Defective viral vectors as agents for gene transfer in the nervous system

Journal of Neuroscience Methods

Volumn 71, Issue 1, 1997, Pages 125-132

  Kaplitt, Michael G ^a   Makimura, Hideo ^a  

^a ROCKEFELLER UNIVERSITY   (United States)

Author keywords

defective viral vectors; gene transfer; nervous system

Indexed keywords

ADENOVIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; GENE EXPRESSION; GENE TRANSFER; HERPES SIMPLEX VIRUS; NERVOUS SYSTEM; NONHUMAN; PRIORITY JOURNAL; REVIEW;

EID: 0030964838     PISSN: 01650270     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0165-0270(96)00132-X     Document Type: Article

References (87)

1. Atchison, R.W., Casto, B.C. et al. (1965) Adenovirus-associated defective virus particles, Science, 149: 754-756.
2. Bartlett, J.S. and Samulski, R.J. (1995) Genetics and biology of adeno-associated virus. In M.G. Kaplitt and A.D. Loewy (Eds.), Viral Vectors, Academic Press, San Diego, CA, pp. 56-76.
3. Berkner, K.L. (1988) Development of adenovirus vectors for the expression of heterologous genes, Biotechniques, 6: 616-629.
4. Berns. K.I., Cheung, A., Ostove, J.M. and Lewis, M. (1982) Adeno-associated virus latent infection. In B.W.J. Mahy, A.C. Minson et al. (Eds.), Virus Persistence, Cambridge University Press, Cambridge, p. 249.
5. Berns, K.I., Pinkerton, T.C. et al. (1975) Detection of adeno-associated virus (AVV) specific nucleotide sequences in DNA isolated from latently infected Detroit 6 cells, Virology, 68: 556-560.
6. Buller, R.M., Janik, J.E., Sebring, E.D. and Rose, J.A. (1981) Herpes simplex virus types 1 and 2 completely help adenovirus-associated virus replication, J. Virol., 40: 241-247.
7. Challberg, M.D. (1991) Herpes simplex virus DNA replication, Semin. Virol., 2: 247-256.
8. Chang, L.-S., Shi, Y. and Shenk, T. (1989) Adeno-associated virus p5 promoter contains an adenovirus EIA inducible element and a binding site for the major large transcription factor, J. Virol., 63: 3479-3488.
9. Cheung, A.K., Hoggan, M.D. et al. (1980) Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells, J. Virol., 33: 739-748.
10. Deiss, L.P., Chou, J. and Frenkel, N. (1986) Functional domains within the a sequence involved in the cleavage/packaging of herpes simplex virus DNA. J. Virol., 59: 605-618.
11. Deiss, L.P. and Frenkel, N. (1986) The herpes simplex virus amplicon: cleavage of concatemeric DNA is linked to packaging and involves amplification of the terminally reiterated a sequence, J. Virol., 57: 933-941.
12. DeLuca, N.A., McCarthy, A.M. and Schaffer, P.A. (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.
13. During, M.J., Naegele, J.R., O'Malley, K.L. and Geller, A.I. (1994a) Long-term behavioral recovery in Parkinsonian rats by an HSV vector expressing tyrosine hydroxylase, Science, 266: 1399-1403.
14. Elias, P. and Lehman, I.R. (1988) Interaction of origin binding protein with an origin of replication of HSV type l, Proc. Natl. Acad. Sci. USA, 85, 2959-2963.
15. Engelhardt, J.F., Ye, X., Doranx, B. and Wilson, J.M. (1994) Ablation of E2A in recombinant adenovirus improves transgene persistence and decreases inflammatory response in mouse liver, Proc. Natl. Acad. Sci. USA, 91: 6196-6200.
16. Federoff, H.J., Geschwind, M.D., Geller, A.I. and Kessler, J.A. (1992) Expression of nerve growth factor in vivo, from a defective HSV1 vector prevents effects of axotomy on sympathetic ganglia, Proc. Natl. Acad. Sci. USA, 89: 1636-1640.
17. Federoff, H.J. (1995) The use of defective herpes simplex virus amplicon vectors to modify neural cells and the nervous system. In: M.G. Kaplitt and A.D. Loew (Eds.), Viral Vectors, pp. 109-118.
18. Flint, J. and Shenk, T. (1989) Adenovirus EIA protein paradigm viral transactivator, Annu. Rev. Genet., 23: 141-161.
19. Flott, T.R., Afione, S.A., Conrad, C., McGrath, S.A., Solow, R., Oka, H., Zeitlies, P.L., Guggino, W.B. and Carter, B.J. (1993) Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with adeno-associated virus, Proc. Natl. Acad. Sci. USA, 90: 10613-10617.
20. Frenkel, N. (Ed.) (1981) Defective interfering herpes virus. In: The Human Herpes Virus - An Interdisciplinary Prospective. Elsevier-North Holland, New York.
21. Frenkel, N., Locker, H., Batterson, B., Hayward, G.S. and Roizman, B. (1976) Anatomy of herpes simplex virus VI. Defective DNA originates from the S component, J. Virol., 20: 527-531.
22. Frenkel, N., Locker, H. and Vlazny, D.A. (1980) Studies of defective herpes simplex viruses, Ann. NY Acad. Sci., 354: 347-370.
23. Geller, A.I. and Breakefield, X.O. (1988) A defective HSV 1 vector expresses E. coli beta-galactosidase in cultured peripheral neurons, Science, 241: 1667-1669.
24. Geller, A.I., During, M.J., Oh, Y.J., Freese, A. and O'Malley, K.L. (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.
25. Geller, A.I., Keyomarsi, K., Bryan, J. and Pardee, A.B. (1990) An efficient deletion mutant packaging system for defective herpes simplex vectors: potential applications to human gene therapy and neuronal physiology, Proc. Natl. Acad. Sci. USA, 87: 8950-8954.
26. Ginsberg, H.S., Moldawer, L.L., Sehgal, P.B., Redington, M., Kilian, P.L., Chanok, R.M. and Prince, G.A. (1991) A mouse model for investigating the molecular pathogenesis of adenovirus pneumonia, Proc. Natl. Acad. Sci. USA, 88: 1651-1655.
27. Goff, S.P. and Shenk, T. (1993) Sleeping with the enemy: viruses as gene transfer agents, Curr. Opin. Genet. Dev., 3: 71-73.
28. Goodman, S., Xiao, X., Donahue, R.E., Moulton, A., Miller, J., Walsh, C., Young, N.S., Samulski, R.J. and Nienhuis, A.W. (1994) Recombinant adeno-associated virus-mediated gene transfer into hematopoietic progenitor cells, Blood, 84 (5): 1492-1500.
29. Graham, F.L. and Prevec, L. (1992) In R.W. Ellis (Ed.), Vaccines: New Approaches to Immunological Problems. Butterworth-Heinemann, Boston, pp. 363-390.
30. Halbert, D.N., Cutt, J.R. and Shenk, T. (1985) Adenovirus early region 4 encodes functions required for efficient DNA replication, late gene expression and host cell shutoff, J. Virol., 56: 250-257.
31. Handa, H., Hiroki, K. et al. (1977) Establishment and characterization of KB cell lines latently infected with adeno-associated virus type 1, Virology, 82: 84-92.
32. Ho, D., Mocarski, E. and Sapolski, R. (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.
33. Hoggan, M.D., Blacklow, N.R. and F.B. Rowe (1966) Studies of small DNA viruses found in various adenovirus preparations: physical biological and immunological characteristics, Proc. Natl. Acad. Sci. USA, 55: 1457-1471.
34. Hoggan, M.D., Thomas, G.F. and Johnson, F.B. (1972) Continuous carriage of adenovirus associated virus genome in cell culture in the absence of helper adenovirus. In Proceedings of the Fourth Lepetite Colloquium, Cocoyac, Mexico.
35. Hermonat, P.L. and Muzyczka, N. (1984) Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells, Proc. Natl. Acad. Sci. USA, 81: 6466-6470.
36. Huang, A.S. (1973) Defective interfering viruses, Annu. Rev. Microbiol., 27: 101-117.
37. Janik, J.E., Huston, M.M., Cho, K. and Rose, J.A. (1989) Efficient synthesis of adeno-associated virus structural proteins requires both adenovirus DNA binding protein and VA I RNA, Virology, 168: 320-329.
38. Jay, F.T., De La Maza, L.M. and Carter, B.J. (1979) Parvovirus RNA transcripts containing sequences not present in mature mRNA: a method for isolation of putative mRNA precursor sequences, Proc. Natl. Acad. Sci. USA, 76: 625-629.
39. Jay, F.T., Laughlin, C.A. and Carter, B.J. (1981) Eukaryotic translational control: adeno-associated virus protein synthesis is arrested by a mutation in the adenovirus DNA binding protein, Proc. Natl. Acad. Sci. USA, 78: 2927-2931.
40. Johnson, F.B., Ozer, H.L. and Hoggan, M.D. (1971) Structural proteins of adenovirus-associated virus type 3, J. Virol., 8: 860-863.
41. Johnson, F.B., Whitaker, C.W. and Hoggan, M.D. (1975). Structural polypeptides of adenovirus associated virus top component, Virology, 65: 196-203.
42. Kaplitt, M.G., Pfaus, J.G., Kleopoulas, S.P., Houlon, B.A., Rabkin, S.O. and Pfaff, D.W. (1991) Expression of a functional foreign gene in adult mammalian brain following in vivo transfer via a herpes simplex virus defective viral vector, Mol. Cell. Neurosci., 2: 320-330.
43. Kaplitt, M.G., Leone, P., Samulski, R.J., Xiao, X., Pfaff, D.W., O'Malley, K.L. and During, M.J. (1994a) Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain, Nature Genet., 8: 148-153.
44. Kaplitt, M.G., Kwong, A.D., Kleopoulos, S.P., Mobbs, C.V., Rabkin, S.D. and Pfaff, D.W. (1994b) 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.
45. Kotin, R.M., Sinisalco, M., Samulskio, R.J., Zhu, X.D., Huuter, L. and Laughlin, C.A. (1990) Site-specific integration by adeno-associated virus, Proc. Natl. Acad. Sci. USA, 87: 2211-2215.
46. Kozarsky, K. and Wilson, J.M. (1993) Gene therapy: adenovirus vectors, Curr. Opin. Genet. Dev., 3: 499-503.
47. Kwong, A.D. and Frenkel, N. (1995) Biology of herpes simplex virus (HSV) defective viruses and development of the Amplicon system. In M.G. Kaplitt and A.D. Loewy (Eds.) Viral Vectors, Academic Press, San Diego, CA, pp. 25-42.
48. Locker, H. and Frenkel, N. (1979) Structure and origin of defective genomes contained in serially passaged herpes simplex virus type 1 (Justin), J. Virol., 29: 1065-1077.
49. Locker, H., Frenkel, N. and Halliburton, I. (1982) Structure and expression of class II defective herpes simplex virus genomes encoding infected cell polypeptide number 8, J. Virol., 43: 574-593.
50. McLaughlin, S.K., Collis, P. and Hermonat, P.L. (1988) Adeno-associated virus general transduction vectors: analysis of proviral structures, J. Virol., 62: 1963-1973.
51. McPherson, R.A., Ginsburg, H.S. and Rose, J.A. (1982) Adeno-associated virus helper activity of adenovirus DNA binding protein, J. Virol., 44: 666-673.
52. McPherson, R.A., Rosenthal, L.J. and Rose, J.A. (1985) Human cytomegalovirus completely helps adeno-associated virus replication, Virology, 147: 217-222.
53. Melnick, J.L., Mayor, H.D., Smith, K.O. and Rapp, F. (1965) Association of 20 millimicron particles with adenoviruses, J. Bacteriol., 90: 271-274.
54. Miller, J.L., Donahue, R.E., Seller, S.E., Sanutski, R.J., Yonne, N.S. and Nienhuis, A.W. (1994) Recombinant adeno-associated virus (rAAV) mediated expression of a human γ-globin gene in human progenitor derived erythyroid cells, Proc. Natl. Acad. Sci. USA, 91: 10183-10187.
55. Myers, M.W., Laughlin, C.A., Jay, F.T. and Carter, B.J. (1980) Adenovirus helper function for growth of adeno-associated virus: effect of temperature-sensitive mutations in adenovirus early gene region 2, J. Virol., 35: 65-75.
56. Olivio, P.D., Nelson, N.J. and Challberg, M.D. (1991) Herpes simplex virus replication: the UL9 gene encodes an origin binding protein, Proc. Natl. Acad. Sci. USA, 85: 5414-5418.
57. Paterson, T. and Everett, R. (1990) A prominent serine rich region in Vmw 175, the major transcriptional regulator protein of herpes simplex virus type 1 is not essential for viral growth in tissue culture, J. Gen. Virol., 71: 1775-1783.
58. Richardson, W.D. and Westphal, W.D. (1981) A cascade of adenovirus early functions is required for expression of adeno-associated virus, Cell, 27: 133-141.
59. Richardson, W.D. and Westphal, W.D. (1984) Requirement for either early region 1A or early region 1B adenovirus gene products in the helper effect for adeno-associated virus, J. Virol., 51: 404-410.
60. Roizman, B. (1990) Herpes viridae: a brief introduction. In B. Fields and D. Knipes (Eds.), Virology, Raven Press, New York, pp. 1787-1794.
61. Roizman, B. and Jenkins, F. (1985) Genetic engineering of novel genomes of large DNA viruses, Science, 229: 1208-1214.
62. Roizman, B. and Sears, A. (1990) Herpes simplex virus and their replication. In B. Fields and D. Knipes (Eds.), Raven Press, New York, pp. 1795-1842.
63. Rose, J.A., Maizel, J.K. et al. (1971) Structural proteins of adenovirus-associated viruses, J. Virol., 8: 766-770.
64. Samulski, R.J., Chang, L.-S. and Shenk, T. (1987) A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication, J. Virol., 61: 3096-3101.
65. Samulski, R.J., Chang, L.-S. and Shenk, T. (1989) Helper-free stock of recombinant adeno-associated viruses: normal integration does not require viral gene expression, J. Virol., 63: 3822-3828.
66. Samulski, R.J., Zhu, X., Xiao, X., Brook, J.D., Housman, D.E., Epstein, N. and Hunter, L.A. (1991) Targeted integration of adeno-associated virus (AAV) into human chromosome 19, EMBO J., 10: 3941-4950.
67. Shenk, T. and Williams, J. (1984) Genetic analysis of adenovirus, Curr. Topics Microbiol. Immunol., 111: 1-39.
68. Shi, M., Arsenakis, M., Tiollais, P. and Roizman, B. (1984) Expression of hepatitis BS gene by a herpes simplex type 1 vectors carrying a-and b-regulated gene chimeras. Proc. Natl. Acad. Sci. USA, 81, 5867-5870.
69. Simon, R.H., Engelhardt, J.F., Yang, Y., Zepeda, M., Weber-Pendleton, S., Grossman, M. and Wilson, J.M. (1993) Adenovirus-mediated transfer of the CFTR gene to lung of non-human primates: toxic study, Hum. Gene Ther., 4: 771-788.
70. Spaete, R.R. and Frenkel, N. (1982) The herpes simplex virus amplicon: a new eucaryotic defective-virus cloning amplifying vector, Cell, 30: 295-304.
71. Spaete, R.R. and Frenkel, N. (1985) The herpes simples virus amplicon: analysis of cis-acting replication functions, Proc. Natl. Acad. Sci. USA, 82: 694-698.
72. Stow, N.D. (1985) Mutagenesis of a herpes simplex virus origin of DNA replication and its effect of viral interference, J. Gen. Virol., 66: 31-42.
73. Stow, N.D. and McMonagle, E.C. (1982) Propagation of foreign DNA sequences linked to a herpes simplex virus origin of replication. In Y. Gluzman (Ed.), Eucaryotic Viral Vectors, Cold Springs Harbor Laboratory Press, Cold Spring Harbor, NY, pp. 199-204.
74. Stow, N.D. and McMonagle, E.C. (1983) Characterization of the TRs/IRs origin of DNA replication of herpes simplex virus type 1, Virology, 130: 427-438.
75. Strauss, S.E., Ginsburg, H.S. and Rose, J.A. (1976) DNA-minus temperature-sensitive mutants of adenovirus type 5 help adenovirus-associated virus replication, J. Virol., 17: 140-148.
76. Strivastava, A., Lusby, E.W. and Berns, K.I. (1983) Nucleotide sequence and organization of the adeno-associated virus 2 genome, J. Virol., 45: 555-564.
77. Tratschin, J.D., Miller, I.L., Smith, M.G. and Carter, B.J. (1985) Adeno associated virus vectors for high frequency integration, expression and rescue of genes in mammalian cells, Mol. Cell Biol., 5: 3251-3260.
78. Tsao, J., Chapman, M.S. et al. (1991) The three-dimensional structure of canine parvovirus and its functional implications, Science, 25: 1456-1464.
79. Verhaagen, J., Hermens, W.T.J.M.C., Holtmaat, A.J.G.D., Oestreicher, A.B., Gispen, W.H. and Kaplitt, M.G. (1995) Viral vector-mediated gene transfer in the nervous system: application to the reconstruction of neural circuits in the injured mammalian brain. In M.G. Kaplitt and A.D. Loew (Eds.) Viral Vectors, Academic Press, San Diego, CA, pp. 119-132.
80. Vlazny, D.A. and Frenkel, N. (1981) Replication of herpes simplex virus DNA: localization of replication recognition signals within defective virus genomes, Proc. Natl. Acad. Sci. USA, 78: 742-746.
81. Vlazny, D.A., Kwong, A.D. and Frenkel, N. (1982) Site specific cleavage/packaging of herpes simplex virus DNA and the selective maturation of nucleocapsids containing full length viral DNA, Proc. Natl. Acad. Sci. USA, 79: 1423-1427.
82. Von Magnus, P. (1954) Incomplete forms of influenza virus, Adv. Vir. Res., 2: 59-78.
83. West, M.H.P., Trempe, J.P. et al. (1987) Gene expression in adeno-associated virus vectors: the effects of chimeric mRNA structure, helper virus and adenovirus VAI RNA, Virology, 160: 38-47.
84. Xu, H., Federoff, H., Maragos, J., Parada, L. and Kessler, J. (1994) Viral transduction of trk A into cultured nodose and spinal motor neurons convey NGF responsiveness, Dev. Biol., 163: 152-161.
85. Yin, J., Kaplitt, M.G. and Pfaff, D.W. (1995) In vivo promoter analysis in the adult central nervous system using viral vectors. In M.G. Kaplitt and A.D. Loew (Eds.), Viral Vectors, Academic Press, San Diego, CA, pp. 157-172.
86. Zabner, J., Peterson, D., Puga, A.P., Graham, S.M., Couture, L.A., Keyes, L.D., Lukason, M.J., St. George, J.A., Gregory, R.J., Smith, A.E. and Welsh, M.J. (1994) Safety and efficacy of repetitive adenovirus-mediated transfer of CFTR cDNA top airway epithelia of primates and cotton rats, Nature Genet., 6: 75-83.
87. Zhou, S.Z., Broxmeyer, H.E. et al. (1993) Adeno-associated virus 2-mediated gene transfer in murine hematopoietic progenitor cells. Exp. Hematol. 21: 928-933.