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Volumn 3, Issue 5, 2008, Pages 491-503

Safety of adeno-associated viral vectors

Author keywords

Adeno assoclated viral vectors; Adeno assoclated virus; Clinical trials; Gene therapy; Vaccine carriers

Indexed keywords

BETA GLUCURONIDASE; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; CYCLOSPORIN; DOUBLE STRANDED DNA; DYSTROPHIN; ERYTHROPOIETIN; ETANERCEPT; EYE PROTEIN; GAG PROTEIN; GENE PRODUCT; GLUTAMATE DECARBOXYLASE; HUMAN IMMUNODEFICIENCY VIRUS VACCINE; MEROZOITE SURFACE PROTEIN 4; MEROZOITE SURFACE PROTEIN 5; MICRORNA; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PARASITE ANTIGEN; PROTEIN RPE 65; REP78 PROTEIN; SIMIAN IMMUNODEFICIENCY VIRUS VACCINE; TRANSCRIPTION FACTOR FOXP3; UNCLASSIFIED DRUG; VIRUS VACCINE;

EID: 55849127475     PISSN: 17460794     EISSN: None     Source Type: Journal    
DOI: 10.2217/17460794.3.5.491     Document Type: Review
Times cited : (2)

References (135)
  • 1
    • 48349133827 scopus 로고    scopus 로고
    • Systemic insulin-like growth factor-1 reverses hypoalgesia and improves mobility in a mouse model of diabetic peripheral neuropathy
    • Chu Q, Moreland R, Yew NS, Foley J, Ziegler R, Scheule RK: Systemic insulin-like growth factor-1 reverses hypoalgesia and improves mobility in a mouse model of diabetic peripheral neuropathy. Mol. Ther. (2008).
    • (2008) Mol. Ther
    • Chu, Q.1    Moreland, R.2    Yew, N.S.3    Foley, J.4    Ziegler, R.5    Scheule, R.K.6
  • 2
    • 46249125272 scopus 로고    scopus 로고
    • Complete correction of hemophilia A with adeno-associated viral vectors containing a full-size expression cassette
    • Lu H, Chen L, Wang J et al.: Complete correction of hemophilia A with adeno-associated viral vectors containing a full-size expression cassette. Hum. Gene Ther. 19(6), 648-654 (2008).
    • (2008) Hum. Gene Ther , vol.19 , Issue.6 , pp. 648-654
    • Lu, H.1    Chen, L.2    Wang, J.3
  • 3
    • 36749033228 scopus 로고    scopus 로고
    • The state of the art of adeno-associated virus-based vectors in gene therapy
    • Coura Rdos S, Nardi NB: The state of the art of adeno-associated virus-based vectors in gene therapy. Virol. 14, 99 (2007).
    • (2007) Virol , vol.14 , pp. 99
    • Coura Rdos, S.1    Nardi, N.B.2
  • 4
    • 0842287787 scopus 로고    scopus 로고
    • Can genes transduced by adeno-associated virus vectors elicit or evade an immune response?
    • Wang CH, Liu DW, Tsao YP, Xiao X, Chen SL: Can genes transduced by adeno-associated virus vectors elicit or evade an immune response? Arch. Virol. 149, 1-15 (2004).
    • (2004) Arch. Virol , vol.149 , pp. 1-15
    • Wang, C.H.1    Liu, D.W.2    Tsao, Y.P.3    Xiao, X.4    Chen, S.L.5
  • 5
    • 21244441929 scopus 로고    scopus 로고
    • Immune responses to adeno-associated virus vectors
    • Zaiss AK, Muruve DA: Immune responses to adeno-associated virus vectors. Curr. Gene Ther. 5, 323-331 (2005).
    • (2005) Curr. Gene Ther , vol.5 , pp. 323-331
    • Zaiss, A.K.1    Muruve, D.A.2
  • 6
    • 0030819372 scopus 로고    scopus 로고
    • Genetic immunization with adeno-associated virus vectors expressing herpes simplex virus type 2 glycoproteins B and D
    • Manning WC, Paliard X, Zhou S et al.: Genetic immunization with adeno-associated virus vectors expressing herpes simplex virus type 2 glycoproteins B and D. J. Virol. 71, 7960-7962 (1997).
    • (1997) J. Virol , vol.71 , pp. 7960-7962
    • Manning, W.C.1    Paliard, X.2    Zhou, S.3
  • 7
    • 0034921072 scopus 로고    scopus 로고
    • A novel recombinant adeno-associated virus vaccine induces a long-term humoral immune response to human immunodeficiency virus
    • Xin KQ, Urabe M, Yang J et al.: A novel recombinant adeno-associated virus vaccine induces a long-term humoral immune response to human immunodeficiency virus. Hum. Gene Ther. 12, 1047-1061 (2001).
    • (2001) Hum. Gene Ther , vol.12 , pp. 1047-1061
    • Xin, K.Q.1    Urabe, M.2    Yang, J.3
  • 8
    • 33748142971 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus expressing the receptor-binding domain of severe acute respiratory syndrome coronavirus S protein elicits neutralizing antibodies: Implication for developing SARS vaccines
    • Du L, He Y, Wang Y et al.: Recombinant adeno-associated virus expressing the receptor-binding domain of severe acute respiratory syndrome coronavirus S protein elicits neutralizing antibodies: implication for developing SARS vaccines. Virology 353, 6-16 (2006).
    • (2006) Virology , vol.353 , pp. 6-16
    • Du, L.1    He, Y.2    Wang, Y.3
  • 9
    • 42649111674 scopus 로고    scopus 로고
    • Activation of cytotoxic T lymphocytes against CML28-bearing tumors by dendritic cells transduced with a recombinant adeno-associated virus encoding the CML28 gene
    • Xie LH, Sin FW, Cheng SC et al.: Activation of cytotoxic T lymphocytes against CML28-bearing tumors by dendritic cells transduced with a recombinant adeno-associated virus encoding the CML28 gene. Cancer Immunol. Immunother. 57(7), 1029-1038 (2008).
    • (2008) Cancer Immunol. Immunother , vol.57 , Issue.7 , pp. 1029-1038
    • Xie, L.H.1    Sin, F.W.2    Cheng, S.C.3
  • 10
    • 30044436425 scopus 로고    scopus 로고
    • Multiple human papillomavirus genes affect the adeno-associated virus life cycle
    • You H, Liu Y, Prasad CK et al.: Multiple human papillomavirus genes affect the adeno-associated virus life cycle. Virology 344, 532-540 (2006).
    • (2006) Virology , vol.344 , pp. 532-540
    • You, H.1    Liu, Y.2    Prasad, C.K.3
  • 11
    • 0014082647 scopus 로고
    • Studies on the relationship between adeno-associated virus type-1 (AAV-1) and adenoviruses: I. Replication of AAV-1 in certain cell cultures and its effect on helper adenovirus
    • Casto BC, Atchison RW, Hammon WM: Studies on the relationship between adeno-associated virus type-1 (AAV-1) and adenoviruses: I. Replication of AAV-1 in certain cell cultures and its effect on helper adenovirus. Virology 32, 52-59 (1967).
    • (1967) Virology , vol.32 , pp. 52-59
    • Casto, B.C.1    Atchison, R.W.2    Hammon, W.M.3
  • 12
    • 0019802153 scopus 로고
    • Herpes simplex virus types 1 and 2 completely help adenovirus-associated virus replication
    • Buller RML, Janik JE, Sebring ED, Rose JA: Herpes simplex virus types 1 and 2 completely help adenovirus-associated virus replication. J. Virol. 40, 241-247 (1981).
    • (1981) J. Virol , vol.40 , pp. 241-247
    • Buller, R.M.L.1    Janik, J.E.2    Sebring, E.D.3    Rose, J.A.4
  • 13
    • 0014247830 scopus 로고
    • Serologic evidence for human infection with adenovirus-associated viruses
    • Blacklow NR, Hoggan MD, Rowe WP: Serologic evidence for human infection with adenovirus-associated viruses. J. Natl Cancer Inst. 40, 319-327 (1968).
    • (1968) J. Natl Cancer Inst , vol.40 , pp. 319-327
    • Blacklow, N.R.1    Hoggan, M.D.2    Rowe, W.P.3
  • 15
    • 22144431595 scopus 로고    scopus 로고
    • Adeno-associated virus: From defective virus to effective vector
    • Goncalves MA: Adeno-associated virus: from defective virus to effective vector. Virol. J. 2, 43 (2005).
    • (2005) Virol. J , vol.2 , pp. 43
    • Goncalves, M.A.1
  • 16
    • 0035229222 scopus 로고    scopus 로고
    • Gene therapy: Recombinant adeno-associated virus vectors
    • Smith-Arica JR, Bartlett JS: Gene therapy: recombinant adeno-associated virus vectors. Curr. Cardiol. Rep. 3, 43-49 (2001).
    • (2001) Curr. Cardiol. Rep , vol.3 , pp. 43-49
    • Smith-Arica, J.R.1    Bartlett, J.S.2
  • 17
    • 0031901455 scopus 로고    scopus 로고
    • Cellular proteins required for adeno-associated virus DNA replication in the absence of adenovirus coinfection
    • Ni T-H, McDonald WF, Zolotukhin I et al.: Cellular proteins required for adeno-associated virus DNA replication in the absence of adenovirus coinfection. J. Virol. 72, 2777-2787 (1998).
    • (1998) J. Virol , vol.72 , pp. 2777-2787
    • Ni, T.-H.1    McDonald, W.F.2    Zolotukhin, I.3
  • 18
    • 0036111820 scopus 로고    scopus 로고
    • Repl Cap gene amplification and high-yield production of AAV in an A549 cell line expressing
    • Gao GP, Lu F, Sanmiguel JC et al.: Repl Cap gene amplification and high-yield production of AAV in an A549 cell line expressing Repl Cap. Mol. Ther. 5, 644-649 (2002).
    • (2002) Repl Cap. Mol. Ther , vol.5 , pp. 644-649
    • Gao, G.P.1    Lu, F.2    Sanmiguel, J.C.3
  • 19
    • 0034295382 scopus 로고    scopus 로고
    • Selective Rep-Cap gene amplification as a mechanism for high-titer recombinant AAV production from stable cell lines
    • Liu X, Voulgaropoulou F, Chen R, Johnson PR, Clark KR: Selective Rep-Cap gene amplification as a mechanism for high-titer recombinant AAV production from stable cell lines. Mol. Ther. 2, 394-403 (2000).
    • (2000) Mol. Ther , vol.2 , pp. 394-403
    • Liu, X.1    Voulgaropoulou, F.2    Chen, R.3    Johnson, P.R.4    Clark, K.R.5
  • 20
    • 0033643006 scopus 로고    scopus 로고
    • Molecular biology of adeno-associated viruses
    • Linden RM, Berns KI: Molecular biology of adeno-associated viruses. Contrib. Microbiol. 4, 68-84 (2000).
    • (2000) Contrib. Microbiol , vol.4 , pp. 68-84
    • Linden, R.M.1    Berns, K.I.2
  • 22
    • 55849146568 scopus 로고
    • Pattison JR Ed, Clinical Research Collaboration, Boca Raton, FL, USA
    • Blackdow NR: Paroviruses and Human Disease. Pattison JR (Ed.). Clinical Research Collaboration, Boca Raton, FL, USA, 117-132 (1988).
    • (1988) Paroviruses and Human Disease , pp. 117-132
    • Blackdow, N.R.1
  • 23
    • 0031691257 scopus 로고    scopus 로고
    • Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue
    • Duan D, Sharma P, Yang J et al..: 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 (1998).
    • (1998) J. Virol , vol.72 , pp. 8568-8577
    • Duan, D.1    Sharma, P.2    Yang, J.3
  • 24
    • 0028307303 scopus 로고
    • Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA
    • Weitzman MD, Kyostio SR, Kotin RM, Owens RA: Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA. Proc. Natl Acad. Sci. USA 91, 5808-5812 (1994).
    • (1994) Proc. Natl Acad. Sci. USA , vol.91 , pp. 5808-5812
    • Weitzman, M.D.1    Kyostio, S.R.2    Kotin, R.M.3    Owens, R.A.4
  • 25
    • 0342313708 scopus 로고    scopus 로고
    • Roles of adeno-associated virus Rep protein and human chromosome 19 in site-specific recombinatton
    • Young SM Jr, McCarty DM, Degtyareva N, Samulski RJ: Roles of adeno-associated virus Rep protein and human chromosome 19 in site-specific recombinatton. J. Virol. 74, 3953-3966 (2000).
    • (2000) J. Virol , vol.74 , pp. 3953-3966
    • Young Jr, S.M.1    McCarty, D.M.2    Degtyareva, N.3    Samulski, R.J.4
  • 26
    • 0031041176 scopus 로고    scopus 로고
    • Binding sites for adeno-associated virus Rep proteins within the human genome
    • Wonderling RS, Owens RA: Binding sites for adeno-associated virus Rep proteins within the human genome. J. Virol. 71, 2528-2534 (1997).
    • (1997) J. Virol , vol.71 , pp. 2528-2534
    • Wonderling, R.S.1    Owens, R.A.2
  • 27
    • 35348849523 scopus 로고    scopus 로고
    • Preferential integration of adeno-associated virus type 2 into a polypyrimidine/polypurine-rich region within AAVS1
    • McAlister VJ, Owens RA: Preferential integration of adeno-associated virus type 2 into a polypyrimidine/polypurine-rich region within AAVS1. J. Virol. 81. 9718-9726 (2007).
    • (2007) J. Virol , vol.81 , pp. 9718-9726
    • McAlister, V.J.1    Owens, R.A.2
  • 28
    • 0033861721 scopus 로고    scopus 로고
    • Characteristics of the adeno-associated virus preintegration site in human chromosome 19: Open chromatin conformation and transcription-competent environment
    • Lamartina S, Sporeno E, Fattori E, Toniatti C: Characteristics of the adeno-associated virus preintegration site in human chromosome 19: open chromatin conformation and transcription-competent environment. J. Virol. 74, 7671-7677 (2000).
    • (2000) J. Virol , vol.74 , pp. 7671-7677
    • Lamartina, S.1    Sporeno, E.2    Fattori, E.3    Toniatti, C.4
  • 29
    • 0344406980 scopus 로고    scopus 로고
    • Packaging of human chromosome 19-specific adeno-associated virus (MV) integration sites in AAV virions during AAV wild-type and recombinant AAV vector production
    • Huser D, Weger S, Heilbronn R: Packaging of human chromosome 19-specific adeno-associated virus (MV) integration sites in AAV virions during AAV wild-type and recombinant AAV vector production. J. Virol. 77, 4881-4887 (2003).
    • (2003) J. Virol , vol.77 , pp. 4881-4887
    • Huser, D.1    Weger, S.2    Heilbronn, R.3
  • 30
    • 34547610960 scopus 로고    scopus 로고
    • AAV vector integration sites in mouse hepatocellular carcinoma
    • Donsante A, Miller DG, Li Y et al.: AAV vector integration sites in mouse hepatocellular carcinoma. Science 317, 477 (2007).
    • (2007) Science , vol.317 , pp. 477
    • Donsante, A.1    Miller, D.G.2    Li, Y.3
  • 31
    • 0034830365 scopus 로고    scopus 로고
    • Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors
    • Donsante A, Vogler C, Muzyczka N et al.: Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. Gene Ther. 8, 1343-1346 (2001).
    • (2001) Gene Ther , vol.8 , pp. 1343-1346
    • Donsante, A.1    Vogler, C.2    Muzyczka, N.3
  • 32
    • 0038054338 scopus 로고    scopus 로고
    • AAV serotype 2 vectors preferentially integrate into active genes in mice
    • Nakai H, Montini E, Fuess S, Storm TA, Grompe M, Kay MA: AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat. Genet. 34, 297-302 (2003).
    • (2003) Nat. Genet , vol.34 , pp. 297-302
    • Nakai, H.1    Montini, E.2    Fuess, S.3    Storm, T.A.4    Grompe, M.5    Kay, M.A.6
  • 34
    • 0029257496 scopus 로고
    • The cryptic life style of adenoassociated virus
    • Kenneth I, Berns RML: The cryptic life style of adenoassociated virus. BioEssays 17, 237-245 (1995).
    • (1995) BioEssays , vol.17 , pp. 237-245
    • Kenneth, I.1    Berns, R.M.L.2
  • 37
    • 2642512201 scopus 로고    scopus 로고
    • Glades of adeno-associated viruses are widely disseminated in human tissues
    • Gao G, Vandenberghe LH, Alvira MR et al.: Glades of adeno-associated viruses are widely disseminated in human tissues. J. Virol. 78, 6381-6388 (2004).
    • (2004) J. Virol , vol.78 , pp. 6381-6388
    • Gao, G.1    Vandenberghe, L.H.2    Alvira, M.R.3
  • 38
    • 0010390169 scopus 로고
    • Cloning of adeno-associated virus into pBR322: Rescue of intact virus from the recombinant plasmid in human calls
    • Samulski RJ, Berns KI, Tan M, Muzyczka N: Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human calls. Proc. Natl Acad. Sci. USA 79, 2077-2081 (1982).
    • (1982) Proc. Natl Acad. Sci. USA , vol.79 , pp. 2077-2081
    • Samulski, R.J.1    Berns, K.I.2    Tan, M.3    Muzyczka, N.4
  • 39
    • 0021690155 scopus 로고
    • Use of adeno-associated virus as a mammalian DNA cloning vector: Transduction of neomycin resistance into mammalian tissue culture cells
    • 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. USA 81, 6466-6470 (1984).
    • (1984) Proc. Natl Acad. Sci. USA , vol.81 , pp. 6466-6470
    • Hermonat, P.L.1    Muzyczka, N.2
  • 40
    • 0032589751 scopus 로고    scopus 로고
    • αVβ5 integrin: A co-receptor for adeno-associated virus type 2 infection
    • Summerford C, Bartlett JS, Samuiski RJ: αVβ5 integrin: a co-receptor for adeno-associated virus type 2 infection. Nat. Med. 5, 78-82 (1999).
    • (1999) Nat. Med , vol.5 , pp. 78-82
    • Summerford, C.1    Bartlett, J.S.2    Samuiski, R.J.3
  • 41
    • 33748494015 scopus 로고    scopus 로고
    • Adeno-associated virus type 2 contains an integrin α5β1 binding domain essential for viral cell entry
    • Asokan A, Hamra JB, Govindasamy L, Agbandje-McKenna M, Samulski RJ: Adeno-associated virus type 2 contains an integrin α5β1 binding domain essential for viral cell entry. J. Virol. 80, 8961-8969 (2006).
    • (2006) J. Virol , vol.80 , pp. 8961-8969
    • Asokan, A.1    Hamra, J.B.2    Govindasamy, L.3    Agbandje-McKenna, M.4    Samulski, R.J.5
  • 42
    • 33748945422 scopus 로고    scopus 로고
    • Akache B, Grimm D, Pandey K, Yant SR, Xu H, Kay MA: The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. J. Virol. 80, 9831-9836 (2006).
    • Akache B, Grimm D, Pandey K, Yant SR, Xu H, Kay MA: The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. J. Virol. 80, 9831-9836 (2006).
  • 43
    • 33847793666 scopus 로고    scopus 로고
    • CD9 promotes adeno-associated virus type 2 infection of mammary carcinoma cells with low cell surface expression of heparan sulphate proteoglycans
    • Kurzeder C, Koppold B, Sauer G, Pabst S, Kreienberg R, Deissler H: CD9 promotes adeno-associated virus type 2 infection of mammary carcinoma cells with low cell surface expression of heparan sulphate proteoglycans. Int. J. Mol. Med. 19, 325-333 (2007).
    • (2007) Int. J. Mol. Med , vol.19 , pp. 325-333
    • Kurzeder, C.1    Koppold, B.2    Sauer, G.3    Pabst, S.4    Kreienberg, R.5    Deissler, H.6
  • 44
    • 45749138773 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus transduction and integration
    • Schultz BR, Chamberlain JS: Recombinant adeno-associated virus transduction and integration. Mol. Ther. 16(7), 1189-1199 (2008).
    • (2008) Mol. Ther , vol.16 , Issue.7 , pp. 1189-1199
    • Schultz, B.R.1    Chamberlain, J.S.2
  • 45
    • 55849115907 scopus 로고    scopus 로고
    • Characterization of a murine adeno-associated virus (AAV-mo. 1) capsid
    • Lochrie M, Tatsuno G, Arbetman A et al.: Characterization of a murine adeno-associated virus (AAV-mo. 1) capsid. Mol. Ther.: 11, S52-S53 (2005).
    • (2005) Mol. Ther , vol.11
    • Lochrie, M.1    Tatsuno, G.2    Arbetman, A.3
  • 46
    • 33750722840 scopus 로고    scopus 로고
    • Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes
    • Wu Z, Asokan A, Grieger JC, Govindasamy L, Agbandje-McKenna M, Samulski RJ: Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J. Virol. 80. 11393-11397 (2006).
    • (2006) J. Virol , vol.80 , pp. 11393-11397
    • Wu, Z.1    Asokan, A.2    Grieger, J.C.3    Govindasamy, L.4    Agbandje-McKenna, M.5    Samulski, R.J.6
  • 47
    • 0033010884 scopus 로고    scopus 로고
    • Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2
    • Qing K, Mah C, Hansen J, Zhou S, Dwarki V, Srivastava A: Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nat. Med. 5, 71-77 (1999).
    • (1999) Nat. Med , vol.5 , pp. 71-77
    • Qing, K.1    Mah, C.2    Hansen, J.3    Zhou, S.4    Dwarki, V.5    Srivastava, A.6
  • 48
    • 0035138205 scopus 로고    scopus 로고
    • Intracellular trafficking of adeno-associated virus vectors: Routing to the late endosomal compartment and proteasome degradation
    • Douar AM, Poulard K, Stockholm D, Danos O: Intracellular trafficking of adeno-associated virus vectors: routing to the late endosomal compartment and proteasome degradation. J. Virol. 75, 1824-1833 (2001).
    • (2001) J. Virol , vol.75 , pp. 1824-1833
    • Douar, A.M.1    Poulard, K.2    Stockholm, D.3    Danos, O.4
  • 49
    • 0033799367 scopus 로고    scopus 로고
    • Sanlioglu S, Benson PK, Yang J, Atkinson EM, Reynolds T. Engelhardt JF: Endocytosis and nuclear trafficking of adeno-associated virus type 2 are controlled by rac1 and phosphatidylinositol-3 kinase activation. J. Virol. 74, 9184-9196 (2000).
    • Sanlioglu S, Benson PK, Yang J, Atkinson EM, Reynolds T. Engelhardt JF: Endocytosis and nuclear trafficking of adeno-associated virus type 2 are controlled by rac1 and phosphatidylinositol-3 kinase activation. J. Virol. 74, 9184-9196 (2000).
  • 50
    • 0033998692 scopus 로고    scopus 로고
    • Infectious entry pathway of adeno-associated virus and, adeno-asaociated virus vectors
    • Bartlett JS, Wilcher R, Samulski RJ: Infectious entry pathway of adeno-associated virus and, adeno-asaociated virus vectors. J. Virol. 74, 2777-2785 (2000).
    • (2000) J. Virol , vol.74 , pp. 2777-2785
    • Bartlett, J.S.1    Wilcher, R.2    Samulski, R.J.3
  • 52
    • 17544383607 scopus 로고    scopus 로고
    • Gene transfer into skeletal muscle using novel AAV serotypes
    • Louboutin JP, Wang L, Wilson JM: Gene transfer into skeletal muscle using novel AAV serotypes. J. Gene Med. 7, 442-451 (2005).
    • (2005) J. Gene Med , vol.7 , pp. 442-451
    • Louboutin, J.P.1    Wang, L.2    Wilson, J.M.3
  • 53
    • 34247271439 scopus 로고    scopus 로고
    • Taymans JM, Vandenberghe LH, Haute CV et al.: Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. Hum. Gene Ther. 18, 195-206 (2007).
    • Taymans JM, Vandenberghe LH, Haute CV et al.: Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. Hum. Gene Ther. 18, 195-206 (2007).
  • 54
    • 33749365644 scopus 로고    scopus 로고
    • Adeno-associated virus (AAV) serotypes 2, 4 and 5 display similar transduction profiles and penetrate solid tumor tissue in models of human glioma
    • Thorsen F, Afione S, Huszthy PC et al.: Adeno-associated virus (AAV) serotypes 2, 4 and 5 display similar transduction profiles and penetrate solid tumor tissue in models of human glioma. J. Gene Med. 8, 1131-1140 (2006).
    • (2006) J. Gene Med , vol.8 , pp. 1131-1140
    • Thorsen, F.1    Afione, S.2    Huszthy, P.C.3
  • 55
    • 32944459983 scopus 로고    scopus 로고
    • Cearley CN, Wolfe JH: Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol. Ther. 13. 528-537 (2006).
    • Cearley CN, Wolfe JH: Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol. Ther. 13. 528-537 (2006).
  • 56
    • 0037344731 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus: Formulation challenges and strategies for a gene therapy vector
    • Wright JF, Qu G, Tang C, Sommer JM: Recombinant adeno-associated virus: formulation challenges and strategies for a gene therapy vector. Curr Opin. Drug Discov. Devel. 6, 174-178 (2003).
    • (2003) Curr Opin. Drug Discov. Devel , vol.6 , pp. 174-178
    • Wright, J.F.1    Qu, G.2    Tang, C.3    Sommer, J.M.4
  • 57
    • 33751255418 scopus 로고    scopus 로고
    • A helper-dependent capsid-modified adenovirus vector expressing adeno-associated virus rep78 mediates site-specific integration of a 27-kiobase transgene cassette
    • Wang H, Lieber A: A helper-dependent capsid-modified adenovirus vector expressing adeno-associated virus rep78 mediates site-specific integration of a 27-kiobase transgene cassette. J. Virol. 80, 11699-11709 (2006).
    • (2006) J. Virol , vol.80 , pp. 11699-11709
    • Wang, H.1    Lieber, A.2
  • 58
    • 21244466776 scopus 로고    scopus 로고
    • Helper functions required for wild type and recombinant adeno-associated virus growth
    • Geoffroy MC, Salvetti A: Helper functions required for wild type and recombinant adeno-associated virus growth. Curr. Gene Ther. 5, 265-271 (2005).
    • (2005) Curr. Gene Ther , vol.5 , pp. 265-271
    • Geoffroy, M.C.1    Salvetti, A.2
  • 59
    • 0346777307 scopus 로고    scopus 로고
    • Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
    • McCarty DM, Fu H, Monahan PE, Toulson CE, Nalk P, Samulski RJ: Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther. 10, 2112-2118 (2003).
    • (2003) Gene Ther , vol.10 , pp. 2112-2118
    • McCarty, D.M.1    Fu, H.2    Monahan, P.E.3    Toulson, C.E.4    Nalk, P.5    Samulski, R.J.6
  • 60
    • 0034882826 scopus 로고    scopus 로고
    • Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
    • McCarty DM, Monahan PE, Samulski RJ: Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther. 8, 1248-1254 (2001).
    • (2001) Gene Ther , vol.8 , pp. 1248-1254
    • McCarty, D.M.1    Monahan, P.E.2    Samulski, R.J.3
  • 61
    • 33947410764 scopus 로고    scopus 로고
    • Self-complementary recombinant adeno-associated viral vectors: Packaging capacity and the role of rep proteins in vector purity
    • Wu J, Zhao W, Zhong L et al.: Self-complementary recombinant adeno-associated viral vectors: packaging capacity and the role of rep proteins in vector purity. Hum. Gene Ther. 18(2), 171-182 (2007).
    • (2007) Hum. Gene Ther , vol.18 , Issue.2 , pp. 171-182
    • Wu, J.1    Zhao, W.2    Zhong, L.3
  • 62
    • 0034121579 scopus 로고    scopus 로고
    • Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization
    • Sun L, Li J, Xiao X: Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization. Nat. Med. 6, 599-602 (2000).
    • (2000) Nat. Med , vol.6 , pp. 599-602
    • Sun, L.1    Li, J.2    Xiao, X.3
  • 63
    • 33846192000 scopus 로고    scopus 로고
    • Hybrid adeno-associated virus bearing nonhomologous inverted terminal repeats enhances dual-vector reconstruction of minigenes in vivo
    • Yan Z, Lei-Butters DC, Zhang Y, Zak R, Engelhardt JF: Hybrid adeno-associated virus bearing nonhomologous inverted terminal repeats enhances dual-vector reconstruction of minigenes in vivo. Hum. Gene Ther. 18, 81-87 (2007).
    • (2007) Hum. Gene Ther , vol.18 , pp. 81-87
    • Yan, Z.1    Lei-Butters, D.C.2    Zhang, Y.3    Zak, R.4    Engelhardt, J.F.5
  • 64
    • 0042890335 scopus 로고    scopus 로고
    • Double strand interaction is the predominant pathway for intermolecular recombination of adeno-associated viral genomes
    • Yue Y, Duan D: Double strand interaction is the predominant pathway for intermolecular recombination of adeno-associated viral genomes. Virology 313, 1-7 (2003).
    • (2003) Virology , vol.313 , pp. 1-7
    • Yue, Y.1    Duan, D.2
  • 65
    • 16844363202 scopus 로고    scopus 로고
    • Serotype-specific replicating AAV helper constructs increase recombinant AAV type 2 vector production
    • Li C, Samulski RJ: Serotype-specific replicating AAV helper constructs increase recombinant AAV type 2 vector production. Virology 335, 10-21 (2005).
    • (2005) Virology , vol.335 , pp. 10-21
    • Li, C.1    Samulski, R.J.2
  • 66
    • 43049119982 scopus 로고    scopus 로고
    • Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice
    • Allocca M, Doria M, Petrillo M et al.: Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J. Clin. Invert. 118, 1955-1964 (2008).
    • (2008) J. Clin. Invert , vol.118 , pp. 1955-1964
    • Allocca, M.1    Doria, M.2    Petrillo, M.3
  • 67
    • 34447636768 scopus 로고    scopus 로고
    • Scalable serum-free production of recombinant adeno-associated virus type 2 by transfection of 293 suspension cells
    • Durocher Y, Pham PL, St-Laurent G et al.: Scalable serum-free production of recombinant adeno-associated virus type 2 by transfection of 293 suspension cells. J. Virol. Methods 144, 32-40 (2007).
    • (2007) J. Virol. Methods , vol.144 , pp. 32-40
    • Durocher, Y.1    Pham, P.L.2    St-Laurent, G.3
  • 68
    • 34547567609 scopus 로고    scopus 로고
    • Enhanced preparation of adeno-associated viral vectors by using high hydrostatic pressure to selectively inactivate helper adenovirus
    • Leonard JN, Ferstl P, Delgado A, Schaffer DV: Enhanced preparation of adeno-associated viral vectors by using high hydrostatic pressure to selectively inactivate helper adenovirus. Biotechnol. Bioeng. 97, 1170-1179 (2007).
    • (2007) Biotechnol. Bioeng , vol.97 , pp. 1170-1179
    • Leonard, J.N.1    Ferstl, P.2    Delgado, A.3    Schaffer, D.V.4
  • 69
    • 4644346385 scopus 로고    scopus 로고
    • Improving rAAV production and purification: Towards the definition of a scaleable process
    • Blouin V, Brument N, Toublanc E, Raimbaud I, Moullier P, Salvetti A: Improving rAAV production and purification: towards the definition of a scaleable process. J. Gene Med. 6(Suppl. 1), S223-S228 (2004).
    • (2004) J. Gene Med , vol.6 , Issue.SUPPL. 1
    • Blouin, V.1    Brument, N.2    Toublanc, E.3    Raimbaud, I.4    Moullier, P.5    Salvetti, A.6
  • 70
    • 0037213292 scopus 로고    scopus 로고
    • Evidence for packaging of rep-cap sequences into adeno-associated virus (AAV) TYPE 2 capsids in the absence of inverted terminal repeats: A model for generation of rep-positive AAV particles
    • Nony P, Chadeuf G, Tessier J, Moullier P, Salvetti A: Evidence for packaging of rep-cap sequences into adeno-associated virus (AAV) TYPE 2 capsids in the absence of inverted terminal repeats: a model for generation of rep-positive AAV particles. J. Virol. 77, 776-781 (2003).
    • (2003) J. Virol , vol.77 , pp. 776-781
    • Nony, P.1    Chadeuf, G.2    Tessier, J.3    Moullier, P.4    Salvetti, A.5
  • 71
    • 25144482280 scopus 로고    scopus 로고
    • Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery
    • Chadeuf G, Ciron C, Moullier P, Salvetti A: Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery. Mol. Ther. 12, 744-753 (2005).
    • (2005) Mol. Ther , vol.12 , pp. 744-753
    • Chadeuf, G.1    Ciron, C.2    Moullier, P.3    Salvetti, A.4
  • 72
    • 0028577595 scopus 로고
    • Adeno-associated virus vectors preferentially transduce cells in S phase
    • Russell DW, Miller AD, Alexander IE: Adeno-associated virus vectors preferentially transduce cells in S phase. Proc. Natl Acad. Sci. USA 91, 8915-8919 (1994).
    • (1994) Proc. Natl Acad. Sci. USA , vol.91 , pp. 8915-8919
    • Russell, D.W.1    Miller, A.D.2    Alexander, I.E.3
  • 73
    • 0029655894 scopus 로고    scopus 로고
    • Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis
    • Fisher KJ, Gao GP, Weitzman MD, DeMatteo R, Burda JF, Wilson JM: Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70, 520-532 (1996).
    • (1996) J. Virol , vol.70 , pp. 520-532
    • Fisher, K.J.1    Gao, G.P.2    Weitzman, M.D.3    DeMatteo, R.4    Burda, J.F.5    Wilson, J.M.6
  • 74
    • 0034033447 scopus 로고    scopus 로고
    • Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors
    • Nakai H, Storm TA, Kay MA: Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nat. Biotechnol. 18, 527-532 (2000).
    • (2000) Nat. Biotechnol , vol.18 , pp. 527-532
    • Nakai, H.1    Storm, T.A.2    Kay, M.A.3
  • 75
    • 3042728872 scopus 로고    scopus 로고
    • Adeno-associated virus vectors integrate at chromosome breakage sites
    • Miller DG, Petek LM, Russell DW: Adeno-associated virus vectors integrate at chromosome breakage sites. Nat. Genet. 36, 767-773 (2004).
    • (2004) Nat. Genet , vol.36 , pp. 767-773
    • Miller, D.G.1    Petek, L.M.2    Russell, D.W.3
  • 76
    • 23844556421 scopus 로고    scopus 로고
    • Large-scale analysis of adeno-associated virus vector integration sites in normal human cells
    • Miller DG, Trobridge GD, Petek LM, Jacobs MA, Kaul R, Russell DW: Large-scale analysis of adeno-associated virus vector integration sites in normal human cells. J. Virol. 79, 11434-11442 (2005).
    • (2005) J. Virol , vol.79 , pp. 11434-11442
    • Miller, D.G.1    Trobridge, G.D.2    Petek, L.M.3    Jacobs, M.A.4    Kaul, R.5    Russell, D.W.6
  • 77
    • 35148863458 scopus 로고    scopus 로고
    • The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice
    • Inagaki K, Ma C, Storm TA, Kay MA, Nakai H: The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice. J. Virol. 81, 11304-11321 (2007).
    • (2007) J. Virol , vol.81 , pp. 11304-11321
    • Inagaki, K.1    Ma, C.2    Storm, T.A.3    Kay, M.A.4    Nakai, H.5
  • 78
    • 0034830365 scopus 로고    scopus 로고
    • Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors
    • Donsante A, Vogler C, Muzyczka N et al.: Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. Gene Ther. 8, 1343-1346 (2001).
    • (2001) Gene Ther , vol.8 , pp. 1343-1346
    • Donsante, A.1    Vogler, C.2    Muzyczka, N.3
  • 79
    • 35148868645 scopus 로고    scopus 로고
    • AAV vectors and tumorigenicity
    • Kay MA: AAV vectors and tumorigenicity. Nat. Biotech. 25, 1111-1113 (2007).
    • (2007) Nat. Biotech , vol.25 , pp. 1111-1113
    • Kay, M.A.1
  • 80
    • 22644433751 scopus 로고    scopus 로고
    • No evidence for tumorigenesis of AAV vectors in a large-scale study in mice
    • Bell P, Wang L, Lebherz C et al.: No evidence for tumorigenesis of AAV vectors in a large-scale study in mice. Mol. Ther. 12, 299-306 (2005).
    • (2005) Mol. Ther , vol.12 , pp. 299-306
    • Bell, P.1    Wang, L.2    Lebherz, C.3
  • 81
    • 0033977047 scopus 로고    scopus 로고
    • Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nonhuman primate muscle
    • Chirmule N, Xiao W, Truneh A et al.: Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nonhuman primate muscle. J. Virol. 74, 2420-2425 (2000).
    • (2000) J. Virol , vol.74 , pp. 2420-2425
    • Chirmule, N.1    Xiao, W.2    Truneh, A.3
  • 82
    • 0034169676 scopus 로고    scopus 로고
    • Route of administration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors
    • Xiao W, Chirmule N. Schnell MA, Tazelaar J, Hughes JV, Wilson JM: Route of administration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors. Mol. Ther. 1, 323-329 (2000).
    • (2000) Mol. Ther , vol.1 , pp. 323-329
    • Xiao, W.1    Chirmule, N.2    Schnell, M.A.3    Tazelaar, J.4    Hughes, J.V.5    Wilson, J.M.6
  • 83
    • 0032826773 scopus 로고    scopus 로고
    • Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model
    • Hernandez YJ, Wang J, Kearns WG, Loiler S, Poirier A, Flotte TR: Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model. J. Virol. 73, 8549-8558 (1999).
    • (1999) J. Virol , vol.73 , pp. 8549-8558
    • Hernandez, Y.J.1    Wang, J.2    Kearns, W.G.3    Loiler, S.4    Poirier, A.5    Flotte, T.R.6
  • 84
    • 0013442744 scopus 로고    scopus 로고
    • AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
    • Manno CS, Chew AJ, Hutchison S et al.: AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101, 2963-2972 (2003).
    • (2003) Blood , vol.101 , pp. 2963-2972
    • Manno, C.S.1    Chew, A.J.2    Hutchison, S.3
  • 85
    • 33744825168 scopus 로고    scopus 로고
    • Immune responses to AAV in a phase I study for Canavan disease
    • McPhee SW, Janson CG, Li C et al.: Immune responses to AAV in a phase I study for Canavan disease. J. Gene Med. 8, 577-588 (2006).
    • (2006) J. Gene Med , vol.8 , pp. 577-588
    • McPhee, S.W.1    Janson, C.G.2    Li, C.3
  • 86
    • 0035877984 scopus 로고    scopus 로고
    • Factors influencing the development of an anti-factor IX (FIX) immune response following administration of adeno-associated virus-FIX
    • Ge Y, Powell S, Van Roey M, McArthur JG: Factors influencing the development of an anti-factor IX (FIX) immune response following administration of adeno-associated virus-FIX. Blood 97, 3733-3737 (2001).
    • (2001) Blood , vol.97 , pp. 3733-3737
    • Ge, Y.1    Powell, S.2    Van Roey, M.3    McArthur, J.G.4
  • 87
    • 34547498762 scopus 로고    scopus 로고
    • + T cells in tolerance to the transgene product following hepatic in vivo gene transfer
    • + T cells in tolerance to the transgene product following hepatic in vivo gene transfer. Blood 110, 1132-1140 (2007).
    • (2007) Blood , vol.110 , pp. 1132-1140
    • Cao, O.1    Dobrzynski, E.2    Wang, L.3
  • 88
    • 0036721008 scopus 로고    scopus 로고
    • Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques
    • Nathwani AC, Davidoff AM, Hanawa H et al.: Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques. Blood 100, 1662-1669 (2002).
    • (2002) Blood , vol.100 , pp. 1662-1669
    • Nathwani, A.C.1    Davidoff, A.M.2    Hanawa, H.3
  • 89
    • 1942457251 scopus 로고    scopus 로고
    • Erythropoietin gene therapy leads to autoimmune anemia in macaques
    • Gao G, Lebherz C, Weiner DJ et al.: Erythropoietin gene therapy leads to autoimmune anemia in macaques. Blood 103, 3300-3302 (2004).
    • (2004) Blood , vol.103 , pp. 3300-3302
    • Gao, G.1    Lebherz, C.2    Weiner, D.J.3
  • 90
    • 1942521650 scopus 로고    scopus 로고
    • Autoimmune anemia in macaques following erythropoietin gene therapy
    • Chenuaud P, Larcher T, Rabinowitz JE et al.: Autoimmune anemia in macaques following erythropoietin gene therapy. Blood 103, 3303-3304 (2004).
    • (2004) Blood , vol.103 , pp. 3303-3304
    • Chenuaud, P.1    Larcher, T.2    Rabinowitz, J.E.3
  • 91
    • 37749038350 scopus 로고    scopus 로고
    • Gene delivery GAD 500 autoantigen by AAV serotype 1 prevented diabetes in mice NOD: Transduction efficiency do not play important roles
    • Han G, Wang R, Chen G et al.: Gene delivery GAD 500 autoantigen by AAV serotype 1 prevented diabetes in mice NOD: transduction efficiency do not play important roles. Immunol. Lett. 115, 110-116 (2008).
    • (2008) Immunol. Lett , vol.115 , pp. 110-116
    • Han, G.1    Wang, R.2    Chen, G.3
  • 92
    • 0037676229 scopus 로고    scopus 로고
    • MV-mediated gene transfer for hemophilia
    • High K: MV-mediated gene transfer for hemophilia. Genet. Med. 4, 56S-61S (2002).
    • (2002) Genet. Med , vol.4
    • High, K.1
  • 93
    • 33644815296 scopus 로고    scopus 로고
    • Adeno-associated virus-based gene therapy for inherited disorders
    • Flotte TR: Adeno-associated virus-based gene therapy for inherited disorders. Pediatr. Res. 58, 1143-1147 (2005).
    • (2005) Pediatr. Res , vol.58 , pp. 1143-1147
    • Flotte, T.R.1
  • 94
    • 55849143553 scopus 로고    scopus 로고
    • From the smallest virus to the biggest gene: Marching towards gene therapy for Duchenne muscular dystrophy
    • Duan D: From the smallest virus to the biggest gene: marching towards gene therapy for Duchenne muscular dystrophy. Discov. Med. 6, 103-108 (2006).
    • (2006) Discov. Med , vol.6 , pp. 103-108
    • Duan, D.1
  • 96
    • 33846604310 scopus 로고    scopus 로고
    • Gene therapy in epilepsy: The focus on NPY
    • Noe F, Nissinen J, Pitkanen A et al.: Gene therapy in epilepsy: the focus on NPY. Peptides 28, 377-383 (2007).
    • (2007) Peptides , vol.28 , pp. 377-383
    • Noe, F.1    Nissinen, J.2    Pitkanen, A.3
  • 98
    • 33846933945 scopus 로고    scopus 로고
    • Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium
    • Le Meur G, Stieger K, Smith AJ et al.: Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium. Gene Ther. 14, 292-303 (2007).
    • (2007) Gene Ther , vol.14 , pp. 292-303
    • Le Meur, G.1    Stieger, K.2    Smith, A.J.3
  • 99
    • 38449089618 scopus 로고    scopus 로고
    • Cancer gene therapy using adeno-associated virus vectors
    • Park K, Kim WJ, Cho YH et al.: Cancer gene therapy using adeno-associated virus vectors. Front. Biosci. 13, 2653-2659 (2008).
    • (2008) Front. Biosci , vol.13 , pp. 2653-2659
    • Park, K.1    Kim, W.J.2    Cho, Y.H.3
  • 100
    • 36348956106 scopus 로고    scopus 로고
    • RAC reviews serious adverse event associated with AAV therapy trial
    • Williams DA: RAC reviews serious adverse event associated with AAV therapy trial. Mol. Ther. 15, 2053-2054 (2007).
    • (2007) Mol. Ther , vol.15 , pp. 2053-2054
    • Williams, D.A.1
  • 101
    • 50649112211 scopus 로고    scopus 로고
    • Safety assessment of intradiscal gene therapy II: Effect of dosing and vector choice
    • Levicoff EA, Kim JS, Sobajima S et al.: Safety assessment of intradiscal gene therapy II: effect of dosing and vector choice. Spine 33, 1509-1516 (2008).
    • (2008) Spine , vol.33 , pp. 1509-1516
    • Levicoff, E.A.1    Kim, J.S.2    Sobajima, S.3
  • 102
    • 33746865345 scopus 로고    scopus 로고
    • In vivo delivery of recombinant viruses to the fetal murine cochlea: Transduction characteristics and long-term effects on auditory function
    • Bedrosian JC, Gratton MA, Brigande JV, Tang W, Landau J, Bennett J: In vivo delivery of recombinant viruses to the fetal murine cochlea: transduction characteristics and long-term effects on auditory function. Mol. Ther. 14, 328-335 (2006).
    • (2006) Mol. Ther , vol.14 , pp. 328-335
    • Bedrosian, J.C.1    Gratton, M.A.2    Brigande, J.V.3    Tang, W.4    Landau, J.5    Bennett, J.6
  • 103
    • 33644820684 scopus 로고    scopus 로고
    • Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
    • Manno CS, Arruda VR, Pierce GF et al.: Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med. 12, 342-347 (2006).
    • (2006) Nat. Med , vol.12 , pp. 342-347
    • Manno, C.S.1    Arruda, V.R.2    Pierce, G.F.3
  • 104
    • 33646863928 scopus 로고    scopus 로고
    • Inadvertent germline transmission of AAV2 vector: Findings in a rabbit model correlate with those in a human clinical trial
    • Schuettrumpf J, Liu JH, Couto LB et al.: Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial. Mol. Ther. 13, 1064-1073 (2006).
    • (2006) Mol. Ther , vol.13 , pp. 1064-1073
    • Schuettrumpf, J.1    Liu, J.H.2    Couto, L.B.3
  • 105
    • 34250683023 scopus 로고    scopus 로고
    • Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson' disease: An open label, Phase I trial
    • Kaplitt MG, Feigin A, Tang C et al.: Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson' disease: an open label, Phase I trial. Lancet 369, 2097-2105 (2007).
    • (2007) Lancet , vol.369 , pp. 2097-2105
    • Kaplitt, M.G.1    Feigin, A.2    Tang, C.3
  • 106
    • 39849110435 scopus 로고    scopus 로고
    • Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer
    • Bennicelli J, Wright JF, Komaromy A et al.: Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. Mol. Ther. 16, 458-465 (2008).
    • (2008) Mol. Ther , vol.16 , pp. 458-465
    • Bennicelli, J.1    Wright, J.F.2    Komaromy, A.3
  • 107
    • 33750591238 scopus 로고    scopus 로고
    • Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis
    • Jacobson SG, Boye SL, Aleman TS et al.: Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Hum. Gene Ther. 17, 845-858 (2006).
    • (2006) Hum. Gene Ther , vol.17 , pp. 845-858
    • Jacobson, S.G.1    Boye, S.L.2    Aleman, T.S.3
  • 108
    • 33744495152 scopus 로고    scopus 로고
    • Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection
    • Jacobson SC, Acland GM, Aguirre GD et al.: Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. Mol. Ther. 13, 1074-1084 (2006).
    • (2006) Mol. Ther , vol.13 , pp. 1074-1084
    • Jacobson, S.C.1    Acland, G.M.2    Aguirre, G.D.3
  • 109
    • 33744825168 scopus 로고    scopus 로고
    • Immune responses to AAV in a Phase I study for Canavan disease
    • McPhee SW, Janson CG, Li C et al.: Immune responses to AAV in a Phase I study for Canavan disease. J. Gene Med. 8, 577-588 (2006).
    • (2006) J. Gene Med , vol.8 , pp. 577-588
    • McPhee, S.W.1    Janson, C.G.2    Li, C.3
  • 110
    • 34548138958 scopus 로고    scopus 로고
    • Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products
    • Yuasa K, Yoshimura M, Urasawa N et al.: Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products. Gene Ther. 14, 1249-1260 (2007).
    • (2007) Gene Ther , vol.14 , pp. 1249-1260
    • Yuasa, K.1    Yoshimura, M.2    Urasawa, N.3
  • 111
    • 34147098413 scopus 로고    scopus 로고
    • + T-cell responses to adeno-associated virus capsid in humans
    • + T-cell responses to adeno-associated virus capsid in humans. Nat. Med. 13, 419-422 (2007).
    • (2007) Nat. Med , vol.13 , pp. 419-422
    • Mingozzi, F.1    Maus, M.V.2    Hui, D.J.3
  • 112
    • 33947196295 scopus 로고    scopus 로고
    • + T cells are unable to eliminate AAV-transduced hepatocytes
    • + T cells are unable to eliminate AAV-transduced hepatocytes. Mol. Ther. 15, 792-800 (2007).
    • (2007) Mol. Ther , vol.15 , pp. 792-800
    • Li, H.1    Murphy, S.L.2    Giles-Davis, W.3
  • 113
    • 34948855431 scopus 로고    scopus 로고
    • Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver
    • Mingozzi F, Hasbrouck NC, Basner-Tschakarjan E et al.: Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood 110, 2334-2341 (2007).
    • (2007) Blood , vol.110 , pp. 2334-2341
    • Mingozzi, F.1    Hasbrouck, N.C.2    Basner-Tschakarjan, E.3
  • 114
    • 33751181882 scopus 로고    scopus 로고
    • Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy
    • Jiang H, Couto LB, Patarroyo-White S et al.: Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 108, 3321-3328 (2006).
    • (2006) Blood , vol.108 , pp. 3321-3328
    • Jiang, H.1    Couto, L.B.2    Patarroyo-White, S.3
  • 115
    • 33846934410 scopus 로고    scopus 로고
    • Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates
    • Nathwani AC, Gray JT, McIntosh J et al.: Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates. Blood 109, 1414-1421 (2007).
    • (2007) Blood , vol.109 , pp. 1414-1421
    • Nathwani, A.C.1    Gray, J.T.2    McIntosh, J.3
  • 116
    • 33845531899 scopus 로고    scopus 로고
    • Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs lentiviral vectors for hemophilia B gene therapy
    • Vandendriessche T, Thorrez L, Acosta-Sanchez A et al.: Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs lentiviral vectors for hemophilia B gene therapy. J. Thromb. Haemost. 5, 16-24 (2007).
    • (2007) J. Thromb. Haemost , vol.5 , pp. 16-24
    • Vandendriessche, T.1    Thorrez, L.2    Acosta-Sanchez, A.3
  • 117
    • 0009963512 scopus 로고    scopus 로고
    • Safety of adeno-associated virus gene therapy vectors a current evaluation
    • Monahan PE, Jooss K, Sands MS: Safety of adeno-associated virus gene therapy vectors a current evaluation. Expert Opin. Drug Saf. 1, 79-91 (2002).
    • (2002) Expert Opin. Drug Saf , vol.1 , pp. 79-91
    • Monahan, P.E.1    Jooss, K.2    Sands, M.S.3
  • 118
    • 33845576104 scopus 로고    scopus 로고
    • AAV vectors encoding malarial antigens stimulate antigen-specific immunity but do not protect from parasite infection
    • Logan GJ, Wang L, Zheng M, Cunningham SC, Coppel RL, Alexander IE: AAV vectors encoding malarial antigens stimulate antigen-specific immunity but do not protect from parasite infection. Vaccine 25, 1014-1022 (2007).
    • (2007) Vaccine , vol.25 , pp. 1014-1022
    • Logan, G.J.1    Wang, L.2    Zheng, M.3    Cunningham, S.C.4    Coppel, R.L.5    Alexander, I.E.6
  • 119
    • 34247200575 scopus 로고    scopus 로고
    • Wang L, Figueredo J, Calcedo R, Lin J. Wilson JM: Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets. Hum. Gene Ther. 18, 185-194 (2007).
    • Wang L, Figueredo J, Calcedo R, Lin J. Wilson JM: Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets. Hum. Gene Ther. 18, 185-194 (2007).
  • 120
    • 19344366672 scopus 로고    scopus 로고
    • Systemic protein delivery by muscle-gene transfer is limited by a local immune response
    • Wang L, Dobrzynski E, Schlachterman A, Cao O, Herzog RW: Systemic protein delivery by muscle-gene transfer is limited by a local immune response. Blood 105, 4226-4234 (2005).
    • (2005) Blood , vol.105 , pp. 4226-4234
    • Wang, L.1    Dobrzynski, E.2    Schlachterman, A.3    Cao, O.4    Herzog, R.W.5
  • 121
    • 0034749458 scopus 로고    scopus 로고
    • Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors
    • Sarukhan A, Camugli S, Gjata B. von Boehmer H, Danos O, Jooss K: Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors. J. Virol. 75, 269-277 (2001).
    • (2001) J. Virol , vol.75 , pp. 269-277
    • Sarukhan, A.1    Camugli, S.2    Gjata, B.3    von Boehmer, H.4    Danos, O.5    Jooss, K.6
  • 122
    • 0036375003 scopus 로고    scopus 로고
    • Oral administration of recombinant adeno-associated virus elicits human immunodeficiency virus-specific immune responses
    • Xin KQ, Ooki T, Mizukami H et al.: Oral administration of recombinant adeno-associated virus elicits human immunodeficiency virus-specific immune responses. Hum. Gene Ther. 13. 1571-1581 (2002).
    • (2002) Hum. Gene Ther , vol.13 , pp. 1571-1581
    • Xin, K.Q.1    Ooki, T.2    Mizukami, H.3
  • 123
    • 0033994625 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus expressing human papillomavirus type 16 E7 peptide DNA fused with heat shock protein DNA as a potential vaccine for cervical cancer
    • Liu DW, Tsao YP, Kung JT et al.: Recombinant adeno-associated virus expressing human papillomavirus type 16 E7 peptide DNA fused with heat shock protein DNA as a potential vaccine for cervical cancer. J. Virol. 74, 2888-2894 (2000).
    • (2000) J. Virol , vol.74 , pp. 2888-2894
    • Liu, D.W.1    Tsao, Y.P.2    Kung, J.T.3
  • 124
    • 34447643995 scopus 로고    scopus 로고
    • Generation of robust cytotoxic T lymphocytes against prostate specific antigen by transduction of dendritic cells using protein and recombinant adeno-associated virus
    • Mahadevan M, Liu Y, You C et al.: Generation of robust cytotoxic T lymphocytes against prostate specific antigen by transduction of dendritic cells using protein and recombinant adeno-associated virus. Cancer Immunol. Immunother. 56, 1615-1624 (2007).
    • (2007) Cancer Immunol. Immunother , vol.56 , pp. 1615-1624
    • Mahadevan, M.1    Liu, Y.2    You, C.3
  • 125
    • 33747194373 scopus 로고    scopus 로고
    • HBV genes induce cytotoxic T-lymphocyte response upon adeno-associated virus (AAV) vector delivery into dendritic cells
    • You H, Liu Y, Cong M et al.: HBV genes induce cytotoxic T-lymphocyte response upon adeno-associated virus (AAV) vector delivery into dendritic cells. J. Viral Hepat. 13, 605-612 (2006).
    • (2006) J. Viral Hepat , vol.13 , pp. 605-612
    • You, H.1    Liu, Y.2    Cong, M.3
  • 126
    • 32544450298 scopus 로고    scopus 로고
    • An autoimmune domain-reduced HCV core gene remains effective in stimulating anti-core cytotoxic T lymphocyte activity
    • Liu Y, Zhou W, You C et al.: An autoimmune domain-reduced HCV core gene remains effective in stimulating anti-core cytotoxic T lymphocyte activity. Vaccine 24, 1615-1624 (2006).
    • (2006) Vaccine , vol.24 , pp. 1615-1624
    • Liu, Y.1    Zhou, W.2    You, C.3
  • 127
    • 20044366384 scopus 로고    scopus 로고
    • Use and specificity of breast cancer antigen/milk protein BA46 for generating anti-self-cytotoxic T lymphocytes by recombinant adeno-associated virus-based gene loading of dendritic cells
    • Liu Y, Chiriva-Internati M, You C et al.: Use and specificity of breast cancer antigen/milk protein BA46 for generating anti-self-cytotoxic T lymphocytes by recombinant adeno-associated virus-based gene loading of dendritic cells. Cancer Gene Ther. 12, 304-312 (2005).
    • (2005) Cancer Gene Ther , vol.12 , pp. 304-312
    • Liu, Y.1    Chiriva-Internati, M.2    You, C.3
  • 128
    • 0036147989 scopus 로고    scopus 로고
    • Efficient generation of cytotoxic T lymphocytes against cervical cancer cells by adeno-associated virus/human papillomavirus type 16 E7 antigen gene transduction into dendritic cells
    • Chiriva-Internati M, Liu V, Salati E et al.: Efficient generation of cytotoxic T lymphocytes against cervical cancer cells by adeno-associated virus/human papillomavirus type 16 E7 antigen gene transduction into dendritic cells. Eur J. Immunol. 32, 30-38 (2002).
    • (2002) Eur J. Immunol , vol.32 , pp. 30-38
    • Chiriva-Internati, M.1    Liu, V.2    Salati, E.3
  • 129
    • 0035207017 scopus 로고    scopus 로고
    • Rapid induction of cytotoxic T-cell response against cervical cancer cells by human papillomavirus type 16 E6 antigen gene delivery into human dendritic cells by an adeno-associated virus vector
    • Liu Y, Chiriva-Internati M, Grizzi F et al.: Rapid induction of cytotoxic T-cell response against cervical cancer cells by human papillomavirus type 16 E6 antigen gene delivery into human dendritic cells by an adeno-associated virus vector. Cancer Gene Ther. 8, 948-957 (2001).
    • (2001) Cancer Gene Ther , vol.8 , pp. 948-957
    • Liu, Y.1    Chiriva-Internati, M.2    Grizzi, F.3
  • 130
    • 19944425988 scopus 로고    scopus 로고
    • Novel adeno-associated virus vector vaccine restricts replication of simian Immunodeficiency virus in macaques
    • Johnson PR, Schnepp BC, Connell MJ et al.: Novel adeno-associated virus vector vaccine restricts replication of simian Immunodeficiency virus in macaques. J. Virol. 79, 955-965 (2005).
    • (2005) J. Virol , vol.79 , pp. 955-965
    • Johnson, P.R.1    Schnepp, B.C.2    Connell, M.J.3
  • 131
    • 36849033989 scopus 로고    scopus 로고
    • A Phase I study to evaluate the safety and immunogenicity of a recombinant adeno-associated virus vaccine
    • Presented at:, Los Angeles, CA, USA, 25-28 February
    • van Lunzen J, Mehendale S, Clumeck N et al..: A Phase I study to evaluate the safety and immunogenicity of a recombinant adeno-associated virus vaccine. Presented at: 14th Conference on Retroviruses and Opportunistic Infections. Los Angeles, CA, USA, 25-28 February 2007.
    • (2007) 14th Conference on Retroviruses and Opportunistic Infections
    • van Lunzen, J.1    Mehendale, S.2    Clumeck, N.3
  • 134
    • 0348038748 scopus 로고    scopus 로고
    • Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo
    • Wang Z, Ma HI, Li J, Sun L, Zhang J, Xiao X: Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther. 10, 2105-2111 (2003).
    • (2003) Gene Ther , vol.10 , pp. 2105-2111
    • Wang, Z.1    Ma, H.I.2    Li, J.3    Sun, L.4    Zhang, J.5    Xiao, X.6
  • 135
    • 38449091796 scopus 로고    scopus 로고
    • Adeno-associated viral vectors engineered for macrolide-adjustable transgene expression in mammalian cells and mice
    • Fluri DA, Baba MD, Fussenegger M: Adeno-associated viral vectors engineered for macrolide-adjustable transgene expression in mammalian cells and mice. BMC Biotechnol. 7, 75 (2007).
    • (2007) BMC Biotechnol , vol.7 , pp. 75
    • Fluri, D.A.1    Baba, M.D.2    Fussenegger, M.3


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