메뉴 건너뛰기




Volumn 10, Issue 6, 2004, Pages 542-546

Advances in cystic fibrosis gene therapy

Author keywords

Airway gene transfer; Cystic fibrosis; Gene therapy; Gene transfer agents

Indexed keywords

2 (DIETHYLAMINO)ETHYL ETHER DEXTRAN; ADENOVIRUS VECTOR; CHLORIDE; CHOLESTEROL; COMPLEMENTARY DNA; CYTOKERATIN 18; DEXTRAN DERIVATIVE; INTERLEUKIN 8; MACROGOL; MESSENGER RNA; NAKED DNA; NEUTRALIZING ANTIBODY; PLASMID DNA; POLYGLACTIN; POLYLYSINE; PROTEASOME INHIBITOR; SIALIC ACID; TRANSMEMBRANE CONDUCTANCE REGULATOR; UBIQUITIN; UBIQUITIN C; UNCLASSIFIED DRUG; VIRUS GLYCOPROTEIN; VIRUS VECTOR;

EID: 7444232085     PISSN: 10705287     EISSN: None     Source Type: Journal    
DOI: 10.1097/01.mcp.0000142102.91202.04     Document Type: Review
Times cited : (35)

References (32)
  • 1
    • 0038488551 scopus 로고    scopus 로고
    • Phase I trial of intranasal and endobranchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: A two-part clinical study
    • Flotte TR, Zeitlin PL, Reynolds TC, et al.: Phase I trial of intranasal and endobranchial 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 2003, 14:1079-1088.
    • (2003) Hum Gene Ther , vol.14 , pp. 1079-1088
    • Flotte, T.R.1    Zeitlin, P.L.2    Reynolds, T.C.3
  • 2
    • 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, 125:509-521. Repeated administration of nonintegrating viral vectors is a crucial component of gene therapy for chronic diseases.
    • (2004) Chest , vol.125 , pp. 509-521
    • Moss, R.B.1    Rodman, D.2    Spencer, L.T.3
  • 3
    • 0042270435 scopus 로고    scopus 로고
    • Single dose escalation study to evaluate safety of nasal administration of CFTR001 gene transfer vector to subjects with cystic fibrosis
    • Konstan M, Wagener J, Hilliard K, et al.: Single dose escalation study to evaluate safety of nasal administration of CFTR001 gene transfer vector to subjects with cystic fibrosis [abstract]. Mol Ther 2004, 7:S386. Nanoparticles as used in this trial are a comparatively novel class of nonviral gene transfer agents. They may be more efficient than traditional lipid and polymer formulation, because their smaller size may allow easier entry into the nucleus via nuclear pore complexes.
    • (2004) Mol Ther , vol.7
    • Konstan, M.1    Wagener, J.2    Hilliard, K.3
  • 4
    • 0037222237 scopus 로고    scopus 로고
    • Enhancement of adenovirus-mediated gene transfer to the airways by DEAE dextran and sodium caprate in vivo
    • Gregory LG, Harbottle RP, Lawrence L, et al.: Enhancement of adenovirus-mediated gene transfer to the airways by DEAE dextran and sodium caprate in vivo. Mol Ther 2003, 7:19-26.
    • (2003) Mol Ther , vol.7 , pp. 19-26
    • Gregory, L.G.1    Harbottle, R.P.2    Lawrence, L.3
  • 5
    • 0037853137 scopus 로고    scopus 로고
    • Reduced inflammation and improved airway expression using helper-dependent adenoviral vectors with a K18 promoter
    • Toietta G, Koehler DR, Finegold MJ, et al.: Reduced inflammation and improved airway expression using helper-dependent adenoviral vectors with a K18 promoter. Mol Ther 2003, 7:649-658.
    • (2003) Mol Ther , vol.7 , pp. 649-658
    • Toietta, G.1    Koehler, D.R.2    Finegold, M.J.3
  • 6
    • 7444271148 scopus 로고    scopus 로고
    • Serological charcterisation of human and non-human primate AAVs
    • Calcedo R, Gallery L, Gao G, et al.: Serological charcterisation of human and non-human primate AAVs [abstract]. Mol Ther 2004, 7:S74.
    • (2004) Mol Ther , vol.7
    • Calcedo, R.1    Gallery, L.2    Gao, G.3
  • 7
    • 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, 99:10405-10410.
    • (2002) Proc Natl Acad Sci U S A , vol.99 , pp. 10405-10410
    • Xie, Q.1    Bu, W.2    Bhatia, S.3
  • 8
    • 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, 7:515-525.
    • (2003) Mol Ther , vol.7 , pp. 515-525
    • Shi, W.1    Bartlett, J.S.2
  • 9
    • 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
  • 10
    • 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, 110:499-504.
    • (2002) J Clin Invest , vol.110 , pp. 499-504
    • Auricchio, A.1    O'Connor, E.2    Weiner, D.3
  • 11
    • 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, et al.: Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration. J Virol 1997, 71:5932-5941.
    • (1997) J Virol , vol.71 , pp. 5932-5941
    • Halbert, C.L.1    Standaert, T.A.2    Aitken, M.L.3
  • 12
    • 0346656769 scopus 로고    scopus 로고
    • Successful transgene expression with serial doses of aerosolized rAAV2 vectors in Rhesus macaques
    • Fischer A, Beck S, Smith C, et al.: Successful transgene expression with serial doses of aerosolized rAAV2 vectors in Rhesus macaques. Mol Ther 2003, 8:918-926.
    • (2003) Mol Ther , vol.8 , pp. 918-926
    • Fischer, A.1    Beck, S.2    Smith, C.3
  • 13
    • 4243458950 scopus 로고    scopus 로고
    • Recombinant Sendai virus-mediated CFTR cDNA transfer
    • Ferrari S, Farley R, Munkonge F, et al.: Recombinant Sendai virus-mediated CFTR cDNA transfer. Mol Ther 2004, 7:S38.
    • (2004) Mol Ther , vol.7
    • Ferrari, S.1    Farley, R.2    Munkonge, F.3
  • 14
    • 0038026258 scopus 로고    scopus 로고
    • A new Sendai virus vector deficient in the matrix gene does not form virus particles and shows extensive cell-to-cell spreading
    • Inoue M, Tokusumi Y, Ban H, et al.: A new Sendai virus vector deficient in the matrix gene does not form virus particles and shows extensive cell-to-cell spreading. J Virol 2003, 77:6419-6429. Recombinant SeV is the most efficient gene transfer agent for airway epithelial cells in vivo.
    • (2003) J Virol , vol.77 , pp. 6419-6429
    • Inoue, M.1    Tokusumi, Y.2    Ban, H.3
  • 15
    • 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, 8:777-789.
    • (2003) Mol Ther , vol.8 , pp. 777-789
    • Medina, M.F.1    Kobinger, G.P.2    Rux, J.3
  • 16
    • 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, et al.: Pseudotyped lentivirus vectors derived from simian immunodeficiency virus SIVagm with envelope glycoproteins from paramyxovirus. J Virol 2003, 77:2607-2614. The H and FN proteins, which are crucial for efficient transfection of airway epithelial cells with SeV have been incorporated into a lentivirus envelope and may improve the transfection efficiency of an integrating vector into airway epithelial cells.
    • (2003) J Virol , vol.77 , pp. 2607-2614
    • Kobayashi, M.1    Iida, A.2    Ueda, Y.3
  • 17
    • 0033540649 scopus 로고    scopus 로고
    • Sugar-mediated uptake of glycosylated polylysines and gene transfer into normal and cystic fibrosis airway epithelial cells
    • Fajac I, Briand P, Monsigny M, et al.: Sugar-mediated uptake of glycosylated polylysines and gene transfer into normal and cystic fibrosis airway epithelial cells. Hum Gene Ther 1999, 10:395-406.
    • (1999) Hum Gene Ther , vol.10 , pp. 395-406
    • Fajac, I.1    Briand, P.2    Monsigny, M.3
  • 18
    • 0012505259 scopus 로고    scopus 로고
    • Uptake of plasmid/glycosylated polymer complexes and gene transfer efficiency in differentiated airway epithelial cells
    • Fajac I, Thevenot G, Bedouet L, et al.: Uptake of plasmid/glycosylated polymer complexes and gene transfer efficiency in differentiated airway epithelial cells. J Gene Med 2003, 5:38-48.
    • (2003) J Gene Med , vol.5 , pp. 38-48
    • Fajac, I.1    Thevenot, G.2    Bedouet, L.3
  • 19
    • 0037221906 scopus 로고    scopus 로고
    • Lactosylated poly-L-lysine targets a potential lactose receptor in cystic fibrosis and non-cystic fibrosis airway epithelial cells
    • Klink D, Yu QC, Glick MC, et al.: Lactosylated poly-L-lysine targets a potential lactose receptor in cystic fibrosis and non-cystic fibrosis airway epithelial cells. Mol Ther 2003, 7:73-80.
    • (2003) Mol Ther , vol.7 , pp. 73-80
    • Klink, D.1    Yu, Q.C.2    Glick, M.C.3
  • 20
    • 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, et al.: 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, 10:1282-1288.
    • (2003) Gene Ther , vol.10 , pp. 1282-1288
    • Stern, M.1    Ulrich, K.2    Geddes, D.M.3
  • 21
    • 0141744705 scopus 로고    scopus 로고
    • Gene insertion and long-term expression in lung mediated by the Sleeping Beauty transposon system
    • Belur LR, Frandsen JL, Dupuy AJ, et al.: Gene insertion and long-term expression in lung mediated by the Sleeping Beauty transposon system. Mol Ther 2003, 8:501-507.
    • (2003) Mol Ther , vol.8 , pp. 501-507
    • Belur, L.R.1    Frandsen, J.L.2    Dupuy, A.J.3
  • 22
    • 0142244084 scopus 로고    scopus 로고
    • Targeting expression of a transgene to the airway surface epithelium using a ciliated cell-specific promoter
    • Ostrowski LE, Hutchins JR, Zakel K, et al.: Targeting expression of a transgene to the airway surface epithelium using a ciliated cell-specific promoter. Mol Ther 2003, 8:637-645. The use of a cell-specific promoter becomes more and more important for gene transfer, and many protocols move away from using strong viral promoters such as CMV.
    • (2003) Mol Ther , vol.8 , pp. 637-645
    • Ostrowski, L.E.1    Hutchins, J.R.2    Zakel, K.3
  • 23
    • 0142168702 scopus 로고    scopus 로고
    • Electroporation-mediated transfer of the Na-K-ATpase b1 subunit safely increases alveolar fluid clearance in rat lungs
    • Machado-Aranda D, Adi RY, Sznajader A, et al.: Electroporation-mediated transfer of the Na-K-ATpase b1 subunit safely increases alveolar fluid clearance in rat lungs [abstract]. Mol Ther 2004, 7:S381.
    • (2004) Mol Ther , vol.7
    • Machado-Aranda, D.1    Adi, R.Y.2    Sznajader, A.3
  • 24
    • 7444231795 scopus 로고    scopus 로고
    • Use of electroporation to enhance plasmid DNA transfer into cells of the murine respiratory epithelia
    • Pringle I, Sumner S, Davies L, et al.: Use of electroporation to enhance plasmid DNA transfer into cells of the murine respiratory epithelia [abstract]. Pediatr Pulmonol 2003, 25(suppl):257.
    • (2003) Pediatr Pulmonol , vol.25 , Issue.SUPPL. , pp. 257
    • Pringle, I.1    Sumner, S.2    Davies, L.3
  • 25
    • 85047695405 scopus 로고    scopus 로고
    • Magnetofection: Enhancing and targeting gene delivery by magnetic force in vitro and in vivo
    • Scherer F, Anton M, Schillinger U, et al.: Magnetofection: enhancing and targeting gene delivery by magnetic force in vitro and in vivo. Gene Ther 2002, 9:102-109. Physical methods such as electroporation and magnetic force may help to overcome the comparatively inefficient nonviral gene transfer to the airways.
    • (2002) Gene Ther , vol.9 , pp. 102-109
    • Scherer, F.1    Anton, M.2    Schillinger, U.3
  • 26
    • 7444223724 scopus 로고    scopus 로고
    • Magnetofection to enhance airway gene transfer
    • Xenariou S, Griesenbach U, Ferrari S, et al.: Magnetofection to enhance airway gene transfer [abstract]. Mol Ther 2004, 9(suppl 1):S180.
    • (2004) Mol Ther , vol.9 , Issue.SUPPL. 1
    • Xenariou, S.1    Griesenbach, U.2    Ferrari, S.3
  • 27
    • 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, 100:15364-15369.
    • (2003) Proc Natl Acad Sci U S A , vol.100 , pp. 15364-15369
    • Koehler, D.R.1    Sajjan, U.2    Chow, Y.H.3
  • 28
    • 1342339826 scopus 로고    scopus 로고
    • Delivery of CFTR by adenoviral vector to cystic fibrosis mouse lung in a model of chronic Pseudomonas aeruginosa lung infection
    • Van Heeckeren AM, Scaria A, Schluchter MD, et al.: Delivery of CFTR by adenoviral vector to cystic fibrosis mouse lung in a model of chronic Pseudomonas aeruginosa lung infection. Am J Physiol Lung Cell Mol Physiol 2004, 286:L717-L726.
    • (2004) Am J Physiol Lung Cell Mol Physiol , vol.286
    • Van Heeckeren, A.M.1    Scaria, A.2    Schluchter, M.D.3
  • 29
    • 4344663919 scopus 로고    scopus 로고
    • Progress toward generating a ferret model of cystic fibrosis by somatic cell nuclear transfer
    • Li Z, Engelhardt JF: Progress toward generating a ferret model of cystic fibrosis by somatic cell nuclear transfer. Reprod Biol Endocrinol 2003, 1:83.
    • (2003) Reprod Biol Endocrinol , vol.1 , pp. 83
    • Li, Z.1    Engelhardt, J.F.2
  • 30
    • 0042856461 scopus 로고    scopus 로고
    • Evaluation of gene targeting by homologous recombination in ovine somatic cells
    • Williams SH, Sahota V, Palmai-Pallag T, et al.: Evaluation of gene targeting by homologous recombination in ovine somatic cells. Mol Reprod Dev 2003, 66:115-125. Generation of CF animal models has proved to be difficult.
    • (2003) Mol Reprod Dev , vol.66 , pp. 115-125
    • Williams, S.H.1    Sahota, V.2    Palmai-Pallag, T.3
  • 31
    • 2442718786 scopus 로고    scopus 로고
    • Increased airway epithelial Na+ absorption produces cystic fibrosis-like lung disease in mice
    • Mall M, Grubb BR, Harkema JR, et al.: Increased airway epithelial Na+ absorption produces cystic fibrosis-like lung disease in mice. Nat Med 2004, 10:487-493. This paper makes a very important contribution to understanding the etiology of CF lung disease. The epithelial sodium channel transgenic mouse may also become a useful model to study pharmacologic inhibitors of the epithelial sodium channel, and to understand and overcome barriers to gene transfer in the CF lung.
    • (2004) Nat Med , vol.10 , pp. 487-493
    • Mall, M.1    Grubb, B.R.2    Harkema, J.R.3
  • 32
    • 7444250414 scopus 로고    scopus 로고
    • Pre-clinical and clinical endpoint assays for cystic fibrosis gene therapy
    • in press
    • Griesenbach U, Boyd A: Pre-clinical and clinical endpoint assays for cystic fibrosis gene therapy. J Cystic Fibrosis 2004, in press.
    • (2004) J Cystic Fibrosis
    • Griesenbach, U.1    Boyd, A.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.