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Volumn 25, Issue 6, 2014, Pages 517-528

Immunological ignorance allows long-term gene expression after perinatal recombinant adeno-associated virus-mediated gene transfer to murine airways

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; BETA GALACTOSIDASE; NEUTRALIZING ANTIBODY; VIRUS ANTIBODY;

EID: 84902981635     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2013.196     Document Type: Article
Times cited : (14)

References (53)
  • 3
    • 78650917070 scopus 로고    scopus 로고
    • Efficient gene transfer into the mouse lung by fetal intratracheal injection of rAAV2/6.2
    • Carlon, M., Toelen, J., Van Der Perren, A., et al. (2010). Efficient gene transfer into the mouse lung by fetal intratracheal injection of rAAV2/6.2. Mol. Ther. 18, 2130-2138.
    • (2010) Mol. Ther. , vol.18 , pp. 2130-2138
    • Carlon, M.1    Toelen, J.2    Van Der Perren, A.3
  • 4
    • 84872117692 scopus 로고    scopus 로고
    • A novel surgical approach for intratracheal administration of bioactive agents in a fetal mouse model
    • Carlon, M.S., Toelen, J., Da Cunha, M.M., et al. (2012). A novel surgical approach for intratracheal administration of bioactive agents in a fetal mouse model. J. Vis. Exp. 68.
    • (2012) J. Vis. Exp. , pp. 68
    • Carlon, M.S.1    Toelen, J.2    Da Cunha, M.M.3
  • 5
    • 0028168641 scopus 로고
    • Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis
    • Crystal, R.G., McElvaney, N.G., Rosenfeld, M.A., et al. (1994). Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat. Genet. 8, 42-51.
    • (1994) Nat. Genet. , vol.8 , pp. 42-51
    • Crystal, R.G.1    McElvaney, N.G.2    Rosenfeld, M.A.3
  • 8
    • 84891803470 scopus 로고    scopus 로고
    • Biosafety of recombinant adeno-associated virus vectors
    • Dismuke, D.J., Tenenbaum, L., and Samulski, R.J. (2013). Biosafety of recombinant adeno-associated virus vectors. Curr. Gene Ther. 13, 434-452.
    • (2013) Curr. Gene Ther. , vol.13 , pp. 434-452
    • Dismuke, D.J.1    Tenenbaum, L.2    Samulski, R.J.3
  • 9
    • 70349481722 scopus 로고    scopus 로고
    • Transient expression of genes delivered to newborn rat liver using recombinant adeno-associated virus 2/8 vectors
    • Flageul, M., Aubert, D., Pichard, V., et al. (2009). Transient expression of genes delivered to newborn rat liver using recombinant adeno-associated virus 2/8 vectors. J. Gene Med. 11, 689-696.
    • (2009) J. Gene Med. , vol.11 , pp. 689-696
    • Flageul, M.1    Aubert, D.2    Pichard, V.3
  • 10
    • 0038488551 scopus 로고    scopus 로고
    • Phase i trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: A two-part clinical study
    • Flotte, T.R., Zeitlin, P.L., Reynolds, T.C., et al. (2003). Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: A two-part clinical study. Hum. Gene Ther. 14, 1079-1088.
    • (2003) Hum. Gene Ther. , vol.14 , pp. 1079-1088
    • Flotte, T.R.1    Zeitlin, P.L.2    Reynolds, T.C.3
  • 14
    • 84864367347 scopus 로고    scopus 로고
    • Manipulating immune tolerance with micro-RNA regulated gene therapy
    • Goudy, K.S., Annoni, A., Naldini, L., and Roncarolo, M.G. (2011). Manipulating immune tolerance with micro-RNA regulated gene therapy. Front. Microbiol. 2, 221.
    • (2011) Front. Microbiol. , vol.2 , pp. 221
    • Goudy, K.S.1    Annoni, A.2    Naldini, L.3    Roncarolo, M.G.4
  • 15
    • 0032962320 scopus 로고    scopus 로고
    • Pathophysiology of gene-targeted mouse models for cystic fibrosis
    • Grubb, B.R., and Boucher, R.C. (1999). Pathophysiology of gene-targeted mouse models for cystic fibrosis. Physiol. Rev. 79, S193-S214. (Pubitemid 29058915)
    • (1999) Physiological Reviews , vol.79 , Issue.SUPPL. 1
    • Grubb, B.R.1    Boucher, R.C.2
  • 16
    • 0030795691 scopus 로고    scopus 로고
    • Transduction by adeno-associated virus vectors in the rabbit airway: Efficiency, persistence, and readministration
    • Halbert, C.L., Standaert, T.A., Aitken, M.L., et al. (1997). Transduction by adeno-associated virus vectors in the rabbit airway: Efficiency, persistence, and readministration. J. Virol. 71, 5932-5941. (Pubitemid 27304930)
    • (1997) Journal of Virology , vol.71 , Issue.8 , pp. 5932-5941
    • Halbert, C.L.1    Standaert, T.A.2    Aitken, M.L.3    Alexander, I.E.4    Russell, D.W.5    Miller, A.D.6
  • 17
    • 0033960160 scopus 로고    scopus 로고
    • Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes
    • DOI 10.1128/JVI.74.3.1524-1532.2000
    • Halbert, C.L., Rutledge, E.A., Allen, J.M., et al. (2000). Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes. J. Virol. 74, 1524- 1532. (Pubitemid 30044103)
    • (2000) Journal of Virology , vol.74 , Issue.3 , pp. 1524-1532
    • Halbert, C.L.1    Rutledge, E.A.2    Allen, J.M.3    Russell, D.W.4    Miller, A.D.5
  • 19
    • 79952122629 scopus 로고    scopus 로고
    • Neonatal helperdependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII
    • Hu, C., Cela, R.G., Suzuki, M., et al. (2011). Neonatal helperdependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII. Proc. Natl. Acad. Sci. U.S.A. 108, 2082-2087.
    • (2011) Proc. Natl. Acad. Sci. U.S.A. , vol.108 , pp. 2082-2087
    • Hu, C.1    Cela, R.G.2    Suzuki, M.3
  • 20
    • 64649100879 scopus 로고    scopus 로고
    • +, in patients with ulcerative colitis during active and quiescent disease
    • Kamikozuru, K., Fukunaga, K., Hirota, S., et al. (2009). The expression profile of functional regulatory T cells, CD4+ CD25high+/forkhead box protein P3+, in patients with ulcerative colitis during active and quiescent disease. Clin. Exp. Immunol. 156, 320-327.
    • (2009) Clin. Exp. Immunol. , vol.156 , pp. 320-327
    • Kamikozuru, K.1    Fukunaga, K.2    Hirota, S.3
  • 21
    • 33947196295 scopus 로고    scopus 로고
    • + T cells are unable to eliminate AAV-transduced hepatocytes
    • Li, H., Murphy, S.L., Giles-Davis, W., et al. (2007). Preexisting AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes. Mol. Ther. 15, 792-800.
    • (2007) Mol. Ther. , vol.15 , pp. 792-800
    • Li, H.1    Murphy, S.L.2    Giles-Davis, W.3
  • 22
    • 40549129747 scopus 로고    scopus 로고
    • Detection of reporter gene expression in murine airways
    • Reporter Genes: A Practical Guide
    • Limberis, M., Bell, P., and Wilson, J.M. (2007). Detection of reporter gene expression in murine airways. Methods Mol. Biol. 411, 25-34. (Pubitemid 351367195)
    • (2007) Methods in Molecular Biology , vol.411 , pp. 25-34
    • Limberis, M.1    Bell, P.2    Wilson, J.M.3
  • 23
    • 33748338996 scopus 로고    scopus 로고
    • Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered
    • DOI 10.1073/pnas.0601433103
    • Limberis, M.P., and Wilson, J.M. (2006). Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered. Proc. Natl. Acad. Sci. U.S.A. 103, 12993-12998. (Pubitemid 44338905)
    • (2006) Proceedings of the National Academy of Sciences of the United States of America , vol.103 , Issue.35 , pp. 12993-12998
    • Limberis, M.P.1    Wilson, J.M.2
  • 24
    • 62549150024 scopus 로고    scopus 로고
    • Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro
    • Limberis, M.P., Vandenberghe, L.H., Zhang, L., et al. (2009). Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. Mol. Ther. 17, 294-301.
    • (2009) Mol. Ther. , vol.17 , pp. 294-301
    • Limberis, M.P.1    Vandenberghe, L.H.2    Zhang, L.3
  • 25
    • 0034294530 scopus 로고    scopus 로고
    • Reexpression following readministration of an adenoviral vector in adult mice after initial in utero adenoviral administration
    • Lipshutz, G.S., Flebbe-Rehwaldt, L., and Gaensler, K.M. (2000). Reexpression following readministration of an adenoviral vector in adult mice after initial in utero adenoviral administration. Mol. Ther. 2, 374-380.
    • (2000) Mol. Ther. , vol.2 , pp. 374-380
    • Lipshutz, G.S.1    Flebbe-Rehwaldt, L.2    Gaensler, K.M.3
  • 26
    • 58149242206 scopus 로고    scopus 로고
    • Recombinant AAV serotype and capsid mutant comparison for pulmonary gene transfer of a-1-antitrypsin using invasive and noninvasive delivery
    • Liqun Wang, R., McLaughlin, T., Cossette, T., et al. (2009). Recombinant AAV serotype and capsid mutant comparison for pulmonary gene transfer of a-1-antitrypsin using invasive and noninvasive delivery. Mol. Ther. 17, 81-87.
    • (2009) Mol. Ther. , vol.17 , pp. 81-87
    • Liqun Wang, R.1    McLaughlin, T.2    Cossette, T.3
  • 27
    • 33847651713 scopus 로고    scopus 로고
    • Thymic development and peripheral homeostasis of regulatory T cells
    • DOI 10.1016/j.coi.2007.02.005, PII S0952791507000131, Lymphocyte development/Tumour immunology
    • Liston, A., and Rudensky, A.Y. (2007). Thymic development and peripheral homeostasis of regulatory T cells. Curr. Opin. Immunol. 19, 176-185. (Pubitemid 46356966)
    • (2007) Current Opinion in Immunology , vol.19 , Issue.2 , pp. 176-185
    • Liston, A.1    Rudensky, A.Y.2
  • 29
    • 79954622209 scopus 로고    scopus 로고
    • Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges
    • Mingozzi, F., and High, K.A. (2011). Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges. Nat. Rev. Genet. 12, 341-355.
    • (2011) Nat. Rev. Genet. , vol.12 , pp. 341-355
    • Mingozzi, F.1    High, K.A.2
  • 31
    • 10744227749 scopus 로고    scopus 로고
    • Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: A multicenter, double-blind, placebo-controlled trial
    • DOI 10.1378/chest.125.2.509
    • Moss, R.B., Rodman, D., Spencer, L.T., et al. (2004). Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: A multicenter, double-blind, placebo-controlled trial. Chest 125, 509-521. (Pubitemid 38223630)
    • (2004) Chest , vol.125 , Issue.2 , pp. 509-521
    • Moss, R.B.1    Rodman, D.2    Spencer, L.T.3    Aitken, M.L.4    Zeitlin, P.L.5    Waltz, D.6    Milla, C.7    Brody, A.S.8    Clancy, J.P.9    Ramsey, B.10    Hamblett, N.11    Heald, A.E.12
  • 33
    • 44049094771 scopus 로고    scopus 로고
    • Clinical gene therapy using recombinant adeno-associated virus vectors
    • DOI 10.1038/gt.2008.68, PII GT200868, Special Issue: AAV Vectors for Clinical GeneTherapy
    • Mueller, C., and Flotte, T.R. (2008). Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther. 15, 858-863. (Pubitemid 351712619)
    • (2008) Gene Therapy , vol.15 , Issue.11 , pp. 858-863
    • Mueller, C.1    Flotte, T.R.2
  • 34
    • 84855161388 scopus 로고    scopus 로고
    • Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
    • Nathwani, A.C., Tuddenham, E.G., Rangarajan, S., et al. (2011). Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med. 365, 2357-2365.
    • (2011) N. Engl. J. Med. , vol.365 , pp. 2357-2365
    • Nathwani, A.C.1    Tuddenham, E.G.2    Rangarajan, S.3
  • 35
    • 0032506749 scopus 로고    scopus 로고
    • Enhanced in vivo airway gene transfer via transient modification of host barrier properties with a surface-active agent
    • Parsons, D.W., Grubb, B.R., Johnson, L.G., and Boucher, R.C. (1998). Enhanced in vivo airway gene transfer via transient modification of host barrier properties with a surface-active agent. Hum. Gene Ther. 9, 2661-2672. (Pubitemid 29027009)
    • (1998) Human Gene Therapy , vol.9 , Issue.18 , pp. 2661-2672
    • Parsons, D.W.1    Grubb, B.R.2    Johnson, L.G.3    Boucher, R.C.4
  • 36
    • 33746413027 scopus 로고    scopus 로고
    • Epithelial stem cells of the lung: Privileged few or opportunities for many?
    • DOI 10.1242/dev.02407
    • Rawlins, E.L., and Hogan, B.L. (2006). Epithelial stem cells of the lung: privileged few or opportunities for many? Development 133, 2455-2465. (Pubitemid 44123396)
    • (2006) Development , vol.133 , Issue.13 , pp. 2455-2465
    • Rawlins, E.L.1    Hogan, B.L.M.2
  • 37
    • 34548068776 scopus 로고    scopus 로고
    • Persistent expression of hF.IX after tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice
    • DOI 10.1038/sj.mt.6300219, PII 6300219
    • Sabatino, D.E., Mackenzie, T.C., Peranteau, W., et al. (2007). Persistent expression of hF.IX after tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice. Mol. Ther. 15, 1677-1685. (Pubitemid 47289012)
    • (2007) Molecular Therapy , vol.15 , Issue.9 , pp. 1677-1685
    • Sabatino, D.E.1    Mackenzie, T.C.2    Peranteau, W.3    Edmonson, S.4    Campagnoli, C.5    Liu, Y.-L.6    Flake, A.W.7    High, K.A.8
  • 38
    • 79751506542 scopus 로고    scopus 로고
    • Gene therapy finds its niche
    • Sheridan, C. (2011). Gene therapy finds its niche. Nat. Biotechnol. 29, 121-128.
    • (2011) Nat. Biotechnol. , vol.29 , pp. 121-128
    • Sheridan, C.1
  • 39
    • 55249121439 scopus 로고    scopus 로고
    • Lentivirus vector can be readministered to nasal epithelia without blocking immune responses
    • DOI 10.1128/JVI.00227-08
    • Sinn, P.L., Arias, A.C., Brogden, K.A., and McCray, P.B., Jr. (2008). Lentivirus vector can be readministered to nasal epithelia without blocking immune responses. J. Virol. 82, 10684-10692. (Pubitemid 352691165)
    • (2008) Journal of Virology , vol.82 , Issue.21 , pp. 10684-10692
    • Sinn, P.L.1    Arias, A.C.2    Brogden, K.A.3    McCray Jr., P.B.4
  • 40
    • 33845200829 scopus 로고    scopus 로고
    • Long-term persistence of gene expression from adeno-associated virus serotype 5 in the mouse airways
    • DOI 10.1038/sj.gt.3302815, PII 3302815
    • Sumner-Jones, S.G., Davies, L.A., Varathalingam, A., et al. (2006). Long-term persistence of gene expression from adenoassociated virus serotype 5 in the mouse airways. Gene Ther. 13, 1703-1713. (Pubitemid 44849035)
    • (2006) Gene Therapy , vol.13 , Issue.24 , pp. 1703-1713
    • Sumner-Jones, S.G.1    Davies, L.A.2    Varathalingam, A.3    Gill, D.R.4    Hyde, S.C.5
  • 41
    • 0035383766 scopus 로고    scopus 로고
    • Induction of stable prenatal tolerance to b-galactosidase by in utero gene transfer into preimmune sheep fetuses
    • Tran, N.D., Porada, C.D., Almeida-Porada, G., et al. (2001). Induction of stable prenatal tolerance to b-galactosidase by in utero gene transfer into preimmune sheep fetuses. Blood 97, 3417-3423.
    • (2001) Blood , vol.97 , pp. 3417-3423
    • Tran, N.D.1    Porada, C.D.2    Almeida-Porada, G.3
  • 42
    • 62549159967 scopus 로고    scopus 로고
    • Tailoring the AAV vector capsid for gene therapy
    • Vandenberghe, L.H., Wilson, J.M., and Gao, G. (2009). Tailoring the AAV vector capsid for gene therapy. Gene Ther. 16, 311-319.
    • (2009) Gene Ther. , vol.16 , pp. 311-319
    • Vandenberghe, L.H.1    Wilson, J.M.2    Gao, G.3
  • 43
    • 79955928806 scopus 로고    scopus 로고
    • Efficient and stable transduction of dopaminergic neurons in rat substantia nigra by rAAV 2/1, 2/2, 2/5, 2/6.2, 2/7, 2/8 and 2/9
    • Van der Perren, A., Toelen, J., Carlon, M., et al. (2011). Efficient and stable transduction of dopaminergic neurons in rat substantia nigra by rAAV 2/1, 2/2, 2/5, 2/6.2, 2/7, 2/8 and 2/9. Gene Ther. 18, 517-527.
    • (2011) Gene Ther , vol.18 , pp. 517-527
    • Van Der Perren, A.1    Toelen, J.2    Carlon, M.3
  • 46
    • 0036377851 scopus 로고    scopus 로고
    • A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies
    • Wagner, J.A., Nepomuceno, I.B., Messner, A.H., et al. (2002). A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Hum. Gene Ther. 13, 1349-1359.
    • (2002) Hum. Gene Ther. , vol.13 , pp. 1349-1359
    • Wagner, J.A.1    Nepomuceno, I.B.2    Messner, A.H.3
  • 47
    • 34247200575 scopus 로고    scopus 로고
    • Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets
    • DOI 10.1089/hum.2007.001
    • Wang, L., Figueredo, J., Calcedo, R., et al. (2007). Crosspresentation 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. (Pubitemid 46626458)
    • (2007) Human Gene Therapy , vol.18 , Issue.3 , pp. 185-194
    • Wang, L.1    Figueredo, J.2    Calcedo, R.3    Lin, J.4    Wilson, J.M.5
  • 48
    • 84861715230 scopus 로고    scopus 로고
    • Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector
    • Wang, L., Wang, H., Bell, P., et al. (2012). Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector. Hum. Gene Ther. 23, 533-539.
    • (2012) Hum. Gene Ther. , vol.23 , pp. 533-539
    • Wang, L.1    Wang, H.2    Bell, P.3
  • 49
    • 84856520424 scopus 로고    scopus 로고
    • Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials
    • Wang, Z., Tapscott, S.J., Chamberlain, J.S., and Storb, R. (2011). Immunity and AAV-mediated gene therapy for muscular dystrophies in large animal models and human trials. Front. Microbiol. 2, 201.
    • (2011) Front. Microbiol. , vol.2 , pp. 201
    • Wang, Z.1    Tapscott, S.J.2    Chamberlain, J.S.3    Storb, R.4
  • 50
    • 77957943865 scopus 로고    scopus 로고
    • Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia
    • Weinstein, D.A., Correia, C.E., Conlon, T., et al. (2010). Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia. Hum. Gene Ther. 21, 903-910.
    • (2010) Hum. Gene Ther. , vol.21 , pp. 903-910
    • Weinstein, D.A.1    Correia, C.E.2    Conlon, T.3
  • 51
    • 0036760213 scopus 로고    scopus 로고
    • Defining critical windows in the development of the human immune system
    • West, L.J. (2002). Defining critical windows in the development of the human immune system. Hum. Exp. Toxicol. 21, 499-505.
    • (2002) Hum. Exp. Toxicol. , vol.21 , pp. 499-505
    • West, L.J.1
  • 52
    • 0027377523 scopus 로고
    • Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis
    • DOI 10.1016/0092-8674(93)80063-K
    • Zabner, J., Couture, L.A., Gregory, R.J., et al. (1993). Adenovirusmediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 75, 207-216. (Pubitemid 23320285)
    • (1993) Cell , vol.75 , Issue.2 , pp. 207-216
    • Zabner, J.1    Couture, L.A.2    Gregory, R.J.3    Graham, S.M.4    Smith, A.E.5    Welsh, M.J.6
  • 53
    • 0034026085 scopus 로고    scopus 로고
    • Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer
    • DOI 10.1128/JVI.74.8.3852-3858.2000
    • Zabner, J., Seiler, M.,Walters, R., et al. (2000). Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer. J. Virol. 74, 3852-3858. (Pubitemid 30180327)
    • (2000) Journal of Virology , vol.74 , Issue.8 , pp. 3852-3858
    • Zabner, J.1    Seiler, M.2    Walters, R.3    Kotin, R.M.4    Fulgeras, W.5    Davidson, B.L.6    Chiorini, J.A.7


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