Toxin-Based Cancer Gene Therapy: Under the Control of Oncofetal H19 Regulatory Sequences

Gene Therapy of Cancer: Translational Approaches from Preclinical Studies to Clinical Implementation: Third Edition

Volumn , Issue , 2013, Pages 107-122

  Ohana, Patricia ^a   Matouk, Imad ^a   Amit, Doron ^a   Gilon, Michal ^a   Hochberg, Abraham ^a  

^a HEBREW UNIVERSITY OF JERUSALEM   (Israel)

Author keywords

Cancer; Diphtheria toxin; DTA H19 plasmid; H19; Hypoxia; Oncofetal; Transcriptional targeting; Tumor specific promoter

Indexed keywords

EID: 84904084903     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1016/B978-0-12-394295-1.00008-1     Document Type: Chapter

References (59)

1. Szakacs G., Paterson J.K., Ludwig J.A., Booth-Genthe C., Gottesman M.M. Targeting multidrug resistance in cancer. Nat Rev Drug Discov 2006, 5(3):219-234.
2. Nettelbeck D.M., Jerome V., Muller R. Gene therapy: designer promoters for tumour targeting. Trends Genet 2000, 16(4):174-181.
3. Weiner L.M., Surana R., Wang S. Monoclonal antibodies: versatile platforms for cancer immunotherapy. Nat Rev 2010, 10(5):317-327.
4. Banet G., Bibi O., Matouk I., Ayesh S., Laster M., Kimber K.M., et al. Characterization of human and mouse H19 regulatory sequences. Mol Biol Rep 2000, 27(3):157-165.
5. Sidi A.A., Ohana P., Benjamin S., Shalev M., Ransom J.H., Lamm D., et al. Phase I/II marker lesion study of intravesical BC-819 DNA plasmid in H19 overexpressing superficial bladder cancer refractory to bacillus Calmette-Guerin. J Urol 2008, 180(6):2379-2383.
6. Amit D., Hochberg A. Development of targeted therapy for bladder cancer mediated by a double promoter plasmid expressing diphtheria toxin under the control of H19 and IGF2-P4 regulatory sequences. J Transl Med 2010, 8(1):134.
7. Amit D., Tamir S., Birman T., Gofrit O.N., Hochberg A. Development of targeted therapy for bladder cancer mediated by a double promoter plasmid expressing diphtheria toxin under the control of IGF2-P3 and IGF2-P4 regulatory sequences. Int J Clin Exp Med 2011, 4(2):91-102.
8. Ohana P., Gofrit O., Ayesh S., Al-Sahref W., Mizrahi A., Birman T., et al. Regulatory sequences of the H19 gene in DNA therapy of bladder cancer. Gene Ther & Mol Biol 2004, 8:181-192.
9. Pachnis V., Belayew A., Tilghman S.M. Locus unlinked to alpha-fetoprotein under the control of the murine raf and Rif genes. Proc Natl Acad Sci USA 1984, 81(17):5523-5527.
10. Ariel I., Ayesh S., Perlman E.J., Pizov G., Tanos V., Schneider T., et al. The product of the imprinted H19 gene is an oncofetal RNA. Mol Pathol 1997, 50(1):34-44.
11. Pachnis V., Brannan C.I., Tilghman S.M. The structure and expression of a novel gene activated in early mouse embryogenesis. EMBO J 1988, 7(3):673-681.
12. Yoshimizu T., Miroglio A., Ripoche M.A., Gabory A., Vernucci M., Riccio A., et al. The H19 locus acts in vivo as a tumor suppressor. Proc Natl Acad Sci USA 2008, 105(34):12417-12422.
13. Cai X., Cullen B.R. The imprinted H19 noncoding RNA is a primary microRNA precursor. RNA 2007, 13(3):313-316.
14. Tsang W.P., Ng E.K., Ng S.S., Jin H., Yu J., Sung J.J., et al. Oncofetal H19-derived miR-675 regulates tumor suppressor RB in human colorectal cancer. Carcinogenesis 2010, 31(3):350-358.
15. Berteaux N., Aptel N., Cathala G., Genton C., Coll J., Daccache A., et al. A novel H19 antisense RNA overexpressed in breast cancer contributes to paternal IGF2 expression. Mol Cell Biol 2008, 28(22):6731-6745.
16. Onyango P., Feinberg A.P. A nucleolar protein, H19 opposite tumor suppressor (HOTS), is a tumor growth inhibitor encoded by a human imprinted H19 antisense transcript. Proc Natl Acad Sci USA 2011, 108(40):16759-16764.
17. Matouk I., Ohana P., Ayesh S., Sidi A., Czerniak A., de Groot N., Hochberg A. The oncofetal H19 RNA in human cancer, from the bench to the patient. Cancer Ther 2005, 3:249-266.
18. Matouk I., Ohana P., Galun E., Hochberg A. The pivotal role of the oncofetal H19 RNA in human cancer: a new hope. Gene therapy and cancer research focus 2008, Nova publisher, :241-60.
19. Matouk I.J., DeGroot N., Mezan S., Ayesh S., Abu-lail R., Hochberg A., et al. The H19 non-coding RNA is essential for human tumor growth. PLoS ONE 2007, 2(9):e845.
20. Gabory A., Jammes H., Dandolo L. The H19 locus: role of an imprinted non-coding RNA in growth and development. Bioessays 2010, 32(6):473-480.
21. Matouk I.J., Mezan S., Mizrahi A., Ohana P., Abu-Lail R., Fellig Y., et al. The oncofetal H19 RNA connection: hypoxia, p53 and cancer. Biochim Biophys Acta 2010, 1803(4):443-451.
22. Ayesh S., Matouk I., Schneider T., Ohana P., Laster M., Al-Sharef W., et al. Possible physiological role of H19 RNA. Mol Carcinog 2002, 35(2):63-74.
23. Dugimont T., Curgy J.J., Wernert N., Delobelle A., Raes M.B., Joubel A., et al. The H19 gene is expressed within both epithelial and stromal components of human invasive adenocarcinomas. Biol Cell 1995, 85(2-3):117-124.
24. Post D.E., Van Meir E.G. A novel hypoxia-inducible factor (HIF) activated oncolytic adenovirus for cancer therapy. Oncogene 2003, 22(14):2065-2072.
25. Harrington K.J., Linardakis E., Vile R.G. Transcriptional control: an essential component of cancer gene therapy strategies?. Adv Drug Deliv Rev 2000, 44(2-3):167-184.
26. Hernandez-Alcoceba R., Pihalja M., Wicha M.S., Clarke M.F. A novel, conditionally replicative adenovirus for the treatment of breast cancer that allows controlled replication of E1a-deleted adenoviral vectors. Hum Gene Ther 2000, 11(14):2009-2024.
27. Nielsen O.S., Horsman M., Overgaard J. A future for hyperthermia in cancer treatment?. Eur J Cancer 2001, 37(13):1587-1589.
28. Robson T., Hirst D.G. Transcriptional targeting in cancer gene therapy. J Biomed Biotechnol 2003, 2003(2):110-137.
29. Abdul-Ghani R., Ohana P., Matouk I., Ayesh S., Ayesh B., Laster M., et al. Use of transcriptional regulatory sequences of telomerase (hTER and hTERT) for selective killing of cancer cells. Mol Ther 2000, 2(6):539-544.
30. Komata T., Kondo Y., Kanzawa T., Hirohata S., Koga S., Sumiyoshi H., et al. Treatment of malignant glioma cells with the transfer of constitutively active caspase-6 using the human telomerase catalytic subunit (human telomerase reverse transcriptase) gene promoter. Cancer Res 2001, 61(15):5796-5802.
31. Ayesh B., Matouk I., Ohana P., Sughayer M.A., Birman T., Ayesh S., et al. Inhibition of tumor growth by DT-A expressed under the control of IGF2 P3 and P4 promoter sequences. Mol Ther 2003, 7(4):535-541.
32. Wu L., Matherly J., Smallwood A., Adams J.Y., Billick E., Belldegrun A., et al. Chimeric PSA enhancers exhibit augmented activity in prostate cancer gene therapy vectors. Gene Ther 2001, 8(18):1416-1426.
33. Li Y., McCadden J., Ferrer F., Kruszewski M., Carducci M., Simons J., et al. Prostate-specific expression of the diphtheria toxin A chain (DT-A): studies of inducibility and specificity of expression of prostate-specific antigen promoter-driven DT-A adenoviral-mediated gene transfer. Cancer Res 2002, 62(9):2576-2582.
34. Kawakami K., Kawakami M., Joshi B.H., Puri R.K. Interleukin-13 receptor-targeted cancer therapy in an immunodeficient animal model of human head and neck cancer. Cancer Res 2001, 61(16):6194-6200.
35. Martin V., Cortes M.L., de Felipe P., Farsetti A., Calcaterra N.B., Izquierdo M. Cancer gene therapy by thyroid hormone-mediated expression of toxin genes. Cancer Res 2000, 60(12):3218-3224.
36. Deharvengt S., Wack S., Aprahamian M., Hajri A. Transcriptional tumor-selective promoter targeting of E. coli purine nucleoside phosphorylase for pancreatic cancer suicide gene therapy. J Gene Med 2005, 7(5):672-680.
37. Rodriguez R., Lim H.Y., Bartkowski L.M., Simons J.W. Identification of diphtheria toxin via screening as a potent cell cycle and p53-independent cytotoxin for human prostate cancer therapeutics. Prostate 1998, 34(4):259-269.
38. Mizrahi A., Hochberg A., Amiur S., Gallula J., Matouk I., Birman T., et al. Targeting diphtheria toxin and TNF alpha expression in ovarian tumors using the H19 regulatory sequences. Int J Clin Exp Med 2010, 3(4):270-282.
39. Marais R., Spooner R.A., Light Y., Martin J., Springer C.J. Gene-directed enzyme prodrug therapy with a mustard prodrug/carboxypeptidase G2 combination. Cancer Res 1996, 56(20):4735-4742.
40. McCormick F. Cancer gene therapy: fringe or cutting edge?. Nat Rev Cancer 2001, 1(2):130-141.
41. Kirn D., Niculescu-Duvaz I., Hallden G., Springer C.J. The emerging fields of suicide gene therapy and virotherapy. Trends Mol Med 2002, 8(4 Suppl):S68-S73.
42. Ohana P., Bibi O., Matouk I., Levy C., Birman T., Ariel I., et al. Use of H19 regulatory sequences for targeted gene therapy in cancer. Int J Cancer 2002, 98(5):645-650.
43. Scaiewicz V., Sorin V., Fellig Y., Birman T., Mizrahi A., Galula J., et al. Use of H19 gene regulatory sequences in DNA-based therapy for pancreatic cancer. J Oncol 2010, 2010:178174.
44. Mizrahi A., Czerniak A., Ohana P., Amiur S., Gallula J., Matouk I., et al. Treatment of ovarian cancer ascites by intra-peritoneal injection of diphtheria toxin A chain-H19 vector: a case report. J Med Case Reports 2010, 4:228.
45. Fellig Y., Ariel I., Ohana P., Schachter P., Sinelnikov I., Birman T., et al. H19 expression in hepatic metastases from a range of human carcinomas. J Clin Pathol 2005, 58(10):1064-1068.
46. Ohana P., Schachter P., Ayesh B., Mizrahi A., Birman T., Schneider T., et al. Regulatory sequences of H19 and IGF2 genes in DNA-based therapy of colorectal rat liver metastases. J Gene Med 2005, 7(3):366-374.
47. Sorin V., Ohana P., Mizrahi A., Matouk I., Birman T., Hochberg A., et al. Regional therapy with DTA-H19 vector suppresses growth of colon adenocarcinoma metastases in the rat liver. Int J Oncol 2011, 39(6):1407-1412.
48. Mizrahi A., Czerniak A., Levy T., Amiur S., Gallula J., Matouk I., et al. Development of targeted therapy for ovarian cancer mediated by a plasmid expressing diphtheria toxin under the control of H19 regulatory sequences. J Transl Med 2009, 7:69.
49. Ohana P., Kopf E., Bibi O., Ayesh S., Schneider T., Laster M., et al. The expression of the H19 gene and its function in human bladder carcinoma cell lines. FEBS Lett 1999, 454(1-2):81-84.
50. Lansigan F., Stearns D.M., Foss F. Role of denileukin diftitox in the treatment of persistent or recurrent cutaneous T-cell lymphoma. Cancer Manage Res 2010, 2:53-59.
51. Hine C.M., Seluanov A., Gorbunova V. Rad51 promoter-targeted gene therapy is effective for in vivo visualization and treatment of cancer. Mol Ther 2011, 20:347-355.
52. Huang Y.H., Zugates G.T., Peng W., Holtz D., Dunton C., Green J.J., et al. Nanoparticle-delivered suicide gene therapy effectively reduces ovarian tumor burden in mice. Cancer Res 2009, 69(15):6184-6191.
53. Ming Cai W.H., Luo W.e.i., Pan S.h.e.n.g., Yin T.a.o. Specific killing effect of diphtheria toxin A fragment under control of DF3 promotor on human breast cancer cells. Chinese-German J Clin Oncol 2007, 6(2):200-203.
54. Horita H., Thorburn J., Frankel A.E., Thorburn A. EGFR-targeted diphtheria toxin stimulates TRAIL killing of glioblastoma cells by depleting anti-apoptotic proteins. J Neurooncol 2009, 95(2):175-184.
55. Kunitomi M., Takayama E., Suzuki S., Yasuda T., Tsutsui K., Nagaike K., et al. Selective inhibition of hepatoma cells using diphtheria toxin A under the control of the promoter/enhancer region of the human alpha-fetoprotein gene. Jpn J Cancer Res 2000, 91(3):343-350.
56. Maxwell I.H., Kaletta C., Naujoks K., Maxwell F. Targeting diphtheria toxin A-chain transcription to activated endothelial cells using an E-selectin promoter. Angiogenesis 2003, 6(1):31-38.
57. Lidor Y.J., Lee W.E., Nilson J.H., Maxwell I.H., Su L.J., Brand E., et al. In vitro expression of the diphtheria toxin A-chain gene under the control of human chorionic gonadotropin gene promoters as a means of directing toxicity to ovarian cancer cell lines. Am J Obstet Gynecol 1997, 177(3):579-585.
58. Showalter S.L., Huang Y.H., Witkiewicz A., Costantino C.L., Yeo C.J., Green J.J., et al. Nanoparticulate delivery of diphtheria toxin DNA effectively kills Mesothelin expressing pancreatic cancer cells. Cancer Biol Ther 2008, 7(10):1584-1590.
59. Peng W., Anderson D.G., Bao Y., Padera R.F., Langer R., Sawicki J.A. Nanoparticulate delivery of suicide DNA to murine prostate and prostate tumors. Prostate 2007, 67(8):855-862.