Adeno-associated virus-mediated, mifepristone-regulated transgene expression in the brain

Molecular Therapy Nucleic Acids

Volumn 2, Issue , 2013, Pages e106-

  Maddalena, Andrea ^a   Tereshchenko, Julia ^a   Bähr, Mathias ^a   Kügler, Sebastian ^a  

^a UNIVERSITY MEDICAL CENTER GÖTTINGEN   (Germany)

Author keywords

AAV; Gene Switch; Regulated expression

Indexed keywords

ENHANCED GREEN FLUORESCENT PROTEIN; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; MIFEPRISTONE; PARVOVIRUS VECTOR; SYNAPSIN I;

ADENO ASSOCIATED VIRUS; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASTROCYTE; BRAIN; GENE EXPRESSION; GENE SWITCHING; GENETIC TRANSDUCTION; GENOME; GLIA CELL; IMMUNOREACTIVITY; IN VITRO STUDY; INVERTED TERMINAL REPEAT; NEUROPROTECTION; NONHUMAN; PARKINSON DISEASE; PRIORITY JOURNAL; PROTEIN EXPRESSION; RAT; TISSUE REACTION; TRANSGENE;

EID: 84880390816     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2013.35     Document Type: Article

References (34)

1. Bartus, RT, Herzog, CD, Chu, Y, Wilson, A, Brown, L, Siffert, J et al. (2011). Bioactivity of AAV2-neurturin gene therapy (CERE-120): Differences between Parkinson's disease and nonhuman primate brains. Mov Disord 26: 27-36.
2. Manfredsson, FP, Bloom, DC and Mandel, RJ (2012). Regulated protein expression for in vivo gene therapy for neurological disorders: Progress, strategies, and issues. Neurobiol Dis 48: 212-221.
3. Naidoo, J and Young, D (2012). Gene regulation systems for gene therapy applications in the central nervous system. Neurol Res Int 2012: 595410.
4. Schönig, K, Bujard, H and Gossen, M (2010). The power of reversibility regulating gene activities via tetracycline-controlled transcription. Meth Enzymol 477: 429-453.
5. Stieger, K, Belbellaa, B, Le Guiner, C, Moullier, P and Rolling, F (2009). In vivo gene regulation using tetracycline-regulatable systems. Adv Drug Deliv Rev 61: 527-541.
6. Laurenti, E, Barde, I, Verp, S, Offner, S, Wilson, A, Quenneville, S et al. (2010). Inducible gene and shRNA expression in resident hematopoietic stem cells in vivo. Stem Cells 28: 1390-1398.
7. Stieger, K, Le Meur, G, Lasne, F, Weber, M, Deschamps, JY, Nivard, D et al. (2006). Long-term doxycycline-regulated transgene expression in the retina of nonhuman primates following subretinal injection of recombinant AAV vectors. Mol Ther 13: 967-975.
8. Latta-Mahieu, M, Rolland, M, Caillet, C, Wang, M, Kennel, P, Mahfouz, I et al. (2002). Gene transfer of a chimeric trans-activator is immunogenic and results in short-lived transgene expression. Hum Gene Ther 13: 1611-1620. (Pubitemid 35034681)
9. Ginhoux, F, Turbant, S, Gross, DA, Poupiot, J, Marais, T, Lone, Y et al. (2004). HLA-A*0201-restricted cytolytic responses to the rtTA transactivator dominant and cryptic epitopes compromise transgene expression induced by the tetracycline on system. Mol Ther 10: 279-289. (Pubitemid 39127368)
10. Xu, ZL, Mizuguchi, H, Mayumi, T and Hayakawa, T (2003). Regulated gene expression from adenovirus vectors: A systematic comparison of various inducible systems. Gene 309: 145-151. (Pubitemid 36577510)
11. Rauschhuber, C, Noske, N and Ehrhardt, A (2012). New insights into stability of recombinant adenovirus vector genomes in mammalian cells. Eur J Cell Biol 91: 2-9.
12. Clark, KR and Penaud-Budloo, M (2011). Evaluation of the fate of rAAV genomes following in vivo administration. Methods Mol Biol 807: 239-258.
13. Lee, Y, Messing, A, Su, M and Brenner, M (2008). GFAP promoter elements required for region-specific and astrocyte-specific expression. Glia 56: 481-493.
14. Flotte, TR, Afione, SA, Solow, R, Drumm, ML, Markakis, D, Guggino, WB et al. (1993). Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter. J Biol Chem 268: 3781-3790. (Pubitemid 23071029)
15. Blits, B, Derks, S, Twisk, J, Ehlert, E, Prins, J and Verhaagen, J (2010). Adeno-associated viral vector (AAV)-mediated gene transfer in the red nucleus of the adult rat brain: Comparative analysis of the transduction properties of seven AAV serotypes and lentiviral vectors. J Neurosci Methods 185: 257-263.
16. Drinkut, A, Tereshchenko, Y, Schulz, JB, Bähr, M and Kügler, S (2012). Efficient gene therapy for Parkinson's disease using astrocytes as hosts for localized neurotrophic factor delivery. Mol Ther 20: 534-543.
17. Kirik, D, Rosenblad, C and Björklund, A (1998). Characterization of behavioral and neurodegenerative changes following partial lesions of the nigrostriatal dopamine system induced by intrastriatal 6-hydroxydopamine in the rat. Exp Neurol 152: 259-277. (Pubitemid 28419264)
18. Sajadi, A, Bauer, M, Thöny, B and Aebischer, P (2005). Long-term glial cell line-derived neurotrophic factor overexpression in the intact nigrostriatal system in rats leads to a decrease of dopamine and increase of tetrahydrobiopterin production. J Neurochem 93: 1482-1486. (Pubitemid 40853271)
19. Georgievska, B, Jakobsson, J, Persson, E, Ericson, C, Kirik, D and Lundberg, C (2004). Regulated delivery of glial cell line-derived neurotrophic factor into rat striatum, using a tetracycline-dependent lentiviral vector. Hum Gene Ther 15: 934-944. (Pubitemid 39372570)
20. Szulc, J and Aebischer, P (2008). Conditional gene expression and knockdown using lentivirus vectors encoding shRNA. Methods Mol Biol 434: 291-309.
21. Yang, X, Mertens, B, Lehtonen, E, Vercammen, L, Bockstael, O, Chtarto, A et al. (2009). Reversible neurochemical changes mediated by delayed intrastriatal glial cell line-derived neurotrophic factor gene delivery in a partial Parkinson's disease rat model. J Gene Med 11: 899-912.
22. Chtarto, A, Bender, HU, Hanemann, CO, Kemp, T, Lehtonen, E, Levivier, M et al. (2003). Tetracycline-inducible transgene expression mediated by a single AAV vector. Gene Ther 10: 84-94. (Pubitemid 36175829)
23. Chtarto, A, Yang, X, Bockstael, O, Melas, C, Blum, D, Lehtonen, E et al. (2007). Controlled delivery of glial cell line-derived neurotrophic factor by a single tetracycline-inducible AAV vector. Exp Neurol 204: 387-399. (Pubitemid 46330286)
24. Szulc, J, Wiznerowicz, M, Sauvain, MO, Trono, D and Aebischer, P (2006). A versatile tool for conditional gene expression and knockdown. Nat Methods 3: 109-116. (Pubitemid 43173309)
25. Manfredsson, FP, Burger, C, Rising, AC, Zuobi-Hasona, K, Sullivan, LF, Lewin, AS et al. (2009). Tight Long-term dynamic doxycycline responsive nigrostriatal GDNF using a single rAAV vector. Mol Ther 17: 1857-1867.
26. Gallagher, P and Young, AH (2006). Mifepristone (RU-486) treatment for depression and psychosis: A review of the therapeutic implications. Neuropsychiatr Dis Treat 2: 33-42.
27. Cress, DE (2008). The need for regulatable vectors for gene therapy for Parkinson's disease. Exp Neurol 209: 30-33.
28. Kordower, JH and Olanow, CW (2008). Regulatable promoters and gene therapy for Parkinson's disease: Is the only thing to fear, fear itself? Exp Neurol 209: 34-40.
29. Freireich, EJ, Gehan, EA, Rall, DP, Schmidt, LH and Skipper, HE (1966). Quantitative comparison of toxicity of anticancer agents in mouse, rat, hamster, dog, monkey, and man. Cancer Chemother Rep 50: 219-244.
30. Reagan-Shaw, S, Nihal, M and Ahmad, N (2008). Dose translation from animal to human studies revisited. FASEB J 22: 659-661. (Pubitemid 351348146)
31. Gallagher, P, Watson, S, Elizabeth Dye, C, Young, AH and Nicol Ferrier, I (2008). Persistent effects of mifepristone (RU-486) on cortisol levels in bipolar disorder and schizophrenia. J Psychiatr Res 42: 1037-1041.
32. Watson S, Gallagher P, Porter RJ, Smith MS, Herron LJ, Bulmer S et al. (2012). A randomized trial to examine the effect of mifepristone on neuropsychological performance and mood in patients with bipolar depression. Biol Psychiatr 72: 943-949.
33. Kügler, S, Meyn, L, Holzmüller, H, Gerhardt, E, Isenmann, S, Schulz, JB et al. (2001). Neuron-specific expression of therapeutic proteins: Evaluation of different cellular promoters in recombinant adenoviral vectors. Mol Cell Neurosci 17: 78-96. (Pubitemid 32113540)
34. Taschenberger, G, Garrido, M, Tereshchenko, Y, Bähr, M, Zweckstetter, M and Kügler, S (2012). Aggregation of aSynuclein promotes progressive in vivo neurotoxicity in adult rat dopaminergic neurons. Acta Neuropathol 123: 671-683.