Spliceosome-mediated RNA Trans-splicing facilitates targeted delivery of suicide genes to cancer cells

Molecular Cancer Therapeutics

Volumn 10, Issue 2, 2011, Pages 233-241

  Gruber, Christina ^a   Gratz, Iris K ^a,e   Murauer, Eva M ^a   Mayr, Elisabeth ^a   Koller, Ulrich ^a   Bruckner Tuderman, Leena ^b   Meneguzzi, Guerrino ^c,d   Hintner, Helmut ^a   Bauer, Johann W ^a  

^a PARACELSUS MEDICAL UNIVERSITY   (Austria)

^b UNIVERSITY OF FREIBURG   (Germany)

^c INSERM   (France)

^d UNIVERSITÉ CÔTE D'AZUR   (France)

^e UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

APOPTOSIS INDUCING FACTOR; GELATINASE B; STREPTOLYSIN O;

ARTICLE; CANCER CELL; CELL DEATH; CONTROLLED STUDY; EMBRYO; EPIDERMOLYSIS BULLOSA DYSTROPHICA; GENE TARGETING; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; HUMAN TISSUE; IMMUNOFLUORESCENCE TEST; MOLECULARLY TARGETED THERAPY; PRIORITY JOURNAL; SPLICEOSOME; SQUAMOUS CELL CARCINOMA; STREPTOCOCCUS PYOGENES; SUICIDE GENE; SUICIDE GENE THERAPY; TRANS SPLICING;

BACTERIAL PROTEINS; BASE SEQUENCE; CARCINOMA, SQUAMOUS CELL; CELL LINE; CELL SURVIVAL; EPIDERMOLYSIS BULLOSA DYSTROPHICA; FLOW CYTOMETRY; GENE ORDER; GENE TRANSFER TECHNIQUES; GENES, TRANSGENIC, SUICIDE; HEK293 CELLS; HUMANS; MATRIX METALLOPROTEINASE 9; MICROSCOPY, FLUORESCENCE; MOLECULAR SEQUENCE DATA; SPLICEOSOMES; STREPTOLYSINS; TRANS-SPLICING;

EID: 79951834142     PISSN: 15357163     EISSN: 15388514     Source Type: Journal    
DOI: 10.1158/1535-7163.MCT-10-0669     Document Type: Article

References (30)

1. Lanschuetzer CM, Fine JD. Classification and molecular basis of hereditary epidermolysis bullosa. In:Fine JD, Hintner H, editors. Life with Epidermolysis Bullosa (EB): Etiology, Diagnosis, Multidisciplinary Patient Care, and Therapy. New York: Wien Springer; 2008. p.6-24.
2. Fine JD, Johnson LB, Weiner M, Li KP, Suchindran C. Epidermolysis bullosa and the risk of life-threatening cancers: The National EB Registry experience, 1986-2006. J Am Acad Dermatol 2009;60: 203-11.
3. Mansfield SG, Chao H, Walsh CE. RNA repair using spliceosome-mediated RNA trans-splicing. Trends Mol Med 2004;10:263-8. (Pubitemid 38725893)
4. Chen HY, Kathirvel P, Yee WC, Lai PS. Correction of dystrophia myotonica type 1 pre-mRNA transcripts by artificial trans-splicing. Gene Ther 2009;16:211-7.
5. Murauer EM, Gache Y, Gratz IK, Klausegger A, Muss W, Gruber C, et al. Functional correction of type VII collagen expression in dystrophic epidermolysis bullosa. J Invest Dermatol 2011;131: 74-83.
6. Wally V, Klausegger A, Koller U, Lochmuller H, Krause S, Wiche G, et al. 50 trans-splicing repair of the PLEC1 gene. J Invest Dermatol 2008;128:568-74.
7. Wang J, Mansfield SG, Cote CA, Du Jiang P, Weng K, Amar MJA, et al. Trans-splicing into highly abundant albumin transcripts for production of therapeutic proteins in vivo. Mol Ther 2009;17:343-51.
8. Garcia-Blanco MA, Puttaraju M, Mansfield SG, Mitchell LG. Spliceosome- mediated RNA trans-splicing in gene therapy and genomics. Gen Ther Reg 2000;1:141-63.
9. Gottesman MM. Cancer gene therapy: an awkward adolescence. Cancer Gene Ther 2003;10:501-8. (Pubitemid 36842007)
10. Natsume A, Yoshida J. Gene therapy for high-grade glioma: current approaches and future directions. Cell Adh Migr 2008;2:186-91.
11. Xu F, Li S, Li XL, Guo Y, Zou BY, Xu R, et al. Phase I and biodistribution study of recombinant adenovirus vector-mediated herpes simplex virus thymidine kinase gene and ganciclovir administration in patients with head and neck cancer and other malignant tumors. Cancer Gene Ther 2009;16:723-30.
12. Puttaraju M, Jamison SF, Mansfield SG, Garcia-Blanco MA, Mitchell LG. Spliceosome-mediated RNA trans-splicing as a tool for gene therapy. Nat Biotechnol 1999;17:246-52. (Pubitemid 29122632)
13. Dallinger G, Puttaraju M, Mitchell LG, Yancey KB, Yee C, Klausegger A, et al. Development of spliceosome-mediated RNA trans-splicing (SMaRT) for the correction of inherited skin diseases. Exp Dermatol 2003;12:37-46. (Pubitemid 36934688)
14. Uphoff CC, Drexler HG. Comparative PCR analysis for detection of mycoplasma infections in continuous cell lines. In Vitro Cell Dev Biol Anim 2002;38:79-85. (Pubitemid 34538199)
15. Yang WS, Park SO, Yoon AR, Yoo JY, Kim MK, Yun CO, et al. Suicide cancer gene therapy using pore-forming toxin, streptolysin O. Mol Cancer Ther 2006;5:1610-9. (Pubitemid 44070499)
16. Kivisaari AK, Kallajoki M, Mirtti T, McGrath JA, Bauer JW, Weber F, et al. Transformation-specific matrix metalloproteinases (MMP)-7 and MMP-13 are expressed by tumour cells in epidermolysis bullosa-associated squamous cell carcinomas. Br J Dermatol 2008;158:778-85. (Pubitemid 351415753)
17. Mitchell LG, McGarrity GJ. Gene therapy progress and prospects: reprograming gene expression by trans-splicing. Gene Ther 2005;12:1477-85. (Pubitemid 41508672)
18. Bhakdi S, Bayley H, Valeva A, Walev I, Walker B, Kehoe M, et al. Staphylococcal alpha-toxin, streptolysin-O, and Escherichia coli hemolysin: prototypes of pore-forming bacterial cytolysins. Arch Microbiol 1996;165:73-9. (Pubitemid 26081834)
19. Chakraborti S, Mandal M, Das S, Mandal A, Chakraborti T. Regulation of matrix metalloproteinases: an overview. Mol Cell Biochem 2003;253:269-85.
20. Rodriguez-Martin T, Garcia-Blanco MA, Mansfield SG, Grover AC, Hutton M, Yu Q, et al. Reprogramming of tau alternative splicing by spliceosome-mediated RNA trans-splicing: implications for tauopathies. Proc Natl Acad Sci U S A 2005;102:15659-64. (Pubitemid 41528112)
21. Zayed H, Xia L, Yerich A, Yant SR, Kay MA, Puttaraju M, et al. Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery. Mol Ther 2007;15:1273-9. (Pubitemid 46965323)
22. Liu X, Jiang Q, Mansfield SG, Puttaraju M, Zhang Y, Zhou W, et al. Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing. Nat Biotechnol 2002;20:47-52. (Pubitemid 34044914)
23. Thomas SM, Grandis JR. The current state of head and neck cancer gene therapy. Hum Gene Ther 2009;20:1565-75.
24. Chao H, Mansfield SG, Bartel RC, Hiriyanna S, Mitchell LG, Garcia- Blanco MA, et al. Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing. Nat Med 2003;9:1015-9. (Pubitemid 37025113)
25. Puttaraju M, DiPasquale J, Baker CC, Mitchell LG, Garcia-Blanco MA. Messenger RNA repair and restoration of protein function by spliceosome- mediated RNA trans-splicing. Mol Ther 2001;4:105-14. (Pubitemid 32776501)
26. Yamaizumi M, Mekada E, Uchida T, Okada Y. One molecule of diphtheria toxin fragment A introduced into a cell can kill the cell. Cell 1978;15:245-50. (Pubitemid 9027527)
27. Wanebo HJ, Blackinton D, Kouttab N, Mehta S. Contribution of serum inhibitory factors and immune cellular defects to the depressed cell-mediated immunity in patients with head and neck cancer. Am J Surg 1993;166:389-94. (Pubitemid 24240994)
28. Whiteside TL. Immunobiology of head and neck cancer. Cancer Metastasis Rev 2005;24:95-105. (Pubitemid 40576066)
29. Matzinger P. An innate sense of danger. Semin Immunol 1998; 10:399-415. (Pubitemid 28499554)
30. Hobohm U, Stanford JL, Grange JM. Pathogen-associated molecular pattern in cancer immunotherapy. Crit Rev Immunol 2008;28:95-107. (Pubitemid 351507428)