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Clinical Pharmacokinetics

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01.10.2002 | Leading Article

Virus-Based Gene Delivery Systems

verfasst von: Cathryn Mah, Barry J. Byrne, Dr Terence R. Flotte

Erschienen in: Clinical Pharmacokinetics | Ausgabe 12/2002

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Abstract

Within the past decade, gene therapy strategies have come to the forefront of novel therapeutics. Tremendous advances in vector technology along with deeper understandings of vector biology and the molecular mechanisms of disease have significantly advanced the field of human gene therapy. This manuscript will discuss the viral-based subset of current gene transfer vectors. In particular, the most established viral vectors to date, including parvovirus, adenovirus, retro-virus, lentivirus, and herpesvirus-based vectors, are described, as well as the current innovative improvements being made to each. From past experience, it has become evident that in addition to optimising the vectors in terms of transgene expression, minimising vector-related immunology, and vector production, methods of vector delivery resulting in optimum vector transduction of target cells need to be established. This review will also illustrate several current improved physical delivery systems for optimal vector administration.

Berns KI. Parvovirus replication. Microbiol Rev 1990; 54(3): 316–29PubMed

Srivastava A, Lusby EW, Berns KI. Nucleotide sequence and organization of the adeno-associated virus 2 genome. J Virol 1983; 45(2): 555–64PubMed

Carter PJ, Samulski RJ. Adeno-associated viral vectors as gene delivery vehicles. Int J Mol Med 2000; 6(1): 17–27PubMed

Hermonat PL, Muzyczka N. Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells. Proc Natl Acad Sci U S A 1984; 81(20): 6466–70PubMedCrossRef

Tratschin JD, West MH, Sandbank T, et al. A human parvovirus, adeno-associated virus, as a eucaryotic vector: transient expression and encapsidation of the procaryotic gene for chloramphenicol acetyltransferase. Mol Cell Biol 1984; 4(10): 2072–81PubMed

Kotin RM, Siniscalco M, Samulski RJ, et al. Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA 1990; 87(6): 2211–5PubMedCrossRef

Kotin RM, Menninger JC, Ward DC, et al. Mapping and direct visualization of a region-specific viral DNA integration site on chromosome 19ql3-qter. Genomics 1991; 10(3): 831–4PubMedCrossRef

Kotin RM, Linden RM, Berns KI. Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination. EMBO J 1992; 11(13): 5071–8PubMed

Samulski RJ, Zhu X, Xiao X, et al. Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J 1991; 10(12): 3941–50PubMed

10.

Samulski RJ. Adeno-associated virus: integration at a specific chromosomal locus. Curr Opin Genet Dev 1993; 3(1): 74–80PubMedCrossRef

11.

Davidson BL, Stein CS, Heth JA, et al. Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. Proc Natl Acad Sci U S A 2000; 97(7): 3428–32PubMedCrossRef

12.

Dong JY, Fan PD, Frizzell RA. Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum Gene Ther 1996; 7(17): 2101–12PubMedCrossRef

13.

Duan D, Yue Y, Yan Z, et al. A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation. Nat Med 2000; 6(5): 595–8PubMedCrossRef

14.

Flotte TR, Afione SA, Zeitlin PL. Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am J Respir Cell Mol Biol 1994; 11(5): 517–21PubMed

15.

Kearns WG, Afione SA, Fulmer SB, et al. Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line. Gene Ther 1996; 3(9): 748–55PubMed

16.

Zabner J, Seiler M, Walters R, et al. Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J Virol 2000; 74(8): 3852–8PubMedCrossRef

17.

Beck SE, Jones LA, Chesnut K, et al. Repeated delivery of adeno-associated virus vectors to the rabbit airway. J Virol 1999; 73(11): 9446–55PubMed

18.

Summerford C, Samulski RJ. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 1998; 72(2): 1438–45PubMed

19.

Qing K, Mah C, Hansen J, et al. Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nat Med 1999; 5(1): 71–7PubMedCrossRef

20.

Summerford C, Bartlett JS, Samulski RJ. AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infection. Nat Med 1999; 5(1): 78–82PubMedCrossRef

21.

Chiorini JA, Kim F, Yang L, et al. Cloning and characterization of adeno-associated virus type 5. J Virol 1999; 73(2): 1309–19PubMed

22.

Srivastava CH, Samulski RJ, Lu L, et al. Construction of a recombinant human parvovirus B19: adeno-associated virus 2 (AAV) DNA inverted terminal repeats are functional in an AAV-B19 hybrid virus. Proc Natl Acad Sci U S A 1989; 86(20): 8078–82PubMedCrossRef

23.

Wu P, Xiao W, Conlon T, et al. Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism. J Virol 2000; 74(18): 8635–47PubMedCrossRef

24.

Drapkin PT, O’Riordan CR, Yi SM, et al. Targeting the uroki-nase plasminogen activator receptor enhances gene transfer to human airway epithelia. J Clin Invest 2000; 105(5): 589–96PubMedCrossRef

25.

Bartlett JS, Kleinschmidt J, Boucher RC, et al. Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab’gamma)2 antibody. Nat Biotechnol 1999; 17(2): 181–6PubMedCrossRef

26.

Flotte T, Carter B, Conrad C, et al. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther 1996; 7(9): 1145–59PubMedCrossRef

27.

Wagner JA, Reynolds T, Moran ML, et al. Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus. Lancet 1998; 351(9117): 1702–3PubMedCrossRef

28.

Wagner JA, Moran ML, Messner AH, et al. A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis. Hum Gene Ther 1998; 9(6): 889–909PubMedCrossRef

29.

Wagner JA, Messner AH, Moran ML, et al. Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus. Laryngoscope 1999; 109 (2 Pt1): 266–74CrossRef

30.

Virella-Lowell I, Poirier A, Chesnut KA, et al. Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients. Gene Ther 2000; 7(20): 1783–9PubMedCrossRef

31.

Kay MA, Manno CS, Ragni MV, et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 2000; 24(3): 257–61PubMedCrossRef

32.

Yan Z, Zhang Y, Duan D, et al. Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy. Proc Natl Acad Sci U S A 2000; 97(12): 6716–21PubMedCrossRef

33.

Knowles MR, Hohneker KW, Zhou Z, et al. A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. N Engl J Med 1995; 333(13): 823–31PubMedCrossRef

34.

Crystal RG, McElvaney NG, Rosenfeld MA, et al. Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat Genet 1994; 8(1): 42–51PubMedCrossRef

35.

Morral N, Parks RJ, Zhou H, et al. High doses of a helper-dependent adenoviral vector yield supraphysiological levels of alphal-antitrypsin with negligible toxicity. Hum Gene Ther 1998; 9(18): 2709–16PubMedCrossRef

36.

Schiedner G, Morral N, Parks RJ, et al. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat Genet 1998; 18(2): 180–3PubMedCrossRef

37.

Bergelson JM, Krithivas A, Celi L, et al. The murine CAR homolog is a receptor for coxsackie B viruses and adenovi-ruses. J Virol 1998; 72(1): 415–9PubMed

38.

Hidaka C, Milano E, Leopold PL, et al. CAR-dependent and CAR-independent pathways of adenovirus vector-mediated gene transfer and expression in human fibroblasts. J Clin Invest 1999; 103(4): 579–87PubMedCrossRef

39.

Bouri K, Feero WG, Myerburg MM, et al. Polylysine modification of adenoviral fiber protein enhances muscle cell transduction. Hum Gene Ther 1999; 10(10): 1633–40PubMedCrossRef

40.

Gonzalez R, Vereecque R, Wickham TJ, et al. Increased gene transfer in acute myeloid leukemic cells by an adenovirus vector containing a modified fiber protein. Gene Ther 1999; 6(3): 314–20PubMedCrossRef

41.

Wickham TJ, Tzeng E, Shears II LL, et al. Increased in vitro and in vivo gene transfer by adenovirus vectors containing chimeric fiber proteins. J Virol 1997; 71(11): 8221–9PubMed

42.

Wickham TJ, Segal DM, Roelvink PW, et al. Targeted adenovirus gene transfer to endothelial and smooth muscle cells by using bispecific antibodies. J Virol 1996; 70(10): 6831–8PubMed

43.

Wickham TJ, Haskard D, Segal D, et al. Targeting endothelium for gene therapy via receptors up-regulated during angiogene-sis and inflammation. Cancer Immunol Immunother 1997; 45(3–4): 149-51

44.

Wickham TJ, Lee GM, Titus JA, et al. Targeted adenovirus-me-diated gene delivery to T cells via CD3. J Virol 1997; 71(10): 7663–9PubMed

45.

Miller AD, Jolly DJ, Friedmann T, et al. A transmissible retro-virus expressing human hypoxanthine phosphoribosyl-transferase (HPRT): gene transfer into cells obtained from humans deficient in HPRT. Proc Natl Acad Sci U S A 1983; 80(15): 4709–13PubMedCrossRef

46.

Miller AD, Ong ES, Rosenfeld MG, et al. Infectious and selectable retrovirus containing an inducible rat growth hormone minigene. Science 1984; 225(4666): 993–8PubMedCrossRef

47.

Mann R, Mulligan RC, Baltimore D. Construction of a retro-virus packaging mutant and its use to produce helper-free defective retrovirus. Cell 1983; 33(1): 153–9PubMedCrossRef

48.

Cepko CL, Roberts BE, Mulligan RC. Construction and applications of a highly transmissible murine retrovirus shuttle vector. Cell 1984; 37(3): 1053–62PubMedCrossRef

49.

Cone RD, Mulligan RC. High-efficiency gene transfer into mammalian cells: generation of helper-free recombinant retrovirus with broad mammalian host range. Proc Natl Acad Sci U S A 1984; 81(20): 6349–53PubMedCrossRef

50.

Williams DA, Orkin SH, Mulligan RC. Retrovirus-mediated transfer of human adenosine deaminase gene sequences into cells in culture and into murine hematopoietic cells in vivo. Proc Natl Acad Sci U S A 1986; 83(8): 2566–70PubMedCrossRef

51.

Palmer TD, Hock RA, Osborne WR, et al. Efficient retrovirus-mediated transfer and expression of a human adenosine deaminase gene in diploid skin fibroblasts from an adenosine deaminase-deficient human. Proc Natl Acad Sci USA 1987; 84(4): 1055–9PubMedCrossRef

52.

Ramsey WJ, Mullen CA, Blaese RM. Retrovirus mediated gene transfer as therapy for adenosine deaminase (ADA) deficiency [abstract]. Leukemia 1995; 9 Suppl. 1: S70PubMed

53.

Miller DG, Adam MA, Miller AD. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 1990; 10(8): 4239–42PubMed

54.

Scharfmann R, Axelrod JH, Verma IM. Long-term in vivo expression of retrovirus-mediated gene transfer in mouse fibro-blast implants. Proc Natl Acad Sci U S A 1991; 88(11): 4626–30PubMedCrossRef

55.

Miller AD, Buttimore C. Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production. Mol Cell Biol 1986; 6(8): 2895–902PubMed

56.

Miller AD. Retrovirus packaging cells. Hum Gene Ther 1990; 1(1): 5–14PubMedCrossRef

57.

Naldini L, Blomer U, Gallay P, et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272(5259): 263–7PubMedCrossRef

58.

Poeschla EM, Wong-Staal F, Looney DJ. Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors. Nat Med 1998; 4(3): 354–7PubMedCrossRef

59.

Kafri T, Blomer U, Peterson DA, et al. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet 1997; 17(3): 314–7PubMedCrossRef

60.

Miyoshi H, Smith KA, Mosier DE, et al. Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 1999; 283(5402): 682–6PubMedCrossRef

61.

Kafri T, van Praag H, Ouyang L, et al. A packaging cell line for lentivirus vectors. J Virol 1999; 73(1): 576–84PubMed

62.

Miyoshi H, Blomer U, Takahashi M, et al. Development of a self-inactivating lentivirus vector. J Virol 1998; 72(10): 8150–7PubMed

63.

Spaete RR, Frenkel N. The herpes simplex virus amplicon: a new eucaryotic defective-virus cloning-amplifying vector. Cell 1982; 30(1): 295–304PubMedCrossRef

64.

Spaete RR, Frenkel N. The herpes simplex virus amplicon: analyses of cis-acting replication functions. Proc Natl Acad Sci USA 1985; 82(3): 694–8PubMedCrossRef

65.

Geller AI, Yu L, Wang Y, et al. Helper virus-free herpes simplex virus-1 plasmid vectors for gene therapy of Parkinson’s disease and other neurological disorders. Exp Neurol 1997; 144(1): 98–102PubMedCrossRef

66.

Kwong AD, Frenkel N. Herpes simplex virus amplicon: effect of size on replication of constructed defective genomes containing eucaryotic DNA sequences. J Virol 1984; 51(3): 595–603PubMed

67.

Geller AI, Keyomarsi K, Bryan J, et al. An efficient deletion mutant packaging system for defective herpes simplex virus vectors: potential applications to human gene therapy and neuronal physiology. Proc Natl Acad Sci U S A 1990; 87(22): 8950–4PubMedCrossRef

68.

Glorioso JC, Goins WF, DeLuca N, et al. Development of herpes simplex virus as a gene transfer vector for the nervous system [abstract]. Gene Ther 1994; 1 Suppl. 1: S39PubMed

69.

Fink DJ, Ramakrishnan R, Marconi P, et al. Advances in the development of herpes simplex virus-based gene transfer vectors for the nervous system. Clin Neurosci 1995; 3(5): 284–91PubMed

70.

Marconi P, Krisky D, Oligino T, et al. Replication-defective herpes simplex virus vectors for gene transfer in vivo. Proc Natl Acad Sci U S A 1996; 93(21): 11319–20PubMedCrossRef

71.

Glorioso JC, Goins WF, Schmidt MC, et al. Engineering herpes simplex virus vectors for human gene therapy. Adv Pharmacol 1997; 40: 103–36PubMedCrossRef

72.

Rooney JF, Wohlenberg C, Cremer KJ, et al. Immunization with a vaccinia virus recombinant expressing herpes simplex virus type 1 glycoprotein D: long-term protection and effect of re-vaccination. J Virol 1988; 62(5): 1530–4PubMed

73.

Tsukamoto H, Wells D, Brown S, et al. Enhanced expression of recombinant dystrophin following intramuscular injection of Epstein-Barr virus (EB V)-based mini-chromosome vectors in mdx mice. Gene Ther 1999; 6(7): 1331–5PubMedCrossRef

74.

Nakanishi M, Mizuguchia H, Ashihara K, et al. Gene transfer vectors based on Sendai virus. J Control Release 1998; 54(1): 61–8PubMedCrossRef

75.

Nakanishi M, Mizuguchi H, Ashihara K, et al. Gene delivery systems using the Sendai virus. Mol Membr Biol 1999; 16(1): 123–7PubMedCrossRef

76.

Herweijer H, Latendresse JS, Williams P, et al. A plasmid-based self-amplifying Sindbis virus vector. Hum Gene Ther 1995; 6(9): 1161–7PubMedCrossRef

77.

Berglund P, Sjoberg M, Garoff H, et al. Semliki Forest virus expression system: production of conditionally infectious recombinant particles. Bio/technology 1993; 11(8): 916–20PubMedCrossRef

78.

Caplen NJ, Higginbotham JN, Scheel JR, et al. Adeno-retroviral chimeric viruses as in vivo transducing agents. Gene Ther 1999; 6(3): 454–9PubMedCrossRef

79.

Fisher KJ, Kelley WM, Burda JF, et al. A novel adenovirus-adeno-associated virus hybrid vector that displays efficient rescue and delivery of the AAV genome. Hum Gene Ther 1996; 7(17): 2079–87PubMedCrossRef

80.

Lohr M, Hoffmeyer A, Kroger J, et al. Microencapsulated cell-mediated treatment of inoperable pancreatic carcinoma. Lancet 2001; 357(9268): 1591–2PubMedCrossRef

81.

Bruder SP, Fink DJ, Caplan AI. Mesenchymal stem cells in bone development, bone repair, and skeletal regeneration therapy. J Cell Biochem 1994; 56(3): 283–94PubMedCrossRef

82.

Horwitz EM, Prockop DJ, Fitzpatrick LA, et al. Transplantabil-ity and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta. Nat Med 1999; 5(3): 309–13PubMedCrossRef

83.

Negishi Y, Kudo A, Obinata A, et al. Multipotency of a bone marrow stromal cell line, TBR31 -2, established from ts-S V40 T antigen gene transgenic mice. Biochem Biophys Res Com-mun 2000; 268(2): 450–5CrossRef

84.

Prockop DJ. Marrow stromal cells as stem cells for non-hematopoietic tissues. Science 1997; 276(5309): 71–4PubMedCrossRef

85.

Pittenger MF, Mackay AM, Beck SC, et al. Multilineage potential of adult human mesenchymal stem cells. Science 1999; 284(5411): 143–7PubMedCrossRef

86.

Pittenger MF, Mosca JD, McIntosh KR. Human mesenchymal stem cells: progenitor cells for cartilage, bone, fat and stroma. Curr Top Microbiol Immunol 2000; 251: 3–11PubMedCrossRef

87.

Toma C, Pittenger MF, Cahill KS, et al. Human mesenchymal stem cells differentiate to a cardiomyocyte pheotype in the adult murine heart. Circulation 2002; 105(1): 93–8PubMedCrossRef

88.

Fukuda K. Development of regenerative cardiomyocytes from mesenchymal stem cells for cardiovascular tissue engineering. Artif Organs 2001; 25(3): 187–93PubMedCrossRef

89.

Nishikawa M, Huang L. Nonviral vectors in the new millennium: delivery barriers in gene transfer. Hum Gene Ther 2001; 12(8): 861–70PubMedCrossRef

90.

Mittereder N, March KL, Trapnell BC. Evaluation of the concentration and bioactivity of adenovirus vectors for gene therapy. J Virol 1996; 70(11): 7498–509PubMed

91.

Mah C, Fraites TJ Jr, Zolotukhim I, et al. Improved method of recombinant AAV2 delivery for systemic targeted gene therapy. Mol Ther 2002; 6(1): 106–12PubMedCrossRef

92.

Weiss DJ, Strandjord TP, Liggitt D, et al. Perflubron enhances adenovirus-mediated gene expression in lungs of transgenic mice with chronic alveolar filling. Hum Gene Ther 1999; 10(14): 2287–93PubMedCrossRef

93.

Weiss DJ, Strandjord TP, Jackson JC, et al. Perfluorochemical liquid-enhanced adenoviral vector distribution and expression in lungs of spontaneously breathing rodents. Exp Lung Res 1999; 25(4): 317–33PubMedCrossRef

94.

Boekstegers P, von Degenfeld G, Giehrl W, et al. Myocardial gene transfer by selective pressure-regulated retroinfusion of coronary veins. Gene Ther 2000; 7(3): 232–40PubMedCrossRef

95.

Miyamoto MI, del Monte F, Schmidt U, et al. Adenoviral gene transfer of SERCA2a improves left-ventricular function in aortic-banded rats in transition to heart failure. Proc Natl Acad Sci U S A 2000; 97(2): 793–8PubMedCrossRef

96.

Svensson EC, Marshall DJ, Woodard K, et al. Efficient and stable transduction of cardiomyocytes after intramyocardial injection or intracoronary perfusion with recombinant adeno-associated virus vectors. Circulation 1999; 99(2): 201–5PubMedCrossRef

97.

Hajjar RJ, Schmidt U, Matsui T, et al. Modulation of ventricular function through gene transfer in vivo. Proc Natl Acad Sci USA 1998; 95(9): 5251–6PubMedCrossRef

98.

Lathi KG, Vale PR, Losordo DW, et al. Gene therapy with vascular endothelial growth factor for inoperable coronary artery disease: anesthetic management and results. Anesth Analg 2001; 92(1): 19–25PubMedCrossRef

Titel: Virus-Based Gene Delivery Systems
verfasst von: Cathryn Mah
Barry J. Byrne
Dr Terence R. Flotte
Publikationsdatum: 01.10.2002
Verlag: Springer International Publishing
Erschienen in: Clinical Pharmacokinetics / Ausgabe 12/2002
Print ISSN: 0312-5963
Elektronische ISSN: 1179-1926
DOI: https://doi.org/10.2165/00003088-200241120-00001

Springer Medizin

Abstract

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