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Adeno-Associated Virus (AAV) Vectors in Gene Therapy pp 145–170Cite as

Adeno-associated Virus Based Vectors As Antivirals

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Part of the book series: Current Topics in Microbiology and Immunology ((CT MICROBIOLOGY,volume 218))

Abstract

Although great strides have been made in the treatment and prevention of human viral infections, antiviral drug therapy still remains problematic when compared to treatment of bacterial infections. The therapeutic toxicity/efficacy ratio of many antivirals is low, resulting in potential risks of regimen related toxicity. The number of effective antivirals is limited, and many important viral infections remain untreatable. In addition, currently used antivirals often suppress rather than cure infections, necessitating repeated, sometimes lifelong, therapy to prevent recurrences. Furthermore, the increasing use of antivirals has resulted in the selection or emergence of drug-resistant strains as has occurred following the widespread use of antibacterial (Field and Biron 1994). Finally, several medically important viruses, most notably human immunodeficiency virus (HIV), have been recalcitrant to either the development of curative chemotherapy or protective vaccines (Schnittman and Fauci 1994). Thus, novel approaches to the treatment or prevention of viral infections are constantly being sought.

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References

  • Alexander IE, Russell DW, Miller AD (1994) DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors. J Virol 68: 8282–8287

    PubMed  CAS  Google Scholar 

  • Allen ND, Cran DG, Barton SC, Hettle S, Reik R, Surani MAH (1988) Transgenes as probes for active chromosomal domains in mouse development. Nature 333: 852–855

    Article  PubMed  CAS  Google Scholar 

  • Anderson WF (1992) Human Gene Therapy. Science 256: 808–813

    Article  PubMed  CAS  Google Scholar 

  • Antoni BA, Rabson AB, Miller IL, Trempe JP, Chejanovsky N, Carter BJ (1991) Adeno-associated virus rep protein inhibits human immuno-deficiency virus type 1 production in human cells. J Virol 65: 396–404

    PubMed  CAS  Google Scholar 

  • Arnheiter H, Skuntz S, Noteborn M, Chang S, Meier E (1990) Transgenic mice with intracellular immunity to influenza virus. Cell 62: 51–61

    Article  PubMed  CAS  Google Scholar 

  • Bahner I, Zhou C, Yu XJ, Hao QL, Guatelli JC, Kohn DB (1993) Comparison of trans-dominant inhibitory mutant human immunodeficiency virus type 1 genes expressed by retroviral vectors in human T lymphocytes. J Virol 67: 3199–3207

    PubMed  CAS  Google Scholar 

  • Balfour HH Jr, Benson C, Braun J, Cassens B, Erice A, Friedman-Kien A, Klein T, Polsky B, Safrin S (1994) Management of acyclovir-resistant herpes simplex and varicella-zoster virus infections. J Acquir Immune Defic Syndr 7: 254–260

    PubMed  Google Scholar 

  • Baltimore D (1988) Intracellular immunization. Nature 335: 395–396

    Article  PubMed  CAS  Google Scholar 

  • Bantel-Schaall U, zur Hausen H (1988) Adeno-associated viruses inhibit SV40 DNA amplification and replication of herpes simplex virus in SV40-transformed hamster cells. Virology 164: 64–74

    Article  Google Scholar 

  • Bass BL, Weintraub H (1988) An unwinding activity that covalently modifies its double-stranded RNA substrate. Cell 55: 1089–1098

    Article  PubMed  CAS  Google Scholar 

  • Bednarik DP, Mosca JD, Raj NBK, Pitha PM (1989) Inhibition of human immunodeficiency virus (HIV) replication by HIV-trans-activated alpha-2 interferon. Proc Natl Acad Sci USA 86: 4958–4962

    Article  PubMed  CAS  Google Scholar 

  • Berns KI, Bohenzky RA (1987) Adeno-associated viruses: an update. Adv Virus Res 32: 243–306

    Article  PubMed  CAS  Google Scholar 

  • Berns KI, Pinkerton TC, Thomas GF, Hoggan MD (1975) Detection of adeno-associated virus (AAV)-spceifie nucleotide sequences in DNA isolated from latently infected Detroit 6 cells. Virology 68: 556–560

    Article  PubMed  CAS  Google Scholar 

  • Bevec D, Volc-Platzer B, Zimmermann K, Dobrovnik M, Hauber J, Veres G, Bohnlein E (1994) Constitutive expression of chimeric neo-Rev response element transcripts suppresses HIV-1 replication in human CD4+ T lymphocytes. Hum Gene Ther 5: 193–201

    Article  PubMed  CAS  Google Scholar 

  • Bertrand EL, Rossi JJ (1994) Facilitation of hammerhead ribozyme catalysis by the nucleocapsid protein of HIV-1 and the heterogeneous nuclear ribonucleoprotein A1. EMBO J 13: 2904–2912

    PubMed  CAS  Google Scholar 

  • Blaese RM (1993) Development of gene therapy for immunodeficiency: adenosine deaminase deficiency. Pediatr Res 33 [Suppl]: S49-S53

    Article  PubMed  CAS  Google Scholar 

  • Bohnlein S, Pirker FP, Hofer L, Zimmermann K, Bachmayer H, Bohnlein E, Hauber J (1991) Transdominant repressors for human T-cell leukemia virus type I rex and human immunodeficiency virus type 1 rev function. J Virol 65: 81–88

    PubMed  CAS  Google Scholar 

  • Brady HJ, Miles CG, Pennington DJ, Dzierzak EA (1994) Specific ablation of human immunodeficiency virus Tat-expressing cells by conditionally toxic retroviruses. Proc Natl Acad Sci USA 91: 365–369

    Article  PubMed  CAS  Google Scholar 

  • Brenner MK, Rill DR, Holladay MS, Heslop HE, Moen RC, Buschle M, Krance RA, Santana VM, Anderson WF, Ihle JN (1993) Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients. Lancet 342: 1134–1137

    Article  PubMed  CAS  Google Scholar 

  • Bruton JK, Koeller JM (1994) Recombinant interleukin-2. Pharmacotherapy 14: 635–656

    PubMed  CAS  Google Scholar 

  • Buchschacher GL, Freed EO, Panganiban AT (1992) Cells induced to express a human immunodeficiency virus type 1 envelope gene mutant inhibit the spread of wild-type virus. Hum Gene Ther 3: 391–397

    Article  PubMed  Google Scholar 

  • Buonocore L, Rose JK (1990) Prevention of HIV-1 glycoprotein transport by soluble CD4 retained in the endoplasmic reticulum. Nature 345: 625–628

    Article  PubMed  CAS  Google Scholar 

  • Buonocore L, Rose JK (1993) Blockade of human immunodeficiency virus type 1 production in CD4+ T cells by an intracellular CD4 expressed under control of the viral long terminal repeat. Proc Natl Acad Sci USA 90: 2695–2699

    Article  PubMed  CAS  Google Scholar 

  • Carter RB, Abrams-Ogg AC, Dick JE, Kruth SA, Valli VE, Kamel-Reid S, Dube ID (1992) Autologous transplantation of canine long-term marrow culture cells genetically marked by retroviral vectors. Blood 79: 356–364

    PubMed  CAS  Google Scholar 

  • Castanotto D, Rossi JJ, Sarver N (1994) Antisense catalytic RNAs as therapeutic agents. Adv Pharm 25: 289–317

    Article  CAS  Google Scholar 

  • Casto BC, Armstrong JA, Atchinson RW, Hammon WMcD (1967) Studies on the relationship between adeno-associated virus type 1 (AAV-1) and adenoviruses. II. Inhibition of adenovirus plaques by AAV; its nature and specificity. Virology 33: 452–458

    Article  PubMed  CAS  Google Scholar 

  • Cech TR, Bass BL (1986) Biological catalysis by RNA. Annu Rev Biochem 55: 599–629

    Article  PubMed  CAS  Google Scholar 

  • Chang L-J, Stoltzfus CM (1987) Inhibition of Rous sarcoma virus replication antisense RNA. J Virol 61: 921–924

    PubMed  CAS  Google Scholar 

  • Chatterjee S, Wong KK Jr (1993) Adeno-associated viral vectors for the delivery of antisense RNA. Methods (a companion to methods. Enzymol) 5: 51–59

    Article  CAS  Google Scholar 

  • Chatterjee S, Johnson PR, Wong KK Jr (1992a) Dual target inhibition of HIV-1 in vitro by means of an adeno-associated virus antisense vector. Science 258: 1485–1488

    Article  PubMed  CAS  Google Scholar 

  • Chatterjee S, Wong KK, Podsakoff G, Zaia J, Forman S (1992b) Adeno-associated virus vectors for high efficiency gene transfer into primary hematopoietic cells. Blood 80: 167a

    Google Scholar 

  • Chatterjee S, Podsakoff G, Wong KK Jr (1994) Gene transfer into terminally differentiated primary human peripheral blood-derived mononuclear cells by adeno-associated virus. Blood 84: 360a

    Google Scholar 

  • Chatterjee S, Wong KK Jr, Lu D, Permana P, Podsakoff G. Novel approaches for efficient gene transfer into hematopoietic progenitor cells: the use of adeno-associated virus vectors. Bone Marrow Transplant (in press)

    Google Scholar 

  • Chatterjee S, Podsakoff G, Forman SJ, Wong KK Jr (1995) Adeno-associated virus-mediated gene transfer into CD34+ primary human hematopoietic progenitor cells is highly efficient and stable (submitted)

    Google Scholar 

  • Chen SY, Khouri Y, Bagley J, Marasco WA (1994) Combined intra- and extracellular immunization against human immunodeficiency virus type 1 infection with a human anti-gp120 antibody. Proc Natl Acad Sci USA 91: 5932–5936

    Article  PubMed  CAS  Google Scholar 

  • Chuah MKL, Vandendriessche T, Chang H-K, Ensoli B, Morgan RA (1994) Inhibition of human immunodeficiency virus type-1 by retroviral vectors expressing antisense-TAR. Hum Gene Ther 5: 1467–1475

    Article  PubMed  CAS  Google Scholar 

  • Curiel TJ, Cook DR, Wang Y, Hahn BH, Ghosh SK, Harrison GS (1993) Long-term inhibition of clinical and laboratory human immunodeficiency virus strains in human T-cell lines containing an HIV-regulated diphtheria toxin A chain gene. Hum Gene Ther 4: 741–747

    Article  PubMed  CAS  Google Scholar 

  • Cuypers HT, Selten G, Quint W, Zijlstra M, Maandag ER, Boelens W, van Wezenbeek P, Melief C, Berns A (1984) Murine leukemia virus-induced T-cell lymphomagensis: integration of proviruses in a distinct chromosomal region. Cell 37: 141–150

    Article  PubMed  CAS  Google Scholar 

  • Daar ES, Li XL, Moudgil T, Ho DD (1990) High concentrations of recombinant soluble CD4 are required to neutralize primary human immunodeficiency virus type 1 isolates. Proc Natl Acad Sci USA 87: 6574–6578

    Article  PubMed  CAS  Google Scholar 

  • Dalgleish AG, Beverley PCL, Clapham PR, Crawford DH, Greaves MF, Weiss RA (1984) The CD4 (T4) antigen is an essential component of the receptor for the AIDS retrovirus. Nature 312: 763–767

    Article  PubMed  CAS  Google Scholar 

  • Donahue RE, Kessler SW, Bodine D, Monagh K, Dunbar C, Goodman S, Agricola B, Byrne E, Raffeid M, Meon R, Backer J, Zsebo KM, Nienhuis AW (1992) Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J Exp Med 176: 1125–1135

    Article  PubMed  CAS  Google Scholar 

  • Duan L, Bagasra O, Laughlin MA, Oakes JW, Pomerantz RJ (1994) Potent inhibition of human immunodeficiency virus type 1 replication by an intracellular anti-rev single chain antibody. Proc Natl Acad Sci USA 91: 5075–5077

    Article  PubMed  CAS  Google Scholar 

  • Dunbar CE, Cottier-Fox M, O’Shaughnessy JA, Dören S, Carter C, Berenson R, Brown S, Moen RC, Greenblatt J, Stewart FM, Leitman SF, Wilson WH, Cowan K, Young NS, Nienhuis AW (1995) Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood 85: 3048–3057

    PubMed  CAS  Google Scholar 

  • Dyson N, Howley PM, Münger K, Harlow E (1989) The human papilloma virus-16 oncoprotein is able to bind to the retinoblastoma gene product. Science 243: 934–937

    Article  PubMed  CAS  Google Scholar 

  • Eguchi Y, Itoh T, Tomizawa J (1991) Antisense RNA. Annu Rev Biochem 60: 631–652

    Article  PubMed  CAS  Google Scholar 

  • Faraji-Shadan F, Stubbs JD, Bowman PD (1990) A putative approach for gene therapy against human immunodeficiency virus (HIV). Med Hypotheses 32: 81–84

    Article  PubMed  CAS  Google Scholar 

  • Field AK, Biron KK (1994) The end of innocence revisited: resistance of herpesviruses to antiviral drugs. Clin Microbiol Rev 7: 1–13

    PubMed  CAS  Google Scholar 

  • Flotte TR, Afione SA, Conrad C, McGrath SA, Solow R, Oka H, Zeitlin PL, Guggino WB, Carter BJ (1993) Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci USA 90: 10613–10617

    Article  PubMed  CAS  Google Scholar 

  • Flotte TR, Afione SA, Zeitlin PL (1994) Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am J Respir Cell Mol Biol 11: 517–521

    PubMed  CAS  Google Scholar 

  • Flotte TR, Barraza-Ortiz X, Solow R, Afione SA, Carter BJ, Guggino WB (1995) An improved system for packaging recombinant adeno-associated virus vector capable of in vivo transduction. Gene Therapy 2: 29–37

    PubMed  CAS  Google Scholar 

  • Friedman AD, Triezenberg SJ, McKnight SL (1988) Expression of a truncated viral transactivator selectively impedes lytic infection by its cognate virus. Nature 335: 452–454

    Article  PubMed  CAS  Google Scholar 

  • Galpin JE, Casciato DA, Richards SB (1994) A phase I clinical trial to evaluate the safety and biological activity of HIV-IT (TAF) (HIV-1 III Benv-transduced, autologous fibroblasts) in asymptomatic HIV-1 infected subjects. Hum Gene Ther 5: 997–1017

    Article  PubMed  CAS  Google Scholar 

  • Goodman S, Xiao X, Donahue RE, Moulton A, Miller J, Walsh C, Young NS, Samulski RJ, Nienhuis AW (1994) Recombinant adeno-associated virus-mediated gene transfer into hematopoietic progenitor cells. Blood 84: 1492–1500

    PubMed  CAS  Google Scholar 

  • Green M, Ishino M, Loewenstein PM (1989) Mutational analysis of HIV-1 Tat minimal domain peptides: identification of trans-dominant mutants that suppress HIV-LTR-driven gene expression. Cell 58: 215–223

    Article  PubMed  CAS  Google Scholar 

  • Green PJ, Pines O, Inouye M (1986) The role of antisense RNA in gene regulation. Annu Rev Biochem 55: 569–597

    Article  PubMed  CAS  Google Scholar 

  • Gresser I (1986) Interferon-induced diseases. In: Notkins AL, Oldstone MBA (eds) Concepts in viral pathogenesis II. Springer, Berlin Heidelberg New York, pp 232–242

    Google Scholar 

  • Haibert CL, Alexander IE, Wolgamot GM, Miller AD (1995) Adeno-associated virus vectors transduce primary cells much less efficiently than immortalized cells. J Virol 69: 1473–1479

    Google Scholar 

  • Han L, Yun JS, Wagner TE (1991) Inhibition of Moloney murine leukemia virus-induced leukemia in transgenic mice expressing antisense RNA complementary to the retroviral packaging sequences. Proc Natl Acad Sci USA 88: 4313–4317

    Article  PubMed  CAS  Google Scholar 

  • Harrison GS, Long CJ, Curiel TJ, Maxwell F, Maxwell IH (1992) Inhibition of human immunodeficiency virus-1 production resulting from transduction with a retrovirus containing an HIV-regulated diphtheria toxin A chain gene. Hum Gene Ther. 3: 461–469

    Article  PubMed  CAS  Google Scholar 

  • Hartung S, Jaenisch R, Breindl M (1986) Retrovirus insertion inactivates mouse al(l) collagen gene by blocking initiation of transcription. Nature 320: 365–367

    Article  PubMed  CAS  Google Scholar 

  • Haseloff J, Gerlach WL (1988) Simple RNA enzymes with new and highly specific endoribonuclease activities. Nature 334: 585–591

    Article  PubMed  CAS  Google Scholar 

  • Hayward WS, Neel BG, Astrin SM (1981) Activation of a cellular oncogene by promoter insertion in ALV-induced lymphoid leukosis. Nature 290: 475–480

    Article  PubMed  CAS  Google Scholar 

  • Helene C, Toulme J-J (1990) Specific regulation of gene expression by antisense, sense and antigene nucleic acids. Biochim Biophys Acta 1049: 99–125

    PubMed  CAS  Google Scholar 

  • Hermonat PL (1989) The adeno-associated virus rep 78 gene inhibits cellular transformation induced by bovine papillomavirus. Virology 172: 253–261

    Article  PubMed  CAS  Google Scholar 

  • Hermonat PL (1991) Inhibition of H-ras expression by the adeno-associated virus Rep78 transformation suppressor gene product. Cancer Res 51: 3373–3377

    PubMed  CAS  Google Scholar 

  • Hermonat PL (1994) Adeno-associated virus inhibits human papillomavirus type 16: a viral interaction implicated in cervical cancer. Cancer Res 54: 2278–2281

    PubMed  CAS  Google Scholar 

  • Hermonat PL, 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

    Article  PubMed  CAS  Google Scholar 

  • Herskowitz I (1987) Functional inactivation of genes by dominant negative mutations. Nature 329: 219–222

    Article  PubMed  CAS  Google Scholar 

  • Huibregtse JM, Scheffner 1994) Mechanisms of tumor suppressor protein inactivation by the human papillomavirus E6 and E7 oncoproteins. Semin Virol 5: 357–367

    Article  CAS  Google Scholar 

  • James W (1991) Towards gene-inhibition therapy: a review of progress and prospects in the field of antiviral antisense nucleic acids and ribozymes. Antiviral Chem Chemother 2: 191–214

    CAS  Google Scholar 

  • Joshi S, van Brunschot A, Asad S, van der Elst I, Read SE, Bernstein A (1991) Inhibition of human immunodeficiency virus type 1 multiplication by antisense and sense RNA expression. J Virol 65: 5524–5530

    PubMed  CAS  Google Scholar 

  • Kaniewski WK, Thomas PE (1993) Field testing of virus resistant transgenic plants. Semin Virol 4: 389–396

    Article  Google Scholar 

  • Kantoff PW, Gillio AP, McKachlin JR, Bordignon C, Eglitis MA, Kernan NA, Moen RC, Kohn DB, Yu S-F, Karson E, Karlsson S, Zwiebel JA, Gilboa E, Blaese RM, Nienhuis A, O’Reilly RJ, Anderson WF (1987) Expression of human adenosine deaminase in nonhuman primates after retrovirusmediated gene transfer. J Exp Med 166: 219–234

    Article  PubMed  CAS  Google Scholar 

  • Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, O’Malley KL, During MJ (1994) Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nature Genet 8: 148–153

    Article  PubMed  CAS  Google Scholar 

  • Kotin RM (1994) Prospects for the use of adeno-associated virus as a vector for human gene therapy. Hum Gene Ther 5: 793–801

    Article  PubMed  CAS  Google Scholar 

  • Kotin RM, Siniscalco M, Samulski RJ, Zhu X, Hunter L, Laughlin CA, McLaughlin S, Muzyczka N, Rocchi M, Berns KI (1990) Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA 87: 2211–2215

    Article  PubMed  CAS  Google Scholar 

  • Kung HJ, Boerkel C, Carter TH (1991) Retroviral mutagenesis of cellular oncogenes: a review with insights into the mechanisms of insertional activation. In: Kung HJ, Vogt PK (eds) Retroviral insertional mutagenesis and oncogene activation. Springer, Berlin Heidelberg New York, pp 1–23 (Current topics in microbiology and immunology, vol 171)

    Chapter  Google Scholar 

  • LaFace D, Hermonat P, Wakeland E, Peck A (1988) Gene transfer into hematopoietic progenitor cells mediated by an adeno-associated virus vector. Virology 162: 483–486

    Article  PubMed  CAS  Google Scholar 

  • Lebkowski JS, McNally MM, Okarma TB, Lerch LB (1988) Adeno-associated virus: a vector system for efficient introduction and integration of DNA into a variety of mammalian cell types. Mol Cell Biol 8: 3988–3996

    PubMed  CAS  Google Scholar 

  • Lee TC, Sullenger BA, Gallardo HF, Ungers GE, Gilboa E (1992) Overexpression of RRE-derived sequences inhibits HIV-1 replication in CEM cells. New Biol 4: 66–74

    PubMed  CAS  Google Scholar 

  • Liem SE, Ramezani A, Li X, Joshi S (1993) The development and testing of retroviral vectors expressing trans-dominant mutants of HIV-1 proteins to confer anti-HIV-1 resistance. Hum Gene Ther 4: 625–634

    Article  PubMed  CAS  Google Scholar 

  • Lin X, Dashti A, Schinazi RF, Tang J (1993) Intracellular diversion of glycoprotein GP160 of human immunodeficiency virus to lysosomes as a strategy of AIDS gene therapy. FASEB J 7: 1070–1080

    PubMed  CAS  Google Scholar 

  • Lisziewicz J, Sun D, Smythe J, Lusso P, Lori F, Louie A, Markham P, Rossi J, Reitz M, Gallo RC (1993) Inhibition of human immunodeficiency virus type 1 replication by regulated expression of a polymeric Tat activation response RNA decoy as a strategy for gene therapy in AIDS. Proc Natl Acad Sci USA 90: 8000–8004

    Article  PubMed  CAS  Google Scholar 

  • Liu J, Woffendin C, Yang A, Nabel GJ (1994) Regulated expression of a dominant negative form of Rev improves resistance to HIV replication in T cells. Gene Ther 1: 32–27

    PubMed  CAS  Google Scholar 

  • Lu D, Chatterjee S, Brar D, Wong KK Jr (1994a) High efficiency in vitro cleavage of transcripts arising from the major transforming genes of human papillomavirus type 16 mediated by ribozymes transcribed from an adeno-associated virus-based vector. Cancer Gene Ther 1: 267–277

    PubMed  CAS  Google Scholar 

  • Lu D, Wong KK Jr, Podsakoff G, Chatterjee S (1994b) Stable and efficient adeno-associated virusmediated gene transfer into primitive human marrow-derived hematopoietic cells in long term culture. Blood 84: 360a

    Google Scholar 

  • Lusso P, Veronese F, Ensoli B, Franchini G, Jemma C, dePocco SE, Kalyanaraman VS, Gallo RC (1990) Expanded HIV-1 cellular tropism by phenotypic mixing with murine endogenous retroviruses. Science 247: 848–852

    Article  PubMed  CAS  Google Scholar 

  • Maciejewski JP, Weichold FF, Young NS, Cara A, Zella D, Reitz MS Jr, Gallo RC (1995) Intracellular expression of antibody fragments directed against HIV reverse transcriptase prevents HIV infection in vitro. Nature Med 1: 667–673

    Article  PubMed  CAS  Google Scholar 

  • Malim MH, Freimuth WW, Liu J, Boyle TJ, Lyerly HK, Cullen BR, Nabel GJ (1992) Stable expression of transdominant Rev protein in human T cells inhibits human immunodeficiency virus replication. J Exp Med 176: 1197–1201

    Article  PubMed  CAS  Google Scholar 

  • Marasco WA, Haseltine WA, Chen SY (1993) Design, intracellular expression, and activity of a human anti-human immunodeficiency virus typel gp120 single-chain antibody. Proc Natl Acad Sci USA 90: 7889–7893

    Article  PubMed  CAS  Google Scholar 

  • Mayor HD (1993) Defective parvoviruses may be good for your health! Prog Med Virol 40: 193–205

    PubMed  CAS  Google Scholar 

  • McLaughlin SK, Collis P, Hermonat PL, Muzyczka N (1988) Adeno-associated virus general transduction vectors: analysis of proviral structures. J Virol 62: 1963–1973

    PubMed  CAS  Google Scholar 

  • Mendelson E, Grossman Z, Mileguir F, Rechavi G, Carter BJ (1992) Replication of adeno-associated virus type 2 in human lymphocytic cells and interaction with HIV-1. Virology 187: 453–463

    Article  PubMed  CAS  Google Scholar 

  • Miller AD (1992a) Retroviral vectors. In: Muzyczka N (ed) Viral expression vectors. Springer, Berlin Heidelberg Neork, pp1–24 (Current topics in microbiology and immunology, vol 158)

    Chapter  Google Scholar 

  • Miller AD (1992b) Human gene therapy comes of age. Nature 357: 455–460

    Article  PubMed  CAS  Google Scholar 

  • Miller DG, Adam MA, Miller AD (1990) Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 10: 4239–4242

    PubMed  CAS  Google Scholar 

  • Miroshnichenko OI, Ponomareva TI, Tikchonenko TI (1989) Inhibition of adenovirus 5 replication in COS-1 cells by antisense RNAs against the viral E1a region. Gene 84: 83–89

    Article  PubMed  CAS  Google Scholar 

  • Morgan RA, Looney DJ, Muenchau DD, Wong-Staal F, Gallo RC, Anderson WF (1990) Retroviral vectors expressing soluble CD4: a potential gene therapy for AIDS. AIDS Res Hum Retroviruses 6: 183–191

    Article  PubMed  CAS  Google Scholar 

  • Mulligan RC (1993) The basic science of gene therapy. Science 260: 926–932

    Article  PubMed  CAS  Google Scholar 

  • Muro-Cacho CA, Samulski RJ, Kaplan D (1992) Gene transfer in human lymphocytes using a vector based on adeno-associated virus. J Immunother 11: 231–237

    Article  PubMed  CAS  Google Scholar 

  • Muzyczka N (1992) Use of AAV as a general transduction vector for mammalian cells. Curr Top Micorb Immunol 158: 97–129

    Article  CAS  Google Scholar 

  • Nabel GJ, Fox BA, Post L, Thompson CB, Woffendin C (1994) A molecular genetic intervention for AIDS-effects of a transdominant negative form of Rev. Hum Gene Ther 5: 79–92

    Article  PubMed  CAS  Google Scholar 

  • Nakagawa Y, Kakimi K, Ling W, Kubo Y, Higo K, Masuda T, Kuribayashi K, Iwashiro M, Komatz Y, Hirama T, Adachi A, Ishimoto A (1994) Inhibition of murine AIDS (MAIDS), development by the transplantation of bone marrow cells carrying the Fv-4 resistance gene to MAIDS virus-infected mice. J Virol 68: 1438–1441

    PubMed  CAS  Google Scholar 

  • Natsoulis G, Seshaiah P, Federspiel MJ, Rein A, Hughes SH, Boeke JD (1995) Targeting of a nuclease to murine leukemia virus capsids inhibits viral multiplication. Proc Natl Acad Sci USA 92: 364–368

    Article  PubMed  CAS  Google Scholar 

  • Oelze I, Rittner K, Sczakiel G (1994) Adeno-associated virus type 2 rep gene-mediated inhibition of basal gene expression of human immunodeficiency virus type 1 involves its negative regulatory functions. J Virol 68: 1229–1233

    PubMed  CAS  Google Scholar 

  • Ojwang JO, Hampel A, Looney DJ, Wong-Staal F, Rappaport J (1992) Inhibition of human immunodeficiency virus type 1 expression by a hairpin ribozyme. Proc Natl Acad Sci USA 89: 10802–10806

    Article  PubMed  CAS  Google Scholar 

  • Ostrove JM, Duckworth DH, Berns KI (1981) Inhibition of adenovirus-transformed cell oncogenicity by adeno-associated virus. Virology 113: 521–533

    Article  PubMed  CAS  Google Scholar 

  • Perriman R, Delves A, Gerlach WL (1992) Extended target-site specificity for a hammerhead ribozyme. Gene 113: 157–163

    Article  PubMed  CAS  Google Scholar 

  • Podsakoff G, Wong KK Jr, Chatterjee S (1994a) Stable and efficient gene transfer into non-dividing cells by adeno-associated virus (AAV)-based vectors. J Virol 68: 5656–5666

    Google Scholar 

  • Podsakoff G, Shaughnessy EA, Lu D, Wong KK Jr, Chatterjee S (1994b) Long term in vivo reconstitution with murine marrow cells transduced with an adeno-associated virus vector. Blood 84: 256a

    Google Scholar 

  • Poeschla E, Wong-Staal F (1994) Antiviral and anticancer ribozymes. Curr Opin Oncol 6: 601–606

    Article  PubMed  CAS  Google Scholar 

  • Ptashne M (1988) How eukaryotic transcriptional activators work. Nature 335: 683–689

    Article  PubMed  CAS  Google Scholar 

  • Rhodes A, James W (1990) Inhibition of human immunodeficiency virus replication in cell culture by endogenously synthesized antisense RNA. J Gen Virol 71: 1965–1974

    Article  PubMed  CAS  Google Scholar 

  • Richman DD (1993) HIV drug resistance. Annu Rev Pharmacol Toxicol 33: 149–164

    Article  PubMed  CAS  Google Scholar 

  • Rittner K, Heilbronn R, Kleinschmidt JA, Sczakiel G (1992) Adeno-associated virus type 2-mediated inhibition of human immunodeficiency virus type 1 (HIV-1) replication: involvement of p78rep/p68 Rep and the HIV-1 long terminal repeat. J Gen Virol 73: 2977–2981

    Article  PubMed  CAS  Google Scholar 

  • Roberts MR, Qin L, Zhang D, Smith DH, Tran AC, Dull TJ, Groopman JE, Capon DJ, Byrn RA, Finer MH (1994) Targeting of human immunodeficiency virus-infected cells by CD8+ T lymphocytes armed with universal T-cell receptors. Blood 84: 2878–2889

    PubMed  CAS  Google Scholar 

  • Roe TY, Reynolds TC, Yu G, Brown PO (1993) Integration of murine leukemia virus DNA depends on mitosis. EMBO J 12: 2099–2108

    PubMed  CAS  Google Scholar 

  • Rommelaere J, Cornelis JJ (1991) Antineoplastic activity of parvoviruses. J Virol Methods 33: 233–251

    Article  PubMed  CAS  Google Scholar 

  • Salter DW, Crittenden LB (1989) Artificial insertion of a dominant gene for resistance to avian leukosis virus into the germ line of the chicken. Theor Appl Genet 77: 457–461

    Article  Google Scholar 

  • Sarver N, Cantin EM, Chang PS, Zaia JA, Ladne PA, Stephens DA, Rossi JJ (1990) Ribozymes as potential anti-HIV-1 therapeutic agents. Science 247: 1222–1225

    Article  PubMed  CAS  Google Scholar 

  • Schlehofer JR (1994) The tumor suppressive properties of adeno-associated viruses. Mutat Res 305: 303–313

    Article  PubMed  CAS  Google Scholar 

  • Schnittman SM, Fauci AS (1994) Human immunodeficiency virus and acquired immunodeficiency syndrome: an update. Adv Intern Med 39: 305–355

    PubMed  CAS  Google Scholar 

  • Sczakiel G, Pawlita M (1991) Inhibition of human immunodeficiency virus type 1 replication in human T cells stably expressing antisense RNA. J Virol 65: 468–472

    PubMed  CAS  Google Scholar 

  • Seif I, de Maeyer E, Riviere I, de Maeyer-Guignard J (1991) Stable antiviral expression in BALB/c 3T3 cells carrying a beta interferon sequence behind a major histocompatibility complex promoter fragment. J Virol 65: 664–671

    PubMed  CAS  Google Scholar 

  • Shepard AA, Polentino P, Duca NA (1990) Trans-dominant inhibition of herpes simplex virus transcriptional regulatory protein ICP4 by heterodimer formation. J Virol 64: 3916–3926

    PubMed  CAS  Google Scholar 

  • Shillitoe EJ, Kamath P, Chen Z (1994) Papillomaviruses as targets for cancer gene therapy. Cancer Genher 1: 193–204

    CAS  Google Scholar 

  • Shimada T, Fujii H, Maier B, Hayashi S, Mitsuya H, Broder S, Nienhuis AW (1991) Trial of antisense RNA inhibition of HIV replication and gene expression. Antiviral Chem Chemother 2: 133–142

    CAS  Google Scholar 

  • Smythe JA, Sun D, Thomson M, Markham PD, Reitz MS Jr, Gallo RC, Lisziewicz J (1994) A Revinducible mutant gag gene stably trasferred into T lymphocytes: an approach to gene therapy against human immunodeficiency virus type 1 infection. Proc Natl Acad Sci USA 91: 3657–3661

    Article  PubMed  CAS  Google Scholar 

  • Staeheli P (1990) Interferon-induced proteins and the antiviral state. Adv Virus Res 38: 147–200

    Article  PubMed  CAS  Google Scholar 

  • Sullenger BA, Lee TC, Smith CA, Ungers GE, Gilboa E (1990a) Expression of chimeric NA-driven antisense transcripts renders NIH 3T3 cells highly resistant to Moloney murine leukemia virus replication. Mol Cell Biol 10: 6512–6523

    PubMed  CAS  Google Scholar 

  • Sullenger BA, Gallardo HF, Ungers GE, Gilboa E (1990b) Overexpression of TAR sequences renders cells resistant to human immunodeficiency virus replication. Cell 63: 601–608

    Article  PubMed  CAS  Google Scholar 

  • Tratschin JD, West MH, Sandbank T, Carter BJ (1984) A human parvovirus, adeno-associated virus, as a eucaryotic vector: transient expression and encapsidation of the procaryotic gene for chloramphenicol acetyltransferase. Mol Cell Biol 4: 2072–2081

    PubMed  CAS  Google Scholar 

  • Trono D, Feinberg MB, Baltimore D (1989) HIV-1 gag mutants can dominantly interfere with the replication of the wild-type virus. Cell 59: 113–120

    Article  PubMed  CAS  Google Scholar 

  • Vaishnav YN, Wong-Staal F (1991) The biochemistry of AIDS. Annu Rev Biochem 60: 577–630

    Article  PubMed  CAS  Google Scholar 

  • Van der Krol AR, Mol JNM, Stuitje AR (1988) Modulation of eukaryotic gene expression by complementary RNA or DNA sequences. Biotechniques 6: 958–976

    PubMed  Google Scholar 

  • Vieillard V, Lauret E, Rousseau V, De Maeyer E (1994) Blocking of retroviral infection at a step prior to reverse transcription in cells transformed to constitutively express interferon beta. Proc Natl Acad Sci USA 91: 2689–2693

    Article  PubMed  CAS  Google Scholar 

  • von Ruden T, Gilboa E (1989) Inhibition of human T-cell leukemia virus type 1 replication in primary human T cells that express antisense RNA. J Virol 63: 677–682

    Google Scholar 

  • Wagner EGH, Simons RW (1994) Antisense RNA control in bacteria, phages, and plasmids. Annu Rev Microbiol 48: 713–742

    Article  PubMed  CAS  Google Scholar 

  • Walsh CE, Nienhuis AW, Samulski RJ, Brown MG, Miller JL, Young NS, Liu JM (1994a) Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector. J Clin Invest 94: 1440–1448

    Article  PubMed  CAS  Google Scholar 

  • Walsh CE, Liu IM, Wang S, Xiao X, Hashmi NJ, Zwerdling T, Agarwal R (1994b) In vivo gene transfer with a novel adeno-associated virus vector to human hematopoietic cells engrafted in SCID-hu mice. Blood 84: 256a

    Google Scholar 

  • Warner JF, Anderson CG, Laube L, Jolly DJ, Townsend K, Chada S, St Louis D (1991) HIV-specific CTL and antibody responses in mice using retroviral vector-transduced cells. Aids Res Hum Retroviruses 7: 645–655

    Article  PubMed  CAS  Google Scholar 

  • Watson RJ, Clemens JB (1980) A herpes simplex virus type 1 function continuously required for early and late virus RNA synthesis. Nature 285: 329–330

    Article  PubMed  CAS  Google Scholar 

  • Weerasinghe M, Liem SE, Asad S, Read SE, Joshi S (1991) Resistance to human immunodeficiency virus type 1 (HIV-1) infection in human CD4+ lymphocyte-derived cell lines conferred by using retroviral vectors expressing an HIV-1 RNA-specific ribozyme. J Virol 65: 5531–5534

    PubMed  CAS  Google Scholar 

  • Weintraub H, Izant JG, Harland RM (1985) Anti-sense RNA as a molecular tool for genetic analysis. Trends Genet 2: 22–25

    Article  Google Scholar 

  • Werge TM, Biocca S, Cattaneo A (1990) Intracellular immunization. Cloning and intracellular expression of a monoclonal antibody to the p21ras protein. FEBS Lett 274: 193–198

    Article  PubMed  CAS  Google Scholar 

  • Werness BA, Levine A, Howley PM (1990) Association of human papilloma virus type 16 and 18 E6 proteins with p53. Science. 248: 76–79

    Article  PubMed  CAS  Google Scholar 

  • Woffendin C, Yang ZY, Udaykumar, Xu L, Yang NS, Sheehy MJ, Nabel GJ (1994) Nonviral and viral delivery of a human immunodeficiency virus protective gene into primary human T cells. Proc Natl Acad Sci USA 91: 11581–11585

    Article  PubMed  CAS  Google Scholar 

  • Wong KK Jr, Chatterjee S (1992) Controlling herpes simplex virus (HSV) infections: intracellular immunization, the way of the future? Curr Top Micro Immunol 179: 159–174

    Article  CAS  Google Scholar 

  • Wong KK Jr, Chatterjee S (1992) In: Rouse BT (ed) Herpes Simplex virus. Springer, Berlin Heidelberg New York, pp 159–174 (Current topics in microbiology and immunology, vol 179)

    Chapter  Google Scholar 

  • Wong KK Jr, Rose JA, Chatterjee S (1991) Restriction of HSV-1 production in cell lines transduced with an antisense viral vector targeting the ICP4 gene. (Vaccine, vol 91) Cold Spring Harbor Laboratory Press, Cold Spring Harbor, New York, pp 183–189

    Google Scholar 

  • Yamada O, Yu M, Yee J-K, Kraus G, Looney D, Wong-Staal F (1994) Intracellular immunization of human T cells with a hairpin ribozyme against human immunodeficiency virus type 1. Gene Ther 1: 38–45

    PubMed  CAS  Google Scholar 

  • Yang Q, Chen F, Trempe JP (1994) Characterization of cell lines that inducibly express the adeno-associated virus Rep proteins. J Virol 68: 4847–4856

    PubMed  CAS  Google Scholar 

  • Yu M, Ojwang J, Yamada O, Hampel A, Rapapport J, Looney D, Wong-Staal F (1993) A hairpin ribozyme inhibits expression of diverse strains of human immunodeficiency virus type 1. Proc Natl Acad Sci USA 90: 6340–6344

    Article  PubMed  CAS  Google Scholar 

  • Yu M, Poeschla E, Wong-Staal F (1994) Progress towards gene therapy for HIV infection. Gene Ther 1: 13–26

    PubMed  CAS  Google Scholar 

  • Zaia JA, Chatterjee S, Wong KK, Elkins D, Taylor NR, Rossi JJ (1992) Status of ribozyme and antisense-based developmental approaches for anti-HIV-1 therapy. Ann N Y Acad Sci 660: 95–105

    Article  PubMed  CAS  Google Scholar 

  • Zhou C, Bahner IC, Larson GP, Zaia JA, Rossi JJ, Kohn DB (1994) Inhibition of HIV-1 in human T-lymphocytes by retrovirally transduced anti-faiand rev hammerhead ribozymes. Gene 149: 33–39

    Article  PubMed  CAS  Google Scholar 

  • Zhou SZ, Cooper S, Kang LY, Ruggieri L, Heimfeld S, Srivastava A, Broxmeyer HE (1994) Adeno-associated virus 2-mediated high efficiency gene transfer into immature and mature subsets of hematopoietic progenitor cells in human umbilical cord blood. J Exp Med 179: 1867–1875

    Article  PubMed  CAS  Google Scholar 

  • zur Hausen H, de Villiers EM (1994) Human papillomaviruses. Annu Rev Microbiol 48: 427–447

    Article  PubMed  Google Scholar 

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  1. Departments of Hematology and Bone Marrow Transplantation, Infectious Diseases, 1500 E. Duarte Road, 91010, Duarte, CA, USA

    K. K. Wong Jr.

  2. Division of Pediatrics, Beckman Research Institute, City of Hope National Medical Center, 1500 E. Duarte Road, 91010, Duarte, CA, USA

    S. Chatterjee

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  1. Hearst Microbiology Research Center, Department of Microbiology, Cornell University Medical College, 1300 York Avenue, Box 62, 10021, New York, NY, USA

    Kenneth I. Berns M.D., Ph.D.  & Catherine Giraud Ph.D.  & 

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Wong, K.K., Chatterjee, S. (1996). Adeno-associated Virus Based Vectors As Antivirals. In: Berns, K.I., Giraud, C. (eds) Adeno-Associated Virus (AAV) Vectors in Gene Therapy. Current Topics in Microbiology and Immunology, vol 218. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-80207-2_9

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