Development of an HSV-based vector for the treatment of Parkinson's disease

Experimental Neurology

Volumn 144, Issue 1, 1997, Pages 103-112

  Fink, David J ^a   Poliani, P Luigi ^a   Oligino, Thomas ^a   Krisky, David M ^a   Goins, William F ^a   Glorioso, Joseph C ^a  

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

Author keywords

[No Author keywords available]

Indexed keywords

NEUROTROPIC AGENT; PLASMID VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; DOPAMINERGIC NERVE CELL; GENE EXPRESSION; GENE THERAPY; HERPES SIMPLEX VIRUS; NONHUMAN; PARKINSON DISEASE; PRIORITY JOURNAL;

EID: 0031105958     PISSN: 00144886     EISSN: None     Source Type: Journal    
DOI: 10.1006/exnr.1996.6395     Document Type: Article

References (87)

1. 1. ACE, C. I., T. A. MCKEE, J. M. RYAN, J. M. CAMERON, AND C. M. PRESTON. 1989. Construction and characterization of a herpes simplex virus type 1 mutant unable to transinduce immediate-early gene expression. J. Virol. 63: 2260-2269.
2. 2. ANDERSON, J., D. GARBER, C. MEANEY, AND X. BREAKFIELD. 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: 487-499.
3. 3. AXELROD, J. D., M. S. REAGAN, AND J. MAJORS. 1993. GAL4 disrupts a repressing nucleosome during activation of GAL 1 transcription in vivo. Genes Dev. 7: 857-869.
4. 4. BATCHELOR, A. H., AND P. O. O'HARE. 1990. Regulation and cell-type-specific activity of a promoter located upstream of the latency-associated transcript of herpes simplex virus type 1. J. Virol. 64: 3269-3279.
5. 5. BATCHELOR, A. H., AND P. O. O'HARE. 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: 3573-3582.
6. 6. BATTLEMAN, D., A. GELLER, AND M. CHAO. 1993. HSV-1 vector-mediated gene transfer of the human nerve growth factor receptor p75hNGFR defines high-affinity NGF binding. J. Neurosci. 13: 941-951.
7. 7. BECK, K., J. VALVERDE, T. ALEXI, K. POULSON, B. MOFFAT, R. VANDLEN, A. ROSENTHAL, AND F. HEFTI. 1995. Mesencephalic dopaminergic neurons protected by GDNF from axotomy-induced degeneration in the adult brain. Nature 373: 339-341.
8. 8. CAREY, M., J. LEATHERWOOD, AND M. PTASHNE. 1990. A potent GAL4 derivative activates transcription at a distance in vitro. Science 247: 710-712.
9. 9. CHASMAN, D. I., M. LEATHERWOOD, M. CAREY, M. PTASHNE, AND R. D. KORNBERG. 1989. Activation of yeast polymerase II transcription by herpesvirus VP16 and GAL4 derivative in vitro. Mol. Cell. Biol. 9: 4746-4749.
10. 10. CHEN, X., M. SCHMIDT, W. GOINS, D. FINK, R. HENDRICKS, AND J. GLORIOSO. 1996. HSV-1 vector-mediated lacZ gene transfer to the nervous system: Long-term gene expression using the viral latency-active promoter. Manuscript in preparation.
11. 11. CHEN, X., M. C. SCHMIDT, W. F. GOINS, AND J. C. GLORIOSO. 1995. Two herpes simplex virus type-1 latency active promoters differ in their contribution to latency-associated transcript expression during lytic and latent infection. J. Virol. 69: 7899-7908.
12. 12. CHRISP, C., J. SUNSTRUM, D. AVERILL, M. LEVINE, AND J. GLORIOSO. 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: 822-830.
13. 13. COTZIAS, G., H. V. WOERT, AND L. SCHIFFER 1967. Aromatic amino acids and modification of parkinsonism. N. Engl. J. Med. 276: 374-379.
14. 14. DELUCA, N. A., A. M. MCCARTHY, AND P. A. SCHAFFER 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: 558-570.
15. 15. DELUCA, N. A., AND P. A. SCHAFFER 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: 1997-2008.
16. 16. DESAI, P., R. RAMAKRISHNAN, Z. W. LIN, B. OZAK, J. C. GLORIOSO, AND M. LEVINE. 1993. The RR1 gene of herpes simplex virus type 1 is uniquely transactivated by ICP0 during infection. J. Virol. 67: 6125-6135.
17. 17. DIXON, R. A. F., AND P. A. SCHAFFER 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.
18. 18. DOBSON, A. T., F. SEDERATI, G. DEVI-RAO, W. M. FLANAGAN, M. J. FARRELL, J. G. STEVENS, E. K. WAGNER, AND L. T. FELDMAN. 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: 3844-3851.
19. 19. DURING, M., M. KAPLITT, P. LOONE, X. XINO, J. ELSWORTH, R. ROTH, J. SLADEK, D. REDMOND, AND K. O'MALLEY. 1994. AAV-mediated transfer of tyrosine hydroxylase and aromatic amino acid decarboxylase into the primate brain: A direct gene therapy approach to Parkinson's disease. Soc. Neurosci. Abstr. 20: 602.10.
20. 20. DURING, M., J. NAEGELE, K. O'MALLEY, AND A. GELLER. 1994. Long-term behavioral recovery in parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 266: 1399-1403.
21. 21. FINK, D. J., L. R. STERNBERG, P. C. WEBER, M. MATA, W. F. GOINS, AND J. C. GLORIOSO. 1992. In vivo expression of β-galactosidase in hippocampal neurons by HSV-mediated gene transfer. Hum. Gene Ther. 3: 11-19.
22. 22. FISHER, K. J., G.-P. GAO, M. D. WEITZMAN, R. DEMATTEO, J. F. BURDA, AND J. M. WILSON. 1996. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70: 520-532.
23. 23. FRAEFEL, C., S. SONG, F. LIM, P. LANG, L. Yu, Y. WANG, AND A. GELLER. 1995. An efficient helper virus-free packaging system for herpes simplex type 1 plasmid vectors. Proc. 20th Int. Herpesvirus Workshop, p. 32. Groningen, The Netherlands.
24. 24. FREED, C., R. BREEZE, N. ROSENBERG, S. SCHNECK, E. KRIEK, AND J.-X. QI. 1993. Survival of implanted dopamine cells and neurologic improvement 12 to 46 months after transplantation for Parkinson's disease. N. Engl. J. Med. 327: 1549-1555.
25. 25. FREESE, A., A. GELLER, AND R. NEVE. 1990. HSV-1 vector mediated neuronal gene delivery: Strategies for molecular neuroscience and neurology. Biochem. Pharmacol. 40: 2189-2199.
26. 26. GASH, D. M., Z. ZHANG, A. OVDARA, et al. 1996. Functional recovery in parkinsonian monkeys treated with GDNF. Nature 380: 252-255.
27. 27. GELLER, A., AND X. BREAKFIELD. 1988. A defective HSV-1 vector expresses Escherichia coli β-galactosidase in cultured peripheral neurons. Science 241: 1667-1669.
28. 28. GELLER, A., M. DURING, J. HAYCOCK, A. FREESE, AND R. NEVE. 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: 7603-7607.
29. 29. GELLER, A., M. DURING, AND R. NEVE. 1991. Molecular analysis of neuronal physiology by gene transfer into neurons with herpes simplex virus vectors. Trends Neurosci. 14: 28-32.
30. 30. GELLER, A., M. DURING, Y. OH, A. FREESE, AND K. O'MALLEY. 1995. An HSV-1 vector expressing tyrosine hydroxylase causes production and release of L-dopa from cultured rat striatal cells. J. Neurochem. 64: 487-496.
31. 31. GELLER, A., AND A. FREESE. 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: 1149-1153.
32. 32. GESCHWIND, M., J. KESSLER, A. GELLER, AND H. FEDEROFF. 1994. Transfer of the nerve growth factor gene into cell lines and cultured neurons using a defective herpes simplex virus vector: Transfer to the NGF gene into cells by a HSV-1 vector. Brain Res. 24: 327-335.
33. 33. GOINS, W. F., L. R. STERNBERG, K. D. CROEN, P. R. KRAUSE, R. L. HENDRICKS, D. J. FINK, S. E. STRAUS, M. LEVINE, AND J. C. GLORIOSO. 1994. A novel latency-active promoter is contained within the herpes simplex virus type 1 UL flanking repeats. J. Virol. 68: 2239-2252.
34. 34. GREENFIELD, J., AND F. BOUSANGUET. 1953. The brain-stem lesions in parkinsonism. J. Neurol. Neurosurg. Psychiatry 16: 213-216.
35. 35. HILL, T. J. 1985. Herpes simplex virus latency. Pages 175-240 in B. Roizman, Ed., The Herpesviruses. Plenum Press, New York.
36. 36. Ho, D., E. MOCARSKI, AND R. SAPOLSKY. 1993. Altering central nervous system physiology with a defective herpes simplex virus vector expressing the glucose transporter gene. Proc. Natl. Acad. Sci. USA 90: 3655-3659.
37. 37. Ho, D. Y., AND E. S. MOCARSKI. 1989. Herpes simplex virus latent RNA (LAT) is not required for latent infection in the mouse. Proc. Natl. Acad. Sci. USA 86: 7596-7600.
38. 38. HOFFER, B., A. HOFFMAN, K. BOWENKAMP, P. HUETTL, J. HUDSON, D. MARTIN, L.-F. LIN, AND G. GERHARD. 1995. GDNF reverses toxin-induced injury to midbrain dopaminergic neurons in vivo. Neurosci. Lett. 182: 107-111.
39. 39. HONESS, R., AND B. ROIZMAN. 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.
40. 40. HORNIGKIEWICZ, O. 1963. Die Topische Lokalisation und das Verhalten van Noradrenalin und Dopamin un der substantia nigra des normalen und Parkinsonkranken Menschen. Wien Klin. Wochenschr. 75: 309-312.
41. 41. ISACSON, O. 1995. Behavioral effects and gene delivery in a rat model of Parkinson's disease. Science 269: 858.
42. 42. JIAO, S., V. GUREVICH, AND J. WOLFF. 1993. Long-term correction of rat model of Parkinson's disease by gene therapy. Nature 362: 450-453.
43. 43. JIAO, S., V. GUREVICH, AND J. A. WOLFF. 1996. Long-term correction of rat model of Parkinson's disease by gene therapy [retraction]. Nature 380: 734.
44. 44. JOHNSON, P., A. MIYANOHARA, F. LEVINE, T. CAHILL, AND T. FRIEDMANN. 1992. Cytotoxicity of a replication-defective mutant herpes simplex virus type 1. J. Virol. 66: 2952-2965.
45. 45. JOHNSON, P., M. WANG, AND T. FRIEDMAN. 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 viron host shutoff function. J. Virol. 68: 6347-6362.
46. 46. KAPLITT, M., A. KWONG, S. KLEOPOULOS, C. MOBBS, S. RABKIN, AND D. PFAFF. 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.
47. 47. KAPLITT, M., P. LEONE, R. SAMULSKI, X. XIAO, D. PFAFF, K. O'MALLEY, AND M. DURING. 1994. Long-term gene expression and phenotype correction using adeno-associated virus vectors in the mammalian brain. Nat. Genet. 8: 148-154.
48. 48. KEARNS, C., AND D. GASH. 1995. GDNF protects nigral dopamine neurons against 6-hydroxydopamine in vivo. Brain Res. 672: 104-111.
49. 49. KRISTIE, T., AND P. SHARP. 1993. Purification of the cellular C1 factor required for the stable recognition of the Oct-1 homeodomain by herpes simplex virus α-trans-induction factor (VP16). J. Biol. Chem. 268: 6525-6534.
50. 50. KWONG, A. D., J. A. KRUPER, AND N. FRENKEL. 1988. Herpes simplex virus virion host shutoff function. J. Virol. 62: 912-921.
51. 51. LEIDEN, J., N. FRENKEL, AND F. RAPP. 1980. Identification of the herpes simplex virus DNA sequences present in six herpes simplex virus thymidine kinase-transformed mouse cell lines. J. Virol. 33: 272-285.
52. 52. LIN, L., D. DOHERTY, J. LILE, S. BEHTESH, AND F. COLLINS. 1993. GDNF: A glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science 260: 1130-1132.
53. 53. LOKENSGARD, J. R., D. C. BLOOM, A. T. DOBSON, AND L. T. FELDMAN. 1994. Long-term promoter activity during herpes simplex virus latency. J. Virol. 68: 7148-7158.
54. 54. MARGOLIS, T. P., D. C. BLOOM, A. T. DOBSON, L. T. FELDMAN, AND J. G. STEVENS. 1993. Decreased reporter gene expression during latent infection with HSV LAT promoter constructs. Virology 197: 585-592.
55. 55. MCCARTHY, A. M., L. MCMAHAN, AND P. A. SCHAFFER. 1989. Herpes simplex virus type 1 ICP27 deletion mutants exhibit altered patterns of transcription and are DNA deficient. J. Virol. 63: 18-27.
56. 56. MEIGNIER, B., R. LONGNECKER, P. MAVROMARA-NAZOS, A. SEARS, AND B. ROIZMAN. 1988. Virulence of and establishment of latency by genetically engineered deletion mutants of herpes simplex virus type 1. Virology 162: 251-254.
57. 57. MESTER, J. C., P. PITHA, AND J. C. GLORIOSO. 1995. Anti-viral activity of herpes simplex virus vectors expressing alpha-interferon. Gene Ther. 3: 187-196.
58. 58. MOURADIAN, M., AND T. CHASE. 1994. Improved dopaminergic therapy of Parkinson's disease. Pages 181-199 in C. D. Marsden and S. Fahn, Eds., Movement Disorders, Vol. 3. Butterworth-Heinemann, Oxford.
59. 59. NICOSIA, M., S. L. DESHMANE, J. M. ZABOLOTNY, T. VALYI-NAGY, AND N. W. FRASER 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: 7276-7283.
60. 60. O'HARE, P., AND C. GODING. 1988. Herpes simplex virus regulatory elements and the immunoglobulin octamer domain bind a common factor and are both targets for virion transactivation. Cell 52: 435-445.
61. 61. OLIGINO, T., P. L. POLIANI, P. MARCONI, M. A. BENDER, M. C. SCHMIDT, D. J. FINK, AND J. C. GLORIOSO. 1996. In vivo transgene activation from an HSV-based gene vector by GAL4:VP16. Submitted for publication.
62. 62. OROSKAR, A., AND G. READ. 1989. Control of mRNA stability by the virion host shutoff function of herpes simplex virus. J. Virol. 63: 1897-1906.
63. 63. PAGE, B., M. PAGE, AND C. NOEL. 1993. A new fluorometric assay for cytotoxicity measurements in vitro. Int. J. Oncol. 3: 473-476.
64. 64. PAK, A., D. N. EVERLY, K. KNIGHT, AND G. S. READ. 1995. The virion host shutoff protein of herpes simplex virus inhibits reporter gene expression in the absence of other viral gene products. Virology 211: 491-506.
65. 65. PATERSON, T., AND R. D. EVERETT. 1990. A prominent serine-rich region in Vmw175, the major regulatory of herpes simplex virus type 1, is not essential for virus growth in tissue culture. J. Gen. Virol. 71: 1775-1783.
66. 66. PRESTON, C., M. FRAME, AND M. CAMPBELL. 1988. A complex formed between cell components and an HSV structural polypeptide binds to a viral immediate early gene regulatory DNA sequence. Cell 52: 425-434.
67. 67. RAMAKRISHNAN, R., D. J. FINK, J. GUIHUA, P. DESAI, J. C. GLORIOSO, AND M. LEVINE. 1994. Competitive quantitative polymerase chain reaction (PCR) analysis of herpes simplex virus type 1 DNA and LAT RNA in latently infected cells of the rat brain. J. Virol. 68: 1864-1870.
68. 68. RASTY, S., W. F. GOINS, AND J. C. GLORIOSO. 1995. Site-specific integration of multigenic shuttle plasmids into the herpes simplex virus type 1 (HSV-1) genome using a cell-free Cre-lox recombination system. Pages 114-130 in K. Adolph, Ed., Methods in Molecular Genetics. Academic Press, San Diego.
69. 69. RASTY, S., P. POLIANI, D. FINK, AND J. GLORIOSO. 1996. Deletion of the S component inverted repeat sequence c′ and the nonessential genes Us1 through Us5 from the herpes simplex virus type 1 genome substantially impairs productive viral infection in cell culture and pathogenesis in the rat central nervous system. Submitted for publication.
70. 70. READ, G. S., AND N. FRENKEL. 1983. Herpes simplex virus mutants defective in the virion-associated shutoff of host polypep tide synthesis and exhibiting abnormal synthesis of a (immediate early) viral polypeptides. J. Virol. 46: 498-512.
71. 71. SADOWSKI, I., J. MA, S. TRIEZENBERG, AND M. PTASHNE. 1988. GAL4/VP16 is an unusually potent transcriptional activator. Nature 335: 563-564.
72. 72. SOARES, M. K., D.-Y. HWANG, M. C. SCHMIDT, D. J. FINK, AND J. C. GLORIOSO. 1996. Cis-acting elements involved in transcriptional regulation of the herpes simplex virus type-1 latency-associated promoter 1 (LAP1) in vitro and in vivo. J. Virol. 70: 5384-5394.
73. 73. SPAETE, R., AND N. FRENKEL. 1982. The herpes simplex virus amplicon: A new eucaryotic defective-virus cloning amplifying vector. Cell 30: 295-304.
74. 74. SPECK, P., AND A. SIMMONS. 1992. Synchronous appearance of antigen-positive and latently infected neurons in spinal ganglia of mice infected with a virulent strain of herpes simplex virus. J. Gen. Virol. 73: 1281-1285.
75. 75. SPECK, P. G., AND A. SIMMONS. 1991. Divergent molecular pathways of productive and latent infection with a virulent strain of herpes simplex virus type 1. J. Virol. 65: 4004-4005.
76. 76. SPENCER, D., R. ROBBINS, F. NAFTOLIN, K. MAREK, T. VOLLMER, AND C. LERANTH. 1993. Unilateral transplantation of human fetal mesencephalic tissue into the caudate nucleus of patients with Parkinson's disease. N. Engl. J. Med. 327: 151-148.
77. 77. STEVENS, J. G. 1989. Human herpesviruses: A consideration of the latent state. Microbiol. Rev. 53: 318-332.
78. 78. TOMAC, A., E. LINQUIST, L.-F. LIN, S. OGREN, D. YOUNG, B. HOFFER, AND L. OLSON. 1995. Protection and repair of the nigro-striatal dopaminergic system by GDNF in vivo. Nature 373: 335-339.
79. 79. VEGETO, E., G. F. ALLAN, W. T. SCHRADER, M.-J. TSAI, D. P. MCDONNELL, AND B. W. O'MALLEY. 1992. The mechanism of RU486 antagonism is dependent on the conformation of the carboxy-terminal tail of the human progesterone receptor. Cell 69: 703-713.
80. 80. WANG, Y., B. O'MALLEY JR., S. TSAI, AND B. O'MALLEY. 1994. A novel regulatory system for gene transfer. Proc. Natl. Acad. Sci. USA 91: 8180-8184.
81. 81. WERSTUCK, G., AND J. CAPONE. 1993. An unusual cellular factor potentiates protein-DNA complex assembly of Oct-1 and Vmw65. J. Biol. Chem. 268: 1272-1278.
82. 82. WHITLEY, R., E. KERN, S. CHATTERJEE, J. CHOU, AND B. ROIZMAN. 1993. Replication establishment of latency, and induced reactivation of herpes simplex virus gl 34.5 deletion mutants in rodent models. J. Clin. Invest. 91: 2837-2843.
83. 83. WIDNER, H., J. TETRUD, S. REHNORONA, B. SNOW, P. BRUNDIN, AND B. GUSTAVII. 1993. Bilateral fetal mesencephalic grafting in two patients with Parkinsonim induced by MPTP. N. Engl. J. Med. 327: 1556-1563.
84. 84. WILSON, A., K. LAMARCO, M. PETERSON, AND W. HERR. 1993. The VP16 accessory protein HCF is a family of polypeptides processed from a large precursor protein. Cell 74: 115-125.
85. 85. WOLFE, J. H., S. L. DESHMANE, AND N. W. FRASER 1992. Herpesvirus vector gene transfer and expression of β-glucuronidase in the central nervous system of MPS VII mice. Nat. Genet. 1: 379-384.
86. 86. Xu, L., W. SCHAFFNER, AND D. RUNGGER. 1993. Transcription activation by recombinant GAL4/VP16 in the Xenopus oocyte. Nucleic Acids Res. 21: 2775.
87. 87. ZWAAGSTRA, J. C., H. GHIASI, S. M. SLANINA, A. B. NESBURN, S. C. WHEATLEY, K. LILLYCROP, J. WOOD, K. LATCHMAN, K. PATEL, AND S. L. WECHSLER 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: 5019-5028.