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Volumn 2, Issue 1, 2010, Pages 159-171

Viral vectors for cystic fibrosis gene therapy: What does the future hold?

Author keywords

AAV; Adenovirus; Cystic fibrosis; Gene therapy; Lentivirus; Sendai virus

Indexed keywords

ADENOVIRUS VECTOR; COMPLEMENTARY DNA; LENTIVIRUS VECTOR; PARVOVIRUS VECTOR; RETROVIRUS VECTOR; TRANSMEMBRANE CONDUCTANCE REGULATOR; VIRUS VECTOR;

EID: 79251471162     PISSN: None     EISSN: 11791624     Source Type: Book Series    
DOI: 10.2147/VAAT.S8887     Document Type: Article
Times cited : (10)

References (116)
  • 1
    • 0001752544 scopus 로고    scopus 로고
    • In: Scriver CR, Beaudet AL, Sly WS, et al, editors. The Metabolic and Molecular Basis of Inherited Disease. New York: McGraw-Hill
    • Welsh M, Ramsey BW, Accurso F, Cutting GR. Cystic fibrosis. In: Scriver CR, Beaudet AL, Sly WS, et al, editors. The Metabolic and Molecular Basis of Inherited Disease. New York: McGraw-Hill; 2001: 5121-5188.
    • (2001) Cystic Fibrosis , pp. 5121-5188
    • Welsh, M.1    Ramsey, B.W.2    Accurso, F.3    Cutting, G.R.4
  • 2
    • 0024424270 scopus 로고
    • Identification of the cystic fibrosis gene: Cloning and characterization of complementary DNA
    • Riordan JR, Rommens JM, Kerem B, et al. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science. 1989;245(4922):1066-1073.
    • (1989) Science , vol.245 , Issue.4922 , pp. 1066-1073
    • Riordan, J.R.1    Rommens, J.M.2    Kerem, B.3
  • 3
    • 0027361622 scopus 로고
    • Non-invasive liposomemediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice
    • Alton EW, Middleton PG, Caplen NJ, et al. Non-invasive liposomemediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nat Genet. 1993;5(2):135-142.
    • (1993) Nat Genet , vol.5 , Issue.2 , pp. 135-142
    • Alton, E.W.1    Middleton, P.G.2    Caplen, N.J.3
  • 4
    • 0025094612 scopus 로고
    • Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer
    • Drumm ML, Pope HA, Cliff WH, et al. Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer. Cell. 1990;62(6): 1227-1233.
    • (1990) Cell , vol.62 , Issue.6 , pp. 1227-1233
    • Drumm, M.L.1    Pope, H.A.2    Cliff, W.H.3
  • 5
    • 60149095412 scopus 로고    scopus 로고
    • Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy
    • . UK Cystic Fibrosis Gene Therapy Consortium
    • Griesenbach U, Alton EW. UK Cystic Fibrosis Gene Therapy Consortium. Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy. Adv Drug Deliv Rev. 2009; 61(2): 128-139.
    • (2009) Adv Drug Deliv Rev , vol.61 , Issue.2 , pp. 128-139
    • Griesenbach, U.1    Alton, E.W.2
  • 7
    • 0042025196 scopus 로고    scopus 로고
    • Poly (D, L-lactide-co- glycolide)/DNA microspheres to facilitate prolonged transgene expression in airway epithelium in vitro, ex vivo and in vivo
    • Stern M, Ulrich K, Geddes DM, Alton EW. Poly (D, L-lactide-co- glycolide)/DNA microspheres to facilitate prolonged transgene expression in airway epithelium in vitro, ex vivo and in vivo. Gene Ther. 2003;10(16):1282-1288.
    • (2003) Gene Ther , vol.10 , Issue.16 , pp. 1282-1288
    • Stern, M.1    Ulrich, K.2    Geddes, D.M.3    Alton, E.W.4
  • 8
    • 0034425984 scopus 로고    scopus 로고
    • Efficient gene transfer to airway epithelium using recombinant Sendai virus
    • Yonemitsu Y, Kitson C, Ferrari S, et al. Efficient gene transfer to airway epithelium using recombinant Sendai virus. Nat Biotechnol. 2000;18(9):970-973.
    • (2000) Nat Biotechnol , vol.18 , Issue.9 , pp. 970-973
    • Yonemitsu, Y.1    Kitson, C.2    Ferrari, S.3
  • 9
    • 18244409874 scopus 로고    scopus 로고
    • Viscoelastic gel formulationsenhance airway epithelial gene transfer with viral vectors
    • Sinn PL, Shah AJ, Donovan MD, McCray PB Jr. Viscoelastic gel formulationsenhance airway epithelial gene transfer with viral vectors. Am J Respir Cell Mol Biol. 2005;32(5):404-410.
    • (2005) Am J Respir Cell Mol Biol , vol.32 , Issue.5 , pp. 404-410
    • Sinn, P.L.1    Shah, A.J.2    Donovan, M.D.3    McCray Jr., P.B.4
  • 10
    • 74449092542 scopus 로고    scopus 로고
    • The use of carboxymethylcellulosegel to increase non-viral gene transfer in mouse airways
    • Griesenbach U, Meng C, Farley R, et al. The use of carboxymethylcellulosegel to increase non-viral gene transfer in mouse airways. Biomaterials. 2010;31(9):2665-2672.
    • (2010) Biomaterials , vol.31 , Issue.9 , pp. 2665-2672
    • Griesenbach, U.1    Meng, C.2    Farley, R.3
  • 11
    • 0032853699 scopus 로고    scopus 로고
    • Immuneresponses to adenovirus and adeno-associated virus in humans
    • Chirmule N, Propert K, Magosin S, Qian Y, Qian R, Wilson J. Immuneresponses to adenovirus and adeno-associated virus in humans. Gene Ther. 1999;6(9):1574-1583.
    • (1999) Gene Ther , vol.6 , Issue.9 , pp. 1574-1583
    • Chirmule, N.1    Propert, K.2    Magosin, S.3    Qian, Y.4    Qian, R.5    Wilson, J.6
  • 12
    • 84889758577 scopus 로고    scopus 로고
    • Repeat aerosol delivery of concentrated PEI/pDNA to the sheep lung [abstract]
    • McLachlan G, Davies LA, Gordon C, et al. Repeat aerosol delivery of concentrated PEI/pDNA to the sheep lung [abstract]. Mol Ther. 2010;18(S1):880.
    • (2010) Mol Ther , vol.18 , Issue.S1 , pp. 880
    • McLachlan, G.1    Davies, L.A.2    Gordon, C.3
  • 13
    • 0033924663 scopus 로고    scopus 로고
    • Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis
    • Hyde SC, Southern KW, Gileadi U, et al. Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis. Gene Ther. 2000;7(13):1156-1165.
    • (2000) Gene Ther , vol.7 , Issue.13 , pp. 1156-1165
    • Hyde, S.C.1    Southern, K.W.2    Gileadi, U.3
  • 14
    • 0031777961 scopus 로고    scopus 로고
    • Limited entry of adenovirus vectors into well-differentiated airway epithelium is responsible for inefficient gene transfer
    • Pickles RJ, McCarty D, Matsui H, Hart PJ, Randell SH, Boucher RC. Limited entry of adenovirus vectors into well-differentiated airway epithelium is responsible for inefficient gene transfer. J Virol. 1998; 72(7):6014-6023.
    • (1998) J Virol , vol.72 , Issue.7 , pp. 6014-6023
    • Pickles, R.J.1    McCarty, D.2    Matsui, H.3    Hart, P.J.4    Randell, S.H.5    Boucher, R.C.6
  • 15
    • 8844258028 scopus 로고    scopus 로고
    • A defectivenontransmissible recombinant Sendai virus mediates efficient gene transfer to airway epithelium in vivo
    • Ferrari S, Griesenbach U, Shiraki-Iida T, et al. A defectivenontransmissible recombinant Sendai virus mediates efficient gene transfer to airway epithelium in vivo. Gene Ther. 2004;11(22): 1659-1664.
    • (2004) Gene Ther , vol.11 , Issue.22 , pp. 1659-1664
    • Ferrari, S.1    Griesenbach, U.2    Shiraki-Iida, T.3
  • 16
    • 65549146847 scopus 로고    scopus 로고
    • Learning from the viral journey: How to enter cells and how to overcome intracellular barriers to reach the nucleus
    • Mudhakir D, Harashima H. Learning from the viral journey: how to enter cells and how to overcome intracellular barriers to reach the nucleus. AAPS J. 2009;11(1):65-77.
    • (2009) AAPS J , vol.11 , Issue.1 , pp. 65-77
    • Mudhakir, D.1    Harashima, H.2
  • 17
    • 77950857472 scopus 로고    scopus 로고
    • Progress and prospects: Nuclear import of nonviral vectors
    • Lam AP, Dean DA. Progress and prospects: nuclear import of nonviral vectors. Gene Ther. 2010;17(4):439-447.
    • (2010) Gene Ther , vol.17 , Issue.4 , pp. 439-447
    • Lam, A.P.1    Dean, D.A.2
  • 18
    • 20744433079 scopus 로고    scopus 로고
    • Intracellular trafficking of adeno-associated viral vectors
    • Ding W, Zhang L, Yan Z, Engelhardt JF. Intracellular trafficking of adeno-associated viral vectors. Gene Ther. 2005;12(11):873-880.
    • (2005) Gene Ther , vol.12 , Issue.11 , pp. 873-880
    • Ding, W.1    Zhang, L.2    Yan, Z.3    Engelhardt, J.F.4
  • 19
    • 77749342887 scopus 로고    scopus 로고
    • Flexible use of nuclear import pathways by HIV-1
    • Lee K, Ambrose Z, Martin TD, et al. Flexible use of nuclear import pathways by HIV-1. Cell Host Microbe. 2010;7(3):221-233.
    • (2010) Cell Host Microbe , vol.7 , Issue.3 , pp. 221-233
    • Lee, K.1    Ambrose, Z.2    Martin, T.D.3
  • 20
    • 43049178300 scopus 로고    scopus 로고
    • Progress and prospects: Gene therapy for genetic diseases with helper-dependent adenoviral vectors
    • Brunetti-Pierri N, Ng P. Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors. Gene Ther. 2008;15(8):553-560.
    • (2008) Gene Ther , vol.15 , Issue.8 , pp. 553-560
    • Brunetti-Pierri, N.1    Ng, P.2
  • 21
    • 0027377523 scopus 로고
    • Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis
    • Zabner J, Couture LA, Gregory RJ, Graham SM, Smith AE, Welsh MJ. Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell. 1993;75(2):207-216.
    • (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
  • 22
    • 0033537837 scopus 로고    scopus 로고
    • Basolateral localization of fiber receptors limits adenovirus infection from the apical surface of airway epithelia
    • Walters RW, Grunst T, Bergelson JM, Finberg RW, Welsh MJ, Zabner J. Basolateral localization of fiber receptors limits adenovirus infection from the apical surface of airway epithelia. J Biol Chem. 1999;274(15):10219-10226.
    • (1999) J Biol Chem , vol.274 , Issue.15 , pp. 10219-10226
    • Walters, R.W.1    Grunst, T.2    Bergelson, J.M.3    Finberg, R.W.4    Welsh, M.J.5    Zabner, J.6
  • 23
    • 0347815187 scopus 로고    scopus 로고
    • Transient increase in lung epithelial tight junction permeability: An additional mechanism for enhancement of lung transgene expression by perfluorochemical liquids
    • Weiss DJ, Beckett T, Bonneau L, Young J, Kolls JK, Wang G. Transient increase in lung epithelial tight junction permeability: an additional mechanism for enhancement of lung transgene expression by perfluorochemical liquids. Mol Ther. 2003;8(6):927-935.
    • (2003) Mol Ther , vol.8 , Issue.6 , pp. 927-935
    • Weiss, D.J.1    Beckett, T.2    Bonneau, L.3    Young, J.4    Kolls, J.K.5    Wang, G.6
  • 24
    • 27944457244 scopus 로고    scopus 로고
    • Aerosol delivery of an enhanced helper-dependent adenovirus formulation to rabbit lung using an intratracheal catheter
    • Koehler DR, Frndova H, Leung K, et al. Aerosol delivery of an enhanced helper-dependent adenovirus formulation to rabbit lung using an intratracheal catheter. J Gene Med. 2005;7(11):1409-1420.
    • (2005) J Gene Med , vol.7 , Issue.11 , pp. 1409-1420
    • Koehler, D.R.1    Frndova, H.2    Leung, K.3
  • 25
    • 35848970333 scopus 로고    scopus 로고
    • Pulmonary delivery of adenovirus vector formulated with dexamethasone-spermine facilitates homologous vector re-administration
    • Price AR, Limberis MP, Wilson JM, Diamond SL. Pulmonary delivery of adenovirus vector formulated with dexamethasone-spermine facilitates homologous vector re-administration. Gene Ther. 2007; 14(22):1594-1604.
    • (2007) Gene Ther , vol.14 , Issue.22 , pp. 1594-1604
    • Price, A.R.1    Limberis, M.P.2    Wilson, J.M.3    Diamond, S.L.4
  • 26
    • 0034056840 scopus 로고    scopus 로고
    • Readministration of adenovirus vector in nonhuman primate lungs by blockade of CD40-CD40 ligand interactions
    • Chirmule N, Raper SE, Burkly L, et al. Readministration of adenovirus vector in nonhuman primate lungs by blockade of CD40-CD40 ligand interactions. J Virol. 2000;74(7):3345-3352.
    • (2000) J Virol , vol.74 , Issue.7 , pp. 3345-3352
    • Chirmule, N.1    Raper, S.E.2    Burkly, L.3
  • 27
    • 0035447558 scopus 로고    scopus 로고
    • Budesonide enhances repeated gene transfer and expression in the lung with adenoviral vectors
    • Kolb M, Inman M, Margetts PJ, Galt T, Gauldie J. Budesonide enhances repeated gene transfer and expression in the lung with adenoviral vectors. Am J Respir Crit Care Med. 2001;164(5):866-872.
    • (2001) Am J Respir Crit Care Med , vol.164 , Issue.5 , pp. 866-872
    • Kolb, M.1    Inman, M.2    Margetts, P.J.3    Galt, T.4    Gauldie, J.5
  • 28
    • 33846271820 scopus 로고    scopus 로고
    • Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis
    • Flotte TR, Ng P, Dylla DE, et al. Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis. Mol Ther. 2007;15(2):229-241.
    • (2007) Mol Ther , vol.15 , Issue.2 , pp. 229-241
    • Flotte, T.R.1    Ng, P.2    Dylla, D.E.3
  • 29
    • 0347364650 scopus 로고    scopus 로고
    • Protection of Cftr knockout mice from acute lung infection by a helper-dependent adenoviral vector expressing Cftr in airway epithelia
    • Koehler DR, Sajjan U, Chow YH, et al. Protection of Cftr knockout mice from acute lung infection by a helper-dependent adenoviral vector expressing Cftr in airway epithelia. Proc Natl Acad Sci U S A. 2003; 100(26):15364-15369.
    • (2003) Proc Natl Acad Sci U S A , vol.100 , Issue.26 , pp. 15364-15369
    • Koehler, D.R.1    Sajjan, U.2    Chow, Y.H.3
  • 31
    • 0031040502 scopus 로고    scopus 로고
    • Reversal of cystic fibrosis phenotype in mice by gene therapy in utero
    • Larson JE, Morrow SL, Happel L, Sharp JF, Cohen JC. Reversal of cystic fibrosis phenotype in mice by gene therapy in utero. Lancet. 1997;349(9052):619-620.
    • (1997) Lancet , vol.349 , Issue.9052 , pp. 619-620
    • Larson, J.E.1    Morrow, S.L.2    Happel, L.3    Sharp, J.F.4    Cohen, J.C.5
  • 32
    • 42549089142 scopus 로고    scopus 로고
    • Intra-amniotic delivery of CFTR-expressing adenovirus does not reverse cystic fibrosis phenotype in inbred CFTR-knockout mice
    • Buckley SM, Waddington SN, Jezzard S, et al. Intra-amniotic delivery of CFTR-expressing adenovirus does not reverse cystic fibrosis phenotype in inbred CFTR-knockout mice. Mol Ther. 2008;16(5): 819-824.
    • (2008) Mol Ther , vol.16 , Issue.5 , pp. 819-824
    • Buckley, S.M.1    Waddington, S.N.2    Jezzard, S.3
  • 33
    • 42549129355 scopus 로고    scopus 로고
    • Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice
    • Davies LA, Varathalingam A, Painter H, et al. Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice. Mol Ther. 2008;16(5):812-818.
    • (2008) Mol Ther , vol.16 , Issue.5 , pp. 812-818
    • Davies, L.A.1    Varathalingam, A.2    Painter, H.3
  • 34
    • 77749237132 scopus 로고    scopus 로고
    • Pathology of gastrointestinal organs in a porcine model of cystic fibrosis
    • Meyerholz DK, Stoltz DA, Pezzulo AA, Welsh MJ. Pathology of gastrointestinal organs in a porcine model of cystic fibrosis. Am J Pathol. 2010;176(3):1377-1389.
    • (2010) Am J Pathol , vol.176 , Issue.3 , pp. 1377-1389
    • Meyerholz, D.K.1    Stoltz, D.A.2    Pezzulo, A.A.3    Welsh, M.J.4
  • 35
    • 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. 1998;72(11):8568-8577.
    • (1998) J Virol , vol.72 , Issue.11 , pp. 8568-8577
    • Duan, D.1    Sharma, P.2    Yang, J.3
  • 36
    • 21244463942 scopus 로고    scopus 로고
    • Recent developments in recombinant AAV-mediated gene therapy for lung diseases
    • Flotte TR. Recent developments in recombinant AAV-mediated gene therapy for lung diseases. Curr Gene Ther. 2005;5(3):361-366.
    • (2005) Curr Gene Ther , vol.5 , Issue.3 , pp. 361-366
    • Flotte, T.R.1
  • 37
    • 10944269753 scopus 로고    scopus 로고
    • Integration of adeno-associated virus (AAV) and recombinant AAV vectors
    • McCarty DM, Young SM Jr, Samulski RJ. Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet. 2004;38:819-845.
    • (2004) Annu Rev Genet , vol.38 , pp. 819-845
    • McCarty, D.M.1    Young Jr., S.M.2    Samulski, R.J.3
  • 38
    • 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
    • Moss RB, Rodman D, Spencer LT, et al. 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. 2004; 125(2):509-521.
    • (2004) Chest , vol.125 , Issue.2 , pp. 509-521
    • Moss, R.B.1    Rodman, D.2    Spencer, L.T.3
  • 39
    • 34548512049 scopus 로고    scopus 로고
    • Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: A randomized placebo-controlled phase 2B trial
    • Moss RB, Milla C, Colombo J, et al. Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Hum Gene Ther. 2007;18(8):726-732.
    • (2007) Hum Gene Ther , vol.18 , Issue.8 , pp. 726-732
    • Moss, R.B.1    Milla, C.2    Colombo, J.3
  • 40
  • 41
    • 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 MP, Vandenberghe LH, Zhang L, Pickles RJ, Wilson JM. Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. Mol Ther. 2009;17(2):294-301.
    • (2009) Mol Ther , vol.17 , Issue.2 , pp. 294-301
    • Limberis, M.P.1    Vandenberghe, L.H.2    Zhang, L.3    Pickles, R.J.4    Wilson, J.M.5
  • 42
    • 0036678455 scopus 로고    scopus 로고
    • The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy
    • Xie Q, Bu W, Bhatia S, et al. The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy. Proc Natl Acad Sci U S A. 2002;99(16):10405-10410.
    • (2002) Proc Natl Acad Sci U S A , vol.99 , Issue.16 , pp. 10405-10410
    • Xie, Q.1    Bu, W.2    Bhatia, S.3
  • 43
    • 0038027345 scopus 로고    scopus 로고
    • RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism
    • Shi W, Bartlett JS. RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism. Mol Ther. 2003;7(4):515-525
    • (2003) Mol Ther , vol.7 , Issue.4 , pp. 515-525
    • Shi, W.1    Bartlett, J.S.2
  • 44
    • 57749190573 scopus 로고    scopus 로고
    • Genetic modification of adeno-associated viral vector type 2 capsid enhances gene transfer efficiency in polarized human airway epithelial cells
    • White AF, Mazur M, Sorscher EJ, Zinn KR, Ponnazhagan S. Genetic modification of adeno-associated viral vector type 2 capsid enhances gene transfer efficiency in polarized human airway epithelial cells. Hum Gene Ther. 2008;19(12):1407-1414.
    • (2008) Hum Gene Ther , vol.19 , Issue.12 , pp. 1407-1414
    • White, A.F.1    Mazur, M.2    Sorscher, E.J.3    Zinn, K.R.4    Ponnazhagan, S.5
  • 45
    • 32344438754 scopus 로고    scopus 로고
    • Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
    • Maheshri N, Koerber JT, Kaspar BK, Schaffer DV. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol. 2006;24(2):198-204.
    • (2006) Nat Biotechnol , vol.24 , Issue.2 , pp. 198-204
    • Maheshri, N.1    Koerber, J.T.2    Kaspar, B.K.3    Schaffer, D.V.4
  • 46
    • 62649174755 scopus 로고    scopus 로고
    • Directed evolution of adeno-associated virus to an infectious respiratory virus
    • Excoffon KJ, Koerber JT, Dickey DD, et al. Directed evolution of adeno-associated virus to an infectious respiratory virus. Proc Natl Acad Sci U S A. 2009;106(10):3865-3870.
    • (2009) Proc Natl Acad Sci U S A , vol.106 , Issue.10 , pp. 3865-3870
    • Excoffon, K.J.1    Koerber, J.T.2    Dickey, D.D.3
  • 47
    • 73849130017 scopus 로고    scopus 로고
    • Generation of novel AAV variants by directed evolution for improved CFTR delivery to human ciliated airway epithelium
    • Li W, Zhang L, Johnson JS, et al. Generation of novel AAV variants by directed evolution for improved CFTR delivery to human ciliated airway epithelium. Mol Ther. 2009;17(12):2067-2077.
    • (2009) Mol Ther , vol.17 , Issue.12 , pp. 2067-2077
    • Li, W.1    Zhang, L.2    Johnson, J.S.3
  • 48
    • 35348836681 scopus 로고    scopus 로고
    • Comparative biology of rAAV transduction in ferret, pig and human airway epithelia
    • Liu X, Luo M, Guo C, Yan Z, Wang Y, Engelhardt JF. Comparative biology of rAAV transduction in ferret, pig and human airway epithelia. Gene Ther. 2007;14(21):1543-1548.
    • (2007) Gene Ther , vol.14 , Issue.21 , pp. 1543-1548
    • Liu, X.1    Luo, M.2    Guo, C.3    Yan, Z.4    Wang, Y.5    Engelhardt, J.F.6
  • 49
    • 0037791759 scopus 로고    scopus 로고
    • Second-strand genome conversion of adeno-associated virus type 2 (AAV-2) and AAV-5 is not rate limiting following apical infection of polarized human airway epithelia
    • Ding W, Yan Z, Zak R, Saavedra M, Rodman DM, Engelhardt JF. Second-strand genome conversion of adeno-associated virus type 2 (AAV-2) and AAV-5 is not rate limiting following apical infection of polarized human airway epithelia. J Virol. 2003;77(13):7361-7366.
    • (2003) J Virol , vol.77 , Issue.13 , pp. 7361-7366
    • Ding, W.1    Yan, Z.2    Zak, R.3    Saavedra, M.4    Rodman, D.M.5    Engelhardt, J.F.6
  • 50
    • 33846192000 scopus 로고    scopus 로고
    • Hybrid adenoassociated 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 adenoassociated virus bearing nonhomologous inverted terminal repeats \enhances dual-vector reconstruction of minigenes in vivo. Hum Gene Ther. 2007;18(1):81-87.
    • (2007) Hum Gene Ther , vol.18 , Issue.1 , pp. 81-87
    • Yan, Z.1    Lei-Butters, D.C.2    Zhang, Y.3    Zak, R.4    Engelhardt, J.F.5
  • 51
    • 10344225049 scopus 로고    scopus 로고
    • Dual therapeutic utility of proteasome modulating agents for pharmaco-gene therapy of the cystic fibrosis airway
    • Zhang LN, Karp P, Gerard CJ, et al. Dual therapeutic utility of proteasome modulating agents for pharmaco-gene therapy of the cystic fibrosis airway. Mol Ther. 2004;10(6):990-1002.
    • (2004) Mol Ther , vol.10 , Issue.6 , pp. 990-1002
    • Zhang, L.N.1    Karp, P.2    Gerard, C.J.3
  • 52
    • 0034756451 scopus 로고    scopus 로고
    • Expanding AAV packaging capacity with trans-splicing or overlapping vectors: A quantitative comparison
    • Duan D, Yue Y, Engelhardt JF. Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison. Mol Ther. 2001;4(4):383-391.
    • (2001) Mol Ther , vol.4 , Issue.4 , pp. 383-391
    • Duan, D.1    Yue, Y.2    Engelhardt, J.F.3
  • 53
    • 0035989639 scopus 로고    scopus 로고
    • Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene
    • Halbert CL, Allen JM, Miller AD. Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nat Biotechnol. 2002;20(7):697-701.
    • (2002) Nat Biotechnol , vol.20 , Issue.7 , pp. 697-701
    • Halbert, C.L.1    Allen, J.M.2    Miller, A.D.3
  • 54
    • 62549141507 scopus 로고    scopus 로고
    • Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing
    • Song Y, Lou HH, Boyer JL, et al. Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing. Hum Gene Ther. 2009;20(3):267-281.
    • (2009) Hum Gene Ther , vol.20 , Issue.3 , pp. 267-281
    • Song, Y.1    Lou, H.H.2    Boyer, J.L.3
  • 55
    • 20044381100 scopus 로고    scopus 로고
    • A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia
    • Ostedgaard LS, Rokhlina T, Karp PH, et al. A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia. Proc Natl Acad Sci U S A. 2005; 102(8):2952-2957.
    • (2005) Proc Natl Acad Sci U S A , vol.102 , Issue.8 , pp. 2952-2957
    • Ostedgaard, L.S.1    Rokhlina, T.2    Karp, P.H.3
  • 56
    • 33947255488 scopus 로고    scopus 로고
    • Expression of a truncated cystic fibrosis transmembrane conductance regulator with an AAV5-pseudotyped vector in primates
    • Fischer AC, Smith CI, Cebotaru L, et al. Expression of a truncated cystic fibrosis transmembrane conductance regulator with an AAV5-pseudotyped vector in primates. Mol Ther. 2007;15(4): s756-763.
    • (2007) Mol Ther , vol.15 , Issue.4
    • Fischer, A.C.1    Smith, C.I.2    Cebotaru, L.3
  • 57
    • 38349091329 scopus 로고    scopus 로고
    • Partial correction of the CFTRdependent ABPA mouse model with recombinant adeno-associated virus gene transfer of truncated CFTR gene
    • Mueller C, Torrez D, Braag S, et al. Partial correction of the CFTRdependent ABPA mouse model with recombinant adeno-associated virus gene transfer of truncated CFTR gene. J Gene Med. 2008; 10(1):51-60.
    • (2008) J Gene Med , vol.10 , Issue.1 , pp. 51-60
    • Mueller, C.1    Torrez, D.2    Braag, S.3
  • 58
    • 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 Invest. 2008;118(5):1955-1964.
    • (2008) J Clin Invest , vol.118 , Issue.5 , pp. 1955-1964
    • Allocca, M.1    Doria, M.2    Petrillo, M.3
  • 59
    • 34248194614 scopus 로고    scopus 로고
    • High-efficiency promoter-dependent transduction by adeno-associated virus type 6 vectors in mouse lung
    • Halbert CL, Lam SL, Miller AD. High-efficiency promoter-dependent transduction by adeno-associated virus type 6 vectors in mouse lung. Hum Gene Ther. 2007;18(4):344-354.
    • (2007) Hum Gene Ther , vol.18 , Issue.4 , pp. 344-354
    • Halbert, C.L.1    Lam, S.L.2    Miller, A.D.3
  • 60
    • 0032853032 scopus 로고    scopus 로고
    • Repeated delivery of adenoassociated virus vectors to the rabbit airway
    • Beck SE, Jones LA, Chesnut K, et al. Repeated delivery of adenoassociated virus vectors to the rabbit airway. J Virol. 1999;73(11): 9446-9455.
    • (1999) J Virol , vol.73 , Issue.11 , pp. 9446-9455
    • Beck, S.E.1    Jones, L.A.2    Chesnut, K.3
  • 61
    • 0030795691 scopus 로고    scopus 로고
    • Transduction by adeno-associated virus vectors in the rabbit airway: Efficiency, persistence, and readministration
    • Halbert CL, Standaert TA, Aitken ML, Alexander IE, Russell DW, Miller AD. Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration. J Virol. 1997;71(8):5932-5941.
    • (1997) J Virol , 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
  • 62
    • 0033960160 scopus 로고    scopus 로고
    • Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes
    • Halbert CL, Rutledge EA, Allen JM, Russell DW, Miller AD. Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes. J Virol. 2000;74(3):1524-1532.
    • (2000) J Virol , 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
  • 63
    • 0036677468 scopus 로고    scopus 로고
    • Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins
    • Auricchio A, O'Connor E, Weiner D, et al. Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins. J Clin Invest. 2002;110(4):499-504.
    • (2002) J Clin Invest , vol.110 , Issue.4 , pp. 499-504
    • Auricchio, A.1    O'Connor, E.2    Weiner, D.3
  • 64
    • 0346656769 scopus 로고    scopus 로고
    • Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaques
    • Fischer AC, Beck SE, Smith CI, et al. Successful transgene expression with serial doses of aerosolized rAAV2 vectors in rhesus macaques. Mol Ther. 2003;8(6):918-926.
    • (2003) Mol Ther , vol.8 , Issue.6 , pp. 918-926
    • Fischer, A.C.1    Beck, S.E.2    Smith, C.I.3
  • 65
    • 77953128609 scopus 로고    scopus 로고
    • Expression of human alpha1-antitrypsin in mice and dogs following AAV6 vector-mediated gene transfer to the lungs
    • Halbert CL, Madtes DK, Vaughan AE, et al. Expression of human alpha1-antitrypsin in mice and dogs following AAV6 vector-mediated gene transfer to the lungs. Mol Ther. 2010;18(6): 1165-1172.
    • (2010) Mol Ther , vol.18 , Issue.6 , pp. 1165-1172
    • Halbert, C.L.1    Madtes, D.K.2    Vaughan, A.E.3
  • 66
    • 63649130139 scopus 로고    scopus 로고
    • Analysis of adeno-associated virus progenitor cell transduction in mouse lung
    • Liu X, Luo M, Guo C, et al. Analysis of adeno-associated virus progenitor cell transduction in mouse lung. Mol Ther. 2009;17(2): 285-293.
    • (2009) Mol Ther , vol.17 , Issue.2 , pp. 285-293
    • Liu, X.1    Luo, M.2    Guo, C.3
  • 67
    • 77952425348 scopus 로고    scopus 로고
    • IL-10 delivery by AAV5 vector attenuates inflammation in mice with pseudomonas pneumonia
    • Buff SM, Yu H, McCall JN, et al. IL-10 delivery by AAV5 vector attenuates inflammation in mice with pseudomonas pneumonia. Gene Ther. 2010;17(5):567-576.
    • (2010) Gene Ther , vol.17 , Issue.5 , pp. 567-576
    • Buff, S.M.1    Yu, H.2    McCall, J.N.3
  • 68
    • 76749131499 scopus 로고    scopus 로고
    • In vitro and in vivo functional characterization of gutless recombinant SV40-derived CFTR vectors
    • Mueller C, Strayer MS, Sirninger J, et al. In vitro and in vivo functional characterization of gutless recombinant SV40-derived CFTR vectors. Gene Ther. 2010;17(2):227-237.
    • (2010) Gene Ther , vol.17 , Issue.2 , pp. 227-237
    • Mueller, C.1    Strayer, M.S.2    Sirninger, J.3
  • 69
    • 0031834248 scopus 로고    scopus 로고
    • Use of SV40 to immunize against hepatitis B surface antigen: Implications for the use of SV40 for gene transduction and its use as an immunizing agent
    • Kondo R, Feitelson MA, Strayer DS. Use of SV40 to immunize against hepatitis B surface antigen: implications for the use of SV40 for gene transduction and its use as an immunizing agent. Gene Ther. 1998;5(5):575-582.
    • (1998) Gene Ther , vol.5 , Issue.5 , pp. 575-582
    • Kondo, R.1    Feitelson, M.A.2    Strayer, D.S.3
  • 70
    • 11144227707 scopus 로고    scopus 로고
    • Infection of ciliated cells by human parainfluenza virus type 3 in an in vitro model of human airway epithelium
    • Zhang L, Bukreyev A, Thompson CI, et al. Infection of ciliated cells by human parainfluenza virus type 3 in an in vitro model of human airway epithelium. J Virol. 2005;79(2):1113-1124.
    • (2005) J Virol , vol.79 , Issue.2 , pp. 1113-1124
    • Zhang, L.1    Bukreyev, A.2    Thompson, C.I.3
  • 71
    • 68049143467 scopus 로고    scopus 로고
    • CFTR delivery to 25% of surface epithelial cells restores normal rates of mucus transport to human cystic fibrosis airway epithelium
    • Zhang L, Button B, Gabriel SE, et al. CFTR delivery to 25% of surface epithelial cells restores normal rates of mucus transport to human cystic fibrosis airway epithelium. PLoS Biol. 2009;7(7):e1000155.
    • (2009) PLoS Biol , vol.7 , Issue.7
    • Zhang, L.1    Button, B.2    Gabriel, S.E.3
  • 72
    • 77954514975 scopus 로고    scopus 로고
    • Respiratory syncytial virus engineered to express the cystic fibrosis transmembrane conductance regulator corrects the bioelectric phenotype of human cystic fibrosis airway epithelium in vitro
    • Kwilas AR, Yednak MA, Zhang L, et al. Respiratory syncytial virus engineered to express the cystic fibrosis transmembrane conductance regulator corrects the bioelectric phenotype of human cystic fibrosis airway epithelium in vitro. J Virol. 2010;84(15):7770-7781.
    • (2010) J Virol , vol.84 , Issue.15 , pp. 7770-7781
    • Kwilas, A.R.1    Yednak, M.A.2    Zhang, L.3
  • 73
    • 0027164674 scopus 로고
    • Sendai virus-induced cell fusion
    • Okada Y. Sendai virus-induced cell fusion. Methods Enzymol. 1993; 221:18-41.
    • (1993) Methods Enzymol , vol.221 , pp. 18-41
    • Okada, Y.1
  • 74
    • 34548730279 scopus 로고    scopus 로고
    • Sendai virus-mediated CFTR gene transfer to the airway epithelium
    • Ferrari S, Griesenbach U, Iida A, et al. Sendai virus-mediated CFTR gene transfer to the airway epithelium. Gene Ther. 2007; 14(19):1371-1379.
    • (2007) Gene Ther , vol.14 , Issue.19 , pp. 1371-1379
    • Ferrari, S.1    Griesenbach, U.2    Iida, A.3
  • 75
    • 0036166302 scopus 로고    scopus 로고
    • The nasal epithelium as a factory for systemic protein delivery
    • Griesenbach U, Cassady RL, Ferrari S, et al. The nasal epithelium as a factory for systemic protein delivery. Mol Ther. 2002;5(2):98-103.
    • (2002) Mol Ther , vol.5 , Issue.2 , pp. 98-103
    • Griesenbach, U.1    Cassady, R.L.2    Ferrari, S.3
  • 76
    • 0033947169 scopus 로고    scopus 로고
    • A cytoplasmic RNA vector derived from nontransmissible Sendai virus with efficient gene transfer and expression
    • Li HO, Zhu YF, Asakawa M, et al. A cytoplasmic RNA vector derived from nontransmissible Sendai virus with efficient gene transfer and expression. J Virol. 2000;74(14):6564-6569.
    • (2000) J Virol , vol.74 , Issue.14 , pp. 6564-6569
    • Li, H.O.1    Zhu, Y.F.2    Asakawa, M.3
  • 77
    • 0037370625 scopus 로고    scopus 로고
    • Nontransmissible virus-like particle formation by F-deficient Sendai virus is temperature sensitive and reduced by mutations in M and HN proteins
    • Inoue M, Tokusumi Y, Ban H, et al. Nontransmissible virus-like particle formation by F-deficient Sendai virus is temperature sensitive and reduced by mutations in M and HN proteins. J Virol. 2003;77(5):3238-3246.
    • (2003) J Virol , vol.77 , Issue.5 , pp. 3238-3246
    • Inoue, M.1    Tokusumi, Y.2    Ban, H.3
  • 78
    • 79951514000 scopus 로고    scopus 로고
    • Validation of recombinant Sendai virus in a non-natural host model
    • In press
    • Griesenbach U, McLachlan G, Owaki T, et al. Validation of recombinant Sendai virus in a non-natural host model. Gene Ther. 2010;In press.
    • (2010) Gene Ther
    • Griesenbach, U.1    McLachlan, G.2    Owaki, T.3
  • 79
    • 33745755929 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: Cystic fibrosis
    • Griesenbach U, Geddes DM, Alton EW. Gene therapy progress and prospects: cystic fibrosis. Gene Ther. 2006;13(14):1061-1067.
    • (2006) Gene Ther , vol.13 , Issue.14 , pp. 1061-1067
    • Griesenbach, U.1    Geddes, D.M.2    Alton, E.W.3
  • 80
    • 47249101463 scopus 로고    scopus 로고
    • HVJ envelope vector, a versatile delivery system: Its development, application, and perspectives
    • Zhang Q, Li Y, Shi Y, Zhang Y. HVJ envelope vector, a versatile delivery system: its development, application, and perspectives. Biochem Biophys Res Commun. 2008;373(3):345-349.
    • (2008) Biochem Biophys Res Commun , vol.373 , Issue.3 , pp. 345-349
    • Zhang, Q.1    Li, Y.2    Shi, Y.3    Zhang, Y.4
  • 81
    • 0036664449 scopus 로고    scopus 로고
    • Hemagglutinating virus of Japan (HVJ) envelope vector as a versatile gene delivery system
    • Kaneda Y, Nakajima T, Nishikawa T, et al. Hemagglutinating virus of Japan (HVJ) envelope vector as a versatile gene delivery system. Mol Ther. 2002;6(2):219-226.
    • (2002) Mol Ther , vol.6 , Issue.2 , pp. 219-226
    • Kaneda, Y.1    Nakajima, T.2    Nishikawa, T.3
  • 82
    • 70450265981 scopus 로고    scopus 로고
    • Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome
    • Fusaki N, Ban H, Nishiyama A, Saeki K, Hasegawa M. Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome. Proc Jpn Acad Ser B Phys Biol Sci. 2009;85(8): 348-362.
    • (2009) Proc Jpn Acad Ser B Phys Biol Sci , vol.85 , Issue.8 , pp. 348-362
    • Fusaki, N.1    Ban, H.2    Nishiyama, A.3    Saeki, K.4    Hasegawa, M.5
  • 83
    • 77957671713 scopus 로고    scopus 로고
    • Generation of induced pluripotent stem cells from human terminally differentiated circulating T cells
    • Seki T, Yuasa S, Oda M, et al. Generation of induced pluripotent stem cells from human terminally differentiated circulating T cells. Cell Stem Cell. 2010;7(1):11-14.
    • (2010) Cell Stem Cell , vol.7 , Issue.1 , pp. 11-14
    • Seki, T.1    Yuasa, S.2    Oda, M.3
  • 84
    • 37349084579 scopus 로고    scopus 로고
    • CFTR gene transfer to human cystic fibrosis pancreatic duct cells using a Sendai virus vector
    • Rakonczay Z Jr, Hegyi P, Hasegawa M, et al. CFTR gene transfer to human cystic fibrosis pancreatic duct cells using a Sendai virus vector. J Cell Physiol. 2008;214(2):442-455.
    • (2008) J Cell Physiol , vol.214 , Issue.2 , pp. 442-455
    • Rakonczay Jr., Z.1    Hegyi, P.2    Hasegawa, M.3
  • 85
    • 71049152079 scopus 로고    scopus 로고
    • CFTR expression but not Cl- transport is involved in the stimulatory effect of bile acids on apical Cl-/HCO3- exchange activity in human pancreatic duct cells
    • Ignath I, Hegyi P, Venglovecz V, et al. CFTR expression but not Cl- transport is involved in the stimulatory effect of bile acids on apical Cl-/HCO3- exchange activity in human pancreatic duct cells. \Pancreas. 2009;38(8):921-929.
    • (2009) Pancreas , vol.38 , Issue.8 , pp. 921-929
    • Ignath, I.1    Hegyi, P.2    Venglovecz, V.3
  • 86
    • 0027099652 scopus 로고
    • In vivo retroviral gene transfer into human bronchial epithelia of xenografts
    • Engelhardt JF, Yankaskas JR, Wilson JM. In vivo retroviral gene transfer into human bronchial epithelia of xenografts. J Clin Invest. 1992;90(6):2598-2607.
    • (1992) J Clin Invest , vol.90 , Issue.6 , pp. 2598-2607
    • Engelhardt, J.F.1    Yankaskas, J.R.2    Wilson, J.M.3
  • 87
    • 0037320020 scopus 로고    scopus 로고
    • Pseudotyped lentivirus vectors derived from simian immunodeficiency virus SIVagm with envelope glycoproteins from paramyxovirus
    • Kobayashi M, Iida A, Ueda Y, Hasegawa M. Pseudotyped lentivirus vectors derived from simian immunodeficiency virus SIVagm with envelope glycoproteins from paramyxovirus. J Virol. 2003;77(4):2607-2614.
    • (2003) J Virol , vol.77 , Issue.4 , pp. 2607-2614
    • Kobayashi, M.1    Iida, A.2    Ueda, Y.3    Hasegawa, M.4
  • 88
    • 77953138185 scopus 로고    scopus 로고
    • Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes
    • Mitomo K, Griesenbach U, Inoue M, et al. Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes. Mol Ther. 2010;18(6):1173-1182.
    • (2010) Mol Ther , vol.18 , Issue.6 , pp. 1173-1182
    • Mitomo, K.1    Griesenbach, U.2    Inoue, M.3
  • 89
    • 55249121439 scopus 로고    scopus 로고
    • Lentivirus vector can be readministered to nasal epithelia without blocking immune responses
    • Sinn PL, Arias AC, Brogden KA, McCray PB Jr. Lentivirus vector can be readministered to nasal epithelia without blocking immune responses. J Virol. 2008;82(21):10684-10692.
    • (2008) J Virol , vol.82 , Issue.21 , pp. 10684-10692
    • Sinn, P.L.1    Arias, A.C.2    Brogden, K.A.3    McCray Jr., P.B.4
  • 90
    • 33645767668 scopus 로고    scopus 로고
    • Influenza M2 envelope protein augments avian influenza hemagglutinin pseudotyping of lentiviral vectors
    • McKay T, Patel M, Pickles RJ, Johnson LG, Olsen JC. Influenza M2 envelope protein augments avian influenza hemagglutinin pseudotyping of lentiviral vectors. Gene Ther. 2006;13(8):715-724.
    • (2006) Gene Ther , vol.13 , Issue.8 , pp. 715-724
    • McKay, T.1    Patel, M.2    Pickles, R.J.3    Johnson, L.G.4    Olsen, J.C.5
  • 91
    • 22744455256 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: Development of improved lentiviral and retroviral vectors-design, biosafety, and production
    • Sinn PL, Sauter SL, McCray PB Jr. Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors-design, biosafety, and production. Gene Ther. 2005;12(14):1089-1098.
    • (2005) Gene Ther , vol.12 , Issue.14 , pp. 1089-1098
    • Sinn, P.L.1    Sauter, S.L.2    McCray Jr., P.B.3
  • 92
    • 0036431592 scopus 로고    scopus 로고
    • Recovery of airway cystic fibrosis transmembrane conductance regulator function in mice with cystic fibrosis after single-dose lentivirus-mediated gene transfer
    • Limberis M, Anson DS, Fuller M, Parsons DW. Recovery of airway cystic fibrosis transmembrane conductance regulator function in mice with cystic fibrosis after single-dose lentivirus-mediated gene transfer. Hum Gene Ther. 2002;13(16):1961-1970.
    • (2002) Hum Gene Ther , vol.13 , Issue.16 , pp. 1961-1970
    • Limberis, M.1    Anson, D.S.2    Fuller, M.3    Parsons, D.W.4
  • 93
    • 77956667210 scopus 로고    scopus 로고
    • Lysophosphatidylcholine as an adjuvant for lentiviral vector mediated gene transfer to airway epithelium: Effect of acyl chain length
    • Cmielewski P, Anson DS, Parsons DW. Lysophosphatidylcholine as an adjuvant for lentiviral vector mediated gene transfer to airway epithelium: effect of acyl chain length. Respir Res. 2010; 11(1):84.
    • (2010) Respir Res , vol.11 , Issue.1 , pp. 84
    • Cmielewski, P.1    Anson, D.S.2    Parsons, D.W.3
  • 94
    • 0347503527 scopus 로고    scopus 로고
    • Lentiviral vectors pseudotyped with minimal filovirus envelopes increased gene transfer in murine lung
    • Medina MF, Kobinger GP, Rux J, et al. Lentiviral vectors pseudotyped with minimal filovirus envelopes increased gene transfer in murine lung. Mol Ther. 2003;8(5):777-789.
    • (2003) Mol Ther , vol.8 , Issue.5 , pp. 777-789
    • Medina, M.F.1    Kobinger, G.P.2    Rux, J.3
  • 96
    • 49049114350 scopus 로고    scopus 로고
    • Ciliated epithelial cell lifespan in the mouse trachea and lung
    • Rawlins EL, Hogan BL. Ciliated epithelial cell lifespan in the mouse trachea and lung. Am J Physiol Lung Cell Mol Physiol. 2008; 295(1):L231-L234.
    • (2008) Am J Physiol Lung Cell Mol Physiol , vol.295 , Issue.1
    • Rawlins, E.L.1    Hogan, B.L.2
  • 97
    • 84889752335 scopus 로고    scopus 로고
    • Repeat administration of Sendai virus-F/HN pseudotyped lentivirus vector to the respiratory epithelium is feasible [abstract]
    • Griesenbach U, Mitomo K, Inoue M, et al. Repeat administration of Sendai virus-F/HN pseudotyped lentivirus vector to the respiratory epithelium is feasible [abstract]. Mol Ther. 2008;1(16):A754.
    • (2008) Mol Ther , vol.1 , Issue.16
    • Griesenbach, U.1    Mitomo, K.2    Inoue, M.3
  • 98
    • 51349158298 scopus 로고    scopus 로고
    • Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
    • Howe SJ, Mansour MR, Schwarzwaelder K, et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest. 2008;118(9):3143-3150.
    • (2008) J Clin Invest , vol.118 , Issue.9 , pp. 3143-3150
    • Howe, S.J.1    Mansour, M.R.2    Schwarzwaelder, K.3
  • 99
    • 0031743608 scopus 로고    scopus 로고
    • Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
    • Zufferey R, Dull T, Mandel RJ, et al. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J Virol. 1998; 72(12):9873-9880.
    • (1998) J Virol , vol.72 , Issue.12 , pp. 9873-9880
    • Zufferey, R.1    Dull, T.2    Mandel, R.J.3
  • 100
    • 65249101260 scopus 로고    scopus 로고
    • The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy
    • Montini E, Cesana D, Schmidt M, et al. The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J Clin Invest. 2009;119(4):964-975.
    • (2009) J Clin Invest , vol.119 , Issue.4 , pp. 964-975
    • Montini, E.1    Cesana, D.2    Schmidt, M.3
  • 101
    • 68249112926 scopus 로고    scopus 로고
    • Integration-deficient lentiviral vectors: A slow coming of age
    • Wanisch K, Yanez-Munoz RJ. Integration-deficient lentiviral vectors: a slow coming of age. Mol Ther. 2009;17(8):1316-1332.
    • (2009) Mol Ther , vol.17 , Issue.8 , pp. 1316-1332
    • Wanisch, K.1    Yanez-Munoz, R.J.2
  • 102
    • 64649102398 scopus 로고    scopus 로고
    • Site-specific integration of retroviral DNA in human cells using fusion proteins consisting of human immunodeficiency virus type 1 integrase and the designed polydactyl zinc-finger protein E2C
    • Su K, Wang D, Ye J, Kim YC, Chow SA. Site-specific integration of retroviral DNA in human cells using fusion proteins consisting of human immunodeficiency virus type 1 integrase and the designed polydactyl zinc-finger protein E2C. Methods. 2009;47(4): 269-276.
    • (2009) Methods , vol.47 , Issue.4 , pp. 269-276
    • Su, K.1    Wang, D.2    Ye, J.3    Kim, Y.C.4    Chow, S.A.5
  • 103
    • 64749107970 scopus 로고    scopus 로고
    • Production, concentration and titration of pseudotyped HIV-1-based lentiviral vectors
    • Kutner RH, Zhang XY, Reiser J. Production, concentration and titration of pseudotyped HIV-1-based lentiviral vectors. Nat Protoc. 2009;4(4):495-505.
    • (2009) Nat Protoc , vol.4 , Issue.4 , pp. 495-505
    • Kutner, R.H.1    Zhang, X.Y.2    Reiser, J.3
  • 105
    • 77950606953 scopus 로고    scopus 로고
    • The Adaptive Aerosol Delivery (AAD) technology:Past, present, and future
    • Denyer J, Dyche T. The Adaptive Aerosol Delivery (AAD) technology:past, present, and future. J Aerosol Med Pulm Drug Deliv. 2010;23 Suppl 1:S1-S10.
    • (2010) J Aerosol Med Pulm Drug Deliv , vol.23 , Issue.1
    • Denyer, J.1    Dyche, T.2
  • 106
    • 0036797279 scopus 로고    scopus 로고
    • Deposition and expression of aerosolized rAAV vectors in the lungs of Rhesus macaques
    • Beck SE, Laube BL, Barberena CI, et al. Deposition and expression of aerosolized rAAV vectors in the lungs of Rhesus macaques. Mol Ther. 2002;6(4):546-554.
    • (2002) Mol Ther , vol.6 , Issue.4 , pp. 546-554
    • Beck, S.E.1    Laube, B.L.2    Barberena, C.I.3
  • 107
    • 48949105555 scopus 로고    scopus 로고
    • Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression
    • Buckley SM, Howe SJ, Sheard V, et al. Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression. Gene Ther. 2008; 15(16):1167-1175.
    • (2008) Gene Ther , vol.15 , Issue.16 , pp. 1167-1175
    • Buckley, S.M.1    Howe, S.J.2    Sheard, V.3
  • 108
    • 79251484053 scopus 로고    scopus 로고
    • Genetic modification of airway progenitors following lentiviral gene delivery to the amniotic fluid of murine fetuses
    • Jun 25 [Epub ahead of print
    • Mishra S, Wang X, Smiley N, et al. Genetic modification of airway progenitors following lentiviral gene delivery to the amniotic fluid of murine fetuses. Am J Respir Cell Mol Biol. 2010: Jun 25 [Epub ahead of print].
    • (2010) Am J Respir Cell Mol Biol
    • Mishra, S.1    Wang, X.2    Smiley, N.3
  • 110
    • 74949132576 scopus 로고    scopus 로고
    • Amelioration of emphysema in mice through lentiviral transduction of long-lived pulmonary alveolar macrophages
    • Wilson AA, Murphy GJ, Hamakawa H, et al. Amelioration of emphysema in mice through lentiviral transduction of long-lived pulmonary alveolar macrophages. J Clin Invest. 2010;120(1): 379-389.
    • (2010) J Clin Invest , vol.120 , Issue.1 , pp. 379-389
    • Wilson, A.A.1    Murphy, G.J.2    Hamakawa, H.3
  • 111
    • 33846907545 scopus 로고    scopus 로고
    • Alpha1-Antitrypsin inhalation reduces airway inflammation in cystic fibrosis patients
    • Griese M, Latzin P, Kappler M, et al. Alpha1-Antitrypsin inhalation reduces airway inflammation in cystic fibrosis patients. Eur Respir J. 2007;29(2):240-250.
    • (2007) Eur Respir J , vol.29 , Issue.2 , pp. 240-250
    • Griese, M.1    Latzin, P.2    Kappler, M.3
  • 112
    • 62249100734 scopus 로고    scopus 로고
    • Short hairpin RNAs against eotaxin or interleukin-5 decrease airway eosinophilia and hyper-responsiveness in a murine model of asthma
    • Huang HY, Lee CC, Chiang BL. Short hairpin RNAs against eotaxin or interleukin-5 decrease airway eosinophilia and hyper-responsiveness in a murine model of asthma. J Gene Med. 2009;11(2): 112-118.
    • (2009) J Gene Med , vol.11 , Issue.2 , pp. 112-118
    • Huang, H.Y.1    Lee, C.C.2    Chiang, B.L.3
  • 113
    • 77953953690 scopus 로고    scopus 로고
    • Lentiviral vector-mediated expression of pre-miRNAs and antagomiRs
    • Scherr M, Venturini L, Eder M. Lentiviral vector-mediated expression of pre-miRNAs and antagomiRs. Methods Mol Biol. 2010;614: 175-185.
    • (2010) Methods Mol Biol , vol.614 , pp. 175-185
    • Scherr, M.1    Venturini, L.2    Eder, M.3
  • 114
    • 2442718786 scopus 로고    scopus 로고
    • Increased airway epithelial Na+ absorption produces cystic fibrosis-like lung disease in mice
    • Mall M, Grubb BR, Harkema JR, O'Neal WK, Boucher RC. Increased airway epithelial Na+ absorption produces cystic fibrosis-like lung disease in mice. Nat Med. 2004;10(5):487-493.
    • (2004) Nat Med , vol.10 , Issue.5 , pp. 487-493
    • Mall, M.1    Grubb, B.R.2    Harkema, J.R.3    O'Neal, W.K.4    Boucher, R.C.5
  • 115
    • 27944483003 scopus 로고    scopus 로고
    • Mutations in the beta-subunit of the epithelial Na+ channel in patients with a cystic fibrosis-like syndrome
    • Sheridan MB, Fong P, Groman JD, et al. Mutations in the beta-subunit of the epithelial Na+ channel in patients with a cystic fibrosis-like syndrome. Hum Mol Genet. 2005;14(22):3493-3498.
    • (2005) Hum Mol Genet , vol.14 , Issue.22 , pp. 3493-3498
    • Sheridan, M.B.1    Fong, P.2    Groman, J.D.3
  • 116
    • 33745759579 scopus 로고    scopus 로고
    • Endpoint assay development and validation of non-viral gene transfer agents in CF mice [abstract]
    • Griesenbach U, Wasowicz MY, Smith SN, et al. Endpoint assay development and validation of non-viral gene transfer agents in CF mice [abstract]. Pediatr Pulmol. 2005;S28:270.
    • (2005) Pediatr Pulmol , vol.S28 , pp. 270
    • Griesenbach, U.1    Wasowicz, M.Y.2    Smith, S.N.3


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