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Volumn 14, Issue 11, 2003, Pages 1079-1088

Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (raav2)-cftr vector in adult cystic fibrosis patients: A two-part clinical study

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; DEOXYRIBONUCLEASE; NEUTRALIZING ANTIBODY; TRANSMEMBRANE CONDUCTANCE REGULATOR;

EID: 0038488551     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/104303403322124792     Document Type: Article
Times cited : (205)

References (24)
  • 4
    • 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, J.F. (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
  • 5
    • 0026687233 scopus 로고
    • Defective regulation of outwardly rectifying C1-channels by protein kinase A corrected by insertion of CFTR
    • EGAN, M., FLOTTE, T., AFIONE, S., SOLOW, R., ZEITLIN, P.L., CARTER, B.J., and GUGGINO, W.B. (1992). Defective regulation of outwardly rectifying C1-channels by protein kinase A corrected by insertion of CFTR [see comments]. Nature 358, 581-584.
    • (1992) Nature , vol.358 , pp. 581-584
    • Egan, M.1    Flotte, T.2    Afione, S.3    Solow, R.4    Zeitlin, P.L.5    Carter, B.J.6    Guggino, W.B.7
  • 6
    • 0032323673 scopus 로고    scopus 로고
    • Adeno-associated virus vectors for gene therapy of cystic fibrosis
    • FLOTTE, T.R., and CARTER, B.J. (1998). Adeno-associated virus vectors for gene therapy of cystic fibrosis. Methods Enzymol. 292, 717-732.
    • (1998) Methods Enzymol. , vol.292 , pp. 717-732
    • Flotte, T.R.1    Carter, B.J.2
  • 9
    • 0027412209 scopus 로고
    • Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter
    • FLOTTE, T.R., AFIONE, S.A., SOLOW, R., DRUMM, M.L., MARKAKIS, D., GUGGINO, W.B., ZEITLIN, P.L., and CARTER, B.J. (1993b). Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter. J. Biol. Chem. 268, 3781-3790.
    • (1993) J. Biol. Chem. , vol.268 , pp. 3781-3790
    • Flotte, T.R.1    Afione, S.A.2    Solow, R.3    Drumm, M.L.4    Markakis, D.5    Guggino, W.B.6    Zeitlin, P.L.7    Carter, B.J.8
  • 10
    • 0029794845 scopus 로고    scopus 로고
    • Gradient of RGD-dependent entry of adenoviral vector in nasal and intrapulmonary epithelia: Implications for gene therapy of cystic fibrosis
    • GOLDMAN, M., SU, Q., and WILSON, J.M. (1996). Gradient of RGD-dependent entry of adenoviral vector in nasal and intrapulmonary epithelia: Implications for gene therapy of cystic fibrosis. Gene Ther. 3, 811-818.
    • (1996) Gene Ther. , vol.3 , pp. 811-818
    • Goldman, M.1    Su, Q.2    Wilson, J.M.3
  • 11
    • 0033857632 scopus 로고    scopus 로고
    • Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element
    • HABERMAN, R.P., MCCOWN, T.J., and SAMULSKI, R.J. (2000). Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element. J. Virol. 74, 8732-8739.
    • (2000) J. Virol. , vol.74 , pp. 8732-8739
    • Haberman, R.P.1    Mccown, T.J.2    Samulski, R.J.3
  • 12
    • 0032826773 scopus 로고    scopus 로고
    • Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model
    • In Process Citation
    • HERNANDEZ, Y.J., WANG, J., KEARNS, W.G., LOILER, S., POIRIER, A., and FLOTTE, T.R. (1999). Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model [In Process Citation]. J. Virol. 73, 8549-8558.
    • (1999) J. Virol. , vol.73 , pp. 8549-8558
    • Hernandez, Y.J.1    Wang, J.2    Kearns, W.G.3    Loiler, S.4    Poirier, A.5    Flotte, T.R.6
  • 13
    • 17344365059 scopus 로고    scopus 로고
    • A double-blind, placebo controlled, dose ranging study to evaluate the safety and biological efficacy of the lipid-DNA complex GR213487B in the nasal epithelium of adult patients with cystic fibrosis
    • KNOWLES, M.R., NOONE, P.G., HOHNEKER, K., JOHNSON, L.G., BOUCHER, R.C., EFTHIMIOU, J., CRAWFORD, C., BROWN, R., SCHWARTZBACH, C., and PEARLMAN, R. (1998). A double-blind, placebo controlled, dose ranging study to evaluate the safety and biological efficacy of the lipid-DNA complex GR213487B in the nasal epithelium of adult patients with cystic fibrosis. Hum. Gene Ther. 9, 249-269.
    • (1998) Hum. Gene Ther. , vol.9 , pp. 249-269
    • Knowles, M.R.1    Noone, P.G.2    Hohneker, K.3    Johnson, L.G.4    Boucher, R.C.5    Efthimiou, J.6    Crawford, C.7    Brown, R.8    Schwartzbach, C.9    Pearlman, R.10
  • 15
    • 0036786467 scopus 로고    scopus 로고
    • Bronchoalveolar fluid is not a major hindrance to virus-mediated gene therapy in cystic fibrosis
    • ROONEY, C.P., DENNING, G.M., DAVIS, B.P., FLAHERTY, D.M., CHIORINI, J.A., and ZABNER, J. (2002). Bronchoalveolar fluid is not a major hindrance to virus-mediated gene therapy in cystic fibrosis. J. Virol. 76, 10437-10443.
    • (2002) J. Virol. , vol.76 , pp. 10437-10443
    • Rooney, C.P.1    Denning, G.M.2    Davis, B.P.3    Flaherty, D.M.4    Chiorini, J.A.5    Zabner, J.6
  • 16
    • 0026070440 scopus 로고
    • Targeted integration of adeno-associated virus (AAV) into human chromosome 19
    • published erratum appears in EMBO J. 1992;11:1228
    • SAMULSKI, R.J., ZHU, X., XIAO, X., BROOK, J.D., HOUSMAN, D.E., EPSTEIN, N., and HUNTER, L.A. (1991). Targeted integration of adeno-associated virus (AAV) into human chromosome 19 [published erratum appears in EMBO J. 1992;11:1228]. EMBO J. 10, 3941-3950.
    • (1991) EMBO J. , vol.10 , pp. 3941-3950
    • Samulski, R.J.1    Zhu, X.2    Xiao, X.3    Brook, J.D.4    Housman, D.E.5    Epstein, N.6    Hunter, L.A.7
  • 19
    • 0033759007 scopus 로고    scopus 로고
    • Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients
    • VIRELLA-LOWELL, I., POIRIER, A., CHESNUT, K.A., BRANTLY, M., and FLOTTE, T.R. (2000). Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients. Gene Ther. 7, 1783-1789.
    • (2000) Gene Ther. , vol.7 , pp. 1783-1789
    • Virella-Lowell, I.1    Poirier, A.2    Chesnut, K.A.3    Brantly, M.4    Flotte, T.R.5
  • 23
    • 0029843596 scopus 로고    scopus 로고
    • Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
    • XIAO, X., LI, J., and SAMULSKI, R.J. (1996). Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70, 8098-8108.
    • (1996) J. Virol. , vol.70 , pp. 8098-8108
    • Xiao, X.1    Li, J.2    Samulski, R.J.3
  • 24
    • 0034612248 scopus 로고    scopus 로고
    • From the cover: Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy
    • YAN, Z., ZHANG, Y., DUAN, D., and ENGELHARDT, J.F. (2000). From the cover: Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy [see comments]. Proc. Natl. Acad. Sci. U.S.A. 97, 6716-6721.
    • (2000) Proc. Natl. Acad. Sci. U.S.A. , vol.97 , pp. 6716-6721
    • Yan, Z.1    Zhang, Y.2    Duan, D.3    Engelhardt, J.F.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.