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Liver Cancer pp 230-245 | Cite as

Gene Therapy for Hepatocellular Carcinoma

  • Dennie V. JonesJr.
Part of the M.D. Anderson Solid Tumor Oncology Series book series (MDA)

Abstract

Hepatocellular carcinoma (HCC) is one of the most common malignancies in the world, with approximately one million new cases recorded annually.1 At present, only complete surgical resection has the potential for producing a cure, and then only in a small subset of patients. Other modalities, such as radiotherapy, chemotherapy, or bioimmunotherapy, have produced response in a few patients, but it is clear that most patients derive little or no benefit from the application of such treatments. Virtually all antineoplastic therapies are associated with substantial toxicities due to the often unacceptable adverse effects on normal tissues. As a result, most patients either receive ineffective therapy, or are unable to tolerate therapy that could potentially eradicate their neoplasm. This situation justifies the evaluation of novel modalities.

Keywords

Gene Therapy Thymidine Kinase Thymidine Kinase Gene Herpes Simplex Virus Genetic Therapy 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

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References

  1. 1.
    Carr BI, Flickinger JC, Lotze MT. Hepatobiliary cancers. In: DeVita VT, Hellman S, Rosenberg SA (eds) Cancer: Principles and Practice of Oncology, 5th ed. Philadelphia, Lippincott-Raven, 1997, pp. 1087–1114Google Scholar
  2. 2.
    Hug P, Sleight RG. Liposomes for the transformation of eukaryotic cells. Biochim Biophys Acta 1097:1, 1991PubMedCrossRefGoogle Scholar
  3. 3.
    Sorrentino BP, Brandt SJ, Bodine D, et al. Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of the human MDR1. Science 257:99, 1992PubMedCrossRefGoogle Scholar
  4. 4.
    Mickisch GH, Aksentijevich I, Schoenlein PV, et al. Transplantation of bone marrow cells from transgenic mice expressing the human MDR1 gene results in long-term protection against the myelosuppressive effect of chemotherapy in mice. Blood 79:1087, 1992PubMedGoogle Scholar
  5. 5.
    May C, Gunther R, McIvor RS. Protection of mice from lethal doses of methotrexate by transplantation with transgenic marrow expressing drug-resistant dihydrofolate-reductase activity. Blood 86:2439, 1995PubMedGoogle Scholar
  6. 6.
    Moritz T, Mackay W, Glassner BJ, Williams DA, Samson L. Retrovirus-mediated expression of DNA repair protein protection in bone marrow protects hematopoietic cells from nitrosourea-induced toxicity in vitro and in vivo. Cancer Res 55:2608, 1995PubMedGoogle Scholar
  7. 7.
    Spencer HT, Sleep SE, Rehg JE, Blakely RL, Sorrentino BR A gene transfer strategy for making bone marrow cells resistant to trimetrexate. Blood 87:2579, 1996PubMedGoogle Scholar
  8. 8.
    Feigner PL, Gadek TR, Holm M, et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 84:7413, 1987CrossRefGoogle Scholar
  9. 9.
    Graham FL, van der Eb AJ. A new technique for the assay of human adenovirus 5 DNA. Virology 52:456, 1973PubMedCrossRefGoogle Scholar
  10. 10.
    Diacumakos EG, Gershey EL. Uncoating and gene expression of simian virus 40 in CV-1 cell nuclei inoculated by microinjection. J Virol 24:903, 1977PubMedGoogle Scholar
  11. 11.
    Graessmann M, Graessmann A. In: Celis JE, Graessmann A, Loyter A (eds) Microinjection and Organelle Transplantation Techniques. London, Academic, 1986, pp. 3–13Google Scholar
  12. 12.
    Fountain JW, Lockwood WK, Collins FS. Transformation of primary human skin fibroblasts by electroporation. Gene 68:167, 1988PubMedCrossRefGoogle Scholar
  13. 13.
    Hama-Inaba H, Nishimoto T, Ohtsubo M, Sato K, Kasai M. Simple and effect tive method of electroporation for introduction of plasmid and cosmid DNAs to mammalian cells. Nucleic Acids Symp Series 19:149, 1988Google Scholar
  14. 14.
    Strauss M. Liver-directed gene therapy: prospects and problems. Gene Ther 1:156, 1994PubMedGoogle Scholar
  15. 15.
    Bowles NE, Eisensmith RC, Mohuiddin R, Pyron M, Woo SLC. A simple and efficient method for the concentration and purification of recombinant retrovirus for increased hepatocyte transduction in vivo. Hum Gene Ther 7:1735, 1996PubMedCrossRefGoogle Scholar
  16. 16.
    Ferry N, Duplessis O, Houssein D, Danos O, Heard J-L. Retroviral-mediated gene transfer into hepatocytes in vivo. Proc Natl Acad Sci USA 88:8377, 1991PubMedCrossRefGoogle Scholar
  17. 17.
    Kay MA, Li Q, Liu TJ, et al. Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo. Hum Gene Ther 3:641, 1992PubMedCrossRefGoogle Scholar
  18. 18.
    Stratford-Perricaudet LD, Levrero M, Chasse JF, Perricaudet M, Briaand P. Evaluation of the transfer and expression in mice of an enzyme-encoding gene using an adenovirus vector. Hum Gene Ther 1:241, 1990PubMedCrossRefGoogle Scholar
  19. 19.
    Levrero M, Barbaann V, Manteca S, et al. Defective and nondefective adenovirus vectors for expressing foreign genes in vitro and in vivo. Gene 101:195, 1991PubMedCrossRefGoogle Scholar
  20. 20.
    Graham FL, Smiley J, Russell WL, Nairn R. Characterization of a human ccell line transformed by DNA from adenovirus 5. J Gen Virol 36:59, 1977PubMedCrossRefGoogle Scholar
  21. 21.
    Yang Y, Li Q, Ertl HCJ, Wilson JM. Cellular and humoral immune responses to viral antigens created barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol 69:2004, 1995Google Scholar
  22. 22.
    Yang Y, Greenough K, Wilson JM. Transient immune blockade prevents formation of neutralizing antibody to recombinant adenovirus and allows repeated gene transfer to mouse liver. Gene Ther 3:412, 1996PubMedGoogle Scholar
  23. 23.
    Yang Y, Ertl HCJ, Wilson JM. MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses. Immunity 1:433, 1994PubMedCrossRefGoogle Scholar
  24. 24.
    Dai Y, Schwarz EM, Gu D, et al. Cellular aand humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of both factor IX and vector antigens allows for long-term expression. Proc Natl Acad Sci USA 92:1401, 1995PubMedCrossRefGoogle Scholar
  25. 25.
    Fang B, Eisensmith RC, Wang H, et al. Gene therapy for hemophilia B: host immunosuppresssion prolongs the therapeutic effect of adenovirus-mediated factor IX expression. Hum Gene Ther 6:1039, 1995PubMedCrossRefGoogle Scholar
  26. 26.
    Li QT, Kay MA, Finegold M, Stratford-Perricaudet LD, Woo SLC. Assessment of recombinaent adenoviral vectors for hepatic gene therapy. Hum Gene Ther 4:403, 1993PubMedCrossRefGoogle Scholar
  27. 27.
    Halbert CL, Alexander IE, Wolgamot GM, Miller AD. Adeno-associated virus vectors transduce primary cells much less efficiently than immortalized cells. J Virol 69:1473, 1995PubMedGoogle Scholar
  28. 28.
    O’Reilly OR, Miller LK, Luckow VA. Virus host interactions. In: Baculovirus Expression Vectors: A Laboratory Manual. Oxford, Oxford University Press, 1994Google Scholar
  29. 29.
    Roth JA, Cristiano RJ. Gene therapy for cancer: what have we done and where are we going? J Natl Cancer Inst 88:21, 1997CrossRefGoogle Scholar
  30. 30.
    Gruenwald S, Heitz J. Baculovirus Expressor System: Procedures and Methods Manual. San Diego, PharMingen, 1993Google Scholar
  31. 31.
    Sandig V, Hofmann C, Steinert S, et al. Gene transfer into hepatocytes and human liver tissue by baculovirus vectors. Hum Gene Ther 7:1937, 1996PubMedCrossRefGoogle Scholar
  32. 32.
    Mukherjee AB, Orloff S, Butler JD, et al. Entrapment of metaphase chromosomes into phospholipid vesicles (lipochromosomes): carrier potential in gene transfer. Proc Natl Acad Sci USA 75:1361, 1978PubMedCrossRefGoogle Scholar
  33. 33.
    Nicolau C, Cudd A. Liposomes as carriers of DNA. Crit Rev Ther Drug Carrier Syst 6:239, 1989PubMedGoogle Scholar
  34. 34.
    Lopez-Berestein G. Treatment of systemic fungal infections with liposomal amphotericin B. In: Liposomes in the Treatment of Infectious Diseases and Cancer. New York, Liss, 1989, pp. 317–327Google Scholar
  35. 35.
    Forssen EA, Tokes ZA. Use of anionic liposomes for the reduction of chronic doxorubicin-induced cardiotoxicity. Proc Natl Acad Sci USA 78:1873, 1981PubMedCrossRefGoogle Scholar
  36. 36.
    Gabizon A, Dagan A, Goren D, Barenholz Y, Fuks Z. Liposomes as in vivo carriers of adriamycin: reduced cardiac uptake and preserved antitumor activity in mice. Cancer Res 42:4734, 1982PubMedGoogle Scholar
  37. 37.
    Perez-Soler R, Lopez-Berestein G, Lautersztain J, et al. Phase I clinical and pharmacological study of liposome-entrapped cis-bis-neodecanoato-trans-R,R-1,2-diaminocyclohexane platinum (II). Cancer Res 50:4254, 1990PubMedGoogle Scholar
  38. 38.
    Lurquin PF. Entrapment of plasmid DNA by liposomes and their interactions with plant protoplasts. Nucleic Acids Res 6:3773, 1979PubMedCrossRefGoogle Scholar
  39. 39.
    Fukunaga Y, Nagata T, Takebe I. Liposome-mediated infection of plant protoplasts with tobacco mosaic virus RNA. Virology 113:752, 1981PubMedCrossRefGoogle Scholar
  40. 40.
    Nicolau C, Sene C. Liposome-mediated DNA transfer in eukaryotic cells: dependence of the transfer efficiency upon the type of liposomes used and the host cell cycle stage. Biochim Biophys Acta 721:185, 1982PubMedCrossRefGoogle Scholar
  41. 41.
    Kaneda Y, Uchida T, Sugawa H, Ishiura M, Okada Y The improved efficient method for introducing macromolecules into cells using HVJ (Sendai virus) liposomes with gangliosides. Exp Cell Res 173:56, 1987PubMedCrossRefGoogle Scholar
  42. 42.
    Lapidot M, Loyter A. Fusion-mediated microinjection of liposome-enclosed DNA into cultured cells with the aid of influenza virus glycoproteins. Exp Cell Res 189:241, 1990PubMedCrossRefGoogle Scholar
  43. 43.
    Vainstein A, Razin A, Graessman A, Loyter A. Methods Enzymol 101:492, 1983PubMedCrossRefGoogle Scholar
  44. 44.
    Jay DG, Gilbert W. Basic protein enhances the incorporation of DNA into lipid vesicles: model for the formation of primordial cells. Proc Natl Acad Sci USA 84:1978, 1987PubMedCrossRefGoogle Scholar
  45. 45.
    Fraley R, Straubinger RM, Rule G, Springer EL, Papahadjopoulos D. Liposome-mediated delivery of deoxyribonucleic acid to cells: enhanced efficiency of delivery related to lipid composition and incubation conditions. Biochemistry 20:6978, 1981PubMedCrossRefGoogle Scholar
  46. 46.
    Straubinger RM, Hong K, Friend DS, Papahadjopoulos D. Endocytosis of liposomes and intracellular fate of encapsulated molecules: encounter with a low pH compartment after internalization in coated vesicles. Cell 32:1069, 1983PubMedCrossRefGoogle Scholar
  47. 47.
    Felgner PL, Ringold GM. Cationic liposome-mediated transfection. Nature 337:387, 1989PubMedCrossRefGoogle Scholar
  48. 48.
    Loeffler J-P, Behr J-P. Gene transfer into primary and established mammalian cell lines with lipopolyamine-coated DNA. Methods Enzymol 217:599, 1993PubMedCrossRefGoogle Scholar
  49. 49.
    Dwarki VJ, Malone RW, Verma IM. Cationic liposome-mediated RNA transfection. Methods Enzymol 217:644, 1993PubMedCrossRefGoogle Scholar
  50. 50.
    Milhaud PG, Machy P, Colote S, Lebleu B, Leserman L. Free and liposome-encapsulated double-stranded RNAs as inducers of interferon, interleukin-6, and cellular toxicity. J Interferon Res 11:261, 1991PubMedCrossRefGoogle Scholar
  51. 51.
    Akhtar S, Basu S, Wickstrom E, Juliano RL. Interactions of antisense DNA oligonucleotide analogs with phospholipid membranes (liposomes). Nucleic Acids Res 20:5551, 1991CrossRefGoogle Scholar
  52. 52.
    Budker V, Zhang G, Knechtle S, Wolff JA. Naked DNA delivered intraportally expresses efficiently in hepatocytes. Gene Ther 3:593, 1996PubMedGoogle Scholar
  53. 53.
    Yang N, Burkholder J, Robert B. In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment. Proc Natl Acad Sci USA 87:9568, 1990PubMedCrossRefGoogle Scholar
  54. 54.
    Zelenin AV, Alimov AA, Titomirov AV, et al. High-velocity mechanical DNA transfer of the chloramphenicol acetyltransferase gene into rodent liver, kidney and mammary gland cells in organ expiants and in vivo. FEBS Lett 280:365, 1991CrossRefGoogle Scholar
  55. 55.
    Furth PA, Shamay A, Wall RJ, Henninghausen L. Gene transfer into somatic tissues by jet injection. Anal Biochem 205;365, 1992PubMedCrossRefGoogle Scholar
  56. 56.
    Cheng L, Ziegelhoffer PR, Yang NS. In vivo promoter activity and transgene expression in mammalian somatic tissues evaluated by using particle bombardment. Proc Natl Acad Sci USA 90:4455, 1993PubMedCrossRefGoogle Scholar
  57. 57.
    Nicolet CM, Burkholder JK, Gan J, et al. Expression of a tumor-reactive antibody-interleukin 2 fusion protein after in vivo particle-mediated gene delivery. Cancer Gene Ther 2:161, 1995PubMedGoogle Scholar
  58. 58.
    Bowling WM, Kennedy SC, Cai S-R, et al. Portal branch occlusion safely facilitates in vivo retroviral vector transduction of rat liver. Hum Gene Ther 7:2113, 1996PubMedCrossRefGoogle Scholar
  59. 59.
    Zern MA, Kresina TF. Hepatic drug delivery and gene therapy. Hepatology 25:484, 1997PubMedCrossRefGoogle Scholar
  60. 60.
    Arbuthnot PB, Bralet M-P, Le Jossic C, et al. In vitro and in vivo hepatoma cell-specific expression of a gene transferred with an adenoviral vector. Hum Gene Ther 7:1503, 1996PubMedCrossRefGoogle Scholar
  61. 61.
    Wu G, Wu C. Receptor-mediated gene delivery and expression in vivo. J Biol Chem 263:14621, 1988PubMedGoogle Scholar
  62. 62.
    Wu G, Wu C. Receptor-mediated in vitro gene transfections by a soluble DNA carrier system. J Biol Chem 262:4429, 1987PubMedGoogle Scholar
  63. 63.
    Neda H, Wu CH, Wu GY. Chemical modification of an ectotropic virus results in redirection of its target cell specificity. J Biol Chem 266:14143, 1991PubMedGoogle Scholar
  64. 64.
    Kasahara N, Dozy AM, Kan YW. Tissue-specific targeting of retroviral vectors through ligand-receptor interactions. Science 266:1373, 1994PubMedCrossRefGoogle Scholar
  65. 65.
    Okuyama T, Huber RM, Bowling W, et al. Liver-directed gene therapy: a retroviral vector with a complete LTR and the ApoE enhancer-α1-antitrypsin promoter dramatically increases expression of human α1-antitrypsin in vivo. Hum Gene Ther 7:637, 1996PubMedCrossRefGoogle Scholar
  66. 66.
    Sandig V, Löser P, Lieber A, Kay MA, Strauss M. HBV-derived promoters direct liver-specific expression of an adenovirally transduced LDL receptor gene. Gene Ther 3:1002, 1996PubMedGoogle Scholar
  67. 67.
    Bao J-J, Zhang W-W, Kuo MT. Adenoviral delivery of recombinant DNA into transgenic mice bearing hepatocellular carcinomas. Hum Gene Ther 7:355, 1996PubMedCrossRefGoogle Scholar
  68. 68.
    Moolten FL, Wells M. Curability of tumors bearing herpes thymidine kinase genes transferred by retroviral vectors. J Natl Cancer Inst 82:297, 1990PubMedCrossRefGoogle Scholar
  69. 69.
    Matthews T, Boehme R. Antiviral activity and mechanism of action of ganciclovir. Rev Infect Dis 10:S490, 1988PubMedCrossRefGoogle Scholar
  70. 70.
    Elshami AA, Saavedra A, Zhang H, et al. Gap junctions play a role in the ‘bystander effect’ of the herpes simplex virus thymidine kinase/ganciclovir system in vitro. Gene Ther 3:85, 1996PubMedGoogle Scholar
  71. 71.
    Kaneko S, Hallenbeck P, Kotani T, et al. Adenovirus-mediated gene therapy of hepatocellular carcinoma using cancer-specific gene expression. Cancer Res 55:5283, 1995PubMedGoogle Scholar
  72. 72.
    Su H, Chang JC, Xu SM, Kan YW. Selective killing of AFP-positive hepatocellular carcinoma cells by adeno-associated virus transfer of the herpes simplex virus thymidine kinase gene. Hum Gene Ther 7:463, 1996PubMedCrossRefGoogle Scholar
  73. 73.
    Deonarain MP, Spooner RA, Epenetos AA. Genetic delivery of enzymes for cancer therapy. Gene Ther 2:235, 1995PubMedGoogle Scholar
  74. 74.
    Huang H, Chen SH, Kosai K, Finegold MJ, Woo SLC. Gene therapy for hepatocellular carcinoma: long-term remission of primary and metastatic tumors in mice by interleukin-2 gene therapy in vivo. Gene Ther 3:980, 1996PubMedGoogle Scholar
  75. 75.
    Cao G, Kuriyama S, Du P, et al. Complete regression of established murine hepatocellular carcinoma by in vivo tumor necrosis factor α gene transfer. Gastroenterology 112:501, 1997PubMedCrossRefGoogle Scholar

Copyright information

© Springer Science+Business Media New York 1998

Authors and Affiliations

  • Dennie V. JonesJr.

There are no affiliations available

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