메뉴 건너뛰기




Volumn 26, Issue 6, 2015, Pages 334-346

Optimization of Recombinant Adeno-Associated Virus-Mediated Expression for Large Transgenes, Using a Synthetic Promoter and Tandem Array Enhancers

Author keywords

[No Author keywords available]

Indexed keywords

CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; PARVOVIRUS VECTOR; FIREFLY LUCIFERASE;

EID: 84932629728     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2015.001     Document Type: Article
Times cited : (53)

References (54)
  • 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;245:1066-1073.
    • (1989) Science , vol.245 , pp. 1066-1073
    • Riordan, J.R.1    Rommens, J.M.2    Kerem, B.3
  • 3
    • 0024453308 scopus 로고
    • Identification of the cystic fibrosis gene: Chromosome walking and jumping
    • Rommens JM, Iannuzzi MC, Kerem B, et al. Identification of the cystic fibrosis gene: chromosome walking and jumping. Science 1989;245:1059-1065.
    • (1989) Science , vol.245 , pp. 1059-1065
    • Rommens, J.M.1    Iannuzzi, M.C.2    Kerem, B.3
  • 4
    • 0025277214 scopus 로고
    • Abnormal regulation of ion channels in cystic fibrosis epithelia
    • Welsh MJ. Abnormal regulation of ion channels in cystic fibrosis epithelia. FASEB J 1990;4:2718-2725.
    • (1990) FASEB J , vol.4 , pp. 2718-2725
    • Welsh, M.J.1
  • 9
    • 60149095412 scopus 로고    scopus 로고
    • Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy
    • Griesenbach U, Alton EW. Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy. Adv Drug Deliv Rev 2009;61:128-139.
    • (2009) Adv Drug Deliv Rev , vol.61 , pp. 128-139
    • Griesenbach, U.1    Alton, E.W.2
  • 10
    • 79251471162 scopus 로고    scopus 로고
    • Viral vectors for cystic fibrosis gene therapy: What does the future hold?
    • Griesenbach U, Inoue M, Hasegawa M, et al. Viral vectors for cystic fibrosis gene therapy: what does the future hold? Virus Adapt Treat 2010;2:159-171.
    • (2010) Virus Adapt Treat , vol.2 , pp. 159-171
    • Griesenbach, U.1    Inoue, M.2    Hasegawa, M.3
  • 11
    • 19644368252 scopus 로고    scopus 로고
    • Adeno-associated virus vectors in clinical trials
    • Carter BJ. Adeno-associated virus vectors in clinical trials. Hum Gene Ther 2005;16:541-650.
    • (2005) Hum Gene Ther , vol.16 , pp. 541-650
    • Carter, B.J.1
  • 12
    • 33746874152 scopus 로고    scopus 로고
    • Adeno-associated virus serotypes: Vector toolkit for human gene therapy
    • Wu Z, Asokan A, Samulski RJ. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 2006;14:316-327.
    • (2006) Mol Ther , vol.14 , pp. 316-327
    • Wu, Z.1    Asokan, A.2    Samulski, R.J.3
  • 13
    • 54249126862 scopus 로고    scopus 로고
    • Gene therapy using adeno-associated virus vectors
    • Daya S, Berns KI. Gene therapy using adeno-associated virus vectors. Clin Microbiol Rev 2008;21:583-593.
    • (2008) Clin Microbiol Rev , vol.21 , pp. 583-593
    • Daya, S.1    Berns, K.I.2
  • 14
    • 0034777713 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus vectors for cystic fibrosis gene therapy
    • Flotte TR. Recombinant adeno-associated virus vectors for cystic fibrosis gene therapy. Curr Opin Mol Ther 2001;3:497-502.
    • (2001) Curr Opin Mol Ther , vol.3 , pp. 497-502
    • Flotte, T.R.1
  • 15
    • 0034779927 scopus 로고    scopus 로고
    • A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease
    • Aitken ML, Moss RB, Waltz DA, et al. A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease. Hum Gene Ther 2001;12:1907-1916.
    • (2001) Hum Gene Ther , vol.12 , pp. 1907-1916
    • Aitken, M.L.1    Moss, R.B.2    Waltz, D.A.3
  • 16
    • 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 JA, Nepomuceno IB, Messner AH, et al. 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 2002;13:1349-1359.
    • (2002) Hum Gene Ther , vol.13 , pp. 1349-1359
    • Wagner, J.A.1    Nepomuceno, I.B.2    Messner, A.H.3
  • 17
    • 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;18:726-732.
    • (2007) Hum Gene Ther , vol.18 , pp. 726-732
    • Moss, R.B.1    Milla, C.2    Colombo, J.3
  • 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, et al. Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J Clin Invest 2000;105:1573-1587.
    • (2000) J Clin Invest , vol.105 , pp. 1573-1587
    • Duan, D.1    Yue, Y.2    Yan, Z.3
  • 19
    • 0032506771 scopus 로고    scopus 로고
    • Polarity influences the efficiency of recombinant adenoassociated virus infection in differentiated airway epithelia
    • Duan D, Yue Y, Yan Z, et al. Polarity influences the efficiency of recombinant adenoassociated virus infection in differentiated airway epithelia. Hum Gene Ther 1998;9:2761-2776.
    • (1998) Hum Gene Ther , vol.9 , pp. 2761-2776
    • Duan, D.1    Yue, Y.2    Yan, Z.3
  • 20
    • 0029741735 scopus 로고    scopus 로고
    • Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung
    • Conrad CK, Allen SS, Afione SA, et al. Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung. Gene Ther 1996;3:658-668.
    • (1996) Gene Ther , vol.3 , pp. 658-668
    • Conrad, C.K.1    Allen, S.S.2    Afione, S.A.3
  • 21
    • 36348993662 scopus 로고    scopus 로고
    • Biological differences in rAAV transduction of airway epithelia in humans and in Old World non-human primates
    • Liu X, Luo M, Trygg C, et al. Biological differences in rAAV transduction of airway epithelia in humans and in Old World non-human primates. Mol Ther 2007;15:2114-2123.
    • (2007) Mol Ther , vol.15 , pp. 2114-2123
    • Liu, X.1    Luo, M.2    Trygg, C.3
  • 22
    • 20744433079 scopus 로고    scopus 로고
    • Intracellular trafficking of adeno-associated viral vectors
    • Ding W, Zhang L, Yan Z, et al. Intracellular trafficking of adeno-associated viral vectors. Gene Ther 2005;12:873-880.
    • (2005) Gene Ther , vol.12 , pp. 873-880
    • Ding, W.1    Zhang, L.2    Yan, Z.3
  • 23
    • 0036171821 scopus 로고    scopus 로고
    • Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors
    • Yan Z, Zak R, Luxton GW, et al. Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors. J Virol 2002;76:2043-2053.
    • (2002) J Virol , vol.76 , pp. 2043-2053
    • Yan, Z.1    Zak, R.2    Luxton, G.W.3
  • 24
    • 33749570518 scopus 로고    scopus 로고
    • Unique biologic properties of recombinant AAV1 transduction in polarized human airway epithelia
    • Yan Z, Lei-Butters DC, Liu X, et al. Unique biologic properties of recombinant AAV1 transduction in polarized human airway epithelia. J Biol Chem 2006;281:29684-29692.
    • (2006) J Biol Chem , vol.281 , pp. 29684-29692
    • Yan, Z.1    Lei-Butters, D.C.2    Liu, X.3
  • 25
    • 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;10:990-1002.
    • (2004) Mol Ther , vol.10 , pp. 990-1002
    • Zhang, L.N.1    Karp, P.2    Gerard, C.J.3
  • 26
    • 0029849257 scopus 로고    scopus 로고
    • Quantitative analysis of the packaging capacity of recombinant adeno-associated virus
    • Dong JY, Fan PD, Frizzell RA. Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum Gene Ther 1996;7:2101-2112.
    • (1996) Hum Gene Ther , vol.7 , pp. 2101-2112
    • Dong, J.Y.1    Fan, P.D.2    Frizzell, R.A.3
  • 27
    • 74049085498 scopus 로고    scopus 로고
    • Effect of genome size on AAV vector packaging
    • Wu Z, Yang H, Colosi P. Effect of genome size on AAV vector packaging. Mol Ther 2010;18:80-86.
    • (2010) Mol Ther , vol.18 , pp. 80-86
    • Wu, Z.1    Yang, H.2    Colosi, P.3
  • 28
    • 0027412209 scopus 로고
    • Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter
    • Flotte TR, Afione SA, Solow R, et al. Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter. J Biol Chem 1993;268:3781-3790.
    • (1993) J Biol Chem , vol.268 , pp. 3781-3790
    • Flotte, T.R.1    Afione, S.A.2    Solow, R.3
  • 29
    • 0037338508 scopus 로고    scopus 로고
    • Analysis of sequential aliquots of hypertonic saline solution-induced sputum from clinically stable patients with cystic fibrosis
    • Aitken ML, Greene KE, Tonelli MR, et al. Analysis of sequential aliquots of hypertonic saline solution-induced sputum from clinically stable patients with cystic fibrosis. Chest 2003;123:792-799.
    • (2003) Chest , vol.123 , pp. 792-799
    • Aitken, M.L.1    Greene, K.E.2    Tonelli, M.R.3
  • 30
    • 0032544053 scopus 로고    scopus 로고
    • Efficient expression of CFTR function with adeno-associated virus vectors that carry shortened cftr genes
    • Zhang L, Wang D, Fischer H, et al. Efficient expression of CFTR function with adeno-associated virus vectors that carry shortened cftr genes. Proc Natl Acad Sci U S A 1998;95:10158-10163.
    • (1998) Proc Natl Acad Sci U S A , vol.95 , pp. 10158-10163
    • Zhang, L.1    Wang, D.2    Fischer, H.3
  • 31
    • 0037022607 scopus 로고    scopus 로고
    • CFTR with a partially deleted R domain corrects the cystic fibrosis chloride transport defect in human airway epithelia in vitro and in mouse nasal mucosa in vivo
    • Ostedgaard LS, Zabner J, Vermeer DW, et al. CFTR with a partially deleted R domain corrects the cystic fibrosis chloride transport defect in human airway epithelia in vitro and in mouse nasal mucosa in vivo. Proc Natl Acad Sci U S A 2002;99:3093-3098.
    • (2002) Proc Natl Acad Sci U S A , vol.99 , pp. 3093-3098
    • Ostedgaard, L.S.1    Zabner, J.2    Vermeer, D.W.3
  • 33
    • 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;102:2952-2957.
    • (2005) Proc Natl Acad Sci U S A , vol.102 , pp. 2952-2957
    • Ostedgaard, L.S.1    Rokhlina, T.2    Karp, P.H.3
  • 34
    • 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;17:2067-2077.
    • (2009) Mol Ther , vol.17 , pp. 2067-2077
    • Li, W.1    Zhang, L.2    Johnson, J.S.3
  • 35
    • 41849102053 scopus 로고    scopus 로고
    • Adeno-associated virus-targeted disruption of the CFTR gene in cloned ferrets
    • Sun X, Yan Z, Yi Y, et al. Adeno-associated virus-targeted disruption of the CFTR gene in cloned ferrets. J Clin Invest 2008;118:1578-1583.
    • (2008) J Clin Invest , vol.118 , pp. 1578-1583
    • Sun, X.1    Yan, Z.2    Yi, Y.3
  • 36
    • 77956379893 scopus 로고    scopus 로고
    • Disease phenotype of a ferret CFTR-knockout model of cystic fibrosis
    • Sun X, Sui H, Fisher JT, et al. Disease phenotype of a ferret CFTR-knockout model of cystic fibrosis. J Clin Invest 2010;120:3149-3160.
    • (2010) J Clin Invest , vol.120 , pp. 3149-3160
    • Sun, X.1    Sui, H.2    Fisher, J.T.3
  • 37
    • 84867166324 scopus 로고    scopus 로고
    • Abnormal endocrine pancreas function at birth in cystic fibrosis ferrets
    • Olivier AK, Yi Y, Sun X, et al. Abnormal endocrine pancreas function at birth in cystic fibrosis ferrets. J Clin Invest 2012;122:3755-3768.
    • (2012) J Clin Invest , vol.122 , pp. 3755-3768
    • Olivier, A.K.1    Yi, Y.2    Sun, X.3
  • 38
    • 84895512997 scopus 로고    scopus 로고
    • Lung phenotype of juvenile and adult cystic fibrosis transmembrane conductance regulator-knockout ferrets
    • Sun X, Olivier AK, Liang B, et al. Lung phenotype of juvenile and adult cystic fibrosis transmembrane conductance regulator-knockout ferrets. Am J Respir Cell Mol Biol 2014;50:502-512.
    • (2014) Am J Respir Cell Mol Biol , vol.50 , pp. 502-512
    • Sun, X.1    Olivier, A.K.2    Liang, B.3
  • 39
    • 84899513890 scopus 로고    scopus 로고
    • Gastrointestinal pathology in juvenile and adult CFTR-knockout ferrets
    • Sun X, Olivier AK, Yi Y, et al. Gastrointestinal pathology in juvenile and adult CFTR-knockout ferrets. Am J Pathol 2014;184:1309-1322.
    • (2014) Am J Pathol , vol.184 , pp. 1309-1322
    • Sun, X.1    Olivier, A.K.2    Yi, Y.3
  • 40
    • 84883725341 scopus 로고    scopus 로고
    • Postentry processing of recombinant adeno-associated virus type 1 and transduction of the ferret lung are altered by a factor in airway secretions
    • Yan Z, Sun X, Evans IA, et al. Postentry processing of recombinant adeno-associated virus type 1 and transduction of the ferret lung are altered by a factor in airway secretions. Hum Gene Ther 2013;24:786-796.
    • (2013) Hum Gene Ther , vol.24 , pp. 786-796
    • Yan, Z.1    Sun, X.2    Evans, I.A.3
  • 42
    • 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, et al. 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 2004;78:2863-2874.
    • (2004) J Virol , vol.78 , pp. 2863-2874
    • Yan, Z.1    Zak, R.2    Zhang, Y.3
  • 43
    • 33645522130 scopus 로고    scopus 로고
    • rAAV2 traffics through both the late and the recycling endosomes in a dose-dependent fashion
    • Ding W, Zhang LN, Yeaman C, et al. rAAV2 traffics through both the late and the recycling endosomes in a dose-dependent fashion. Mol Ther 2006;13:671-682.
    • (2006) Mol Ther , vol.13 , pp. 671-682
    • Ding, W.1    Zhang, L.N.2    Yeaman, C.3
  • 44
    • 35348836681 scopus 로고    scopus 로고
    • Comparative biology of rAAV transduction in ferret, pig and human airway epithelia
    • Liu X, Luo M, Guo C, et al. Comparative biology of rAAV transduction in ferret, pig and human airway epithelia. Gene Ther 2007;14:1543-1548.
    • (2007) Gene Ther , vol.14 , pp. 1543-1548
    • Liu, X.1    Luo, M.2    Guo, C.3
  • 45
    • 0036367311 scopus 로고    scopus 로고
    • An in vitro model of differentiated human airway epithelia: Methods for establishing primary cultures
    • Karp PH, Moninger TO, Weber SP, et al. An in vitro model of differentiated human airway epithelia: methods for establishing primary cultures. Methods Mol Biol 2002;188:115-137.
    • (2002) Methods Mol Biol , vol.188 , pp. 115-137
    • Karp, P.H.1    Moninger, T.O.2    Weber, S.P.3
  • 46
    • 0037405173 scopus 로고    scopus 로고
    • Development of cystic fibrosis and noncystic fibrosis airway cell lines
    • Zabner J, Karp P, Seiler M, et al. Development of cystic fibrosis and noncystic fibrosis airway cell lines. Am J Physiol Lung Cell Mol Physiol 2003;284:L844-L854.
    • (2003) Am J Physiol Lung Cell Mol Physiol , vol.284 , pp. L844-L854
    • Zabner, J.1    Karp, P.2    Seiler, M.3
  • 47
    • 84890129810 scopus 로고    scopus 로고
    • A novel chimeric adenoassociated virus 2/human bocavirus 1 parvovirus vector efficiently transduces human airway epithelia
    • Yan Z, Keiser NW, Song Y, et al. A novel chimeric adenoassociated virus 2/human bocavirus 1 parvovirus vector efficiently transduces human airway epithelia. Mol Ther 2013;21:2181-2194.
    • (2013) Mol Ther , vol.21 , pp. 2181-2194
    • Yan, Z.1    Keiser, N.W.2    Song, Y.3
  • 48
    • 33847664265 scopus 로고    scopus 로고
    • Bioelectric properties of chloride channels in human, pig, ferret, and mouse airway epithelia
    • Liu X, Luo M, Zhang L, et al. Bioelectric properties of chloride channels in human, pig, ferret, and mouse airway epithelia. Am J Respir Cell Mol Biol 2007;36:313-323.
    • (2007) Am J Respir Cell Mol Biol , vol.36 , pp. 313-323
    • Liu, X.1    Luo, M.2    Zhang, L.3
  • 49
    • 0141627611 scopus 로고    scopus 로고
    • A sensitive, realtime, RNA-specific PCR method for the detection of recombinant AAV-CFTR vector expression
    • Gerard CJ, Dell'aringa J, Hale KA, et al. A sensitive, realtime, RNA-specific PCR method for the detection of recombinant AAV-CFTR vector expression. Gene Ther 2003; 10:1744-1753.
    • (2003) Gene Ther , vol.10 , pp. 1744-1753
    • Gerard, C.J.1    Dell'Aringa, J.2    Hale, K.A.3
  • 50
    • 84862969739 scopus 로고    scopus 로고
    • Native molecular state of adeno-associated viral vectors revealed by single-molecule sequencing
    • Kapranov P, Chen L, Dederich D, et al. Native molecular state of adeno-associated viral vectors revealed by single-molecule sequencing. Hum Gene Ther 2012;23:46-55.
    • (2012) Hum Gene Ther , vol.23 , pp. 46-55
    • Kapranov, P.1    Chen, L.2    Dederich, D.3
  • 51
    • 64049096002 scopus 로고    scopus 로고
    • N-Glycans are direct determinants of CFTR folding and stability in secretory and endocytic membrane traffic
    • Glozman R, Okiyoneda T, Mulvihill CM, et al. N-Glycans are direct determinants of CFTR folding and stability in secretory and endocytic membrane traffic. J Cell Biol 2009;184:847-862.
    • (2009) J Cell Biol , vol.184 , pp. 847-862
    • Glozman, R.1    Okiyoneda, T.2    Mulvihill, C.M.3
  • 52
    • 84862648349 scopus 로고    scopus 로고
    • Comparative processing and function of human and ferret cystic fibrosis transmembrane conductance regulator
    • Fisher JT, Liu X, Yan Z, et al. Comparative processing and function of human and ferret cystic fibrosis transmembrane conductance regulator. J Biol Chem 2012;287:21673-21685.
    • (2012) J Biol Chem , vol.287 , pp. 21673-21685
    • Fisher, J.T.1    Liu, X.2    Yan, Z.3
  • 53
    • 80052538896 scopus 로고    scopus 로고
    • Comparative biology of cystic fibrosis animal models
    • Fisher JT, Zhang Y, Engelhardt JF. Comparative biology of cystic fibrosis animal models. Methods Mol Biol 2011;742:311-334.
    • (2011) Methods Mol Biol , vol.742 , pp. 311-334
    • Fisher, J.T.1    Zhang, Y.2    Engelhardt, J.F.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.