Stable transgene expression from HSV amplicon vectors in the brain: Potential involvement of immunoregulatory signals

Molecular Therapy

Volumn 16, Issue 10, 2008, Pages 1727-1736

  Suzuki, Masataka ^a,b   Chiocca, E Antonio ^a   Saeki, Yoshinaga ^a  

^a OHIO STATE UNIVERSITY   (United States)

^b BAYLOR COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CYTOKINE; HERPESVIRUS VECTOR; INTERFERON; MOLECULAR MARKER; STAT1 PROTEIN;

ADAPTIVE IMMUNITY; AMPLICON; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BRAIN REGION; CELL INFILTRATION; CONTROLLED STUDY; CORPUS STRIATUM; CYTOKINE PRODUCTION; FEMALE; GENE EXPRESSION; GENE SILENCING; GENETIC STABILITY; IMMUNE RESPONSE; IMMUNOCOMPETENT CELL; IMMUNOREGULATION; INFLAMMATION; INJECTION SITE; LIVER PARENCHYMA; MOUSE; NONHUMAN; PROTEIN INDUCTION; REGULATORY T LYMPHOCYTE; STEREOTACTIC PROCEDURE; TARGET ORGAN; TRANSCRIPTION REGULATION; TRANSGENE; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; BASE SEQUENCE; BRAIN; CORPUS STRIATUM; DNA PRIMERS; GENE EXPRESSION; GENETIC VECTORS; MICE; MICE, INBRED C57BL; SIMPLEXVIRUS; TRANSGENES;

MUS; SIMPLEXVIRUS;

EID: 54849392000     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2008.175     Document Type: Article

References (60)

1. Inoue, R, Moghaddam, KA, Ranasinghe, M, Saeki, Y, Chiocca, EA and Wade-Martins, R (2004). Infectious delivery of the 132 kb CDKN2A/CDKN2B genomic DNA region results in correctly spliced gene expression and growth suppression in glioma cells. Gene Ther 11: 1195-1204.
2. Wade-Martins, R, Saeki, Y and Chiocca, EA (2003). Infectious delivery of a 135-kb LDLR genomic locus leads to regulated complementation of low-density lipoprotein receptor deficiency in human cells. Mol Ther 7: 604-612.
3. Wade-Martins, R, Smith, ER, Tyminski, E, Chiocca, EA and Saeki, Y (2001). An infectious transfer and expression system for genomic DNA loci in human and mouse cells. Nat Biotechnol 19: 1067-1070.
4. Kasai, K and Saeki, Y (2006). DNA-based methods to prepare helper virus-free herpes amplicon vectors and versatile design of amplicon vector plasmids. Curr Gene Ther 6: 303-314.
5. Bowers, WJ, Mastrangelo, MA, Howard, DF, Southerland, HA, Maguire-Zeiss, KA and Federoff, HJ (2006). Neuronal precursor-restricted transduction via in utero CNS gene delivery of a novel bipartite HSV amplicon/ transposase hybrid vector. Mol Ther 13: 580-588.
6. Heister, T, Heid, I, Ackermann, M and Fraefel, C (2002). Herpes simplex virus type 1/adeno-associated virus hybrid vectors mediate site-specific integration at the adeno-associated virus preintegration site, AAVS1, on human chromosome 19. J Virol 76: 7163-7173.
7. Savard, N, Cosset, FL and Epstein, AL (1997). Defective herpes simplex virus type 1 vectors harboring gag, pol, and env genes can be used to rescue defective retrovirus vectors. J Virol 71: 4111-4117.
8. Sena-Esteves, M, Saeki, Y, Camp, SM, Chiocca, EA and Breakefield, XO (1999). Single-step conversion of cells to retrovirus vector producers with herpes simplex virus-Epstein-Barr virus hybrid amplicons. J Virol 73: 10426-10439.
9. Wang, S and Vos, JM (1996). A hybrid herpesvirus infectious vector based on Epstein-Barr virus and herpes simplex virus type 1 for gene transfer into human cells in vitro and in vivo. J Virol 70: 8422-8430.
10. Wang, Y, Camp, SM, Niwano, M, Shen, X, Bakowska, JC, Breakefield, XO et al. (2002). Herpes simplex virus type 1/adeno-associated virus rep(+) hybrid amplicon vector improves the stability of transgene expression in human cells by site-specific integration. J Virol 76: 7150-7162.
11. Saeki, Y, Breakefield, XO arid Chiocca, EA (2003). Improved HSV-1 amplicon packaging system using ICP27-deleted, oversized HSV-1 BAC DNA. Methods Mol Med 76: 51-60.
12. Saeki, Y, Fraefel, C, Ichikawa, T, Breakefield, XO and Chiocca, EA (2001). Improved helper virus-free packaging system for HSV amplicon vectors using an ICP27-deleted, oversized HSV-1 DNA in a bacterial artificial chromosome. Mol Ther 3: 591-601.
13. Suzuki, M, Chiocca, EA and Saeki, Y (2007). Early STAT1 activation after systemic delivery of HSV amplicon vectors suppresses transcription of the vector-encoded transgene. Mol Ther 15: 2017-2026.
14. Olschowka, JA, Bowers, WJ, Hurley, SD, Mastrangelo, MA and Federoff, HJ (2003). Helper-free HSV-1 amplicons elicit a markedly less robust innate immune response in the CNS. Mol Ther 7: 218-227.
15. Sun, M, Kong, L, Wang, X, Holmes, C, Gao, Q, Zhang, GR et al. (2004). Coexpression of tyrosine hydroxylase, GTP cyclohydrolase I, aromatic amino acid decarboxylase, and vesicular monoamine transporter 2 from a helper virps-free herpes simplex virus type 1 vector supports high-level, long-term biochemical and behavioral correction of a rat model of Parkinson's disease. Hum Gene Ther 15: 1177-1196.
16. Zhang, GR, Wang, X, Yang, T, Sun, M, Zhang, W, Wang, Y et al. (2000). A tyrosine hydroxylase-neurofilament chimeric promoter enhances long-term expression in rat forebrain neurons from helper virus-free HSV-1 vectors. Brain Res Mol Brain Res 84: 17-31.
17. Asselin-Paturel, C, Boonstra, A, Dalod, M, Durand, I, Yessaad, N, Dezutter-Dambuyant, C et al. (2001). Mouse type I IFN-producing cells are immature APCs with plasmacytoid morphology. Nat Immunol 2: 1144-1150.
18. McKenna, K, Beignon, AS and Bhardwaj, N (2005). Plasmacytoid dendritic cells: Linkiing innate and adaptive immunity. J Virol 79: 17-27.
19. Clark-Lewis, I, Mattioli, I, Gong, JH and Loetscher, P (2003). Structure-function relationship between the human chemokine receptor CXCR3 and its ligands. J Biol Chem 278: 289-295.
20. Inngjerdingen, M, Damaj, B and Maghazachi, AA (2001). Expression and regulation of chemokine receptors in human natural killer cells. Blood 97: 367-375.
21. Crispe, IN (2003). Hepatic T cells and liver tolerance. Nat Rev Immunol 3: 51-62.
22. Colonna, M, Krug, A and Celia, M (2002). Interferon-producing cells: On the front line in immune responses against pathogens. Curr Opin Immunol 14: 373-379.
23. Wilson, SB and Delovitch, TL (2003). Janus-like role of regulatory iNKT cells in autoimmune disease and tumour immunity. Nat Rev Immunol 3: 211-222.
24. Dijkstra, IM, Hulshof, S, van der Valk, P, Boddelke, HW and Biber, K (2004). Cutting edge: Activity of human adult microglia in response to CC chemokine ligand 21. J Immunol 172: 2744-2747.
25. Niwa, H, Yamamura, K and Miyazaki, J (1991). Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 108: 193-199.
26. Hara, M, Kingsley, CI, Niimi, M, Read, S, Turvey, SE, Bushell, AR et al. (2001). IL-10 is required for regulatory T cells to mediate tolerance to alloantigens in vivo. J Immunol 166: 3789-3796.
27. Piccirillo, CA, Letterio, JJ, Thornton, AM, McHugh, RS, Mamura, M, Mizuhara, H et al. (2002). CD4(+)CD25(+) regulatory T cells can mediate suppressor function in the absence of transforming growth factor betal production and responsiveness. J Exp Med 196: 237-246.
28. Bettelli, E, Carrier, Y, Gao, W, Korn, T, Strom, TB, Oukka, M et al. (2006). Reciprocal developmental pathways for the generation of pathogenic effector TH17 and regulatory T cells. Nature 441: 235-238.
29. Levings, MK, Sangregorio, R and Roncarolo, MG (2001). Human cd25(+)cd4(+) t regulatory cells suppress naive and memory T cell proliferation and can be expanded in vitro without loss of function. J Exp Med 193: 1295-1302.
30. Nociari, M, Ocheretina, O, Schoggins, JW and Falck-Pedersen, E (2007), Sensing infection by adenovirus: Toll-like receptor-independent viral DNA recognition signals activation of the interferon regulatory factor 3 master regulator. J Virol 81: 4145-4157.
31. Zhu, J, Huang, X and Yang, Y (2007). Innate immune response to adenoviral vectors is mediated by both Toll-like receptor-dependent and -independent pathways. J Virol 81: 3170-3180.
32. Torseth, JW, Cohen, GH, Eisenberg, RJ, Berman, PW, Lasky, LA, Cerini, CP et al. (1987). Native and recombinant herpes simplex virus type 1 envelope proteins induce human immune T-lymphocyte responses. J Virol 61: 1532-1539.
33. Tang, J, Yang, T, Ghosh, HP and Geller, AI (2001). Helper virus-free HSV-1 vectors packaged both in the presence of VSV G protein and in the absence of HSV-1 glycoprotein B support gene transfer into neurons in the rat striatum. J Neurovirol 7: 548-555.
34. McMenamin, PG (1999). Distribution and phenotype of dendritic cells and resident tissue macrophages in the dura mater, leptomeninges, and choroid plexus of the rat brain as demonstrated in wholemount preparations. J Camp Neurol 405: 553-562.
35. Bechmann, I, Galea, I and Perry, VH (2007). What is the blood-brain barrier (not)? Trends Immunol 28: 5-11.
36. Barcia, C, Jimenez-Dalmaroni, M, Kroeger, KM, Puntel, M, Rapaport, AJ, Larocque, D et al. (2007). One-year expression from high-capacity adenoviral vectors in the brains of animals with pre-existing anti-adenoviral immunity: Clinical implications. Mol Ther 15 2154-2163.
37. Diebold, SS, Montoya, M, Unger, H, Alexopoulou, L, Roy, P Haswell, LE et al. (2003). Viral infection switches non-plasmacytoid dendritic cells into high interferon producers. Nature 424: 324-328.
38. Zermansky, AJ, Bolognani, F, Stone, D, Cowsill, CM, Morrissey, G, Castro, MG et al. (2001). Towards global and long-term neurological gene therapy: Unexpected transgene dependent, high-level, and widespread distribution of HSV-1 thymidine kinase throughout the CNS. Mol Ther 4: 490-A98.
39. Ellermann-Eriksen, S (2005). Macrophages and cytokines in the early defence against herpes simplex virus. Virol 12: 59.
40. Rasmussen, M, Kong, L, Zhang, GR, Liu, M, Wang, X, Szabo G et al. (2007). Glutamatergic or GABAergic neuron-specific, long-term expression in neocortical neurons from helper virus-free HSV-1 vectors containing the phosphate-activated glutaminase, vesicular glutamate transporter-1, or glutamic acid decarboxylase promoter. Brain Res 1144: 19-32.
41. Kaplitt, MG, Kwong, AD, Kleopoulos, SP, Mobbs, CV, Rabkin, SD and Pfaff, DW (1994). Preproenkephalin promoter yields region-specific and long-term expression in adult brain after direct in vivo gene transfer via a defective herpes simplex viral vector. Proc Natl Acad Sci USA 91: 8979-8983.
42. Cao, H, Zhang, GR, Wang, X, Kong, L and Geller, AI (2008). Enhanced nigrostriatal neuron-specific, long-term expression by using neural-specific promoters in combination with targeted gene transfer by modified helper virus-free HSV-1 vector particles. BMC Neurosci 9: 37.
43. Loiacono, CM, Myers, R and Mitchell, WJ (2002). Neurons differentially activate the herpes simplex virus type 1 immediate-early gene ICP0 and ICP27 promoters in transgenic mice. J Virol 76: 2449-2459.
44. Mitchell, WJ (1995). Neurons differentially control expression of a herpes simplex virus type 1 immediate-early promoter in transgenic mice. J Virol 69: 7942-7950.
45. Wang, Y, Yu, L and Geller, AI (1999). Diverse stabilities of expression in the rat brain from different cellular promoters in a helper virus-free herpes simplex virus type 1 vector system. Hum Gene Ther 10: 1763-1771.
46. Morris, MM, Dyson, H, Baker, D, Harbige, LS, Fazakerley, JK and Amor, S (1997). Characterization of the cellular and cytokine response in the central nervous system following Semliki Forest virus infection. J Neuroimmunol 74: 185-197.
47. Halford, WP, Gebhardt, BM and Carr, DJ (1996). Persistent cytokine expression in trigeminal ganglion latently infected with herpes simplex virus type 1. J Immunol 157: 3542-3549.
48. Saeki, Y, Ichikawa, T, Saeki, A, Chiocca, EA, Tobler, K, Ackermann, M et al. (1998). Herpes simplex virus type 1 DNA amplified as bacterial artificial chromosome in Escherichio coli: Rescue of replication-competent virus progeny and packaging of amplicon vectors. Hum Gene Ther 9: 2787-2794.
49. von Boehmer, H (2005). Mechanisms of suppression by suppressor T cells. Nat Immunol 6: 338-344.
50. Miyara, M and Sakaguchi, S (2007). Natural regulatory T cells: mechanisms of suppression. Trends Mol Med 13: 108-116.
51. Sawitzki, B, Kingsley, CI, Oliveira, V, Karim, M, Herber, M and Wood, KJ (2005). IFN-gamma production by alloantigen-reactive regulatory T cells is important for their regulatory function in vivo. J Exp Med 201: 1925-1935.
52. Carr, DJ, Noisakran, S, Halford, WP, Lukacs, N, Asensio, V and Campbell, IL (1998). Cytokine and chemokine production in HSV-1 latently infected trigeminal ganglion cell cultures: Effects of hyperthermic stress. J Neuroimmunol 85: 111-121.
53. Kreutzberg, GW (1996). Microglia: A sensor for pathological events in the CNS. Trends Neurosci 19: 312-318.
54. Lee, YB, Nagai, A and Kim, SU (2002). Cytokines, chemokines, and cytokine receptors in human microglia. J Neurosci Res 69: 94-103.
55. Fulci, G, Dmitrieva, N, Gianni, D, Fontana, EJ, Pan, X, Lu, Y et al. (2007). Depletion of peripheral macrophages and brain microglia increases brain tumor titers of oncolytic viruses. Cancer Res 67: 9398-9406.
56. Brubaker, JO, Thompson, CM, Morrison, LA, Knipe, DM, Siber, GR and Finberg, RW (1996). Th1-associated immune responses to beta-galactosidase expressed by a replication-defective herpes simplex virus. J Immunol 157: 1598-1604.
57. Smith, IL, Hardwicke, MA and Sandri-Goldin, RM (1992). Evidence that the herpes simplex virus immediate early protein ICP27 acts post-transcriptionally during infection to regulate gene expression. Virology 186: 74-86.
58. Paxinos, G and Franklin, K (2003). The Mouse Brain in Stereotaxic Coordinates, 2nd edn. Academic: San Diego.
59. Suzuki, M, Kasai, K and Saeki, Y (2006). Plasmid DNA sequences present in conventional herpes simplex virus amplicon vectors cause rapid transgene silencing by forming inactive chromatin. J Virol 80 3293-3300.
60. Tang, Y, Shah, K, Messerli, SM, Snyder, E, Breakefield, X and Weissleder, R (2003). in vivo tracking of neural progenitor cell migration to glioblastomas. Hum Gene Ther 14: 1247-1254.