A role for adeno-associated viral vectors in gene therapy

Genetics and Molecular Biology

Volumn 31, Issue 1, 2008, Pages 1-11

  dos Santos Coura, Renata ^a   Nardi, Nance Beyer ^a  

^a FEDERAL UNIVERSITY OF RIO GRANDE DO SUL   (Brazil)

Author keywords

AAV based recombinant vectors; Adeno associated virus; Gene therapy

Indexed keywords

ADENOVIRUS VECTOR; DRUG VEHICLE; LENTIVIRUS VECTOR; LIPOPLEX; NAKED DNA; PARVOVIRUS VECTOR; RETROVIRUS VECTOR;

CELL GROWTH; DNA MODIFICATION; ELECTROPORATION; ENDOSOME; GENE EXPRESSION; GENE OVEREXPRESSION; GENE TRANSFER; GENETIC DISORDER; GENETIC ENGINEERING; HUMAN; IMMUNOGENICITY; LONG TERM CARE; MICROINJECTION; NONVIRAL GENE DELIVERY SYSTEM; REVIEW; SAFETY; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS ISOLATION; VIRUS RECOMBINANT; VIRUS VIRULENCE;

ADENO-ASSOCIATED VIRUS;

EID: 44649172999     PISSN: 14154757     EISSN: 16784685     Source Type: Journal    
DOI: 10.1590/s1415-47572008000100001     Document Type: Review

References (102)

1. Arbetman AE, Lochrie M, Zhou S, Wellman J, Scallan C, Doroudchi MM, Radley B, Patarroyo-White S, Liu T, Smith P, et al. (2005) Novel caprine adeno-associated virus (AAV) capsid (AAVGo1) is closely related to the primate AAV-5 and has unique tropism and neutralization properties. J Virol 79:15238-15245.
2. Atchison RW, Casto BC and Hammon M (1965) Adenovirus-associated defective virus particles. Science 149:754-756.
3. Bantel-Schaal U and zur Hausen H (1984) Characterization of the DNA of a defective human parvovirus isolated from a genital site. Virology 134:52-63.
4. Bartlett JS, Wilcher R and Samulski RJ (2000) Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors. J Virol 74:2777-2785.
5. Berns KI, Pinkerton TC, Thomas GF and Hoggan MD (1975) Detection of adeno-associated virus (AAV)-specific nucleotide sequences in DNA isolated from latently infected Detroit 6 cells. Virology 68:556-560.
6. Berns K (1990) Parvovirus replication. Microbiol Rev 54:316-329.
7. Berns KI and Adler S (1972) Separation of two types of adeno-associated virus particles containing complementary polynucleotide chains. J Virol 9:394-396.
8. Berns KI and Linden RM (1995) The cryptic life style of adeno-associated virus. Bioessays 17:237-245.
9. Berns KI and Giraud C (1996) Biology of adeno-associated virus. Curr Top Microbiol Immunol 218:1-23.
10. Blacklow NR, Hoggan MD and Rowe WP (1968) Serologic evidence for human infection with adenovirus-associated viruses. J Natl Cancer Inst 40:319-327.
11. Blaese RM, Culver KC, Miller AD, Carter CS, Fleisher T, Sheare G, Chang L, Chiang Y, Tolstoshev P, Greenblatt JJ et al. (1995) T lymphocyte-directed gene therapy for ADASCID: Initial trial results after 4 years. Science 270:475-480.
12. Bleker S, Sonntag F and Kleinschmidt JA (2005) Mutational analysis of narrow pores at the fivefold symmetry axes of adeno-associated virus type 2 capsids reveals a dual role in genome packaging and activation of phospholipase A2 activity. J Virol 79:2528-2540.
13. Blenman KR, Duan B, Xu Z, Wan S, Atkinson MA, Flotte TR, Croker BP and Morel L (2006) IL-10 regulation of lupus in the NZM2410 murine model. Lab Invest 86:1136-1148.
14. Bossis I and Chiorini JA (2003) Cloning of an avian adeno-associated virus (AAV) and generation of recombinant AAV particles. J Virol 77:6799-6810.
15. Burger C, Gorbatyuk OS, Velardo MJ, Peden CS, Williams P, Zolotukhin S, Reier PJ, Mandel RJ and Muzyczka N (2004) Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system. Mot Ther 10:302-317.
16. Campbell EM and Hope TJ (2005) Gene therapy progress and prospects: Viral trafficking during infection. Gene Ther 12:1353-1359.
17. Chejanovsky N and Carter BJ (1989) Mutagenesis of an AUG codon in the adeno-associated virus rep gene: Effects on viral DNA replication. Virology 173:120-128.
18. Collaco RF, Kalman-Maltese V, Smith AD, Dignam JD and Trempe JP (2003) A biochemical characterization of the adeno-associated virus rep40 helicase. J Biol Chem 278:34011-34017.
19. Coura RD and Nardi NB (2007) The state of the art of adeno-associated virus-based vectors in gene therapy. Virol J 4:99.
20. Di Pasquale G, Davidson BL, Stein CS, Martins I, Scudiero D, Monks A and Chiorini JA (2003) Identification of PDGFR as a receptor for AAV-5 transduction. Nat Med 9:1306-1312.
21. Ding W, Zhang L, Yan Z and Engelhardt JF (2005) Intracellular trafficking of adeno-associated viral vectors. Gene Ther 12:873-880.
22. Duan D, Li Q, Kao AW, Yue Y, Pessin JE and Engelhardt JF (1999) Dynamin is required for recombinant adeno-associated virus type 2 infection. J Virol 73:10371-10376.
23. Duan D, Yue Y, Yan Z, Yang J and Engelhardt JF (2000) Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J Clin Invest 105:1573-1587.
24. Dutta SK (1975) Isolation and characterization of an adenovirus and isolation of its adenovirus-associated virus in cell culture from foals with respiratory tract disease. Am J Vet Res 36:247-250.
25. Edelstein ML, Abedi MR, Wixon J and Edelstein RM (2004) Gene therapy clinical trials worldwide 1989-2004-an overview. J Gene Med 6:597-602.
26. El-Aneed A (2004) Current strategies in cancer gene therapy. Eur J Pharmacol 498:1-8.
27. Erles K, Sebokova P and Schlehofer JR (1999) Update on the prevalence of serum antibodies (IgG and IgM) to adeno-associated virus (AAV). J Med Virol 59:406-411.
28. Farkas SL, Zadori Z, Benko M, Essbauer S, Harrach B and Tijssen P (2004) A parvovirus isolated from royal python (Python regius) is a member of the genus Dependovirus. J Gen Virol 85:555-561.
29. Gao G, Alvira MR, Wang L, Calcedo R, Johnston J and Wilson JM (2002) Novel adeno-associated viruses ftorn rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99:11854-11859.
30. Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, Zhou X and Wilson JM (2004) Clades of adeno-associated viruses are widely disseminated in human tissues. J Virol 78:6381-6388.
31. Gao G, Vandenberghe LH and Wilson JM (2005) New recombinant serotypes of AAV vectors. Curr Gene Ther 5:285-297.
32. Gao X, Kim K-S and Liu D (2007) Nonviral gene delivery: What we know and what is next. AAPS J 9:E92-E104.
33. Georg-Fries B, Biederlack S, Wolf J and zur Hausen H (1984) Analysis of proteins, helper dependence, and seroepidemiology of a new human parvovirus. Virology 134:64-71.
34. Girod A, Wobus CE, Zádori Z, Ried M, Leike K, Tijssen P, Kleinschmidt JA and Hallek M (2002) The VPI capsid protein of adeno-associated virus type 2 is carrying a phospholipase A2 domain required for virus infectivity. J Gen Virol 83:973-978.
35. Goverdhana S, Puntel M, Xiong W, Zirger JM, Barcia C, Curtin JF, Soffer EB, Mondkar S, King GD, Hu J et al. (2005) Regulatable gene expression systems for gene therapy applications: Progress and future challenges. Mot Ther 12:189-211.
36. Grimm D and Kay MA (2003) From virus evolution to vector revolution: Use of naturally-occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Curr Gene Ther 3:281-304.
37. Halbert CL, Rutledge EA, Allen JM, Russell DW and Miller AD (2000) Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes. J Virol 74:1524-1532.
38. Hansen J, Qing K and Srivastava A (2001) Adeno-associated virus type 2-mediated gene transfer: Altered endocytic processing enhances transduction efficiency in murine fibroblasts. J Virol 75:4080-4090.
39. Hermonat PL and Muzyczka N (1984) Use of adeno-associated virus as a mammalian DNA cloning vector: Transduction of neomycin resistance into mammalian tissue cultured cells. Proc Natl Acad Sci USA 81:6466-6470.
40. Hildinger M and Auricchio A (2004) Advances in AAV-mediated gene transfer for the treatment of inherited disorders. Eur J Hum Genet 12:263-271.
41. Hoggan MD, Blacklow NR and Rowe WP (1966) Studies of small DNA viruses found in various adenovirus preparations: Physical, biological and immunological characteristics. Proc Natl Acad Sci USA 55:1467-1472.
42. Ideno J, Mizukami H, Kakehashi A, Saito Y, Okada T, Urabe M, Kume A, Kuroki M, Kawakami M, Ishibashi S et al. (2007) Prevention of diabetic retinopathy by intraocular soluble flt-1 gene transfer in a spontaneously diabetic rat model. Int J Mol Med 19:75-79.,
43. Im DS and Muzyczka N (1989) Factors that bind to adeno-associated virus terminal repeats. J Virol 63:3095-3104.
44. Im DS and Muzyczka N (1990) The AAV origin binding protein Rep68 is an TP-dependent site-specific endonuclease with DNA helicase activity. Cell 61:447-457.
45. Jacobson ER, Kopit W, Kennedy FA and Funk RS (1996) Coinfection of a bearded dragon, Pogona vitticeps, with adenovirus- and dependovirus-like viruses. Vet Pathol 33:343-346.
46. Jazwa A, Jozkowicz A and Dulak J (2007) New vectors and strategies for cardiovascular gene therapy. Curr Gene Ther 7:7-23.
47. Kaludov N, Brown KE, Walters RW, Zabner J and Chiorini JA (2001) Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and efficient transduction but differ in sialic acid linkage specificity. J Virol 75:6884-6893.
48. Kashiwakura Y, Tamayose K, Iwabuchi K, Hirai Y, Shimada T, Matsumoto K, Nakamura T, Watanabe M, Oshimi K and Daida H (2005) Hepatocyte growth factor receptor is a coreceptor for adeno-associated virus type 2 infection. J Virol 79:609-614.
49. King JA, Dubielzig R, Grimm D and Kleinschmidt JA (2001) DNA helicasemediated packaging of adeno-associated virus type 2 genomes into preformed capsids. EMBO J 20:3282-3291.
50. Koczot FJ, Carter BJ, Garon CF and Rose JA (1973) Self-complementarity of terminal sequences within plus or minus strands of adenovirus-associated virus DNA. Proc Natl Acad Sci USA 70:215-219.
51. Kotin RM, Siniscalco M, Samulski RJ, Zhu XD, Hunter L, Laughlin CA, McLaughlin S, Muzyczka N, Rocchi M and Berns KI (1990) Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA 87:2211-2215.
52. Kotin RM, Menninger JC, Ward DC and Berns KI (1991) Mapping and direct visualization of a region-specific viral DNA integration site on chromosome 19q13-qter. Genomics 10:831-834.
53. Laughlin CA, Tratschin JD, Coon H and Carter BJ (1983) Cloning of infectious adeno-associated virus genomes in bacterial plasmids. Gene 23:65-73.
54. Linden RM, Ward P, Giraud C, Winocour E and Berns KI (1996) Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA 93:11288-11294.
55. Liu Y, Li D, Chen J, Xie J, Bandyopadhyay S, Zhang D, Nemarkommula AR, Liu H, Mehta JL and Hermonat PL (2006) Inhibition of atherogenesis in LDLR knockout mice by systemic delivery of adeno-associated virus type 2-hIL-10. Atherosclerosis 188:19-27.
56. Martin KR, Klein RL and Quigley HA (2002) Gene delivery to the eye using adeno-associated viral vectors. Methods 28:267-275.
57. McCarty DM, Young Jr SM and Samulski RJ (2004) Integration of adeno-associatcd virus (AAV) and recombinant AAV vectors. Annu Rev Genet 38:819-845.
58. Mori S, Wang L, Takeuchi T and Kanda T (2004) Two novel adeno-associated viruses from cynomolgus monkey: Pseudotyping characterization of capsid protein. Virology 330:375-383.
59. Nakai H, Montini E, Fuess S, Storm TA, Grompe M and Kay MA (2003) AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet 34:297-302.
60. Ng SS, Gao Y, Chau DH, Li GH, Lai LH, Huang PT, Huang CF. Huang JJ, Chen YC, Kung HF et al. (2007) A novel glioblastoma cancer gene therapy using AAV-mediated long-term expression of human TERT C-terminal polypeptide. Cancer Gene Ther 14:561-572.
61. Ni T-H, McDonald WF, Zolotukhin I, Melendy T, Waga S, Stillman B and Muzyczka N (1998) Cellular proteins required for adeno-associated virus DNA replication in the absence of adenovirus coinfection. J Virol 72:2777-2787.
62. Olson EJ, Haskell SR, Frank RK, Lehmkuhl HD, Hobbs LA, Warg JV, Landgraf JG and Wunschmann A (2004) Isolation of an adenovirus and an adeno-associated virus from goat kids with enteritis. J Vet Diagn Invest 16:461-464.
63. Parks WP, Green M, Pina M and Melnick IL (1967) Physicochemical characterization of adeno-associated satellite virus type 4 and its nucleic acid. J Virol 5:980-987.
64. Parks WP, Boucger DW, Melnich JL, Taber LH and Yow MD (1970) Seroepidemiological and ecological studies of the adeno-associated satellite viruses. Infect Immun 2:716-722.
65. Paterna JC, Leng A, Weber E, Feldon J and Büeler H (2007) DJ-1 and Parkin modulate dopamine-dependent behavior and inhibit MPTP-induced nigral dopamine neuron loss in mice. Mol Ther 15:698-704.
66. Peden CS, Burger C, Muzyczka N and Mandel RJ (2004) Circulating anti-wildtype adeno-associated virus type 2 (AAV2) antibodies inhibit recombinant AAV2 (rAAV2)-mediated, but not rAAV5-mediated, gene transfer in the brain. J Virol 78:6344-6359.
67. Pereira DJ, McCarty DM and Muzyczka N (1997) The adeno-associated virus (AAV) Rep protein acts as both a repressor and an activator to regulate AAV transcription during a productive infection. J Virol 71:1079-1088.
68. Pfeifer A and Verma IM (2001) Gene therapy: Promises and problems. Annu Rev Genomics Hum Genet 2:177-211.
69. Philpott NJ, Gomos J and Falck-Pedersen E (2004) Transgene expression after rep-mediated site-specific integration into chromosome 19. Hum Gene Ther 15:47-61.
70. Qing K, Mah C, Hansen J, Zhou S, Dwarki V and Srivastava A (1999) Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nat Med 5:71-77.
71. Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, Karson EM, Lotze MT, Yang JC and Topalian SL (1990) Gene transfer into humans - Immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 323:570-578.
72. Rubanyi GM (2001) The future of human gene therapy. Mol Aspects Med 22:113-142.
73. Russell DW (2003) AAV loves an active genome. Nat Genet 34:241-242.
74. Rutledge EA, Halbert CL and Russell DW (1998) Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J Virol 72:309-319.
75. Salani B, Damonte P, Zingone A, Barbieri O, Chou JY, D'Costa J, Arya SK, Eva A and Varesio L (2005) Newborn liver gene transfer by an HIV-2-based lentiviral vector. Gene Ther 12:803-814.
76. Samulski RJ, Berns KI, Tan M and Muzyczka N (1982) Cloning of infectious adeno-associated virus into pBR322: Rescue of intact virus from the recombinant plasmid in human cells. Proc Natl Acad Sci USA 79:2077-2081.
77. Samulski RJ, Zhu X, Xiao X, Brook JD, Housman DE, Epstein N and Hunter LA (1991) Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J 10:3941-3950.
78. Sandalon Z, Bruckheimer EM, Lustig KH and Burstein H (2007) Long-term suppression of experimental arthritis following intramuscular administration of a pseudotyped AAV2/1-TNFR:Fc vector. Mol Ther 15:264-269.
79. Schmidt M, Katano H, Bossis I and Chiorini JA (2004) Cloning and characterization of a bovine adeno-associated virus. J Virol 78:6509-6516.
80. Schmidt M, Grot E, Cervenka P, Wainer S, Buck C and Chiorini JA (2006) Identification and characterization of novel adeno-associated virus isolates in ATCC virus stocks. J Virol 80:5082-5085.
81. Seisenberger G, Ried MU, Endress T, Buning H, Hallek M and Brauchle C (2001) Real-time single-molecule imaging of the infection pathway of an adenoassociated virus. Science 294:1929-1932.
82. Shenouda SM, Johns C, Kintsurashvili E, Gavras I and Gavras H (2006) Long-term inhibition of the central alpha(2B)-adrenergic receptor gene via recombinant AAV-delivered anti-sense in hypertensive rats. Am J Hypertens 19:1135-1143.
83. Smith KR (2003) Gene therapy: Theoretical and bioethical concepts. Arch Med Res 34:247-268.
84. Smith RH and Kotin RM (1998) The Rep52 gene product of adeno-associated virus is a DNA helicase with 3′-to-5′ polarity. J Virol 72:4874-4881.
85. Smith JR, Verwaerde C, Rolling F, Naud MC, Delanoye A, Thillaye-Goldenberg B, Apparailly F and De Kozak Y (2005) Tetracycline-inducible viral interleukin-10 intraocular gene transfer, using adeno-associated virus in experimental autoimmune uveoretinitis. Hum Gene Ther 16:1037-1046.
86. Stender S, Murphy M, O'Brien T, Stengaard C, Ulrich-Vinther M, Soballe K and Barry F (2007) Adeno-associated viral vector transduction of human mesenchymal stem cells. Eur Cell Mater 13:93-99.
87. Summerford C and Samulski RJ (1998) Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 72:1438-1445.
88. Summerford C, Bartlett JS and Samulski RJ (1999) AlphaVbeta5 integrin: A coreceptors for adeno-associated virus type 2 infection. Nat Med 5:78-82.
89. Tat J (2000) Adeno-associated virus-based vectors in gene therapy. J Biomed Sci 7:279-291.
90. Verma IM and Somia N (1997) Gene therapy - Promises, problems and prospects. Nature 389:239-242.
91. Vincent KA, Piraino ST and Wadsworth SC (1997) Analysis of recombinant adeno-associated virus packaging and requirements for rep and cap gene products. J Virol 71:1897-1905.
92. Wang H and Lieber A (2006) A helper-dependent capsid-modified adenovirus vector expressing adeno-associated virus rep78 mediates site-specific integration of a 27-kilobase transgene cassette. J Virol 80:11699-11709.
93. Wiley (2007) Gene Therapy Clinical Trials Worldwide. http://www.wiley.co.uk/genetherapy/clinical/ (June, 2007).
94. Wistuba A, Kern A, Weger S, Grimm D and Kleinschmidt J (1997) Subcellular compartmentalization of adeno-associated virus type 2 assembly. J Virol 71:1341-1352.
95. Worgall S (2005) A realistic chance for gene therapy in the near future. Pediatr Nephrol 20:118-124.
96. Wu Z, Asokan A and Samulski RJ (2006) Adeno-associated virus serotypes: Vector toolkit for human gene therapy. Mol Ther 14:316-327.
97. Xiao X, Li J and Samulski RJ (1998) Production of high-titer recombinant adenoassociated virus vectors in the absence of helper adenovirus. J Virol 72:2224-2232.
98. Xiao W, Chirmule N, Berta SC, McCullough B, Gao G and Wilson JM (1999), Gene therapy vectors based on adenoassociated virus type 1. J Virol 73:3994-4003.
99. Xiao W, Warrington Jr KH, Hearing P, Hughes J and Muzyczka N (2002) Adenovirus-facilitated nuclear translocation of adeno-associated virus type 2. J Virol 76:11505-11517.
100. Zádori Z, Szelei J, Lacoste M-C, Li Y, Gariépy S, Raymond P, Allaire M, Nabi IR and Tijssen P (2001) A viral phospholipase A2 is required for parvovirus infectivity. Dev Cell 1:291-302.
101. Zolotukhin S, Potter M, Zolotukhin I, Sakai Y, Loiler S, Fraites TJ Jr, Chiodo VA, Phillipsberg T, Muzyczka N, Hauswirth WW et al. (2002) Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods 28:158-67.
102. Zubler RH (2006) Ex vivo expansion of haematopoietic stem cells and gene therapy development. Swiss Med Wkly 136:795-799.