RANGE: Gene transfer of reversibly controlled polycistronic genes

Molecular Therapy Nucleic Acids

Volumn 2, Issue , 2013, Pages e85-

  Chen, Yiwei ^a   Cao, Liji ^b   Luo, Chonglin ^b   Ditzel, Désirée A W ^a   Peter, Jörg ^b   Sprengel, Rolf ^a  

^a MAX PLANCK INSTITUTE FOR MEDICAL RESEARCH   (Germany)

^b GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

Author keywords

2A peptide bridge; Conditional gene expression; RAAV; Tet system

Indexed keywords

BETA ACTIN; DOXYCYCLINE; PARVOVIRUS VECTOR; SYNAPSIN I; TETRACYCLINE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BIOLUMINESCENCE; CISTRON; CONTROLLED STUDY; GENE CONTROL; GENE EXPRESSION; GENE FUNCTION; GENE SWITCHING; GENE TARGETING; GENE TRANSFER; HUMAN; HUMAN CELL; IN VITRO STUDY; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; REPORTER GENE; SILENCER ELEMENT;

EID: 84878859915     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2013.15     Document Type: Article

References (46)

1. Davidson, BL and Breakefield, XO (2003). Viral vectors for gene delivery to the nervous system. Nat Rev Neurosci 4: 353-364. (Pubitemid 37280174)
2. Mingozzi, F and High, KA (2011). Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet 12: 341-355.
3. Summerford, C and Samulski, RJ (1999). Viral receptors and vector purification: new approaches for generating clinical-grade reagents. Nat Med 5: 587-588.
4. Grimm, D, Kay, MA and Kleinschmidt, JA (2003). Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6. Mol Ther 7: 839-850. (Pubitemid 36790261)
5. 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.
6. Gossen, M, Freundlieb, S, Bender, G, Müller, G, Hillen, W and Bujard, H (1995). Transcriptional activation by tetracyclines in mammalian cells. Science 268: 1766-1769.
7. Lewandoski, M (2001). Conditional control of gene expression in the mouse. Nat Rev Genet 2: 743-755. (Pubitemid 33685572)
8. Sprengel, R and Hasan, MT (2007). Tetracycline-controlled genetic switches. Handb Exp Pharmacol: 49-72.
9. Vanrell, L, Di Scala, M, Blanco, L, Otano, I, Gil-Farina, I, Baldim, V et al. (2011). Development of a liver-specific Tet-on inducible system for AAV vectors and its application in the treatment of liver cancer. Mol Ther 19: 1245-1253.
10. Fitzsimons, HL, Mckenzie, JM and During, MJ (2001). Insulators coupled to a minimal bidirectional tet cassette for tight regulation of rAAV-mediated gene transfer in the mammalian brain. Gene Ther 8: 1675-1681. (Pubitemid 34030700)
11. Haberman, R, Criswell, H, Snowdy, S, Ming, Z, Breese, G, Samulski, R et al. (2002). Therapeutic liabilities of in vivo viral vector tropism: adeno-associated virus vectors, NMDAR1 antisense, and focal seizure sensitivity. Mol Ther 6: 495-500. (Pubitemid 35214871)
12. McGee Sanftner, LH, Rendahl, KG, Quiroz, D, Coyne, M, Ladner, M, Manning, WC et al. (2001). Recombinant AAV-mediated delivery of a tet-inducible reporter gene to the rat retina. Mol Ther 3(5 Pt 1): 688-696. (Pubitemid 32509866)
13. 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.
14. Lhériteau, E, Libeau, L, Mendes-Madeira, A, Deschamps, JY, Weber, M, Le Meur, G et al. (2010). Regulation of retinal function but nonrescue of vision in RPE65-deficient dogs treated with doxycycline-regulatable AAV vectors. Mol Ther 18: 1085-1093.
15. Campbell, M, Humphries, MM, Kiang, AS, Nguyen, AT, Gobbo, OL, Tam, LC et al. (2011). Systemic low-molecular weight drug delivery to pre-selected neuronal regions. EMBO Mol Med 3: 235-245.
16. Gafni, Y, Pelled, G, Zilberman, Y, Turgeman, G, Apparailly, F, Yotvat, H et al. (2004). Gene therapy platform for bone regeneration using an exogenously regulated, AAV-2-based gene expression system. Mol Ther 9: 587-595. (Pubitemid 38578809)
17. Wang, JJ, Niu, DB, Zhang, T, Wang, K, Xue, B and Wang, XM (2005). A tetracycline-regulatable adeno-associated virus vector for double-gene transfer. Neurosci Lett 378: 106-110. (Pubitemid 40354296)
18. Cambridge, SB, Geissler, D, Calegari, F, Anastassiadis, K, Hasan, MT, Stewart, AF et al. (2009). Doxycycline-dependent photoactivated gene expression in eukaryotic systems. Nat Methods 6: 527-531.
19. Rendahl, KG, Quiroz, D, Ladner, M, Coyne, M, Seltzer, J, Manning, WC et al. (2002). Tightly regulated long-term erythropoietin expression in vivo using tet-inducible recombinant adeno-associated viral vectors. Hum Gene Ther 13: 335-342. (Pubitemid 34164116)
20. Haberman, RP and McCown, TJ (2002). Regulation of gene expression in adeno-associated virus vectors in the brain. Methods 28: 219-226. (Pubitemid 35341085)
21. Freundlieb, S, Schirra-Müller, C and Bujard, H (1999). A tetracycline controlled activation/repression system with increased potential for gene transfer into mammalian cells. J Gene Med 1: 4-12. (Pubitemid 129631058)
22. Margolin, JF, Friedman, JR, Meyer, WK, Vissing, H, Thiesen, HJ and Rauscher, FJ 3rd (1994). Kruppel-associated boxes are potent transcriptional repression domains. Proc Natl Acad Sci USA 91: 4509-4513.
23. Witzgall, R, O'Leary, E, Leaf, A, Onaldi, D and Bonventre, JV (1994). The Kruppel-associated box-A (KRAB-A) domain of zinc finger proteins mediates transcriptional repression. Proc Natl Acad Sci USA 91: 4514-4518.
24. Tang, W, Ehrlich, I, Wolff, SB, Michalski, AM, Wölfl, S, Hasan, MT et al. (2009). Faithful expression of multiple proteins via 2A-peptide self-processing: a versatile and reliable method for manipulating brain circuits. J Neurosci 29: 8621-8629.
25. Donnelly, ML, Luke, G, Mehrotra, A, Li, X, Hughes, LE, Gani, D et al. (2001). Analysis of the aphthovirus 2A/2B polyprotein 'cleavage' mechanism indicates not a proteolytic reaction, but a novel translational effect: a putative ribosomal 'skip'. J Gen Virol 82(Pt 5): 1013-1025. (Pubitemid 32454602)
26. Karasawa, S, Araki, T, Nagai, T, Mizuno, H and Miyawaki, A (2004). Cyan-emitting and orange-emitting fluorescent proteins as a donor/acceptor pair for fluorescence resonance energy transfer. Biochem J 381(Pt 1): 307-312. (Pubitemid 38932237)
27. Sawicki, JA, Morris, RJ, Monks, B, Sakai, K and Miyazaki, J (1998). A composite CMV-IE enhancer/beta-actin promoter is ubiquitously expressed in mouse cutaneous epithelium. Exp Cell Res 244: 367-369. (Pubitemid 28480885)
28. Kügler, S, Kilic, E and Bähr, M (2003). Human synapsin 1 gene promoter confers highly neuron-specific long-term transgene expression from an adenoviral vector in the adult rat brain depending on the transduced area. Gene Ther 10: 337-347. (Pubitemid 36305602)
29. Shevtsova, Z, Malik, JM, Michel, U, Bähr, M and Kügler, S (2005). Promoters and serotypes: targeting of adeno-associated virus vectors for gene transfer in the rat central nervous system in vitro and in vivo. Exp Physiol 90: 53-59.
30. Jordan, M and Wurm, F (2004). Transfection of adherent and suspended cells by calcium phosphate. Methods 33: 136-143. (Pubitemid 38569658)
31. Ohki, EC, Tilkins, ML, Cice, VC and Price, PJ (2001). Improving the transfection efficiency of post-mitotic neurons. J Neurosci Methods 112: 95-99. (Pubitemid 33051112)
32. Zincarelli, C, Soltys, S, Rengo, G and Rabinowitz, JE (2008). Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 16: 1073-1080. (Pubitemid 351737072)
33. Shifera, AS and Hardin, JA (2010). Factors modulating expression of Renilla luciferase from control plasmids used in luciferase reporter gene assays. Anal Biochem 396: 167-172.
34. Sudol, KL, Mastrangelo, MA, Narrow, WC, Frazer, ME, Levites, YR, Golde, TE et al. (2009). Generating differentially targeted amyloid-beta specific intrabodies as a passive vaccination strategy for Alzheimer's disease. Mol Ther 17: 2031-2040.
35. 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)
36. Fisher, KJ, Jooss, K, Alston, J, Yang, Y, Haecker, SE, High, K et al. (1997). Recombinant adeno-associated virus for muscle directed gene therapy. Nat Med 3: 306-312. (Pubitemid 27112928)
37. Nakai, H, Storm, TA and Kay, MA (2000). Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo. J Virol 74: 9451-9463.
38. McCarty, DM, Young, SM Jr and Samulski, RJ (2004). Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet 38: 819-845. (Pubitemid 40013747)
39. Teplow, D (2012). Molecular Biology of Neurodegenerative Diseases. Academic Press: Salt lake city. pp. 498.
40. Berkel, S, Marshall, CR, Weiss, B, Howe, J, Roeth, R, Moog, U et al. (2010). Mutations in the SHANK2 synaptic scaffolding gene in autism spectrum disorder and mental retardation. Nat Genet 42: 489-491.
41. Berkel, S, Tang, W, Treviño, M, Vogt, M, Obenhaus, HA, Gass, P et al. (2012). Inherited and de novo SHANK2 variants associated with autism spectrum disorder impair neuronal morphogenesis and physiology. Hum Mol Genet 21: 344-357.
42. Chen, C and Okayama, H (1987). High-efficiency transformation of mammalian cells by plasmid DNA. Mol Cell Biol 7: 2745-2752.
43. Klugmann, M, Symes, CW, Leichtlein, CB, Klaussner, BK, Dunning, J, Fong, D et al. (2005). AAV-mediated hippocampal expression of short and long Homer 1 proteins differentially affect cognition and seizure activity in adult rats. Mol Cell Neurosci 28: 347-360. (Pubitemid 40202595)
44. Smith, RH, Levy, JR and Kotin, RM (2009). A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells. Mol Ther 17: 1888-1896.
45. Cetin, A, Komai, S, Eliava, M, Seeburg, PH and Osten, P (2006). Stereotaxic gene delivery in the rodent brain. Nat Protoc 1: 3166-3173.
46. Fang, J, Qian, JJ, Yi, S, Harding, TC, Tu, GH, VanRoey, M et al. (2005). Stable antibody expression at therapeutic levels using the 2A peptide. Nat Biotechnol 23: 584-590 (Pubitemid 41724897)