Gene transfer to the nigrostriatal system by hybrid herpes simplex virus/adeno-associated virus amplicon vectors

Human Gene Therapy

Volumn 10, Issue 15, 1999, Pages 2481-2494

  Costantini, L C ^a   Jacoby, D R ^b   Wang, S ^b   Fraefel, C ^b,c   Breakefield, X O ^b   Isacson, O ^a,b  

^a MCLEAN HOSPITAL   (United States)

^b MASSACHUSETTS GENERAL HOSPITAL   (United States)

^c UNIVERSITY OF ZURICH   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

GREEN FLUORESCENT PROTEIN; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ARTICLE; CELL FREE SYSTEM; CYTOTOXIC T LYMPHOCYTE; GENE TRANSFER; HERPES SIMPLEX VIRUS; HYBRID; IMMUNE RESPONSE; MOUSE; NERVE CELL CULTURE; NIGRONEOSTRIATAL SYSTEM; NONHUMAN; RETROGRADE NERVE FIBER TRANSPORT; SIGNAL TRANSDUCTION; SUBSTANTIA NIGRA;

ANIMALS; BIOLOGICAL MARKERS; CELLS, CULTURED; CORPUS STRIATUM; CROSSES, GENETIC; DEPENDOVIRUS; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; RATS; SIMPLEXVIRUS; SUBSTANTIA NIGRA; TRANSDUCTION, GENETIC;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; ANIMALIA; DNA VIRUSES; HERPES; HUMAN HERPESVIRUS 1; SIMPLEXVIRUS;

EID: 0033544015     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950016825     Document Type: Article

References (105)

1. ABERCROMBIE, M. (1946). Estimation of nuclear populations from microtome sections. Anat. Rec. 94, 239-247.
2. ANDERSEN, J.K., FRIM, D.M., ISACSON, O., and BREAKFIELD, X.O. (1993). Herpesvirus-mediated gene delivery into the rat brain: Specificity and efficiency of the neuron-specific enolase promoter. Cell. Mol. Neurobiol. 13, 503-515.
3. BALAGUE, C., KALLA, M., and ZHANG, W.W. (1997). Adeno-associated virus Rep78 protein and terminal repeats enhance integration of DNA sequences into the cellular genome. J. Virol. 71, 3299-3306.
4. BARTLETT, J.S., SAMULSKI, R.J., and MCCOWN, T. (1998). Selective and rapid uptake of adeno-associated virus type 2 in brain. Hum. Gene Ther. 9, 1181-1186.
5. BASKAR, J., SMITH, P., NILAVER, G., JUPP, R., HOFFMANN, S., PEFFER, N., TENNEY, D., COLBERG-POLEY, A., GHAZAL, P., and NELSON, J. (1996). The enhancer domain of the human cytomegalovirus major immediately-early promoter determines cell type-specific expression in transgenic mice. J. Virol. 70, 3207-3214.
6. BERGOLD, P.J., CASACCIA-BONNEFIL, P., XIU-LIU, Z., and FEDEROFF, H.J. (1993). Trans-synaptic neuronal loss induced in hippocampal slice cultures by a herpes simplex virus vector expressing the GluR6 subunit of the kainate receptor. Proc. Natl. Acad. Sci. U.S.A. 90, 6165-6169.
7. BERTRAN, J., YANG, Y., HARGROVE, P., VANIN, E.F., and NIENHUIS, A.W. (1998). Target integration of a recombinant globin gene adeno-associated viral vector into human chromosome 19. Ann. N.Y. Acad. Sci. 850, 163-177.
8. BILANG-BLEUEL, A., REVAH, F., COLIN, P., LOCQUET, I., ROBERT, J.J., MALLET, J., and HORELLOU, P. (1997). Itrastriatal injection of an adenoviral vector expressing glial-cell-line-derived neurotrophic factor prevents dopaminergic neuron degeneration and behavioral impairment in a rat model of Parkinson disease. Proc. Natl. Acad. Sci. U.S.A. 94, 8818-8823.
9. BLÖMER, U., NALDINI, L., KAFRI, T., TRONO, D., VERMA, I.M., and GAGE, F.H. (1997). Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. J. Virol. 71, 6641-6649.
10. BOVIATSIS, E.J., CHASE, M., WEI, M.X., TAMIYA, T., HURFORD, R.K., JR., KOWALL, N.W., TEPPER, R.I., BREAKFIELD, X.O., and CHIOCCA, E.A. (1994). Gene transfer into experimental brain tumors mediated by adenovirus, herpes simplex virus, and retrovirus vectors. Hum. Gene Ther. 5, 183-191.
11. BOWERS, W., HOWARD, D., and FEDEROFF, H. (1997). Gene therapeutic strategies for neuroprotection: Implications for Parkinson's disease. Exp. Neurol. 144, 58-68.
12. BYRNES, A.P., RUSBY, J.E., WOOD, M.J.A., and CHARLTON, H.M. (1995). Adenovirus gene transfer causes inflammation in the brain. Neuroscience 66, 1015-1024.
13. BYRNES, A.P., MACLAREN, R.E., and CHARLTON, H.M. (1996). Immunological instability of persistent adenovirus vectors in the brain: Peripheral exposure to vector leads to renewed inflammation, reduced gene expression, and demyelination. J. Neurosci. 16, 3045-3055.
14. CHIORINI, J., WEITZMAN, M., OWENS, R., URCELAY, E., SAFER, B., and KOTIN, R. (1994). Biologically active Rep proteins of adeno-associated virus type 2 produced as fusion proteins in Escherichia coli. J. Virol. 68, 797-804.
15. CHOI-LUNDBERG, D., LIN, Q., CHANG, Y., HAY, C., MOHAJERI, H., DAVIDSON, D., and BOHN, M. (1997). Dopaminergic neurons protected from degeneration by GDNF gene therapy. Science 275, 838-841.
16. COSTANTINI, L.C., LIN, L., and ISACSON, O. (1997). Medial fetal ventral mesencephalon: A preferred source for dopamine neuron grafts. NeuroReport 8, 2253-2257.
17. COSTANTINI, L., CHATURVEDI, P., ARMISTEAD, D., MCCAFFREY, P., DEACON, T., and ISACSON, O. (1998). A novel immunophilin ligand: Distinct branching effects on dopaminergic neurons in culture and neurotrophic actions after oral administration in an animal model of Parkinson's disease. Neurobiol. Dis. 5, 97-106.
18. DAVAR, G., KRAMER, M.F., GARBER, D., ROCA, A.L., ANDERSEN, J.K., BEBRIN, W., COEN, D.M., KOSZ-VNENCHAK, M., KNIPE, D.M., BREAKFIELD, X.O., and ISACSON, O. (1994). Comparative efficacy of expression of genes delivered to mouse sensory neurons with herpes virus vectors. J. Comp. Neurol. 339, 3-11.
19. DAVIDSON, B.L., and BOHN, M.C. (1997). Recombinant adenovirus: A gene transfer vector for study and treatment of CNS diseases. Exp. Neurol. 144, 125-130.
20. DEACON, T., SCHUMACHER, J., DINSMORE, J., THOMAS, C., PALMER, P., KOTT, S., EDGE, A., PENNEY, D., KASSISSIEH, S., DEMPSEY, P., and ISACSON, O. (1997). Histological evidence of fetal pig neural cell survival after transplantation into a patient with Parkinson's disease. Nature Med. 130, 350-353.
21. DUAN, D., FISHER, K., BURDA, J., and ENGELHARDT, J. (1997). Structural and functional heterogeneity of integrated recombinant AAV genomes. Virus Res. 48, 41-56.
22. DUAN, D., SHARMA, P., YANG, J., YUE, Y., DUDUS, L., ZHANG, Y., FISHER, K., and ENGELHARDT, J. (1998). Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue. J. Virol. 72, 8568-8577.
23. DURING, M., NAEGELE, J., O'MALLEY, K., and GELLER, A. (1994). Long-term behavioral recovery in parkinsonian rats by an HSV vector expressing tyrosine hydroxylase. Science 266, 1399-1403.
24. DURING, M.J., SAMULSKI, R.J., ELSWORTH, J.D., KAPLITT, M.G., LEONE, P., XIAO, X., LI, J., FREESE, A., TAYLOR, J.R., ROTH, R.H., SLADEK, J.R., JR., O'MALLEY, K.L., and REDMOND, D.E., JR. (1998). In vivo expression of therapeutic human genes for dopamine production in the caudate of MPTP-treated monkeys using an AAV vector. Gene Ther. 5, 820-827.
25. FEDEROFF, H.J., GESCHWIND, M.D., GELLER, A.I., and KESSLER, J.A. (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. U.S.A. 89, 1636-1640.
26. FERRARI, F.K., XIAO, X., MCCARTY, D., and SAMULSKI, R.J. (1997). New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors. Nature Med. 3, 1295-1297.
27. FINK, D.J., STERNBERG, L.R., WEBER, P.C., MATA, M., GOINS, W.F., and GLORIOSO, J.C. (1992). In vivo expression of beta-galactosidase in hippocampal neurons by HSV-mediated gene transfer. Hum. Gene Ther. 3, 11-19.
28. FOTAKI, M.E., PINK, J.R., and MOUS, J. (1997). Tetracycline-responsive gene expression in mouse brain after amplicon-mediated gene transfer. Gene Ther. 4, 901-908.
29. FRAEFEL, C., SONG, S., LIM, F., LANG, P., YU, L., WANG, Y., WILD, P., and GELLER, A. (1996). Helper virus-free transfer of herpes simplex virus type 1 plasmid vectors into neural cells. J. Virol. 70, 7190-7197.
30. FRAEFEL, C., JACOBY, D.R., LAGE, C., HILDERBRAND, H., CHOU, J.Y., ALT, F.W., BREAKFIELD, X.O., and MAJZOUB, J.A. (1997). Gene transfer into hepatocytes mediated by helper virus-free HSV/AAV hybrid vectors. Mol. Med. 3, 813-825.
31. FRAEFEL, C., JACOBY, D.R., and BREAKFIELD, X.O. (1998). Recent developments on herpes simplex virus type 1-based amplicon vector systems. Adv. Virus Res. (in press).
32. FREED, C.R., BREEZR, R.E., ROSENBERG, N.L., SCHNECK, S.A., KRIEK, E., QI, J.X., LONE, T., ZHANG, Y.B., SNYDER, J.A., WELLS, T.H., RAMIG, L.O., THOMPSON, L., MAZZIOTTA, J.C., HUANG, S.C., GRAFTON, S.T., BROOKS, D., SAWLE, G., SCHROTER, G., and ANSARI, A.A. (1992). Survival of implanted fetal dopamine cells and neurologic improvement 12 and 46 months after transplantation for Parkinson's disease. N. Engl. J. Med. 327, 1549-1555.
33. GELLER, A.I., and BREAKEFIELD, X.O. (1988). A defective HSV-1 vector expresses Escherichia coli beta-galactosidase in cultured peripheral neurons. Science 241, 1667-1669.
34. GELLER, A.I., and 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 beta-galactosidase. Proc. Natl. Acad. Sci. U.S.A. 87, 1149-1153.
35. GELLER, A.I., DURING, M.J., HAYCOCK, J.W., FREESE, A., and 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. U.S.A. 90, 7603-7607.
36. GELLER, A.I., DURING, M.J., OH, Y.J., FREESE, A., and O'MALLEY, K. (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.
37. GESCHWIND, M.D., KESSLER, J.A., GELLER, A.I., and FEDEROFF, H.J. (1994). Transfer of the nerve growth factor gene into cell lines and cultured neurons using a defective herpes simplex virus vector: Transfer of the NGF gene into cells by a HSV-1 vector. Mol. Brain Res. 24, 327-335.
38. GOLDSTEIN, D.J., and WELLER, S.K. (1988). Herpes simplex virus type 1-inducible ribonucleotide reductase activity is dispensible for virus growth and DNA synthesis: Isolation and characterization of an ICP6 lacZ insertion mutant. J. Virol. 62, 196-205.
39. GOODMAN, S., XIAO, X., DONAHUE, R., MOULTON, A., MILLER, J., WALSH, C., YOUNG, N., SAMULSKI, R., and NIENHUIS, A. (1994). Recombinant adeno-associated and virus-mediated gene transfer into hematopoietic progenitor cells. Blood 84, 1492-1500.
40. HARDING, T.C., GEDDES, B.J., NOEL, J.D., MURPHY, D., and UNEY, J.B. (1997). Tetracycline-regulated transgene expression in hippocampal neurones following transfection with adenoviral vectors. J. Neurochem. 69, 2620-2623.
41. HERMONAT, P. (1994). Down-regulation of the human c-fos and c-myr proto-oncogene promoters by adeno-associated virus Rep78. Cancer Lett. 81, 129-136.
42. HO, D.Y., MOCARSKI, E.S., and SAPOLSKI, R.M. (1993). Altering central nervous system physiology with a defective herpes simplex virus vector expressing the glucose transporter gene. Proc. Natl. Acad. Sci. U.S.A. 90, 3655-3659.
43. HO, D.Y., MCLAUGHLIN, J.R., and SAPOLSKY, R.M. (1996). Inducible gene expression from defective herpes simplex virus vectors using tetracycline responsive promoter system. Brain Res. Mol. Brain Res. 41, 200-209.
44. HORER, M., WEGER, S., BUTZ, K., HOPPE-SEYLER, F., GEISEN, C., and KLEINSCHMIDT, J. (1995). Mutational analysis of adeno-associated virus Rep protein-mediated inhibition of heterologous and homologous promoters. J. Virol. 69, 5485-5496.
45. HUANG, Q., VONSATTEL, J.P., SCHAFFER, P.A., MARTUZA, R.L., BREAKEFIELD, X.O., and DIFIGLIA, M. (1992). Introduction of a foreign gene (Escherichia coli lacZ) into rat neostriatal neurons using herpes simplex virus mutants: A light and electron microscopic study. Exp. Neurol. 115, 303-316.
46. ISACSON, O. (1995). Behavioral effects and gene delivery in a rat model of Parkinson's disease; technical comments. Science 269, 856-857.
47. ISACSON, O., DEACON, T., and SCHUMACHER, J. (1999). Immunobiology and Neuroscience of Xenotransplantation in Neurological Disease, Chapter 13. M. Tuszynski and J. Kordower, eds. (Academic Press, San Diego, CA).
48. JIN, B.K., BELLONI, M., CONTI, B., FEDEROFF, H.J., STARR, R., SON, J.H., BAKER, H., and JOH, T.H. (1996). Prolonged in vivo gene expression driven by a tyrosine hydroxylase promoter in a defective herpes simplex virus amplicon vector. Hum. Gene Ther. 7, 2015-2024.
49. JOHNSTON, K., JACOBY, D., PECHAN, P., FRAEFEL, C., BORGHESANI, P., SCHUNACK, D., DUNN, R., SMITH, F., and BREAKEFIELD, X. (1997). HSV/AAV hybrid amplicon vectors extend transgene expression in human glioma cells. Hum. Gene Ther. 8, 359-370.
50. KANG, W., WILSON, M.A., BENDER, M.A., GLORIOSO, J.C., and WILSON, S.P. (1998). Herpes virus-mediated preproenkephalin gene transfer to the amygdala is antinociceptive. Brain Res. 792, 133-135.
51. KAPLITT, M.G., KWONG, A.D., KLEOPOULOS, S.P., MOBBS, C.V., RABKIN, S.D., and PFAFF, D.W. (1994a). Proenkephalin promoter yield region-specific and long-term expression in adult brain after in vivo gene transfer via a defective herpes simplex viral vector. Proc. Natl. Acad. Sci. U.S.A. 91, 8979-8983.
52. KAPLITT, M.G., LEONE, P., SAMULSKI, R.J., XIAO, X., PFAFF, D.W., O'MALLEY, K.L., and DURING, M.J. (1994b). Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nature Genet. 8, 148-154.
53. KLEIN, R.L., MEYER, E.M., PEEL, A.L., ZOLOTUKHIN, S., MEYERS, C., MUZYCZKA, N., and KING, M.A. (1998). Neuron-specific transduction in the rat septohippocampal or nigrostriatal pathway by recombinant adeno-associated virus vectors. Exp. Neurol. 150, 183-194.
54. KORDOWER, J.H., FREEMAN, T.B., SNOW, B.J., VINGERHOETS, F.J.G., MUFSON, E.J., SANBERG, P.R., HAUSER, R.A., SMITH, D.A., NAUERT, M., PERL, D.P., and OLANOW, C.W. (1995). Neuropathological evidence of graft survival and striatal reinnervation after the transplantation of fetal mesencephalic tissue in a patient with Parkinson's disease. N. Engl. J. Med. 332, 1118-1124.
55. KOTIN, R.M., LINDEN, R.M., and BERNS, K.I. (1992). Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination. EMBO J. 11, 5071-5078.
56. KUO, H., INGRAM, D.K., CRYSTAL, R.G., and MASTRANGELI, A. (1995). Retrograde transfer of replication deficient recombinant adenovirus vector in the central nervous system for tracing studies. Brain Res. 705, 31-38.
57. KWONG, A.D., and FRENKEL, N. (1985). Efficient expression of chimeric chicken ovalbumin gene amplified with defective virus genomes. Virology 142, 421-425.
58. KYOSTIO, S., OWENS, R., WEITZMAN, M., ANTONI, B., CHEJANOVSKY, N., and CARTER, B. (1994). Analysis of adeno-associated virus (AAV) wild-type and mutant Rep proteins for their abilities to negatively regulate AAV p5 and p19 mRNA levels. J. Virol. 68, 2947-2957.
59. LAMARTINA, S., ROSCILLI, G., RINAUDO, D., DELMASTRO, P., and TONIATTI, C. (1998). Lipofection of purified adeno-associated virus Rep68 protein: Toward a chromosome-targeting nonviral particle. J. Virol. 72, 7652-7658.
60. LAWRENCE, M.S., HO, D.Y., DASH, R., and SAPOLSKY, R.M. (1995). Herpes simplex virus vectors overexpressing the glucose transporter gene protect against seizure-induced neuron loss. Proc. Natl. Acad. Sci. U.S.A. 92, 7247-7251.
61. LAWRENCE, M.S., HO, D.Y., SUN, G.H., STEINBERG, G.K., and SAPOLSKY, R.M. (1996). Overexpression of Bcl-2 with herpes simplex virus vectors protects CNS neurons against neurological insults in vitro and in vivo. J. Neurosci. 16, 486-496.
62. LAWRENCE, M.S., MCLAUGHLIN, J.R., SUN, G.H., HO, D.Y., MCINTOSH, L., KUNIS, D.M., SAPOLSKY, R.M., and STEINBERG, G.K. (1997). Herpes simplex viral vectors expressing Bcl-2 are neuroprotective when delivered after a stroke. J. Cereb. Blood Flow Metab. 17, 740-744.
63. LEWIS, P., HENSEL, M., and EMERMAN, M. (1992). Human immunodeficiency virus infection of cells arrested in the cell cycle. EMBO J. 11, 3053-3058.
64. LU, B., and FEDEROFF, H.J. (1995). Herpes simplex virus type I amplicon vectors with glucocorticoid-inducible gene expression. Hum. Gene Ther. 6, 419-428.
65. MAIDMENT, N.T., TAN, A.M., BLOOM, D.C., ANTON, B., FELDMAN, L.T., and STEVENS, J.G. (1996). Expression of the lacZ reporter gene in the rat basal forebrain, hippocampus, and nigrostriatal pathway using a nonreplicating herpes simplex vector. Exp. Neurol. 139, 107-114.
66. MANDEL, R.J., SPRATT, S.K., SNYDER, R.O., and LEFF, S.E. (1997). Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor proteins nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats. Proc. Natl. Acad. Sci. 9, 14083-14088.
67. MANDEL, R., RENDAHL, K., SPRATT, S., SYNDER, R., COHEN, L., and LEFF, S. (1998). Characterization of intrastriatal recombinant adeno-associated virus-mediated gene transfer of human tyrosine hydroxylase and human GTP-cyclohydrolase I in a rat model of Parkinson's disease. J. Neurosci. 18, 4271-4284.
68. MANOME, Y., KUNIEDA, T., WEN, P.Y., KOGA, T., KUFE, D.W., and OHNO, T. (1998). Transgene expression in malignant glioma using a replication-defective adenoviral vector containing the Egr-1 promoter: Activation by ionizing radiation or uptake of radioactive iododeoxyuridine. Hum. Gene Ther. 9, 1409-1417.
69. MCCOWN, T.J., XIAO, X., LI, J., BREESE, G.R., and SAMULSKI, R.J. (1996). Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res. 713, 99-107.
70. MIYOSHI, H., BLÖMER, U., TAKAHASHI, M., GAGE, F.H., and VERMA, I.M. (1998). Development of a self-inactivating lentivirus vector. J. Virol. 72, 8150-8157.
71. MONTGOMERY, R., WARNER, M., LUM, B., and SPEAR, P. (1996). Herpes simplex virus-1 entry into cells mediated by a novel member of the TNF/NGF receptor family. Cell 87, 427-436.
72. MUZYCZKA, N. (1992). Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr. Top. Microbiol. Immunol. 158, 97-129.
73. NALDINI, L., BLÖMER, U., GALLAY, P., ORY, D., MULLIGAN, R., GAGE, F.H., VERMA, I.M., and TRONO, D. (1996). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263-267.
74. NILAVER, G., MULDOON, L.L., KROLL, R.A., PAGEL, M.A., BREAKFIELD, X.O., DAVIDSON, B.L., and NEUWELT, E.A. (1995). Delivery of herpesvirus and adenovirus to nude rat intracerebral tumors after osmotic blood-brain barrier distribution. Proc. Natl. Acad. Sci. U.S.A. 92, 9829-9833.
75. OLIGINO, T., POLIANI, P.L., WANG, Y., TSAI, S.Y., O'MALLEY, B.W., FINK, D.J., and GLORIOSO, J.C. (1998). Drug inducible transgene expression in brain using a herpes simplex virus vector. Gene Ther. 5, 491-496.
76. PAKZABAN, P., GELLER, A.I., and ISACSON, O. (1994). Effect of exogenous nerve growth factor on neurotoxicity of and neuronal gene delivery by a herpes simplex amplicon vector in the rat brain. Hum. Gene Ther. 5, 987-995.
77. PAKZABAN, P., DEACON, T., BURNS, L., DINSMORE, J., and ISACSON, O. (1995). A novel mode of immunoprotection of neural xenotransplants: Masking of donor major histocompatibility complex class I enhances transplant survival in the central nervous system. Neuroscience 65, 983-996.
78. PALOMBO, F., MANCIOTTI, A., RECCHIA, A., CORTESE, R., CILIBERTO, G., and MONICA, N. (1998). Site-specific integration in mammalian cells mediated by a new hybrid baculovirus-adeno-associated virus vector. J. Virol. 72, 5025-5034.
79. PARR, M.J., WEN, P.Y., SCHAUB, M., KHOURY, S.J., SAYEGH, M.H., and FINE, H.A. (1998). Immune parameters affecting adenoviral vector gene therapy in the brain. J. Neurovirol. 4, 194-203.
80. PAXINOS, G., and WATSON, C. (1986). The Rat Brain in Stereotaxic Coordinates, Edition 2. (Academic Press, San Diego, CA).
81. PECHAN, P., FOTAKI, M., THOMPSON, R., DUNN, R., CHASE, M., CHIOCCA, E., and BREAKEFIELD, X.O. (1996). A novel "piggyback" packaging system for herpes simplex virus amplicon vectors. Hum. Gene Ther. 7, 2003-2013.
82. PEEL, A.L., ZOLOTUKHIN, S., SCHRIMSHER, G.W., MUZYCZKA, N., and REIER, P.J. (1997). Efficient transduction of green fluorescent protein in spinal cord neurons using adeno-associated virus vectors containing cell type-specific promoters. Gene Ther. 4, 16-24.
83. PRESTON, C. (1979). Control of herpes simplex virus type I mRNA synthesis in cells infected with wild type or temperature sensitive mutant trK. J. Virol. 29, 257-284.
84. RENDAHL, K., LEFF, S., OTTEN, G., SPRATT, S., BOHL, D., CAN ROEY, M., DONAHUE, B., COHEN, L., MANDEL, R., DANOS, O., and SNYDER, R. (1998). Regulation of gene expression in vivo following transduction by two separate rAAV vectors. Nature Biotechnol. 16, 757-761.
85. SAEKI, Y., ICHIKAWA, T., SAEKI, A., CHIOCCA, A., TOBLER, K., ACKERMANN, M., BREAKEFIELD, X.O., and FRAEFEL, C. (1998). Herpes simplex virus type 1 DNA amplified as bacterial artificial chromosome in Escherichia coli: Rescue of replication-competent virus progeny and packaging of amplicon vectors. Hum. Gene Ther. 9, 2787-2794.
86. SAMULSKI, R., CHANG, L., 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.
87. SANES, J., RUBENSTEIN, J., and NICOLAS, J. (1986). Use of a recombinant retrovirus to study postimplantation lineage in mouse embryo. EMBO J. 5, 3133-3142.
88. SNYDER, R.O., XIAO, X., and SAMULSKI, R.J. (1996). Production of Recombinant Adeno-Associated Viral Vectors, Vol. 12. N. Dracopoli, J. Haines, B. Krof, D. Moir, C. Morton, C. Seidman, J. Seidman, and D. Smith, eds. (John Wiley & Sons, New York).
89. SODEIK, B., EBERSOLD, M., and HELENIUS, A. (1997). Microtubule-mediated transport of incoming herpes simplex virus 1 capsids to the nucleus. J. Cell Biol. 136, 1007-1021.
90. SONG, S., WANG, Y., BAK, S.-Y., LANG, P., ULLREY, D., NEVE, R.L., O'MALLEY, K.L., and GELLER, A.I. (1997). An HSV-1 vector containing the rat tyrosine hydroxylase promoter enhances both long-term and cell type-specific expression in the midbrain. J. Neurochem. 68, 1792-1803.
91. SONG, S., WANG, Y., BAK, S.-Y., DURING, M.J., BRYAN, J., ASHE, O., ULLREY, D.B., TRASK, L.E., GRANT, F.D., O'MALLEY, K.L., RIEDEL, H., GOLDSTEIN, D.S., NEVE, K.A., LAHOSTE, G.J., MARSHALL, J.F., HAYCOCK, J.W., NEVE, R.L., and GELLER, A.I. (1998). Modulation of rat rotational behavior by direct gene transfer of constitutively active protein kinase C into nigrostriatal neurons. J. Neurosci. 18, 4119-4132.
92. VERMA, I., and SOMIA, N. (1997). Gene therapy: Promises, problems, and prospects. Nature (London) 389, 239-242.
93. WALKER, S.L., WONDERLING, R.S., and OWENS, R.A. (1997). Mutational analysis of the adeno-associated virus Rep68 protein: Identification of critical residues necessary for site-specific endonuclease activity. J. Virol. 71, 2722-2730.
94. WANG, A., ALLEN, P., PROTASI, F., MOORE, R., FESSENDEN, J., PESSAH, I., BREAKEFIELD, X., and FRAEFEL, C. (1999). Helper virus-free HSV-1 amplicon vectors are highly efficient in delivering large genes to skeletal muscle, myoblasts and myotubes in culture. (Submitted.)
95. WARD, P.L., and ROIZMAN, B. (1994). Herpes simplex genes: The blueprint of a successful human pathogen. Trends Genet. 10, 267-274.
96. WEITZMAN, M.D., KYOSTIO, S.R., KOTIN, R.M., and OWENS, R.A. (1994). Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA. Proc. Natl. Acad. Sci. U.S.A. 91, 5808-5812.
97. WIDNER, H. (1998). The Lund Transplant Program for Parkinson's Disease and Patients with MPTP-Induced Parkinsonism. T. Freeman and H. Widner, eds. (Humana Press, Totowa, NJ).
98. WOOD, M., BYRNES, A., PFAFF, D., RABKIN, S., and CHARLTON, H. (1994). Inflammatory effects of gene transfer into the CNS with defective HSV-1 vectors. Gene Ther. 1, 283-291.
99. WU, P., PHILLIPS, M.I., BUI, J., and TERWILLIGER, E.F. (1998). Adeno-associated virus vector-mediated transgene integration into neurons and other nondividing cell targets. J. Virol. 72, 5919-5926.
100. XIAO, X., MCCOWN, T.J., LI, J., BREESE, G.R., MORROW, A.L., and SAMULSKI, R.J. (1997). Adeno-associated virus (AAV) vector antisense gene transfer in vivo decreases GABA(A) alphal containing receptors and increases inferior collicular seizure sensitivity. Brain Res. 756, 76-83.
101. XIAO, X., LI, J., and SAMULSKI, R.J. (1998). Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol. 72, 2224-2232.
102. YANG, C.C., XIAO, X., ZHU, X., ANSARDI, D.C., EPSTEIN, N.D., FREY, M.R., MATERA, A.G., and SAMULSKI, R.J. (1997). Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro. J. Virol. 71, 9231-9247.
103. YANG, W., MASON, W., and SUMMERS, J. (1996). Covalently closed circular viral DNA formed from two types of linear DNA in woodchuck hepatitis virus-infected liver. J. Virol. 70, 4567-4575.
104. YANG, Y., JOOSS, K., SU, Q., ERTL, H., and WILSON, J. (1996). Immune response to viral antigens versus transgene product in the elimination of recombinant adenovirus-infected hepatocytes in vivo. Gene Ther. 3, 137-144.
105. ZUFFEREY, R., NAGY, D., MANDEL, R.J., NALDINI, L., and TRONO, D. (1997). Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nature Biotechnol. 15, 871-875.