Post-transcriptionally regulated expression system in human xenogeneic transplantation models

Molecular Therapy

Volumn 19, Issue 9, 2011, Pages 1645-1655

  Lee, Hui Ling Rose ^a,b   Chen, Chiann Chyi ^a   Baasov, Timor ^c   Ron, Yacov ^a   Dougherty, Joseph P ^a  

^a RUTGERS ROBERT WOOD JOHNSON MEDICAL SCHOOL   (United States)

^b RUTGERS UNIVERSITY   (United States)

^c TECHNION ISRAEL INSTITUTE OF TECHNOLOGY   (Israel)

Author keywords

[No Author keywords available]

Indexed keywords

FIREFLY LUCIFERASE; GENTAMICIN; HUMAN IMMUNODEFICIENCY VIRUS 2 VECTOR; LENTIVIRUS VECTOR; PUROMYCIN; RENILLA LUCIFERIN 2 MONOOXYGENASE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL POPULATION; CONTROLLED STUDY; GENE; GENE EXPRESSION; GENE EXPRESSION REGULATION; GENE EXPRESSION SYSTEM; GENE INDUCTION; HUMAN; HUMAN CELL; IN VIVO GENE TRANSFER; MOUSE; NONHUMAN; POST TRANSCRIPTIONALLY REGULATED EXPRESSION SYSTEM; PREMATURE TERMINATION CODON; PROMOTER REGION; STOP CODON; SYNAPSIN 1 GENE; TRANSGENE; TUMOR XENOGRAFT; VIRAL GENE DELIVERY SYSTEM;

EID: 80052395852     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.90     Document Type: Article

References (34)

1. Brinster, RL, Chen, HY, Warren, R, Sarthy, A and Palmiter, RD (1982). Regulation of metallothionein-thymidine kinase fusion plasmids injected into mouse eggs. Nature 296: 39-42 (Pubitemid 12159989)
2. Gossen, M and Bujard, H (1992). Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc Natl Acad Sci USA 89: 5547-5551
3. Christopherson, KS, Mark, MR, Bajaj, V and Godowski, PJ (1992). Ecdysteroiddependent regulation of genes in mammalian cells by a Drosophila ecdysone receptor and chimeric transactivators. Proc Natl Acad Sci USA 89: 6314-6318
4. Rivera, VM, Clackson, T, Natesan, S, Pollock, R, Amara, JF, Keenan, T et al. (1996). A humanized system for pharmacologic control of gene expression. Nat Med 2: 1028-1032 (Pubitemid 26306843)
5. Murphy, GJ, Mostoslavsky, G, Kotton, DN and Mulligan, RC (2006). Exogenous control of mammalian gene expression via modulation of translational termination. Nat Med 12: 1093-1099 (Pubitemid 44353392)
6. Harrell, L, Melcher, U and Atkins, JF (2002). Predominance of six different hexanucleotide recoding signals 3' of read-through stop codons. Nucleic Acids Res 30: 2011-2017 (Pubitemid 34537598)
7. Nilsson, M and Rydén-Aulin, M (2003). Glutamine is incorporated at the nonsense codons UAG and UAA in a suppressor-free Escherichia coli strain. Biochim Biophys Acta 1627: 1-6 (Pubitemid 36570465)
8. Feng, YX, Copeland, TD, Oroszlan, S, Rein, A, Levin, JG (1990). Identification of amino acids inserted during suppression of UAA and UGA termination codons at the gag-pol junction of Moloney murine leukemia virus. Proc Natl Acad Sci USA 87: 8860-8863 (Pubitemid 120007608)
9. Urban, C, Zerfass, K, Fingerhut, C and Beier, H (1996). UGA suppression by tRNACmCATrp occurs in diverse virus RNAs due to a limited influence of the codon context. Nucleic Acids Res 24: 3424-3430 (Pubitemid 26294455)
10. Zerfass, K and Beier, H (1992). The leaky UGA termination codon of tobacco rattle virus RNA is suppressed by tobacco chloroplast and cytoplasmic tRNAs(Trp) with CmCA anticodon. EMBO J 11: 4167-4173
11. Welch, EM, Barton, ER, Zhuo, J, Tomizawa, Y, Friesen, WJ, Trifillis, P et al. (2007). PTC124 targets genetic disorders caused by nonsense mutations. Nature 447: 87-91 (Pubitemid 46685839)
12. Howard, MT, Shirts, BH, Petros, LM, Flanigan, KM, Gesteland, RF and Atkins, JF (2000). Sequence specificity of aminoglycoside-induced stop condon readthrough: potential implications for treatment of Duchenne muscular dystrophy. Ann Neurol 48: 164-169 (Pubitemid 30617034)
13. Mukherjee, S, Lee, HL, Pacchia, AL, Ron, Y and Dougherty, JP (2007). A HIV-2-based self-inactivating vector for enhanced gene transduction. J Biotechnol 127: 745-757 (Pubitemid 44959114)
14. Tannous, BA, Kim, DE, Fernandez, JL, Weissleder, R and Breakefield, XO (2005). Codon-optimized Gaussia luciferase cDNA for mammalian gene expression in culture and in vivo. Mol Ther 11: 435-443 (Pubitemid 40277320)
15. Nudelman, I, Glikin, D, Smolkin, B, Hainrichson, M, Belakhov, V and Baasov, T (2010). Repairing faulty genes by aminoglycosides: development of new derivatives of geneticin (G418) with enhanced suppression of diseases-causing nonsense mutations. Bioorg Med Chem 18: 3735-3746
16. Nudelman, I, Rebibo-Sabbah, A, Cherniavsky, M, Belakhov, V, Hainrichson, M, Chen, F et al. (2009). Development of novel aminoglycoside (NB54) with reduced toxicity and enhanced suppression of disease-causing premature stop mutations. J Med Chem 52: 2836-2845
17. Sangkuhl, K, Schulz, A, Römpler, H, Yun, J, Wess, J and Schöneberg, T (2004). Aminoglycoside-mediated rescue of a disease-causing nonsense mutation in the V2 vasopressin receptor gene in vitro and in vivo. Hum Mol Genet 13: 893-903 (Pubitemid 38628426)
18. Xi, B, Guan, F and Lawrence, DS (2004). Enhanced production of functional proteins from defective genes. J Am Chem Soc 126: 5660-5661 (Pubitemid 38621392)
19. Thorne, N, Inglese, J and Auld, DS (2010). Illuminating insights into firefly luciferase and other bioluminescent reporters used in chemical biology. Chem Biol 17: 646-657
20. Tannous, BA (2009). Gaussia luciferase reporter assay for monitoring biological processes in culture and in vivo. Nat Protoc 4: 582-591
21. Zingman, LV, Park, S, Olson, TM, Alekseev, AE and Terzic, A (2007). Aminoglycosideinduced translational read-through in disease: overcoming nonsense mutations by pharmacogenetic therapy. Clin Pharmacol Ther 81: 99-103 (Pubitemid 46050873)
22. Bar-Nun, S, Shneyour, Y and Beckmann, JS (1983). G-418, an elongation inhibitor of 80 S ribosomes. Biochim Biophys Acta 741: 123-127 (Pubitemid 13010172)
23. James, PD, Raut, S, Rivard, GE, Poon, MC, Warner, M, McKenna, S et al. (2005). Aminoglycoside suppression of nonsense mutations in severe hemophilia. Blood 106: 3043-3048 (Pubitemid 41565897)
24. Malik, V, Rodino-Klapac, LR, Viollet, L, Wall, C, King, W, Al-Dahhak, R et al. (2010). Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy. Ann Neurol 67: 771-780
25. Wilschanski, M, Yahav, Y, Yaacov, Y, Blau, H, Bentur, L, Rivlin, J et al. (2003). Gentamicin-induced correction of CFTR function in patients with cystic fibrosis and CFTR stop mutations. N Engl J Med 349: 1433-1441 (Pubitemid 37211058)
26. Hainrichson, M, Nudelman, I and Baasov, T (2008). Designer aminoglycosides: the race to develop improved antibiotics and compounds for the treatment of human genetic diseases. Org Biomol Chem 6: 227-239
27. Peltz, SW, Welch, EM, Trotta, CR, Davis, T and Jacobson, A (2009). Targeting posttranscriptional control for drug discovery. RNA Biol 6: 329-334
28. Allamand, V, Bidou, L, Arakawa, M, Floquet, C, Shiozuka, M, Paturneau-Jouas, M et al. (2008). Drug-induced readthrough of premature stop codons leads to the stabilization of laminin alpha2 chain mRNA in CMD myotubes. J Gene Med 10: 217-224 (Pubitemid 351267163)
29. Arakawa, M, Shiozuka, M, Nakayama, Y, Hara, T, Hamada, M, Kondo, S et al. (2003). Negamycin restores dystrophin expression in skeletal and cardiac muscles of mdx mice. J Biochem 134: 751-758 (Pubitemid 38031275)
30. Du, L, Damoiseaux, R, Nahas, S, Gao, K, Hu, H, Pollard, JM et al. (2009). Nonaminoglycoside compounds induce readthrough of nonsense mutations. J Exp Med 206: 2285-2297
31. Harel-Sharvit, L, Eldad, N, Haimovich, G, Barkai, O, Duek, L and Choder, M (2010). RNA polymerase II subunits link transcription and mRNA decay to translation. Cell 143: 552-563
32. Preker, P and Jensen, TH (2010). Translation by remote control. Cell 143: 501-502
33. Röther, S and Strässer, K (2007). The RNA polymerase II CTD kinase Ctk1 functions in translation elongation. Genes Dev 21: 1409-1421. (Pubitemid 46871043)
34. Yi Shu, Mathieu Cinier, Sejal R Fox, Nira Ben-Johnathan, Peixuan Guo (2011). Assembly of therapeutic pRNA-siRNA nanoparticles using bipartite approach. Mol Ther 19: 1304-1311