Gene transfer to neurons using herpes simplex virus-based vectors

Annual Review of Neuroscience

Volumn 19, Issue , 1996, Pages 265-287

  Fink, D J ^a,b,c   DeLuca, N A ^b   Goins, W F ^b   Glorioso, J C ^b  

^a UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF PITTSBURGH   (United States)

^c VETERANS AFFAIRS MEDICAL CENTER   (United States)

Author keywords

gene therapy; gene transfer; neurons

Indexed keywords

VIRUS VECTOR;

GENE EXPRESSION; GENE TARGETING; GENE TRANSFER; GENETIC ENGINEERING; HERPES SIMPLEX VIRUS; MOLECULAR BIOLOGY; NONHUMAN; PRIORITY JOURNAL; RECOMBINANT DNA TECHNOLOGY; REVIEW; TATA BOX; TRANSGENE; VIRUS EXPRESSION;

EID: 0029925070     PISSN: 0147006X     EISSN: None     Source Type: Book Series    
DOI: 10.1146/annurev.ne.19.030196.001405     Document Type: Review

References (110)

1. Acsadi G, Jani A, Massie B, Simoneau M, Holland P, et al. 1994. A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity. Hum. Mol. Genet. 3:579-84
2. Akli S, Cailland C, Vigne E, Stratford-Perricaudet LD, Poenaru L, et al. 1993. Transfer of a foreign gene into the brain using adenovirus vectors. Nat. Genet. 3: 224-28
3. Andersen JK, Garber DA, Meaney CA, Breakfield XO. 1992. Gene transfer into mammalian central nervous system using herpes virus vectors: extended expression of bacterial lacZ in neurons using the neuron-specific enolase promoter. Hum. Gene Ther. 3:487-99
4. Anderson WF. 1992. Human gene therapy. Science 256:803-13
5. Bak IJ, Markham CH, Cook ML. 1977. Intraaxonal transport of herpes simplex virus in the rat central nervous system. Brain Res. 136:415-29
6. Batchelor AH, O'Hare PO. 1990. Regulation of cell-type-specific activity of a promoter located upstream of the latency-associated transcript of herpes simplex virus type 1. J. Virol. 64:3269-79
7. Batchelor AH, O'Hare PO. 1992. Localization of cis-acting sequence requirements in the promoter of the latency-associated transcript of herpes simplex virus type 1 required for cell-type-specific activity. J. Virol. 66:3573-82
8. Battleman DS, Geller AI. 1993. HSV-1 vector-mediated gene transfer of the human nerve growth factor receptor p75hNGFR defines high-affinity NGF binding. J. Neurosci. 13: 941-51
9. Casaccia-Bonnefil P, Benedikz E, Shen H, Stelzer A, Edelstein D, et al. 1993. Localized gene transfer into organotypic hippocampal slice cultures and acute hippocampal slices. J. Neurosci. Methods 50:341-51
10. Chrisp CE, Sunstrum JC, Averill DR, Levine M, Glorioso JC. 1989. Characterization of encephalitis in adult mice induced by intracerebral inoculation of herpes simplex virus type 1 (KOS) and comparison with mutants showing decreased virulence. Lab. Invest. 60:822-30
11. Chung TP, Wymer JP, Smith CC, Kulka M, Aurelian L. 1989. Protein kinase activity associated with the large subunit of herpes simplex virus type 2 ribonucleotide reductase (ICP6). J. Virol. 63:3389-98
12. Creedon DJ, Greenland LS, Johnson EM Jr. 1993. Characterization of a defective herpes simplex virus vector for ectopic gene expression in cultured neurons. Soc. Neurosci. 19(1): 886 (Abstr.)
13. Croen KD, Ostrove JM, Dragovic LJ, Smialek JE, Straus SE. 1987. Latent herpes simplex virus in human trigeminal ganglia. Detection of an immediate early gene "anti-sense" transcript by in situ hybridization. New Engl. J. Med. 317:1427-32
14. Davidson BL, Allen EE, Kozarsky KF, Wilson JM, Roessler BJ. 1993. A model system for in vivo gene transfer into the central nervous system using an adenoviral vector. Nat. Genet. 3:219-23
15. Deatly AM, Spivack JG, Lavi E, O'Boyle D, Fraser NW. 1988. Latent herpes simplex virus type 1 transcripts in peripheral and central nervous systems tissues of mice map to similar regions of the viral genome. J. Virol. 62:749-56
16. DeLuca NA, McCarthy AM, Schaffer PA. 1985. Isolation and characterization of deletion mutants of herpes simplex virus type 1 in the gene encoding immediate-early regulatory protein ICP4. J. Virol. 56:558-70
17. DeLuca NA, Schaffer PA. 1985. Activation of immediate-early, early, and late promoters by temperature-sensitive and wild-type forms of herpes simplex virus type 1 protein ICP4. Mol. Cell Biol. 5:558-70
18. Deshmane SL, Fraser NW. 1989. During latency, herpes simplex virus type 1 DNA is associated with nucleosomes in a chromation structure. J. Virol. 63:943-47
19. Devi-Rao GB, Goddart SA, Hecht LM, Rochford R, Rice MK, Wagner EK. 1991. Relationship between polyadenylated and nonpolyadenylated, HSV type 1 latency-associated transcripts. J. Gen. Virol. 65: 2179-90
20. Dixon RAF, Schaffer PA. 1980. Fine-structure mapping and functional analysis of temperature-sensitive mutants in the gene encoding the herpes simplex virus type 1 immediate early protein VP175. J. Virol. 36:189-203
21. Dobson AT, Margolis TP, Sederati F, Stevens JG, Feldman TL. 1990. A latent, nonpathogenic HSV-1-derived vector stably expresses beta-galactosidase in mouse neurons. Neuron 5:353-60
22. Dobson AT, Sederati F, Devi-Rao G, Flanagan WM, Farrell MJ, et al. 1989. Identification of the latency-associated transcript promoter by expression of rabbit β-globin mRNA in mouse sensory nerve ganglia latently infected with a recombinant herpes simplex virus. J. Virol. 63:3844-51
23. During MJ, Naegele JR, O'Malley KL, Geller AI. 1994. Long-term behavioral recovery in Parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 266:1399-403
24. Efstathiou S, Minson AC, Field HJ, Anderson JR, Wildy P. 1986. Detection of herpes simplex virus-specific DNA sequences in latently infected mice and in human. J. Virol. 57:446-55
25. Everett RD. 1984. Transactivation of transcription by herpes virus products: requirements for two HSV-1 immediate-early polypetides for maximum activity. EMBO J. 3:3135-41
26. Everett RD. 1987. The regulation of transcription of viral and cellular genes by herpes-virus immediate-early gene products. Anticancer Res. 7:580-604
27. Fareed MU, Spivack JG. 1994. Two open reading frames (ORF1 and ORF2) within the 2.0-kilobase latency-associated transcript of herpes simplex virus type 1 are not essential for reactivation from latency. J. Virol. 68: 8071-81
28. Farrell MJ, Dobson AT, Feldman LT. 1991. Herpes simplex virus latency-associated transcript is a stable intron. Proc. Natl. Acad. Sci. USA 88:790-94
29. Federoff HJ, Geschwind MD, Geller AI, Kessler JA. 1992. Expression of nerve growth factor in vivo from a defective herpes simplex virus 1 vector prevents effects of axotomy on sympathetic ganglia. Proc. Natl. Acad. Sci. USA 89:1636-40
30. Fink DJ, Sternberg LR, Weber PC, Mata M, Goins WF, Glorioso JC. 1992. In vivo expression of β-galactosidase in hippocampal neurons by HSV-mediated gene transfer. Hum. Gene Ther. 3:11-19
31. Freese A, Geller AI, Neve R. 1990. HSV-1 vector mediated neuronal gene delivery. Strategies for molecular neuroscience and neurology. Biochem. Pharm. 40:2189-99
32. Gaffney DF, McLauchlin J, Whitton JL, Clements JB. 1985. A modular system for the assay of transcription regulatory signals: the sequence TAATGARAT is required for herpes simplex virus immediate early gene activation. Nucleic Acids Res. 13: 7847-63
33. Geller AI, Breakefield XO. 1988. A defective HSV-1 vector expresses Escherichia coli β-galactosidase in cultured peripheral neurons. Science 241:1667-69
34. Geller AI, During MJ, Haycock JW, Freese A, Neve R. 1993. Long-term increases in neurotransmitter release from neuronal cells expressing a constitutively active adenylate cyclase from a herpes simplex virus type 1 vector. Proc. Natl. Acad. Sci. USA 90:7603-7
35. Geller AI, Freese A. 1990. Infection of cultured central nervous system neurons with a defective herpes simplex virus 1 vector results in stable expression of Escherichia coli β-galactosidase. Proc. Natl. Acad. Sci. USA 87: 1149-53
36. Geller AI, Keyomarsi K, Bryan J, Pardee AB. 1990. An efficient deletion mutant packaging system for defective herpes simplex virus vectors: potential applications to human gene therapy and neuronal physiology. Proc. Natl. Acad. Sci. USA 87:8950-54
37. Gelman IH, Silverstein S. 1985. Identification of immediate-early genes from herpes simplex virus that transactivate the virus thymidine kinase gene. Proc. Natl. Acad. Sci. USA 82:5265-69
38. Geschwind MD, Kessler JA, Geller AI, Federoff HJ. 1994. Transfer of the nerve growth factor gene into cell lines and cultured neurons using a defective herpes simplex virus vector. Brain Res. 24:327-35
39. L flanking repeats. J. Virol. 68:2239-52
40. Hill JM, Sedarati F, Javier RT, Wagner EK, Stevens JG. 1990. Herpes simplex virus latent phase transcription facilitates in vivo reactivation. Virology 174:117-25
41. Ho DY, Mocarski ES. 1989. Herpes simplex virus latent RNA (LAT) is not required for latent infection in the mouse. Proc. Natl. Acad. Sci. USA 87:7596-600
42. Honess RW, Roizman B. 1974. Regulation of herpes simplex virus macromolecular synthesis. I. Cascade regulation of the synthesis of three groups of viral proteins. J. Virol. 14:8-19
43. Jacob RJ, Morse LS, Roizman B. 1979. Anatomy of herpes simplex virus DNA. XII. Accumulation of head-to-tail concatemers in nuclei of infected cells and their role in the generation of the four isomeric arrangements of viral DNA. J. Virol. 29:448-57
44. Javier RT, Stevens JG, Dissette VB, Wagner EK. 1988. A herpes simplex virus transcript abundant in latently infected neurons is dispensable for establishment of the latent state. Virology 166:254-57
45. Jiao S, Gurevich V, Wolff JA. 1993. Long-term correction of rat model of Parkinson's disease by gene therapy. Nature 362:450-53
46. Johnson PA, Miyanohara A, Levine F, Cahill T, Friedmann T. 1992. Cytotoxicity of a replication-defective mutant herpes simplex virus type 1. J. Virol. 66:2952-65
47. Johnson PA, Wang MJ, Friedmann T. 1994. Improved cell survival by the reduction of immediate-early gene expression in replication-defective mutants of herpes simplex virus type 1 but not by mutation of the virion host shutoff function. J. Virol. 68:6347-62
48. Kaplitt MG, Kwong AD, Kleopoulos SP, Mobbs CV, Rabkin SD, Pfaff DW. 1994a. 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 91:8979-83
49. Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, et al. 1994b. Long-term gene expression and phenotype correction using adeno-associated virus vectors in the mammalian brain. Nat. Genet. 8:148-54
50. Katz JP, Bodin ET, Coen DM. 1990. Quantitative polymerase chain reaction analysis of herpes simplex virus DNA in ganglia of mice infected with replication-incompetent mutants. J. Virol. 64:4288-95
51. Le Gal La Salle G, Robert JJ, Berrard S, Ridoux V, Stratford-Perricaudet LD, et al. 1993. An adenovirus vector for gene transfer into neurons and glia in the brain. Science 259:988-90
52. Leib DA, Coen MD, Bogard CL, Hicks KA, Yager DR, Knipe DM. 1989. Immediate-early regulatory gene mutants define different stages in the establishment and reactivation of herpes simplex virus latency. J. Virol. 63:759-68
53. Leiden JM, Frenkel N, Rapp F. 1980. Identification of the herpes simplex virus DNA sequences present in six herpes simples virus thymidine kinase-transformed mouse cell lines. J. Virol. 33:272-85
54. Lokensgard JR, Bloom DC, Dobson AT, Feldman TL. 1994. Long-term promoter activity during herpes simplex virus latency. J. Virol. 68:7148-58
55. Mackem S, Roizman B. 1982a. Differentiation between alpha promoter and regulatory regions of herpes simplex virus type 1: the functional domains and sequence of a movable alpha regulator. Proc. Natl. Acad. Sci. USA 79:4917-21
56. Mackem S, Roizman B. 1982b. Structural features of the herpes simplex virus alpha gene 4, 0, and 27 promoter-regulatory sequences which confer alpha regulation on chimeric thymidine kinase. J. Virol. 44:939-49
57. MacLean AR, ul-Fareed M, Robertson L, Harland J, Brown SM. 1991. Herpes simplex virus type 1 deletion variants 1714 and 1716 pinpoint neurovirulenece-related sequences in Glasgow strain 17+ between immediate early gene 1 and the "a" sequence. J. Gen. Virol. 72:631-39
58. Margolis TP, Bloom DC, Dobson AT, Feldman LT, Stevens JG. 1993. Decreased reporter gene expression during latent infection with herpes simplex virus latency-associated transcript promoter constructs. Virology 197: 585-92
59. Martuza RL, Malick A, Markert JM, Ruffner KL, Coen DM. 1991. Experimental therapy of human glioma by means of a genetically engineered virus mutant. Science 252:854-86
60. McCarthy AM, McMahan L, Schaffer PA. 1989. Herpes simplex virus type 1 ICP27 deletion mutants exhibit altered patterns of transcription and are DNA deficient. J. Virol. 63:18-27
61. McFarland DJ, Sikora E, Hotchkin J. 1986. The production of focal herpes encephalitis in mice by stereotaxic inoculation of virus. Anatomical and behavioral effects. J. Neurol. Sci. 72:307-18
62. Meignier B, Longnecker R, Mavromara-Nazos P, Sears AE, Roizman B. 1988. Virulence of and establishment of latency by genetically engineered deletion mutants of herpes simplex virus type 1. Virology 162:251-54
63. Mellerick DM, Fraser NW. 1987. Physical state of the latent herpes simplex virus genome in a mouse model system: evidence suggesting an episomal state. Virology 158:265-75
64. Miller AD. 1992. Retroviral vectors. Curr. Top. Microbiol. Immunol. 158:1-24
65. Nicosia M, Deshmane SL, Zabolotny JM, Valyi-Nagy T, Fraser NW. 1993. Herpes simplex virus type 1 latency-associated transcript (LAT) promoter deletion mutants can express a 2-kilobase transcript mapping to the LAT region. J. Virol. 67:7276-83
66. Oroskar AA, Read GS. 1989. Control of mRNA stability by the virion host shutoff function of herpes simplex virus. J. Virol. 63:1897-906
67. Palella TD, Hidaka Y, Silverman LJ, Levine M, Glorioso JC, Kelley WM. 1989. Expression of human HPRT mRNA in brains of mice infected with a recombinant herpes simplex virus type 1 vector. Gene 80:137-44
68. Palella TD, Silverman LJ, Schroll CT, Homa FL, Levine M, Kelley WM. 1988. Herpes simplex virus-mediated human hypoxanthine-guanine phosphoribosyl-transferase gene transfer into neuronal cells. Mol. Cell Biol. 8:457-60
69. Panning B, Smiley JR. 1989. Regulation of cellular genes transduced by herpes simplex virus. J. Virol. 63:1929-37
70. Preston CM. 1979a. Control of herpes simplex virus type 1 mRNA synthesis in cells infected with wild-type virus or the temperature-sensitive mutant tsK. J. Virol. 29: 275-84
71. Preston C. 1979b. Abnormal properties of an immediate early polypeptide in cells infected with the herpes simplex virus type 1 mutant tsK. J. Virol. 32:357-69
72. Quantin B, Perricaudet LD, Tajbakhsh S, Mandel J-L. 1992. Adenovirus as an expression vector in muscle cells in vivo. Proc. Natl. Acad. Sci. USA 89:2581-84
73. Quinlan MP, Knipe DM. 1985. Stimulation of expression of a herpes simplex virus DNA-binding protein by two viral functions. Mol. Cell. Biol. 5:957-63
74. Ragot T, Vincent N, Chafey P, Gilgenkrantz H, Couton D, et al. 1993. Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice. Nature 321:647-50
75. Ramakrishnan R, Fink DJ, Jiang G, Desai P, Glorioso JC, Levine M. 1994. Competitive quantitative polymerase chain reaction (PCR) analysis of herpes simplex virus type 1 DNA and LAT RNA in latently infected cells of brain. J. Virol. 68:1864-70
76. Rice SA, Knipe DM. 1990. Genetic evidence for two different transactivation functions of the herpes simplex virus and protein ICP27. J. Virol. 64:1704-15
77. Rice SA, Long MC, Lam V, Spencer CA. 1994. RNA polymerase II is aberrantly phosphorylated and localized to viral replication compartments following herpes simplex virus infection. J. Virol. 68:988-1001
78. Rock DL, Fraser NW. 1983. Detection of HSV-1 genome in the central nervous system of latently infected mice. Nature 302:523-25
79. Rock DL, Fraser NW. 1985. Latent herpes simplex virus type 1 DNA contains two copies of the virion DNA joint region. J. Virol. 55: 849-52
80. Rock DL, Nesburn AB, Ghiasi H, Ong J, Lewis TL, et al. 1987. Detection of latency-related viral RNAs in trigeminal ganglia of rabbits infect with herpes simplex virus type 1. J. Virol. 61:3820-26
81. Roizman B, Sears AE. 1990. Herpes simplex viruses and their replication. In Virology, ed. B Fields, pp. 1795-841. New York: Raven
82. Sacks WR, Greene CC, Aschman DP, Schaffer PA. 1985. Herpes simplex virus type 1 ICP27 is essential regulatory protein. J. Virol. 55:796-805
83. Sacks WR, Schaffer PA. 1987. Deletion mutants in the gene encoding the herpes simplex virus type 1 immediate-early protein ICP0 exhibit impaired growth in cell culture. J. Virol. 61:829-39
84. Sawtell NM, Thompson RL. 1992. Herpes simplex virus type 1 latency-associated transcription unit promotes anatomical site-dependent establishment and reactivation from latency. J. Virol. 66:2157-69
85. Shin M-F, Arsenakis M, Tiollais P, Roizman B. 1984. Expression of hepatitis B virus S gene by herpes simplex virus type 1 vectors carrying alpha- and beta-regulated gene chimeras. Proc. Natl. Acad. Sci. USA 81:5867-70
86. Smiley JR, Smibert C, Everett RD. 1987. Expression of a cellular gene cloned in herpes simplex virus: rabbit beta-globin is regulated as an early viral gene in infected fibroblasts. J. Virol. 61:2368-77
87. Smith IL, Hardwicke MA, Sandri-Goldin RM. 1992. Evidence that herpes simplex virus immediate early protein ICP27 acts post-transcriptionally during infection to regulate gene expression. Virology 168:74-86
88. Spaete RR, Frenkel N. 1982. The herpes simplex virus amplicon: a new eucaryotic defective-virus cloning-amplifying vector. Cell 30:295-304
89. Spear PG. 1993. Membrane fusion induced by herpes simplex virus. In Viral Fusion Mechanisms, ed. J Bentz, pp. 201-32. Boca Raton: CRC
90. Spivack JG, Fraser NW. 1987. Detection of herpes simplex virus type 1 transcripts during latent infection in mice. J. Virol. 61: 3841-47
91. Spivack JG, Fraser NW. 1988. Expression of herpes simplex virus type 1 latency-associated transcripts in trigeminal ganglia of mice during acute infection and reactivation of latent infection. J. Virol. 62:1479-85
92. Spivack JG, Woods CM, Fraser NW. 1991. Identification of a novel latency-specific splice donor signal within HSV type 1 2.0-kilobase latency-associated transcript (LAT): translation inhibition of LAT open reading frames by the intron within the 2.0-kilobase LAT. J. Virol. 65:6800-10
93. Steiner I, Spivack JG, Lirette RP, Brown SM, MacLean AR, Subak-Sharpe JH. 1989. Herpes simplex virus type 1 latency-assoicated transcripts are evidently not essential for latent infection. EMBO J. 8:505-11
94. Stevens JG. 1989. Human herpesviruses: a consideration of the latent state. Microbiol. Rev. 53:318-32
95. Stevens JG, Wagner EK, Devi-Rao GB, Cook ML, Feldman LT. 1987. RNA complementary to a herpesviruses a gene mRNA is prominent in latently infected neurons. Science 255:1056-59
96. Stow ND, Stow EC. 1986. Isolation and characterization of a herpes simplex virus type 1 mutant containing a deletion within the gene encoding the immediate early polypeptide Vmw 110. J. Gen. Virol. 67: 2571-85
97. Sunstrum JC, Chrisp CE, Levine M, Glorioso JC. 1988. Pathogenicity of glycoprotein C negative mutants in the mouse central nervous system. Virus Res. 11:17-32
98. Takamiya Y, Short MP, Ezzeddine ZD, Moolton FL, Breakefield XO, Martuza RL. 1992. Gene therapy of malignant brain tumors: a rat glioma line bearing the herpes simplex virus type 1 thymidine kinase gene and wild type retrovirus kills other tumor cells. J. Neurosci. Res. 33:493-503
99. Trousdale MD, Steiner I, Spivack JG, Deshmane SM, Brown S, MacLean AR. 1991. In vivo and in vitro reactivation impairment of a herpes simplex virus type 1 latency-associated transcript variant in a rabbit eye model. J. Virol. 65:6989-93
100. Vincent N, Ragot T, Gilgenkrantz H, Couton D, Chafey P, et al. 1993. Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene. Nat. Genet. 5:130-34
101. Wagner EK, Flanagan WM, Devi-Rao GB, Zhang YF, Hill JM, et al. 1988. The herpes simplex virus latency-associated transcript is spliced during the latent phase of infection. J. Virol. 62:4577-85
102. Watson RJ, Clements JB. 1980. A herpes simplex virus type 1 function continuously required for early and late virus RNA synthesis. Nature 285:329-30
103. 1 34.5 deletion mutants in rodent models. J. Clin. Invest. 91:2837-43
104. Wilcox CL, Crnic LS, Pizer LI. 1992. Replication, latent infection, and reactivation in neuronal culture with a herpes simplex virus thymidine kinase-negative mutant. Virology 187:348-52
105. Wilcox CL, Johnson EM Jr. 1987. Nerve growth factor deprivation results in the reactivation of latent herpes simplex virus in vitro. J. Virol. 61:2311-15
106. Wilcox CL, Johnson EM Jr. 1988. Characterization of nerve growth factor-dependent herpes simplex virus latency in neurons in vitro. J. Virol. 62:393-99
107. Wilcox CL, Smith RL, Freed CR, Johnson EM Jr. 1990. Nerve growth factor-dependence of herpes simplex virus latency in peripheral sympathetic and sensory neruons in vitro. J. Neurosci. 10:1268-75
108. Yang Y, Nunes FA, Berencis K, Gonczol E, Engelhardt JF, Wilson JM. 1994. Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis. Nat. Genet. 7:362-69
109. York IA, Roo C, Andrews DW, Riddell SR, Graham L, Johnson DC. 1994. A cytosolic herpes simplex virus protein inhibits antigen presentation to CD8+ T lymphocytes. Cell 77:525-35
110. Zwaagstra JC, Ghiasi H, Slanina SM, Nesburn AB, Wheatley SC, et al. 1990. Activity of herpes simplex virus type 1 latency-associated transcript (LAT) promoter in neuron-derived cells: evidence for neuron specificity and for a large LAT transcript. J. Virol. 64: 5019-28