메뉴 건너뛰기




Volumn 17, Issue 12, 2009, Pages 2067-2077

Generation of novel aav variants by directed evolution for improved cftr delivery to human ciliated airway epithelium

Author keywords

[No Author keywords available]

Indexed keywords

CHLORIDE ION; PARVOVIRUS VECTOR; TRANSMEMBRANE CONDUCTANCE REGULATOR;

EID: 73849130017     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2009.155     Document Type: Article
Times cited : (78)

References (42)
  • 1
    • 0027404779 scopus 로고
    • Pathogenesis of cystic fbrosis
    • Koch, C and Høiby, N (1993). Pathogenesis of cystic fbrosis. Lancet 341: 1065-1069.
    • (1993) Lancet , vol.341 , pp. 1065-1069
    • Koch, C.1    Høiby, N.2
  • 2
    • 34548512049 scopus 로고    scopus 로고
    • Repeated aerosolized AAV-CFTR for treatment of cystic fbrosis: A randomized placebo-controlled phase 2B trial
    • Moss, RB, Milla, C, Colombo, J, Accurso, F, Zeitlin, PL, Clancy, JP et al. (2007). Repeated aerosolized AAV-CFTR for treatment of cystic fbrosis: a randomized placebo-controlled phase 2B trial. Hum Gene Ther 18: 726-732.
    • (2007) Hum Gene Ther , vol.18 , pp. 726-732
    • Moss, R.B.1    Milla, C.2    Colombo, J.3    Accurso, F.4    Zeitlin, P.L.5    Clancy, J.P.6
  • 3
    • 21244463942 scopus 로고    scopus 로고
    • Recent developments in recombinant AAV-mediated gene therapy for lung diseases
    • Flotte, TR (2005). Recent developments in recombinant AAV-mediated gene therapy for lung diseases. Curr Gene Ther 5: 361-366.
    • (2005) Curr Gene Ther , vol.5 , pp. 361-366
    • Flotte, T.R.1
  • 4
    • 0034121397 scopus 로고    scopus 로고
    • A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation
    • Duan, D, Yue, Y, Yan, Z and Engelhardt, JF (2000). A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation. Nat Med 6: 595-598.
    • (2000) Nat Med , vol.6 , pp. 595-598
    • Duan, D.1    Yue, Y.2    Yan, Z.3    Engelhardt, J.F.4
  • 5
    • 33749570518 scopus 로고    scopus 로고
    • Unique biologic properties of recombinant AAV1 transduction in polarized human airway epithelia
    • Yan, Z, Lei-Butters, DC, Liu, X, Zhang, Y, Zhang, L, Luo, M et al. (2006). Unique biologic properties of recombinant AAV1 transduction in polarized human airway epithelia. J Biol Chem 281: 29684-29692.
    • (2006) J Biol Chem , vol.281 , pp. 29684-29692
    • Yan, Z.1    Lei-Butters, D.C.2    Liu, X.3    Zhang, Y.4    Zhang, L.5    Luo, M.6
  • 7
    • 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
    • Zabner, J, Seiler, M, Walters, R, Kotin, RM, Fulgeras, W, Davidson, BL 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.
    • (2000) J Virol , vol.74 , pp. 3852-3858
    • Zabner, J.1    Seiler, M.2    Walters, R.3    Kotin, R.M.4    Fulgeras, W.5    Davidson, B.L.6
  • 8
    • 0034992340 scopus 로고    scopus 로고
    • Adeno-associated virus type 6 (AAV6) vectors mediate effcient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors
    • Halbert, CL, Allen, JM and Miller, AD (2001). Adeno-associated virus type 6 (AAV6) vectors mediate effcient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors. J Virol 75: 6615-6624.
    • (2001) J Virol , vol.75 , pp. 6615-6624
    • Halbert, C.L.1    Allen, J.M.2    Miller, A.D.3
  • 9
    • 33748338996 scopus 로고    scopus 로고
    • Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered
    • Limberis, MP and Wilson, JM (2006). Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered. Proc Natl Acad Sci USA 103: 12993-12998.
    • (2006) Proc Natl Acad Sci USA , vol.103 , pp. 12993-12998
    • Limberis, M.P.1    Wilson, J.M.2
  • 10
    • 45749099260 scopus 로고    scopus 로고
    • Engineering and selection of shuffed AAV genomes: A new strategy for producing targeted biological nanoparticles
    • Li, W, Asokan, A, Wu, Z, Van Dyke, T, DiPrimio, N, Johnson, JS et al. (2008). Engineering and selection of shuffed AAV genomes: a new strategy for producing targeted biological nanoparticles. Mol Ther 16: 1252-1260.
    • (2008) Mol Ther , vol.16 , pp. 1252-1260
    • Li, W.1    Asokan, A.2    Wu, Z.3    Van Dyke, T.4    Diprimio, N.5    Johnson, J.S.6
  • 11
    • 44949131860 scopus 로고    scopus 로고
    • In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses
    • Grimm, D, Lee, JS, Wang, L, Desai, T, Akache, B, Storm, TA et al. (2008). In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J Virol 82: 5887-5911.
    • (2008) J Virol , vol.82 , pp. 5887-5911
    • Grimm, D.1    Lee, J.S.2    Wang, L.3    Desai, T.4    Akache, B.5    Storm, T.A.6
  • 13
    • 20044381100 scopus 로고    scopus 로고
    • A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fbrosis airway epithelia
    • Ostedgaard, LS, Rokhlina, T, Karp, PH, Lashmit, P, Afone, S, Schmidt, M et al. (2005). A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fbrosis airway epithelia. Proc Natl Acad Sci USA 102: 2952-2957.
    • (2005) Proc Natl Acad Sci USA , vol.102 , pp. 2952-2957
    • Ostedgaard, L.S.1    Rokhlina, T.2    Karp, P.H.3    Lashmit, P.4    Afone, S.5    Schmidt, M.6
  • 14
    • 0018581742 scopus 로고
    • Isolation of adenovirus type 5 host range deletion mutants defective for transformation of rat embryo cells
    • Jones, N and Shenk, T (1979). Isolation of adenovirus type 5 host range deletion mutants defective for transformation of rat embryo cells. Cell 17: 683-689. (Pubitemid 9242344)
    • (1979) Cell , vol.17 , Issue.3 , pp. 683-689
    • Jones, N.1    Shenk, T.2
  • 15
    • 0031777961 scopus 로고    scopus 로고
    • Limited entry of adenovirus vectors into well-differentiated airway epithelium is responsible for ineffcient gene transfer
    • Pickles, RJ, McCarty, D, Matsui, H, Hart, PJ, Randell, SH and Boucher, RC (1998). Limited entry of adenovirus vectors into well-differentiated airway epithelium is responsible for ineffcient gene transfer. J Virol 72: 6014-6023.
    • (1998) J Virol , vol.72 , 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
  • 16
    • 0033537837 scopus 로고    scopus 로고
    • Basolateral localization of fber receptors limits adenovirus infection from the apical surface of airway epithelia
    • Walters, RW, Grunst, T, Bergelson, JM, Finberg, RW, Welsh, MJ and Zabner, J (1999). Basolateral localization of fber receptors limits adenovirus infection from the apical surface of airway epithelia. J Biol Chem 274: 10219-10226.
    • (1999) J Biol Chem , vol.274 , 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
  • 17
    • 0033747755 scopus 로고    scopus 로고
    • Enhanced epithelial gene transfer by modulation of tight junctions with sodium caprate
    • Coyne, CB, Kelly, MM, Boucher, RC and Johnson, LG (2000). Enhanced epithelial gene transfer by modulation of tight junctions with sodium caprate. Am J Respir Cell Mol Biol 23: 602-609.
    • (2000) Am J Respir Cell Mol Biol , vol.23 , pp. 602-609
    • Coyne, C.B.1    Kelly, M.M.2    Boucher, R.C.3    Johnson, L.G.4
  • 18
    • 0034091579 scopus 로고    scopus 로고
    • Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus
    • Duan, D, Yue, Y, Yan, Z, Yang, J and Engelhardt, JF (2000). Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J Clin Invest 105: 1573-1587.
    • (2000) J Clin Invest , vol.105 , pp. 1573-1587
    • Duan, D.1    Yue, Y.2    Yan, Z.3    Yang, J.4    Engelhardt, J.F.5
  • 19
    • 1542287505 scopus 로고    scopus 로고
    • Distinct classes of proteasome-modulating agents cooperatively augment recombinant adeno-associated virus type 2 and type 5-mediated transduction from the apical surfaces of human airway epithelia
    • Yan, Z, Zak, R, Zhang, Y, Ding, W, Godwin, S, Munson, K et al. (2004). Distinct classes of proteasome-modulating agents cooperatively augment recombinant adeno-associated virus type 2 and type 5-mediated transduction from the apical surfaces of human airway epithelia. J Virol 78: 2863-2874.
    • (2004) J Virol , vol.78 , pp. 2863-2874
    • Yan, Z.1    Zak, R.2    Zhang, Y.3    Ding, W.4    Godwin, S.5    Munson, K.6
  • 20
    • 0032992225 scopus 로고    scopus 로고
    • Transduction of well-differentiated airway epithelium by recombinant adeno-associated virus is limited by vector entry
    • Bals, R, Xiao, W, Sang, N, Weiner, DJ, Meegalla, RL and Wilson, JM (1999). Transduction of well-differentiated airway epithelium by recombinant adeno-associated virus is limited by vector entry. J Virol 73: 6085-6088.
    • (1999) J Virol , vol.73 , pp. 6085-6088
    • Bals, R.1    Xiao, W.2    Sang, N.3    Weiner, D.J.4    Meegalla, R.L.5    Wilson, J.M.6
  • 21
    • 57749190573 scopus 로고    scopus 로고
    • Genetic modifcation of AAV2 capsid enhances gene transfer effciency in polarized human airway epithelial cells
    • epub ahead of print
    • White, AF, Mazur, M, Sorscher, EJ, Zinn, K and Ponnazhagan, S (2008). Genetic modifcation of AAV2 capsid enhances gene transfer effciency in polarized human airway epithelial cells. Hum Gene Ther (epub ahead of print).
    • (2008) Hum Gene Ther
    • White, A.F.1    Mazur, M.2    Sorscher, E.J.3    Zinn, K.4    Ponnazhagan, S.5
  • 22
    • 0036140216 scopus 로고    scopus 로고
    • Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specifcity
    • Rabinowitz, JE, Rolling, F, Li, C, Conrath, H, Xiao, W, Xiao, X et al. (2002). Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specifcity. J Virol 76: 791-801.
    • (2002) J Virol , vol.76 , pp. 791-801
    • Rabinowitz, J.E.1    Rolling, F.2    Li, C.3    Conrath, H.4    Xiao, W.5    Xiao, X.6
  • 23
    • 0037213899 scopus 로고    scopus 로고
    • Marker rescue of adeno-associated virus (AAV) capsid mutants: A novel approach for chimeric AAV production
    • Bowles, DE, Rabinowitz, JE and Samulski, RJ (2003). Marker rescue of adeno-associated virus (AAV) capsid mutants: a novel approach for chimeric AAV production. J Virol 77: 423-432.
    • (2003) J Virol , vol.77 , pp. 423-432
    • Bowles, D.E.1    Rabinowitz, J.E.2    Samulski, R.J.3
  • 24
    • 32344438754 scopus 로고    scopus 로고
    • Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
    • Maheshri, N, Koerber, JT, Kaspar, BK and Schaffer, DV (2006). Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol 24: 198-204.
    • (2006) Nat Biotechnol , vol.24 , pp. 198-204
    • Maheshri, N.1    Koerber, J.T.2    Kaspar, B.K.3    Schaffer, D.V.4
  • 25
    • 32844470411 scopus 로고    scopus 로고
    • Combinatorial engineering of a gene therapy vector: Directed evolution of adeno-associated virus
    • Perabo, L, Endell, J, King, S, Lux, K, Goldnau, D, Hallek, M et al. (2006). Combinatorial engineering of a gene therapy vector: directed evolution of adeno-associated virus. J Gene Med 8: 155-162.
    • (2006) J Gene Med , vol.8 , pp. 155-162
    • Perabo, L.1    Endell, J.2    King, S.3    Lux, K.4    Goldnau, D.5    Hallek, M.6
  • 26
    • 0042360384 scopus 로고    scopus 로고
    • Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors
    • Müller, OJ, Kaul, F, Weitzman, MD, Pasqualini, R, Arap, W, Kleinschmidt, JA et al. (2003). Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors. Nat Biotechnol 21: 1040-1046.
    • (2003) Nat Biotechnol , vol.21 , pp. 1040-1046
    • Müller, O.J.1    Kaul, F.2    Weitzman, M.D.3    Pasqualini, R.4    Arap, W.5    Kleinschmidt, J.A.6
  • 27
    • 0029962053 scopus 로고    scopus 로고
    • In vitro assessment of variables affecting the effciency and effcacy of adenovirus-mediated gene transfer to cystic fbrosis airway epithelia
    • Johnson, LG, Pickles, RJ, Boyles, SE, Morris, JC, Ye, H, Zhou, Z et al. (1996). In vitro assessment of variables affecting the effciency and effcacy of adenovirus-mediated gene transfer to cystic fbrosis airway epithelia. Hum Gene Ther 7: 51-59.
    • (1996) Hum Gene Ther , vol.7 , pp. 51-59
    • Johnson, L.G.1    Pickles, R.J.2    Boyles, S.E.3    Morris, J.C.4    Ye, H.5    Zhou, Z.6
  • 28
    • 68049143467 scopus 로고    scopus 로고
    • CFTR delivery to 25% of surface epithelial cells restores normal rates of mucus transport to human cystic fbrosis airway epithelium
    • in press
    • Liqun, Z, Brian, B, Sherif, EG, Susan, B, Yu, Y, Skiadopoulos, MH et al. (2009). CFTR delivery to 25% of surface epithelial cells restores normal rates of mucus transport to human cystic fbrosis airway epithelium. PLoS Biol (in press).
    • (2009) PLoS Biol
    • Liqun, Z.1    Brian, B.2    Sherif, E.G.3    Susan, B.4    Yu, Y.5    Skiadopoulos, M.H.6
  • 29
    • 0035989639 scopus 로고    scopus 로고
    • Effcient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene
    • Halbert, CL, Allen, JM and Miller, AD (2002). Effcient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nat Biotechnol 20: 697-701.
    • (2002) Nat Biotechnol , vol.20 , pp. 697-701
    • Halbert, C.L.1    Allen, J.M.2    Miller, A.D.3
  • 30
    • 0034756451 scopus 로고    scopus 로고
    • Expanding AAV packaging capacity with trans-splicing or overlapping vectors: A quantitative comparison
    • Duan, D, Yue, Y and Engelhardt, JF (2001). Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison. Mol Ther 4: 383-391.
    • (2001) Mol Ther , vol.4 , pp. 383-391
    • Duan, D.1    Yue, Y.2    Engelhardt, J.F.3
  • 31
    • 0032544053 scopus 로고    scopus 로고
    • Effcient expression of CFTR function with adeno-associated virus vectors that carry shortened CFTR genes
    • Zhang, L, Wang, D, Fischer, H, Fan, PD, Widdicombe, JH, Kan, YW et al. (1998). Effcient expression of CFTR function with adeno-associated virus vectors that carry shortened CFTR genes. Proc Natl Acad Sci USA 95: 10158-10163.
    • (1998) Proc Natl Acad Sci USA , vol.95 , pp. 10158-10163
    • Zhang, L.1    Wang, D.2    Fischer, H.3    Fan, P.D.4    Widdicombe, J.H.5    Kan, Y.W.6
  • 32
    • 4544377145 scopus 로고    scopus 로고
    • Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fbrosis transmembrane conductance regulator vector
    • Sirninger, J, Muller, C, Braag, S, Tang, Q, Yue, H, Detrisac, C et al. (2004). Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fbrosis transmembrane conductance regulator vector. Hum Gene Ther 15: 832-841.
    • (2004) Hum Gene Ther , vol.15 , pp. 832-841
    • Sirninger, J.1    Muller, C.2    Braag, S.3    Tang, Q.4    Yue, H.5    Detrisac, C.6
  • 33
    • 0032905185 scopus 로고    scopus 로고
    • Effcient CFTR expression from AAV vectors packaged with promoters--the second generation
    • Wang, D, Fischer, H, Zhang, L, Fan, P, Ding, RX and Dong, J (1999). Effcient CFTR expression from AAV vectors packaged with promoters--the second generation. Gene Ther 6: 667-675.
    • (1999) Gene Ther , vol.6 , pp. 667-675
    • Wang, D.1    Fischer, H.2    Zhang, L.3    Fan, P.4    Ding, R.X.5    Dong, J.6
  • 34
    • 24944551962 scopus 로고    scopus 로고
    • Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fbrosis transmembrane conductance regulator function to polarized human cystic fbrosis airway epithelial cells
    • Liu, X, Luo, M, Zhang, LN, Yan, Z, Zak, R, Ding, W et al. (2005). Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fbrosis transmembrane conductance regulator function to polarized human cystic fbrosis airway epithelial cells. Hum Gene Ther 16: 1116-1123.
    • (2005) Hum Gene Ther , vol.16 , pp. 1116-1123
    • Liu, X.1    Luo, M.2    Zhang, L.N.3    Yan, Z.4    Zak, R.5    Ding, W.6
  • 35
    • 10344225049 scopus 로고    scopus 로고
    • Dual therapeutic utility of proteasome modulating agents for pharmaco-gene therapy of the cystic fbrosis airway
    • Zhang, LN, Karp, P, Gerard, CJ, Pastor, E, Laux, D, Munson, K et al. (2004). Dual therapeutic utility of proteasome modulating agents for pharmaco-gene therapy of the cystic fbrosis airway. Mol Ther 10: 990-1002.
    • (2004) Mol Ther , vol.10 , pp. 990-1002
    • Zhang, L.N.1    Karp, P.2    Gerard, C.J.3    Pastor, E.4    Laux, D.5    Munson, K.6
  • 36
    • 2642642141 scopus 로고    scopus 로고
    • Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus
    • Xiao, X, Li, J and Samulski, RJ (1998). Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol 72: 2224-2232.
    • (1998) J Virol , vol.72 , pp. 2224-2232
    • Xiao, X.1    Li, J.2    Samulski, R.J.3
  • 38
    • 0042622380 scopus 로고    scopus 로고
    • SWISS-MODEL: An automated protein homology-modeling server
    • Schwede, T, Kopp, J, Guex, N and Peitsch, MC (2003). SWISS-MODEL: An automated protein homology-modeling server. Nucleic Acids Res 31: 3381-3385.
    • (2003) Nucleic Acids Res , vol.31 , pp. 3381-3385
    • Schwede, T.1    Kopp, J.2    Guex, N.3    Peitsch, M.C.4
  • 39
    • 84889120137 scopus 로고
    • Improved methods for building protein models in electron density maps and the location of errors in these models
    • Jones, TA, Zou, JY, Cowan, SW and Kjeldgaard, M (1991). Improved methods for building protein models in electron density maps and the location of errors in these models. Acta Crystallogr, A, Found Crystallogr 47 (Pt 2): 110-119.
    • (1991) Acta Crystallogr, A, Found Crystallogr , vol.47 , Issue.PART 2 , pp. 110-119
    • Jones, T.A.1    Zou, J.Y.2    Cowan, S.W.3    Kjeldgaard, M.4
  • 40
    • 0036469060 scopus 로고    scopus 로고
    • Unraveling hot spots in binding interfaces: Progress and challenges
    • DeLano, WL (2002). Unraveling hot spots in binding interfaces: progress and challenges. Curr Opin Struct Biol 12: 14-20.
    • (2002) Curr Opin Struct Biol , vol.12 , pp. 14-20
    • Delano, W.L.1
  • 41
    • 11144227707 scopus 로고    scopus 로고
    • Infection of ciliated cells by human parainfuenza virus type 3 in an in vitro model of human airway epithelium
    • Zhang, L, Bukreyev, A, Thompson, CI, Watson, B, Peeples, ME, Collins, PL et al. (2005). Infection of ciliated cells by human parainfuenza virus type 3 in an in vitro model of human airway epithelium. J Virol 79: 1113-1124.
    • (2005) J Virol , vol.79 , pp. 1113-1124
    • Zhang, L.1    Bukreyev, A.2    Thompson, C.I.3    Watson, B.4    Peeples, M.E.5    Collins, P.L.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.