRefGoogle Scholar
  46. Kirch HC, Ruschen S, Brockmann D, Esche H, Horikawa I, Barrett JC, Opalka B, Hengge UR. Tumor-specific activation of hTERT-derived promoters by tumor suppressive E1A-mutants involves recruitment of p300/CBP/H AT and suppression of HDAC-1 and defines a combined tumor targeting and suppression system. Oncogene 2002; 21: 7991–8000PubMedCrossRefGoogle Scholar
  47. Kloeckner J, Prasmickaite L, Hogset A, Berg K, Wagner E. Photochemically enhanced gene delivery of EGF receptor-targeted DNA polyplexes. J Drug Target 2004; 12: 205–213PubMedCrossRefGoogle Scholar
  48. Kobinger GP, Weiner DJ, Yu QC, Wilson JM. Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo. Nat Biotechnol 2001; 19: 225–230PubMedCrossRefGoogle Scholar
  49. Kong WP, Xu L, Stadler K, Ulmer JB, Abrignani S, Rappuoli R, Nabel GJ. Modulation of the immune response to the severe acute respiratory syndrome spike glycoprotein by gene-based and inactivated virus immunization. J Virol 2005; 79: 13915–13923PubMedCrossRefGoogle Scholar
  50. Kyte JA, Mu L, Aamdal S, Kvalheim G, Dueland S, Hauser M, Guilestad HP, Ryder T, Lislerud K, Hammerstad H, Gaudernack G. Phase I/II trial of melanoma therapy with dendritic cells transfected with autologous tumor-mRNA. Cancer Gene Ther 2006; 13: 905–918PubMedCrossRefGoogle Scholar
  51. Leitch J, Fraser K, Lane C, Putzu K, Adema GJ, Zhang QJ, Jefferies WA, Bramson JL, Wan Y. CTL-dependent and-independent antitumor immunity is determined by the tumor not the vaccine. J Immunol 2004; 172: 5200–5205PubMedGoogle Scholar
  52. 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 HG, Fehse B, Baum C. Murine leukemia induced by retroviral gene marking. Science 2002; 296: 497PubMedCrossRefGoogle Scholar
  53. Lillicrap D, Vandendriessche T, High K. Cellular and genetic therapies for haemophilia. Haemophilia 2006; 12(Suppl 3): 36–41PubMedCrossRefGoogle Scholar
  54. Lin E, Nemunaitis J. Oncolytic viral therapies. Cancer Gene Ther 2004; 11: 643–664PubMedCrossRefGoogle Scholar
  55. Liu YL, Mingozzi F, Rodriguez-Colon SM, Joseph S, Dobrzynski E, Suzuki T, High KA, Herzog RW. Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector. Hum Gene Ther 2004a; 15: 783–792PubMedCrossRefGoogle Scholar
  56. Liu Y, Ye T, Sun D, Maynard J, Deisseroth A. Conditionally replicationcompetent adenoviral vectors with enhanced infectivity for use in gene therapy of melanoma. Hum Gene Ther 2004b; 15: 637–647PubMedCrossRefGoogle Scholar
  57. Lonchay C, van der Bruggen P, Connerotte T, Hanagiri T, Coulie P, Colau D, Lucas S, Van Pel A, Thielemans K, van Baren N, Boon T. Correlation between tumor regression and T cell responses in melanoma patients vaccinated with a MAGE antigen. Proc Natl Acad Sci USA 2004; 101(Suppl 2): 14631–14638PubMedCrossRefGoogle Scholar
  58. Lorence RM, Pecora AL, Major PP, Hotte SJ, Laurie SA, Roberts MS, Groene WS, Bamat MK. Overview of phase I studies of intravenous administration of PV701, an oncolytic virus. Curr Opin Mol Ther 2003; 5: 618–624PubMedGoogle Scholar
  59. Lundstrom K. Biology and application of alphaviruses in gene therapy. Gene Ther 2005; 12: S92–97PubMedCrossRefGoogle Scholar
  60. Mangeat B, Trono D. Lentiviral vectors and antiretroviral intrinsic immunity. Hum Gene Ther 2005; 16: 913–920PubMedCrossRefGoogle Scholar
  61. Manno CS, Arruda VR, Pierce GF, Glader B, Ragni M, Rasko J, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006; 12: 342–347PubMedCrossRefGoogle Scholar
  62. Mastrobattista E, van der Aa MA, Hennink WE, Crommelin DJ. Artificial viruses: a nanotechnological approach to gene delivery. Nat Rev Drug Discov 2006; 5: 115–121PubMedCrossRefGoogle Scholar
  63. Mautino MR, Morgan RA. Gene therapy of HIV-1 infection using lentiviral vectors expressing anti-HIV-1 genes. AIDS Patient Care STDS 2002; 16: 11–26PubMedCrossRefGoogle Scholar
  64. Meykadeh N, Mirmohammadsadegh A, Wang Z, Basner-Tschakarjan E, Hengge UR. Topical application of plasmid DNA to mouse and human skin. J Mol Med 2005; 83: 897–903PubMedCrossRefGoogle Scholar
  65. Michienzi A, Castanotto D, Lee N, Li S, Zaia JA, Rossi JJ. RNA-mediated inhibition of HIV in a gene therapy setting. Ann NY Acad Sci 2003; 1002: 63–71PubMedCrossRefGoogle Scholar
  66. Morizono K, Xie Y, Ringpis GE, Johnson M, Nassanian H, Lee B, Wu L, Chen IS. Lentiviral vector retargeting to P-glycoprotein on metastatic melanoma through intravenous injection. Nat Med 2005; 11: 346–352PubMedCrossRefGoogle Scholar
  67. Morris KV, Gilbert J, Wong-Staal F, Gasmi M, Looney DJ. Transduction of cell lines and primary cells by FIV-packaged HIV vectors. Mol Ther 2004; 10: 181–190PubMedCrossRefGoogle Scholar
  68. Morris KV, Rossi JJ. Lentiviral-mediated delivery of siRNAs for antiviral therapy. Gene Ther 2006; 13: 553–558PubMedCrossRefGoogle Scholar
  69. Muul LM, Tuschong LM, Soenen SL Jagadeesh GJ, Ramsey WJ, Long Z, Carter CS, Garabedian EK, Alleyne M, Brown M, Bernstein W, Schurman SH, Fleisher TA, Leitman SF, Dunbar CE, Blaese RM, Candotti F. 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: 2563–2569PubMedCrossRefGoogle Scholar
  70. Muzyczka N, Warrington KH Jr. Custom adeno-associated virus capsids: the next generation of recombinant vectors with novel tropism. Hum GeneTher 2005; 16: 408–416Google Scholar
  71. Nabel GJ. Vaccine for AIDS and Ebola virus infection. Virus Res 2003; 92: 213–217PubMedCrossRefGoogle Scholar
  72. Naldini L, Verma IM. Lentiviral vectors. AdvVirus Res 2000; 55: 599–609Google Scholar
  73. Nestle FO, Alijagic S, Gilliet M, Sun Y, Grabbe S, Dummer R, Burg G, Schadendorf D. Vaccination of melanoma patients with peptide-or tumor lysate-pulsed dendritic cells. Nat Med 1998; 4: 328–332PubMedCrossRefGoogle Scholar
  74. Niculescu-Duvaz I, Springer CJ. Introduction to the background, principles, and state of the art in suicide gene therapy. Mol Biotechnol 2005; 30: 71–88PubMedCrossRefGoogle Scholar
  75. Ogris M, Walker G, Blessing T, Kircheis R, Wolschek M, Wagner E. Tumor-targeted gene therapy: strategies for the preparation of ligand-polyethyleneglycol-polyethylenimine/DNAcomplexes. J Control Release 2003; 91: 173–181PubMedCrossRefGoogle Scholar
  76. Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kuhlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins NA, Copeland NG, Luthi U, Hassan M, Thrasher AJ, Hoelzer D, von Kalle C, Seger R, Grez M. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDMI6 or SETBP1. Nat Med 2006; 12: 401–409PubMedCrossRefGoogle Scholar
  77. Pataer A, Fanale MA, Roth JA, Swisher SG, Hunt KK. Induction of apoptosis in human lung cancer cells following treatment with amifostine and an adenoviral vector containing wild-type p53. Cancer Gene Ther 2006; 13: 806–814PubMedCrossRefGoogle Scholar
  78. Prud’homme GJ. DNA vaccination against tumors. J Gene Med 2005; 7: 3–17PubMedCrossRefGoogle Scholar
  79. Puerta-Fernandez E, Barroso-DelJesus A, Berzal-Herranz A. Anchoring hairpin ribozymes to long target RNAs by loop-loop RNA interactions. Antisense Nucleic Acid Drug Dev 2002; 12: 1–9PubMedCrossRefGoogle Scholar
  80. Ramezani A, Ma XZ, Nazari R, Joshi S. Development and testing of retroviral vectors expressing multimeric hammerhead ribozymes targeted against all major clades of HIV-1. Front Biosci 2002; 7: a29–36PubMedCrossRefGoogle Scholar
  81. Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao GP, Wilson JM, Batshaw ML. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 2003; 80: 148–158PubMedCrossRefGoogle Scholar
  82. Reynolds L, Ullman C, Moore M, Isalan M, West MJ, Clapham P, Klug A, Choo Y. Repression of the HIV-1 5’ LTR promoter and inhibition of HIV-1 replication by using engineered zincfinger transcription factors. Proc Natl Acad Sci USA 2003; 100: 1615–1620PubMedCrossRefGoogle Scholar
  83. Rini B. Recent clinical development of dendritic cell-based immunotherapy for prostate cancer. Expert Opin Biol Ther 2004; 4: 1729–1734PubMedCrossRefGoogle Scholar
  84. Riviere C, Danos O, Douar AM. Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice. Gene Ther 2006; 13: 1300–1308PubMedCrossRefGoogle Scholar
  85. Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, Karson EM, Lotze MT, Yang JC, Topalian SL, et al. Gene transfer into humans-immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 1990; 323: 570–578PubMedGoogle Scholar
  86. Roth JA. Adenovirus p53 gene therapy. Expert Opin Biol Ther 2006; 6: 55–61PubMedCrossRefGoogle Scholar
  87. Saenz DT, Loewen N, Peretz M, Whitwam T, Barraza R, Howell KG, Holmes JM, Good M, Poeschla EM. Unintegrated lentivirus DNA persistenceandaccessibilitytoexpression in nondividing cells: analysis with class I integrase mutants. J Virol 2004; 78: 2906–2920PubMedCrossRefGoogle Scholar
  88. Sahin U, Tureci O, Schmitt H, Cochlovius B, Johannes T, Schmits R, Stenner F, Luo G, Schobert I, Pfreundschuh M. Human neoplasms elicit multiple specific immune responses in the autologous host. Proc Natl Acad Sci USA 1995; 92:11810–11813PubMedCrossRefGoogle Scholar
  89. Sauter ER, Takemoto R, Litwin S, Herlyn M. p53 alone or in combination with antisense cyclin D1 induces apoptosis and reduces tumor size in human melanoma. Cancer Gene Ther 2002; 9: 807–812PubMedCrossRefGoogle Scholar
  90. Schoensiegel F, Paschen A, Sieger S, Eskerski H, Mier W, Rothfels H, Kleinschmidt J, Schadendorf D, Haberkorn U. MIA (melanoma inhibitory activity) promoter mediated tissue-specific suicide gene therapy of malignant melanoma. Cancer GeneTher 2004; 11: 408–818CrossRefGoogle Scholar
  91. See RH, Zakhartchouk AN, Petric M, Lawrence DJ, Mok CP, Hogan RJ, Rowe T, Zitzow LA, Karunakaran KP, Hitt MM, Graham FL, Prevec L, Mahony JB, Sharon C, Auperin TC, Rini JM, Tingle AJ, Scheifele DW, Skowronski DM, Patrick DM, Voss TG, Babiuk LA, Gauldie J, Roper RL, Brunham RC, Finlay BB. Comparative evaluation of two severe acute respiratory syndrome (SARS) vaccine candidates in mice challenged with SARS coronavirus. J Gen Virol 2006; 87: 641–650PubMedCrossRefGoogle Scholar
  92. Segal DJ, Goncalves J, Eberhardy S, Swan CH, Torbett BE, Li X, Barbas CF 3rd. Attenuation of HIV-1 replication in primary human cells with a designed zinc finger transcription factor. J Biol Chem 2004; 279: 14509–14519PubMedCrossRefGoogle Scholar
  93. Serganova I, Blasberg R. Reporter gene imaging: potential impact on therapy. Nucl Med Biol 2005; 32: 763–780PubMedCrossRefGoogle Scholar
  94. Sumimoto H, Miyagishi M, Miyoshi H, Yamagata S, Shimizu A, Taira K, Kawakami Y. Inhibition of growth and invasive ability of melanoma by inactivation of mutated BRAF with lentivirusmediated RNA interference. Oncogene 2004; 23: 6031–6039PubMedCrossRefGoogle Scholar
  95. Thurner B, Haendle I, Roder C, Dieckmann D, Keikavoussi P, Jonuleit H, Bender A, Maczek C, Schreiner D, von den Driesch P, Brocker EB, Steinman RM, Enk A, Kampgen E, Schuler G. Vaccination with mage-3A1 peptide-pulsed mature, monocyte-derived dendritic cells expands specific cytotoxic T cells and induces regression of some metastases in advanced stage IV melanoma. J Exp Med 1999; 190: 1669–1678PubMedCrossRefGoogle Scholar
  96. Tong AW, Nemunaitis J, Su D, Zhang Y, Cunningham C, Senzer N, Netto G, Rich D, Mhashilkar A, Parker K, Coffee K, Ramesh R, Ekmekcioglu S, Grimm EA, van Wart Hood J, Merritt J, Chada S. Intratumoral injection of INGN 241, a nonreplicating adenovector expressing the melanoma-differentiation associated gene-7 (mda-7/IL24): biologic outcome in advanced cancer patients. Mol Ther 2005; 11: 160–172PubMedCrossRefGoogle Scholar
  97. Triozzi PL, Strong TV, Bucy RP, Allen KO, Carlisle RR, Moore SE, Lobuglio AF, Conry RM. Intratumoral administration of a recombinant canarypox virus expressing interleukin 12 in patients with metastatic melanoma. Hum GeneTher 2005; 16: 91–100Google Scholar
  98. Trono D. Lentiviral vectors: turning a deadly foe into a therapeutic agent. Gene Ther 2000; 7: 20–23PubMedCrossRefGoogle Scholar
  99. Unwalla HJ, Li MJ, Kim JD, Li HT, Ehsani A, Alluin J, Rossi JJ. Negative feedback inhibition of HIV-1 by TAT-inducible expression of siRNA. Nat Biotechnol 2004; 22: 1573–1578PubMedCrossRefGoogle Scholar
  100. van Dillen IJ, Mulder NH, Vaalburg W, de Vries EF, Hospers GA. Influence of the bystander effect on HSV-tk/GCV gene therapy. A review. Curr Gene Ther 2002; 2: 307–322PubMedCrossRefGoogle Scholar
  101. Vargas J Jr, Gusella GL, Najfeld V, Klotman ME, Cara A. Novel integrase-defective lentiviral episomal vectors for gene transfer. Hum GeneTher 2004; 15: 361–972Google Scholar
  102. Vasileva A, Jessberger R. Precise hit: adeno-associated virus in gene targeting. Nat Rev Microbiol 2005; 3: 837–847PubMedCrossRefGoogle Scholar
  103. Vassaux G, Nitcheu J, Jezzard S, Lemoine NR. Bacterial gene therapy strategies. J Pathol 2006; 208: 290–298PubMedCrossRefGoogle Scholar
  104. Volpers C, Kochanek S. Adenoviral vectors for gene transfer and therapy. J Gene Med 2004; 6: S164–171PubMedCrossRefGoogle Scholar
  105. Walker PS, Scharton-Kersten T, Rowton ED, Hengge U, Bouloc A, Udey MC, Vogel JC. Genetic immunization with glycoprotein 63 cDNA results in a helper T cell type 1 immune response and protection in a murine model of leishmaniasis. Hum GeneTher 1998; 9: 1899–1907Google Scholar
  106. Wang R, Doolan DL, Le TP, Hedstrom RC, Coonan KM, Charoenvit Y, Jones TR, Hobart P, Margalith M, Ng J, Weiss WR, Sedegah M, de Taisne C, Norman JA, Hoffman SL. Induction of antigen-specific cytotoxic T lymphocytes in humans by a malaria DNA vaccine. Science 1998; 282: 476–480PubMedCrossRefGoogle Scholar
  107. Wang Z, Yuan Z, Matsumoto M, Hengge UR, Chang YF. Immune responses with DNA vaccines encoded different gene fragments of severe acute respiratory syndrome coronavirus in BALB/c mice. Biochem Biophys Res Commun 2005; 327: 130–135PubMedCrossRefGoogle Scholar
  108. Willemsen R, Ronteltap C, Heuveling M, Debets R, Bolhuis R. Redirecting human CD4+ T lymphocytes to the MHC class I-restricted melanoma antigen MAGE-A1 byTCR alphabeta gene transfer requires CD8alpha. Gene Ther 2005; 12: 140–146PubMedCrossRefGoogle Scholar
  109. Wolff JA, Budker V. The mechanism of naked DNA uptake and expression. Adv Genet 2005; 54: 3–20PubMedGoogle Scholar
  110. Woods NB, Bottero V, Schmidt M, von Kalle C, Verma IM. Gene therapy: therapeutic gene causing lymphoma. Nature 2006; 440: 1123PubMedCrossRefGoogle Scholar
  111. Yanez-Munoz RJ, Balaggan KS, Macneil A, Howe SJ, Schmidt M, Smith AJ, Buch P, Maclaren RE, Anderson PN, Barker SE, Duran Y, Bartholomae C, von Kalle C, Heckenlively JR, Kinnon C, Ali RR, Thrasher AJ. Effective gene therapy with nonintegrating lentiviral vectors. Nat Med 2006; 12: 348–353PubMedCrossRefGoogle Scholar
  112. Yang G, Zhong Q, Huang W, Reiser J, Schwarzen berger P. Retrovirus molecular conjugates: a versatile and efficient gene transfer vector system for primitive human hematopoietic progenitor cells. Cancer Gene Ther 2006; 13: 460–468PubMedCrossRefGoogle Scholar

Copyright information

© Springer-Verlag Berlin Heidelberg 2008

Authors and Affiliations

  • Ulrich R. Hengge
    • 1
  1. 1.HautklinikUniversitätsklinikum DüsseldorfDüsseldorf

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