Validation of tissue-specific promoter-driven tumor-targeting trans-splicing ribozyme system as a multifunctional cancer gene therapy device in vivo

Cancer Gene Therapy

Volumn 16, Issue 2, 2009, Pages 113-125

  Song, M S ^a   Jeong, J S ^a,b   Ban, G ^a   Lee, J H ^a   Won, Y S ^a   Cho, K S ^b   Kim, I H ^c   Lee, S W ^a  

^a Dankook University   (South Korea)

^b Dong A University College of Medicine   (South Korea)

^c National Cancer Center   (South Korea)

Author keywords

Hepatocellular carcinoma; hTERT; Tissue selective promoter; trans splicing ribozyme

Indexed keywords

ADENOVIRUS VECTOR; BETA GALACTOSIDASE; GANCICLOVIR; PHOSPHOENOLPYRUVATE CARBOXYKINASE (GTP); RIBOZYME; TELOMERASE REVERSE TRANSCRIPTASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CANCER GENE THERAPY; CANCER INHIBITION; CARCINOMATOUS PERITONITIS; CONTROLLED STUDY; DRUG TARGETING; HELA CELL; HISTOPATHOLOGY; HUMAN; HUMAN TISSUE; LIVER TOXICITY; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; RNA SPLICING; TISSUE SPECIFICITY; TRANSSEXUALISM; TUMOR XENOGRAFT; VALIDATION STUDY;

ADENOVIRIDAE; ANIMALS; CARCINOMA, HEPATOCELLULAR; CELL LINE, TUMOR; GENE EXPRESSION REGULATION, NEOPLASTIC; GENE THERAPY; GENETIC VECTORS; HELA CELLS; HUMANS; LIVER NEOPLASMS; MALE; MICE; MICE, INBRED BALB C; MICE, NUDE; RNA, CATALYTIC; RNA, NEOPLASM; TELOMERASE; TRANS-SPLICING;

ADENOVIRIDAE; MUS; MUS MUSCULUS;

EID: 58349111839     PISSN: 09291903     EISSN: 14765500     Source Type: Journal    
DOI: 10.1038/cgt.2008.64     Document Type: Article

References (39)

1. Sullenger BA, Cech TR. Ribozyme-mediated repair of defective mRNA by targeted, trans-splicing. Nature 1994; 371: 619-622.
2. Jones JT, Lee SW, Sullenger BA. Tagging ribozyme reaction sites to follow trans-splicing in mammalian cells. Nat Med 1996; 2: 643-648.
3. Lewin AS, Hauswirth WW. Ribozyme gene therapy: applications for molecular medicine. Trends Mol Med 2001; 7: 221-228.
4. Long MB, Jones III JP, Sullenger BA, Byun J. Ribozyme-mediated revision of RNA and DNA. J Clin Invest 2003; 112: 312-318.
5. Fiammengo R, Jaschke A. Nucleic acid enzymes. Curr Opin Biotechnol 2005; 16: 614.
6. Lan N, Howrey RP, Lee SW, Smith CA, Sullenger BA. Ribozyme-mediated repair ofsickle beta-globin mRNAs in erythrocyte precursors. Science 1998; 280: 1593-1596.
7. Phylactou LA, Darrah C, Wood MJ. Ribozyme-mediated trans-splicing ofa trinucleotide repeat. Nat Genet 1998; 18: 378-381.
8. Rogers CS, Vanoye CG, Sullenger BA, George Jr AL. Functional repair of a mutant chloride channel using a trans-splicing ribozyme. J Clin Invest 2002; 110: 1783-1789.
9. Shin KS, Sullenger BA, Lee SW. Ribozyme-mediated induction of apoptosis in human cancer cells by targeted repair ofmutant p53 RNA. Mol Ther 2004; 10: 365-372.
10. Kastanos E, Hjiantoniou E, Phylactou LA. Restoration of protein synthesis in pancreatic cancer cells by trans-splicing ribozymes. Biochem Biophys Res Commun 2004; 322: 930-934.
11. Kwon BS, Jung HS, Song MS, Cho KS, Kim SC, Kimm K et al. Specific regression of human cancer cells by ribozyme-mediated targeted replacement of tumor-specific transcript. Mol Ther 2005; 12: 824-834.
12. Hong SH, Jeong JS, Lee YJ, Jung HI, Cho KS, Kim CM et al. In vivo Reprogramming of hTERT by trans-splicing ribozyme to target tumor cells. Mol Ther 2008; 16: 74-80.
13. Jeong JS, Lee SW, Hong SH, Lee YJ, Jung HI, Cho KS et al. Antitumor effects of systemically delivered adenovirus harboring trans-splicing ribozyme in intrahepatic colon cancer mouse model. Clin Cancer Res 2008; 14: 281-290.
14. Thomson JA, Itskovitz-Eldor J, Shapiro SS, Waknitz MA, Swiergiel JJ, Marshall VS et al. Embryonic stem cell lines derived from human blastocysts. Science 1998; 282: 1145-1147.
15. Yui J, Chiu CP, Lansdorp PM. Telomerase activity in candidate stem cells from fetal liver and adult bone marrow. Blood 1998; 91: 3255-3262.
16. Forsyth NR, Wright WE, Shay JW. Telomerase and differentiation in multicellular organisms: turn it off, turn it on, and turn it off again. Differentiation 2002; 69: 188-197.
17. Kotoula V, Hytiroglou P, Pyrpasopoulou A, Saxena R, Thung SN, Papadimitriou CS. Expression of human telomerase reverse transcriptase in regenerative and precancerous lesions of cirrhotic livers. Liver 2002; 22: 57-69.
18. Fukazawa T, Maeda Y, Sladek FM, Owen-Schaub LB. Development of a cancer-targeted tissue-specific promoter system. Cancer Res 2004; 64: 363-369.
19. Song MS, Lee SW. Cancer-selective induction ofcytotoxicity by tissue-specific expression of targeted trans-splicing ribozyme. FEBS Lett 2006; 580: 5033-5043.
20. Kim NW, Piatyszek MA, Prowse KR, Harley CB, West MD, Ho PL et al. Specific association of human telomerase activity with immortal cells and cancer. Science 1994; 266: 2011-2015.
21. Meyerson M, Counter CM, Eaton EN, Ellisen LW, Steiner P, Caddle SD et al. hEST2, the putative human telomerase catalytic subunit gene, is up-regulated in tumor cells and during immortalization. Cell 1997; 90: 785-795.
22. Smith LL, Coller HA, Roberts JM. Telomerase modulates expression of growth-controlling genes and enhances cell proliferation. Nat Cell Biol 2003; 5: 474-479.
23. Roesler WJ, McFie PJ, Dauvin C. The liver-enriched transcription factor D-site-binding protein activates the promoter ofthe phosphoenolpyruvate carboxykinase gene in hepatoma cells. J Biol Chem 1992; 267: 21235-21243.
24. Jaffe HA, Danel C, Longenecker G, Metzger M, Setoguchi Y, Rosenfeld MA et al. Adenovirus-mediated in vivo gene transfer and expression in normal rat liver. Nat Genet 1992; 1: 372-378.
25. Hendrie PC, Russell DW. Gene targeting with viral vectors. Mol Ther 2005; 12: 9-17.
26. Homma H, Doi T, Mezawa S, Takada K, Kukitsu T, Oku T et al. A novel arterial infusion chemotherapy for the treatment of patients with advanced pancreatic carcinoma after vascular supply distribution via superselective embolization. Cancer 2000; 89: 303-313.
27. Crettaz J, Berraondo P, Mauleon I, Ochoa L, Shankar V, Barajas M et al. Intrahepatic injection of adenovirus reduces inflammation and increases gene transfer and therapeutic effect in mice. Hepatology 2006; 44: 623-632.
28. Lee JS, Thorgeirsson SS. Genome-scale profiling of gene expression in hepatocellular carcinoma: classification, survival prediction, and identification of therapeutic targets. Gastroenterology 2004; 127: S51-S55.
29. Greenberg RA, Allsopp RC, Chi L, Morin GB, Dephino RA. Expression of mouse telomerase reverse transcriptase during development differentiation and proliferation. Oncogene 1998; 16: 1723-1730.
30. Horikawa I, Chiang YJ, Patterson T, Feigenbaum L, Leem SH, Michishita E et al. Differential cis-regulation of human versus mouse TERT gene expression in vivo: identification of a human-specific repressive element. Proc Natl Acad Sci USA 2005; 102: 18437-18442.
31. Sadeghi H, Hitt MM. Transcriptionally targeted adenovirus vectors. Curr Gene Ther 2005; 5: 411-427.
32. Wu L, Johnson M, Sato M. Transcriptionally targeted gene therapy to detect and treat cancer. Trends Mol Med 2003; 9: 421-429.
33. Folini M, Berg K, Millo E, Villa R, Prasmickaite L, Daidone MG et al. Photochemical internalization of a peptide nucleic acid targeting the catalytic subunit of human telomerase. Cancer Res 2003; 63: 3490-3494.
34. Li S, Rosenberg JE, Donjacour AA, Botchkina IL, Hom YK, Cunha GR et al. Rapid inhibition of cancer cell growth induced by lentiviral delivery and expression of mutant template telomerase RNA and anti-telomerase short-interfering RNA. Cancer Res 2004; 64: 4833-4840.
35. Li S, Crothers J, Haqq CM, Blackburn EH. Cellular and gene expression responses involved in the rapid growth inhibition of human cancer cells by RNA interference mediated depletion of telomerase RNA. J Biol Chem 2005; 280: 23709-23717.
36. White LK, Wright WE, Shay JW. Telomerase inhibitors. Trends Biotechnol 2001; 19: 114-120.
37. Jung HS, Lee SW. Ribozyme-mediated selective killing of cancer cells expressing carcinoembryonic antigen RNA by targeted trans-splicing. Biochem Biophys Res Commun 2006; 349: 556-563.
38. Won YS, Lee SW. Targeted retardation of hepatocarcinoma cells by specific replacement of alpha-fetoprotein RNA. J Biotechnol 2007; 129: 614-619.
39. Kim A, Bam G, Song MS, Bae CD, Park J, Lee SW. Selective regression of cells expressing mouse cytoskeleton-associated protein 2 transcript by trans-splicing ribozyme. Oligonucleotides 2007; 17: 95-103.