-
1
-
-
0034779927
-
A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease
-
AITKEN, M.L., MOSS, R.B., WALTZ, D.A., DOVEY, M.E., TONELLI, M.R., McNAMARA, S.C., GIBSON, R.L., RAMSEY, B.W., CARTER, B.J., and REYNOLDS, T.C. (2001). A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease. Hum. Gene Then 12, 1907-1916.
-
(2001)
Hum. Gene Then
, vol.12
, pp. 1907-1916
-
-
Aitken, M.L.1
Moss, R.B.2
Waltz, D.A.3
Dovey, M.E.4
Tonelli, M.R.5
McNamara, S.C.6
Gibson, R.L.7
Ramsey, B.W.8
Carter, B.J.9
Reynolds, T.C.10
-
2
-
-
0033586375
-
Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: A double-blind placebo-controlled trial
-
ALTON, E.W.F.W., STERN, M., FARLEY, R., JAFFEE, A., CHADWICK, S.L., PHILLIPS, J., DAVIES, J., SMITH, S.N., BROWNING, J., DAVIES, M.G., HODSON, M.E., DURHAM, S.R. LI, D., JEFFERY, P.K., SCALLAN, M., BALFOUR, R., EASTMAN, S.J., CHENG, S.H., SMITH, A.E., MEEKER, D., and GEDDES, D.M. (1999). Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: A double-blind placebo-controlled trial. Lancet 353, 947-954.
-
(1999)
Lancet
, vol.353
, pp. 947-954
-
-
Alton, E.W.F.W.1
Stern, M.2
Farley, R.3
Jaffee, A.4
Chadwick, S.L.5
Phillips, J.6
Davies, J.7
Smith, S.N.8
Browning, J.9
Davies, M.G.10
Hodson, M.E.11
Durham, S.R.12
Li, D.13
Jeffery, P.K.14
Scallan, M.15
Balfour, R.16
Eastman, S.J.17
Cheng, S.H.18
Smith, A.E.19
Meeker, D.20
Geddes, D.M.21
more..
-
3
-
-
0026641473
-
Expression of the cystic fibrosis transmembrane conductance regulator gene can be regulated by protein kinase C
-
BARGON, J., TRAPNELL, B.C., YOSHIMURA, K., DALEMANS, W., PAVIRANI, A., LECOCQ, J.P., and CRYSTAL, R.G. (1992). Expression of the cystic fibrosis transmembrane conductance regulator gene can be regulated by protein kinase C. J. Biol. Chem. 267, 16056-16060.
-
(1992)
J. Biol. Chem.
, vol.267
, pp. 16056-16060
-
-
Bargon, J.1
Trapnell, B.C.2
Yoshimura, K.3
Dalemans, W.4
Pavirani, A.5
Lecocq, J.P.6
Crystal, R.G.7
-
4
-
-
0028077815
-
Interferon-γ downregulates CFTR gene expression in epithelial cells
-
BESANÇON, F., PRZEWLOCKI, G., BARO, I., HONGRE, A.S., ESCANDE, D., and EDELMAN, A. (1994). Interferon-γ downregulates CFTR gene expression in epithelial cells. Am. J. Physiol. 267, C1398-C1404.
-
(1994)
Am. J. Physiol.
, vol.267
-
-
Besançon, F.1
Przewlocki, G.2
Baro, I.3
Hongre, A.S.4
Escande, D.5
Edelman, A.6
-
5
-
-
0033557267
-
Status of gene therapy for cystic fibrosis lung disease
-
BOUCHER, R.C. (1999). Status of gene therapy for cystic fibrosis lung disease. J. Clin. Invest. 103, 441-445.
-
(1999)
J. Clin. Invest.
, vol.103
, pp. 441-445
-
-
Boucher, R.C.1
-
6
-
-
0035199694
-
Examining basal chloride transport using the nasal potential difference response in a murine model
-
BRADY, K.G., KELLEY, T.J., and DRUMM, M.L. (2001). Examining basal chloride transport using the nasal potential difference response in a murine model. Am. J. Physiol. Lung Cell Mol. Physiol. 281, L1173-L1179.
-
(2001)
Am. J. Physiol. Lung Cell Mol. Physiol.
, vol.281
-
-
Brady, K.G.1
Kelley, T.J.2
Drumm, M.L.3
-
7
-
-
0027221955
-
Protein kinase C mediates down-regulation of cystic fibrosis transmembrane conductance regulator levels in epithelial cells
-
BREUER, W., GLICKSTEIN, H., KARTNER, N., RIORDAN, J.R., AUSIELLO, D.A., and CABANTCHIK, I.Z. (1993). Protein kinase C mediates down-regulation of cystic fibrosis transmembrane conductance regulator levels in epithelial cells. J. Biol. Chem. 268, 13935-13939.
-
(1993)
J. Biol. Chem.
, vol.268
, pp. 13935-13939
-
-
Breuer, W.1
Glickstein, H.2
Kartner, N.3
Riordan, J.R.4
Ausiello, D.A.5
Cabantchik, I.Z.6
-
8
-
-
0034695758
-
Interleukin-1β regulates CFTR expression in human intestinal T84 cells
-
CAFFERATA, E.G., GONZALEZ-GUERRICO, A.M., GIORDANO, L., PIVETTA, O.H., and SANTA-COLOMA, T.A. (2000). Interleukin-1β regulates CFTR expression in human intestinal T84 cells. Biochim. Biophys. Acta 1500, 241-248.
-
(2000)
Biochim. Biophys. Acta
, vol.1500
, pp. 241-248
-
-
Cafferata, E.G.1
Gonzalez-Guerrico, A.M.2
Giordano, L.3
Pivetta, O.H.4
Santa-Coloma, T.A.5
-
9
-
-
0028913411
-
Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis
-
CAPLEN, N.J., ALTON, E.W., MIDDLETON, P.C., DORIN, J.R., STEVENSON, B.J., GAO, X., DURHAM, S.R., JEFFERY, P.K., HODSON, M.E., COUTELLE, C., HUANG, L., PORTEOUS, D.J., WILLIAMSON, R., and GEDDES, D.M. (1995). Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat. Med. 1, 39-46.
-
(1995)
Nat. Med.
, vol.1
, pp. 39-46
-
-
Caplen, N.J.1
Alton, E.W.2
Middleton, P.C.3
Dorin, J.R.4
Stevenson, B.J.5
Gao, X.6
Durham, S.R.7
Jeffery, P.K.8
Hodson, M.E.9
Coutelle, C.10
Huang, L.11
Porteous, D.J.12
Williamson, R.13
Geddes, D.M.14
-
10
-
-
0026659050
-
IgG antibody response to polyethylene glycol-modified adenosine deaminase in patients with adenosine deaminase deficiency
-
CHAFFEE, S., MARY, A., STIEHM, E.R., GIRAULT, D., FISCHER, A., and HERSHFIELD, M.S. (1992). IgG antibody response to polyethylene glycol-modified adenosine deaminase in patients with adenosine deaminase deficiency. J. Clin. Invest. 89, 1643-1651.
-
(1992)
J. Clin. Invest.
, vol.89
, pp. 1643-1651
-
-
Chaffee, S.1
Mary, A.2
Stiehm, E.R.3
Girault, D.4
Fischer, A.5
Hershfield, M.S.6
-
11
-
-
0000700054
-
Reactivation of silenced, virally transduced genes by inhibitors of histone deacetylase
-
CHEN, W.Y., BAILEY, E.C., McCUNE, S.L., DONG, J.-Y., and TOWNES, T.M. (1997). Reactivation of silenced, virally transduced genes by inhibitors of histone deacetylase. Proc. Natl. Acad. Sci. U.S.A. 94, 5798-5803.
-
(1997)
Proc. Natl. Acad. Sci. U.S.A.
, vol.94
, pp. 5798-5803
-
-
Chen, W.Y.1
Bailey, E.C.2
McCune, S.L.3
Dong, J.-Y.4
Townes, T.M.5
-
12
-
-
0033977047
-
Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nonhuman primate muscle
-
CHIRMULE, N., XIAO, W., TRUNEH, A., SCHNELL, M.A., HUGHES, J.V., ZOLTICK, P., and WILSON, J.M. (2000). Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nonhuman primate muscle. J. Virol. 74, 2420-2425.
-
(2000)
J. Virol.
, vol.74
, pp. 2420-2425
-
-
Chirmule, N.1
Xiao, W.2
Truneh, A.3
Schnell, M.A.4
Hughes, J.V.5
Zoltick, P.6
Wilson, J.M.7
-
13
-
-
0029741735
-
Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung
-
CONRAD, C.K., ALLEN, S.S., AFIONE, S.A., REYNOLDS, T.C., BECK, S.E., FEE-MAKI, M., BARRAZZA-ORTIZ, X., ADAMS, R., ASKIN, F.B., CARTER, B.J., GUGGINO, W.B., and FLOTTE, T.R. (1996). Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung. Gene Ther. 3, 658-668.
-
(1996)
Gene Ther.
, vol.3
, pp. 658-668
-
-
Conrad, C.K.1
Allen, S.S.2
Afione, S.A.3
Reynolds, T.C.4
Beck, S.E.5
Fee-Maki, M.6
Barrazza-Ortiz, X.7
Adams, R.8
Askin, F.B.9
Carter, B.J.10
Guggino, W.B.11
Flotte, T.R.12
-
14
-
-
0027476449
-
Cationic proteins induce airway hyperresponsiveness dependent on charge interactions
-
COYLE, A.J., ACKERMAN, S.J., and IRVIN, C.G. (1993). Cationic proteins induce airway hyperresponsiveness dependent on charge interactions. Am. Rev. Respir. Dis. 147, 896-900.
-
(1993)
Am. Rev. Respir. Dis.
, vol.147
, pp. 896-900
-
-
Coyle, A.J.1
Ackerman, S.J.2
Irvin, C.G.3
-
15
-
-
0028168641
-
Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis
-
CRYSTAL, R.G., MCELVANEY, N.G., ROSENFELD, M.A., CHU, C.S., MASTRANGELI, A., HAY, J.G., BRODY, S.L., JAFFE, H.A., EISSA, N.T., and DANEL, C. (1994). Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nat. Genet. 8, 42-51.
-
(1994)
Nat. Genet.
, vol.8
, pp. 42-51
-
-
Crystal, R.G.1
Mcelvaney, N.G.2
Rosenfeld, M.A.3
Chu, C.S.4
Mastrangeli, A.5
Hay, J.G.6
Brody, S.L.7
Jaffe, H.A.8
Eissa, N.T.9
Danel, C.10
-
16
-
-
0029823833
-
Cystic fibrosis
-
DAVIS, P.B., DRUMM, M., and KONSTAN, M.W. (1996). Cystic fibrosis. Am. J. Respir. Crit. Care Med. 154, 1229-1256.
-
(1996)
Am. J. Respir. Crit. Care Med.
, vol.154
, pp. 1229-1256
-
-
Davis, P.B.1
Drumm, M.2
Konstan, M.W.3
-
17
-
-
4143074815
-
Relation of sweat chloride concentration to severity of lung disease in cystic fibrosis
-
DAVIS, P.B., SCHLUCHTER, M.D., and KONSTAN, M.W. (2004). Relation of sweat chloride concentration to severity of lung disease in cystic fibrosis. Pediatr. Pulmonol. 38, 204-209.
-
(2004)
Pediatr. Pulmonol.
, vol.38
, pp. 204-209
-
-
Davis, P.B.1
Schluchter, M.D.2
Konstan, M.W.3
-
18
-
-
0029813969
-
A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction
-
DORIN, J.R., PARLEY, R., WEBB, S., SMITH, S.N., FARINI, E., DELANEY, S.J., WAINWRIGHT, B.J., ALTON, E.W., and PORTEOUS, D.J. (1996). A demonstration using mouse models that successful gene therapy for cystic fibrosis requires only partial gene correction. Gene Ther. 3, 797-801.
-
(1996)
Gene Ther.
, vol.3
, pp. 797-801
-
-
Dorin, J.R.1
Parley, R.2
Webb, S.3
Smith, S.N.4
Farini, E.5
Delaney, S.J.6
Wainwright, B.J.7
Alton, E.W.8
Porteous, D.J.9
-
19
-
-
0025094612
-
Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer
-
DRUMM, M.L., POPE, H.A., CLIFF, W.H., ROMMENS, J.M., MARVIN, S.A., TSUI, L.C., COLLINS, F.S., FRIZZELL, R.A., and WILSON, J.M. (1990). Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer. Cell 62, 1227-1233.
-
(1990)
Cell
, vol.62
, pp. 1227-1233
-
-
Drumm, M.L.1
Pope, H.A.2
Cliff, W.H.3
Rommens, J.M.4
Marvin, S.A.5
Tsui, L.C.6
Collins, F.S.7
Frizzell, R.A.8
Wilson, J.M.9
-
20
-
-
0029761375
-
Immunologic responses to gene transfer into mice via the polymeric immunoglobulin receptor
-
FERKOL, T., PELLICENA-PALLE, A., ECKMAN, E., PERALES, J.C., TRZASKA, T., TOSI, M., REDLINE, R., and DAVIS, P.B. (1996). Immunologic responses to gene transfer into mice via the polymeric immunoglobulin receptor. Gene Ther. 8, 669-678.
-
(1996)
Gene Ther.
, vol.8
, pp. 669-678
-
-
Ferkol, T.1
Pellicena-Palle, A.2
Eckman, E.3
Perales, J.C.4
Trzaska, T.5
Tosi, M.6
Redline, R.7
Davis, P.B.8
-
21
-
-
0038488551
-
Phase I trial of intranasal and endobronchial administration of a recombinant adenoassociated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: A two-part clinical study
-
FLOTTE, T.R., ZEITLIN, P.L., REYNOLDS, T.C., HEALD, A.E., PEDERSEN, P., BECK, S., CONRAD, C.K., BRASS-ERNST, L., HUMPHRIES, M., SULLIVAN, K., WETZEL, R., TAYLOR, G., CARTER, B.J., and GUGGINO, W.B. (2003). Phase I trial of intranasal and endobronchial administration of a recombinant adenoassociated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: A two-part clinical study. Hum. Gene Ther. 14, 1079-1088.
-
(2003)
Hum. Gene Ther.
, vol.14
, pp. 1079-1088
-
-
Flotte, T.R.1
Zeitlin, P.L.2
Reynolds, T.C.3
Heald, A.E.4
Pedersen, P.5
Beck, S.6
Conrad, C.K.7
Brass-Ernst, L.8
Humphries, M.9
Sullivan, K.10
Wetzel, R.11
Taylor, G.12
Carter, B.J.13
Guggino, W.B.14
-
22
-
-
8244238394
-
A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis
-
GILL, D.R., SOUTHERN, K.W., MOFFORD, K.A., SEDDON, T., HUANG, L., SORGI, F., THOMSON, A., MACVINISH, L.J., RATCLIFF, R., BILTON, D., LANE, D.J., LITTLEWOOD, J.M., WEBB, A.K., MIDDLETON, P.G., COLLEDGE, W.H., CUTHBERT, A.W., EVANS, M.J., HIGGINS, C.F., and HYDE, S.C. (1997). A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther. 4, 199-209.
-
(1997)
Gene Ther.
, vol.4
, pp. 199-209
-
-
Gill, D.R.1
Southern, K.W.2
Mofford, K.A.3
Seddon, T.4
Huang, L.5
Sorgi, F.6
Thomson, A.7
Macvinish, L.J.8
Ratcliff, R.9
Bilton, D.10
Lane, D.J.11
Littlewood, J.M.12
Webb, A.K.13
Middleton, P.G.14
Colledge, W.H.15
Cuthbert, A.W.16
Evans, M.J.17
Higgins, C.F.18
Hyde, S.C.19
-
23
-
-
0035166381
-
Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1α promoter
-
GILL, D.R., SMYTH, S.E., GODDARD, C.A., PRINGLE, I.A., HIGGINS, C.F., COLLEDGE, W.H., and HYDE, S.C. (2001). Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1α promoter. Gene Ther. 8, 1539-1546.
-
(2001)
Gene Ther.
, vol.8
, pp. 1539-1546
-
-
Gill, D.R.1
Smyth, S.E.2
Goddard, C.A.3
Pringle, I.A.4
Higgins, C.F.5
Colledge, W.H.6
Hyde, S.C.7
-
24
-
-
0028878052
-
Gene therapy in a xenograft model of cystic fibrosis lung corrects chloride transport more effectively than the sodium defect
-
GOLDMAN, M.J., YANG, Y., and WILSON, J.M. (1995). Gene therapy in a xenograft model of cystic fibrosis lung corrects chloride transport more effectively than the sodium defect. Nat. Genet. 9, 126-131.
-
(1995)
Nat. Genet.
, vol.9
, pp. 126-131
-
-
Goldman, M.J.1
Yang, Y.2
Wilson, J.M.3
-
25
-
-
0032718592
-
Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus
-
HARVEY, B.G., LEOPOLD, P.L., HACKETT, N.R., GRASSO, T.M., WILLIAMS, P.M., TUCKER, A.L., KANER, R.J., FERRIS, B., GONDA, I., SWEENEY, T.D., RAMALINGAM, R., KOVESDI, I., SHAK, S., and CRYSTAL, R.G. (1999). Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus. J. Clin. Invest. 104, 1245-1255.
-
(1999)
J. Clin. Invest.
, vol.104
, pp. 1245-1255
-
-
Harvey, B.G.1
Leopold, P.L.2
Hackett, N.R.3
Grasso, T.M.4
Williams, P.M.5
Tucker, A.L.6
Kaner, R.J.7
Ferris, B.8
Gonda, I.9
Sweeney, T.D.10
Ramalingam, R.11
Kovesdi, I.12
Shak, S.13
Crystal, R.G.14
-
26
-
-
0028802876
-
Modification of nasal epithelial potential differences of individuals with cystic fibrosis consequent to local administration of a normal CFTR cDNA adenovirus gene transfer vector
-
HAY, J.G., MCELVANEY, N.G., HERENA, J., and CRYSTAL, R.G. (1995). Modification of nasal epithelial potential differences of individuals with cystic fibrosis consequent to local administration of a normal CFTR cDNA adenovirus gene transfer vector. Hum. Gene Ther. 6, 1487-1496.
-
(1995)
Hum. Gene Ther.
, vol.6
, pp. 1487-1496
-
-
Hay, J.G.1
Mcelvaney, N.G.2
Herena, J.3
Crystal, R.G.4
-
27
-
-
0035013543
-
The ΔF508 mutation shortens the biochemical half-life of plasma membrane CFTR in polarized epithelial cells
-
HEDA, G.D., TANWANI, M., and MARINO, C.R. (2001). The ΔF508 mutation shortens the biochemical half-life of plasma membrane CFTR in polarized epithelial cells. Am. J. Physiol. Cell Physiol. 280, C166-C174.
-
(2001)
Am. J. Physiol. Cell Physiol.
, vol.280
-
-
Heda, G.D.1
Tanwani, M.2
Marino, C.R.3
-
28
-
-
0027475701
-
Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy
-
HYDE, S.C., GILL, D.R., HIGGINS, C.F., TREZISE, A.E., MACVINISH, L.J., CUTHBERT, A.W., RATCLIFF, R., EVANS, M.J., and COLLEDGE, W.H. (1993). Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy. Nature 362, 250-255.
-
(1993)
Nature
, vol.362
, pp. 250-255
-
-
Hyde, S.C.1
Gill, D.R.2
Higgins, C.F.3
Trezise, A.E.4
Macvinish, L.J.5
Cuthbert, A.W.6
Ratcliff, R.7
Evans, M.J.8
Colledge, W.H.9
-
29
-
-
0026918861
-
Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis
-
JOHNSON, L.G., OLSEN, J.C., SARKADI, B., MOORE, K.L., SWANSTROM, R., and BOUCHER, R.C. (1992). Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nat. Genet. 2, 21-25.
-
(1992)
Nat. Genet.
, vol.2
, pp. 21-25
-
-
Johnson, L.G.1
Olsen, J.C.2
Sarkadi, B.3
Moore, K.L.4
Swanstrom, R.5
Boucher, R.C.6
-
30
-
-
0028912888
-
Normalization of raised sodium absorption and raised calcium-mediated chloride secretion by adenovirus-mediated expression of cystic fibrosis transmembrane conductance regulator in primary human cystic fibrosis airway epithelial cells
-
JOHNSON, L.G., BOYLES, S.E., WILSON, J., and BOUCHER, R.C. (1995). Normalization of raised sodium absorption and raised calcium-mediated chloride secretion by adenovirus-mediated expression of cystic fibrosis transmembrane conductance regulator in primary human cystic fibrosis airway epithelial cells. J. Clin. Invest. 95, 1377-1382.
-
(1995)
J. Clin. Invest.
, vol.95
, pp. 1377-1382
-
-
Johnson, L.G.1
Boyles, S.E.2
Wilson, J.3
Boucher, R.C.4
-
31
-
-
0038805302
-
Immunity to adenovirus and adeno-associated viral vectors: Implication for gene therapy
-
JOOSS, K., and CHIRMULE, N. (2003). Immunity to adenovirus and adeno-associated viral vectors: Implication for gene therapy. Gene Ther. 10, 955-963.
-
(2003)
Gene Ther.
, vol.10
, pp. 955-963
-
-
Jooss, K.1
Chirmule, N.2
-
32
-
-
0024423668
-
Identification of the cystic fibrosis gene: Genetic analysis
-
KEREM, B., ROMMENS, J.M., BUCHANAN, J.A., MARKIEWICZ, D., COX, T.K., CHAKRAVARTI, A., BUCHWALD, M., and TSUI, L.-C. (1989). Identification of the cystic fibrosis gene: Genetic analysis. Science 245, 1073-1080.
-
(1989)
Science
, vol.245
, pp. 1073-1080
-
-
Kerem, B.1
Rommens, J.M.2
Buchanan, J.A.3
Markiewicz, D.4
Cox, T.K.5
Chakravarti, A.6
Buchwald, M.7
Tsui, L.-C.8
-
33
-
-
0012176095
-
Three-dimensional imaging of EGFP expression in mouse lung using compacted DNA nanoparticles
-
KERSCHMANN, R,. ZIADY, A., PAYNE, J.M., GEDEON, C., MUHAMMAD, O., KOWALCZYK, T.H., PASUMARTHY, M.K., HYATT, S., MOEN, R.C., and COOPER, M.J. (2001). Three-dimensional imaging of EGFP expression in mouse lung using compacted DNA nanoparticles. Mol. Ther. 3, S404.
-
(2001)
Mol. Ther.
, vol.3
-
-
Kerschmann, R.1
Ziady, A.2
Payne, J.M.3
Gedeon, C.4
Muhammad, O.5
Kowalczyk, T.H.6
Pasumarthy, M.K.7
Hyatt, S.8
Moen, R.C.9
Cooper, M.J.10
-
34
-
-
0029100239
-
A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis
-
KNOWLES, M.R., HOHNEKER, K.W., ZHOU, Z., OLSEN, J.C., NOAH, T.L., HU, P.C., LEIGH, M.W., ENGELHARDT, J.F., EDWARDS, L.J., JONES, K.R., GROSSMAN, M., WILSON, J.M., JOHNSON, L.G., and BOUCHER, R.C. (1995a). A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. N. Engl. J. Med. 333, 823-831.
-
(1995)
N. Engl. J. Med.
, vol.333
, pp. 823-831
-
-
Knowles, M.R.1
Hohneker, K.W.2
Zhou, Z.3
Olsen, J.C.4
Noah, T.L.5
Hu, P.C.6
Leigh, M.W.7
Engelhardt, J.F.8
Edwards, L.J.9
Jones, K.R.10
Grossman, M.11
Wilson, J.M.12
Johnson, L.G.13
Boucher, R.C.14
-
35
-
-
0028991826
-
In vivo nasal potential difference: Techniques and protocols for assessing efficacy of gene transfer in cystic fibrosis
-
KNOWLES, M.R., PARADISO, A.M., and BOUCHER, R.C. (1995b). In vivo nasal potential difference: Techniques and protocols for assessing efficacy of gene transfer in cystic fibrosis. Hum. Gene Ther. 6, 445-455.
-
(1995)
Hum. Gene Ther.
, vol.6
, pp. 445-455
-
-
Knowles, M.R.1
Paradiso, A.M.2
Boucher, R.C.3
-
37
-
-
0037027948
-
Pharmacological approaches for the discovery and development of new anti-inflammatory agents for the treatment of cystic fibrosis
-
KONSTAN, M.W., and DAVIS, P.B. (2002). Pharmacological approaches for the discovery and development of new anti-inflammatory agents for the treatment of cystic fibrosis. Adv. Drug Deliv. Rev. 54, 1409-1423.
-
(2002)
Adv. Drug Deliv. Rev.
, vol.54
, pp. 1409-1423
-
-
Konstan, M.W.1
Davis, P.B.2
-
38
-
-
10744220021
-
Ubiquitin promoter generates prolonged high level gene expression following intrapulmonary administration of compacted DNA nanoparticles
-
KOWALCZYK, T.H., GEDEON, C.R., OETTE, S.M., MUHAMMAD, O., HYATT, S.L., PEISCHL, A.R., FINK, T.L., KLEPCYK, P.J., MOEN, R.C., COOPER, M.J., and GILL, D.R. (2003). Ubiquitin promoter generates prolonged high level gene expression following intrapulmonary administration of compacted DNA nanoparticles. Mol. Ther. 7, S324.
-
(2003)
Mol. Ther.
, vol.7
-
-
Kowalczyk, T.H.1
Gedeon, C.R.2
Oette, S.M.3
Muhammad, O.4
Hyatt, S.L.5
Peischl, A.R.6
Fink, T.L.7
Klepcyk, P.J.8
Moen, R.C.9
Cooper, M.J.10
Gill, D.R.11
-
39
-
-
0347388017
-
Intracellular trafficking of non-targeted stabilized molecular conjugates in human airway epithelia cells
-
KUBE, D., and DAVIS, P.B. (2003). Intracellular trafficking of non-targeted stabilized molecular conjugates in human airway epithelia cells. Mol. Ther. 7, S371.
-
(2003)
Mol. Ther.
, vol.7
-
-
Kube, D.1
Davis, P.B.2
-
40
-
-
0032962975
-
Metabolic instability of plasmid DNA in the cytosol: A potential barrier to gene transfer
-
LECHARDEUR, D., SOHN, K.J., HAARDT, M., JOSHI, P.B., MONCK, M., GRAHAM, R.W., BEATTY, B., SQUIRE, J., O'BRODUVICH, H., and LUKACS, G.L. (1999). Metabolic instability of plasmid DNA in the cytosol: A potential barrier to gene transfer. Gene Ther. 6, 482-497.
-
(1999)
Gene Ther.
, vol.6
, pp. 482-497
-
-
Lechardeur, D.1
Sohn, K.J.2
Haardt, M.3
Joshi, P.B.4
Monck, M.5
Graham, R.W.6
Beatty, B.7
Squire, J.8
O'Broduvich, H.9
Lukacs, G.L.10
-
41
-
-
0042859742
-
Nanoparticles of compacted DNA transfect post-mitotic cells
-
LIU, G., LI, D., PASUMARTHY, M.K., KOWALCZYK, T.H., GEDEON, C., HYATT, S., PAYNE, J.M., MILLER, T.J., BRUNOVSKIS, P., MOEN, R.C., HANSON, R.W., and COOPER, M.J. (2003). Nanoparticles of compacted DNA transfect post-mitotic cells. J. Biol. Chem. 278, 32578-32586.
-
(2003)
J. Biol. Chem.
, vol.278
, pp. 32578-32586
-
-
Liu, G.1
Li, D.2
Pasumarthy, M.K.3
Kowalczyk, T.H.4
Gedeon, C.5
Hyatt, S.6
Payne, J.M.7
Miller, T.J.8
Brunovskis, P.9
Moen, R.C.10
Hanson, R.W.11
Cooper, M.J.12
-
42
-
-
0027380236
-
The ΔF508 mutation decreases the stability of cystic fibrosis transmembrane conductance regulator in the plasma membrane: Determination of functional half-lives on transfected cells
-
LUKACS, G.L., CHANG, X.B., BEAR, C., KARTNER, N., MOHAMED, A., RIORDAN, J.R., and GRINSTEIN, S. (1993). The ΔF508 mutation decreases the stability of cystic fibrosis transmembrane conductance regulator in the plasma membrane: Determination of functional half-lives on transfected cells. J. Biol. Chem. 268, 21592-21598.
-
(1993)
J. Biol. Chem.
, vol.268
, pp. 21592-21598
-
-
Lukacs, G.L.1
Chang, X.B.2
Bear, C.3
Kartner, N.4
Mohamed, A.5
Riordan, J.R.6
Grinstein, S.7
-
44
-
-
0028899392
-
IL-6 release and airway administration of human CFTR cDNA adenovirus vector
-
McELVANEY, N.G., and CRYSTAL, R.G. (1995). IL-6 release and airway administration of human CFTR cDNA adenovirus vector. Nat. Med. 1, 182-184.
-
(1995)
Nat. Med.
, vol.1
, pp. 182-184
-
-
McElvaney, N.G.1
Crystal, R.G.2
-
45
-
-
0028020058
-
Protocols for in vivo measurement of the ion transport defects in cystic fibrosis nasal epithelium
-
MIDDLETON, P.C., GEDDES, D.M., and ALTON, E.W. (1994). Protocols for in vivo measurement of the ion transport defects in cystic fibrosis nasal epithelium. Eur. Respir. J. 7, 2050-2056.
-
(1994)
Eur. Respir. J.
, vol.7
, pp. 2050-2056
-
-
Middleton, P.C.1
Geddes, D.M.2
Alton, E.W.3
-
46
-
-
10744227749
-
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, R.B., RODMAN, D., SPENCER, L.T., AITKEN, M.L., ZEITLIN, P.L., WALTZ, D., MILLA, C., BRODY, A.S., CLANCY, J.P., RAMSEY, B., HAMBLETT, N., and HEALD, A.E. (2004). 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 125, 509-521.
-
(2004)
Chest
, vol.125
, pp. 509-521
-
-
Moss, R.B.1
Rodman, D.2
Spencer, L.T.3
Aitken, M.L.4
Zeitlin, P.L.5
Waltz, D.6
Milla, C.7
Brody, A.S.8
Clancy, J.P.9
Ramsey, B.10
Hamblett, N.11
Heald, A.E.12
-
47
-
-
0026619710
-
Tumor necrosis factor modulation of expression of the cystic fibrosis transmembrane conductance regulator gene
-
NAKAMURA, H., YOSHIMURA, K., BAJOCCHI, G., TRAPNELL, B.C., PAVIRANI, A., and CRYSTAL, R.G. (1992). Tumor necrosis factor modulation of expression of the cystic fibrosis transmembrane conductance regulator gene. FEBS Lett. 314, 366-370.
-
(1992)
FEBS Lett.
, vol.314
, pp. 366-370
-
-
Nakamura, H.1
Yoshimura, K.2
Bajocchi, G.3
Trapnell, B.C.4
Pavirani, A.5
Crystal, R.G.6
-
48
-
-
0033660570
-
Safety and biological efficacy of a lipid-CFTR complex for gene transfer in the nasal epithelium of adult patients with cystic fibrosis
-
NOONE, P.G., HOHNEKER, K.W., ZHOU, Z., JOHNSON, L.G., FOY, C., GIPSON, C., JONES, K., NOAH, T.L., LEIGH, M.W., SCHWARTZBACH, C., EFTHIMIOU, J., PEARLMAN, R., BOUCHER, R.C., and KNOWLES, M.R. (2000). Safety and biological efficacy of a lipid-CFTR complex for gene transfer in the nasal epithelium of adult patients with cystic fibrosis. Mol. Ther. 1, 105-114.
-
(2000)
Mol. Ther.
, vol.1
, pp. 105-114
-
-
Noone, P.G.1
Hohneker, K.W.2
Zhou, Z.3
Johnson, L.G.4
Foy, C.5
Gipson, C.6
Jones, K.7
Noah, T.L.8
Leigh, M.W.9
Schwartzbach, C.10
Efthimiou, J.11
Pearlman, R.12
Boucher, R.C.13
Knowles, M.R.14
-
49
-
-
11144241287
-
Aerosols of compacted DNA nanoparticles retain structural integrity and biological activity
-
OETTE, S.M., GEDEON, C.R., KOWALCZYK, T.H., FINK, T.L., HYATT, S.L., MOEN, R.C., and COOPER, M.J. (2004). Aerosols of compacted DNA nanoparticles retain structural integrity and biological activity. Mol. Ther. 9, S190.
-
(2004)
Mol. Ther.
, vol.9
-
-
Oette, S.M.1
Gedeon, C.R.2
Kowalczyk, T.H.3
Fink, T.L.4
Hyatt, S.L.5
Moen, R.C.6
Cooper, M.J.7
-
50
-
-
0029789793
-
Activation of the complement system by synthetic DNA complexes: A potential barrier for intravenous gene delivery
-
PLANK, C., MECHTLER, K., SZOKA, P.C., and WAGNER, E. (1996). Activation of the complement system by synthetic DNA complexes: A potential barrier for intravenous gene delivery. Hum. Gene Ther. 7, 1437-1446.
-
(1996)
Hum. Gene Ther.
, vol.7
, pp. 1437-1446
-
-
Plank, C.1
Mechtler, K.2
Szoka, P.C.3
Wagner, E.4
-
51
-
-
0035287881
-
2+-sensitive cytosolic nucleases prevent efficient delivery to the nucleus of injected plasmids
-
2+-sensitive cytosolic nucleases prevent efficient delivery to the nucleus of injected plasmids. J. Gene Med. 3, 153-164.
-
(2001)
J. Gene Med.
, vol.3
, pp. 153-164
-
-
Pollard, J.1
Toumaniantz, G.2
Amos, J.L.3
Avet-Loiseau, H.4
Guihard, G.5
Behr, J.P.6
Escande, D.7
-
52
-
-
8244245761
-
Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis
-
PORTEOUS, D.J., DORIN, J.R., McLACHLAN, G., DAVIDSON-SMITH, H., DAVIDSON, H., STEVENSON, B.J., CAROTHERS, A.D., WALLACE, W.A., MORALEE, S., HOENES, C., KALLMEYER, G., MICHAELIS, U., NAUJOKS, K., HO, L.P., SAMWAYS, J.M., IMRIE, M., GREENING, A.P., and INNES, J.A. (1997). Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther. 4, 210-218.
-
(1997)
Gene Ther.
, vol.4
, pp. 210-218
-
-
Porteous, D.J.1
Dorin, J.R.2
McLachlan, G.3
Davidson-Smith, H.4
Davidson, H.5
Stevenson, B.J.6
Carothers, A.D.7
Wallace, W.A.8
Moralee, S.9
Hoenes, C.10
Kallmeyer, G.11
Michaelis, U.12
Naujoks, K.13
Ho, L.P.14
Samways, J.M.15
Imrie, M.16
Greening, A.P.17
Innes, J.A.18
-
53
-
-
0030010409
-
Inactivation of the very strong HCMV immediate early promoter by DNA CpG methylation in vitro
-
PROSCH, S., STEIN, J., STAAK, K., LIEBENTHAL, C., VOLK, H,-D., and KRUGER, D.H. (1996). Inactivation of the very strong HCMV immediate early promoter by DNA CpG methylation in vitro. Biol. Chem. Hoppe-Seyler 377, 195-201.
-
(1996)
Biol. Chem. Hoppe-Seyler
, vol.377
, pp. 195-201
-
-
Prosch, S.1
Stein, J.2
Staak, K.3
Liebenthal, C.4
Volk, H.-D.5
Kruger, D.H.6
-
54
-
-
0031581174
-
Promoter attenuation in gene therapy: Interferon-γ and tumor necrosis factor-α inhibit transgene expression
-
QIN, L., DING, Y., PAHUD, D.R., CHANG, E., IMPERIALE, M.J., and BROMBERG, J.S. (1997). Promoter attenuation in gene therapy: Interferon-γ and tumor necrosis factor-α inhibit transgene expression. Hum. Gene Ther. 8, 2019-2029.
-
(1997)
Hum. Gene Ther.
, vol.8
, pp. 2019-2029
-
-
Qin, L.1
Ding, Y.2
Pahud, D.R.3
Chang, E.4
Imperiale, M.J.5
Bromberg, J.S.6
-
55
-
-
0029934215
-
Management of pulmonary disease in patients with cystic fibrosis
-
RAMSEY, B.W. (1996). Management of pulmonary disease in patients with cystic fibrosis. N. Engl. J. Med. 335, 179-188.
-
(1996)
N. Engl. J. Med.
, vol.335
, pp. 179-188
-
-
Ramsey, B.W.1
-
56
-
-
0024424270
-
Identification of the cystic fibrosis gene: Cloning and characterization of complementary DNA
-
RIORDAN, J.R., ROMMENS, J.M., KEREM, B., ALON, N., ROZMAHEL, R., GRZELCZAK, Z., ZIELENSKI, J., LOK, S., PLAVSIC, N., CHOU, J.L., DRUMM, M.L., IANNUZZI, M.C., COLLINS, F.S., and TSUI, L.-C. (1989). Identification of the cystic fibrosis gene: Cloning and characterization of complementary DNA. Science 245, 1066-1073.
-
(1989)
Science
, vol.245
, pp. 1066-1073
-
-
Riordan, J.R.1
Rommens, J.M.2
Kerem, B.3
Alon, N.4
Rozmahel, R.5
Grzelczak, Z.6
Zielenski, J.7
Lok, S.8
Plavsic, N.9
Chou, J.L.10
Drumm, M.L.11
Iannuzzi, M.C.12
Collins, F.S.13
Tsui, L.-C.14
-
57
-
-
0024453308
-
Identification of the cystic fibrosis gene: Chromosome walking and jumping
-
ROMMENS, J.M., IANNUZZI, M.C., KEREM, B., DRUMM, M.L., MELMER, G., DEAN, M., ROZMAHEL, R., COLE, J.L., KENNEDY, D., HIDAKA, N., ZSIGA, M., BUCHWALD, M., RIORDAN, J.R., TSUI, L.-C., and COLLINS, F.S. (1989). Identification of the cystic fibrosis gene: Chromosome walking and jumping. Science 245, 1059-1065.
-
(1989)
Science
, vol.245
, pp. 1059-1065
-
-
Rommens, J.M.1
Iannuzzi, M.C.2
Kerem, B.3
Drumm, M.L.4
Melmer, G.5
Dean, M.6
Rozmahel, R.7
Cole, J.L.8
Kennedy, D.9
Hidaka, N.10
Zsiga, M.11
Buchwald, M.12
Riordan, J.R.13
Tsui, L.-C.14
Collins, F.S.15
-
58
-
-
0034915570
-
A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis
-
RUIZ, F.E., CLANCY, J.P., PERRICONE, M.A., BEBOK, Z., HONG, J.S., CHENG, S.H., MEEKER, D.P., YOUNG, K.R., SCHOUMACHER, R.A., WEATHERLY, M.R., WING, L., MORRIS, J.E., SINDEL, L., ROSENBERG, M., van GINKEL, F.W., McGHEE, J.R., KELLY, D., LYRENE, R.K., and SORSCHER, E.J. (2001). A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis. Hum. Gene Ther. 12, 751-761.
-
(2001)
Hum. Gene Ther.
, vol.12
, pp. 751-761
-
-
Ruiz, F.E.1
Clancy, J.P.2
Perricone, M.A.3
Bebok, Z.4
Hong, J.S.5
Cheng, S.H.6
Meeker, D.P.7
Young, K.R.8
Schoumacher, R.A.9
Weatherly, M.R.10
Wing, L.11
Morris, J.E.12
Sindel, L.13
Rosenberg, M.14
Van Ginkel, F.W.15
McGhee, J.R.16
Kelly, D.17
Lyrene, R.K.18
Sorscher, E.J.19
-
59
-
-
16944364834
-
Basis of pulmonary toxicity associated with cationic lipid-mediated gene transfer to the mammalian lung
-
SCHEULE, R.K., ST. GEORGE, J.A., BAGLEY, R.G., MARSHALL, J., KAPLAN, J.M., AKITA, G.Y., WANG, K.X., LEE, E.R., HARRIS, D.J., JIANG, C., YEW, N.S., SMITH, A.E., and CHENG, S.H. (1997). Basis of pulmonary toxicity associated with cationic lipid-mediated gene transfer to the mammalian lung. Hum. Gene Ther. 8, 689-707.
-
(1997)
Hum. Gene Ther.
, vol.8
, pp. 689-707
-
-
Scheule, R.K.1
St. George, J.A.2
Bagley, R.G.3
Marshall, J.4
Kaplan, J.M.5
Akita, G.Y.6
Wang, K.X.7
Lee, E.R.8
Harris, D.J.9
Jiang, C.10
Yew, N.S.11
Smith, A.E.12
Cheng, S.H.13
-
60
-
-
0030859292
-
CpG motifs in bacterial DNA cause inflammation in the lower respiratory tract
-
SCHWARTZ, D.A., QUINN, T.J., THORNE, P.S., SAYEED, S., YI, A.K., and KRIEG, A.M. (1997). CpG motifs in bacterial DNA cause inflammation in the lower respiratory tract. J. Clin. Invest. 100, 68-73.
-
(1997)
J. Clin. Invest.
, vol.100
, pp. 68-73
-
-
Schwartz, D.A.1
Quinn, T.J.2
Thorne, P.S.3
Sayeed, S.4
Yi, A.K.5
Krieg, A.M.6
-
61
-
-
2442453783
-
Standardized procedure for measurement of nasal potential difference: An outcome measure in multicenter cystic fibrosis clinical trials
-
STANDAERT, T.A., BOITANO, L., EMERSON, J., MILGRAM, L.J.H., KONSTAN, M.W., HUNTER, J., BERCLAZ, P.-Y., BRASS, L., ZEITLIN, P.L., HAMMOND, K., DAVIES, Z., FOY, C., NOONE, P.C., and KNOWLES, M.R. (2004). Standardized procedure for measurement of nasal potential difference: An outcome measure in multicenter cystic fibrosis clinical trials. Pediatr. Pulmonol. 37, 385-392.
-
(2004)
Pediatr. Pulmonol.
, vol.37
, pp. 385-392
-
-
Standaert, T.A.1
Boitano, L.2
Emerson, J.3
Milgram, L.J.H.4
Konstan, M.W.5
Hunter, J.6
Berclaz, P.-Y.7
Brass, L.8
Zeitlin, P.L.9
Hammond, K.10
Davies, Z.11
Foy, C.12
Noone, P.C.13
Knowles, M.R.14
-
62
-
-
0037088577
-
Genistein restores functional interactions between AFSOS-CFTR and ENaC in Xenopus oocytes
-
SUAUD, L., LI, J., JIANG, Q., RUBENSTEIN, R.C., and KLEYMAN, T.R. (2002). Genistein restores functional interactions between AFSOS-CFTR and ENaC in Xenopus oocytes. J. Biol. Chem. 277, 8928-8933.
-
(2002)
J. Biol. Chem.
, vol.277
, pp. 8928-8933
-
-
Suaud, L.1
Li, J.2
Jiang, Q.3
Rubenstein, R.C.4
Kleyman, T.R.5
-
63
-
-
0038805301
-
Immune responses to adeno-associated virus and its recombinant vectors
-
SUN, J.Y., ANAND-JAWA, V., CHATTERJEE, S., and WONG, K.K. (2003). Immune responses to adeno-associated virus and its recombinant vectors. Gene Ther. 10, 964-976.
-
(2003)
Gene Ther.
, vol.10
, pp. 964-976
-
-
Sun, J.Y.1
Anand-Jawa, V.2
Chatterjee, S.3
Wong, K.K.4
-
64
-
-
0026043883
-
Expression of the cystic fibrosis transmembrane conductance regulator gene in the respiratory tract of normal individuals and individuals with cystic fibrosis
-
TRAPNELL, B.C., CHU, C.S., PAAKKO, P.K., BANKS, T.C., YOSHIMURA, K., FERRANS, V.J., CHERNICK, M.S., and CRYSTAL, R.G. (1991). Expression of the cystic fibrosis transmembrane conductance regulator gene in the respiratory tract of normal individuals and individuals with cystic fibrosis. Proc. Natl. Acad. Sci. U.S.A. 88, 6565-6569.
-
(1991)
Proc. Natl. Acad. Sci. U.S.A.
, vol.88
, pp. 6565-6569
-
-
Trapnell, B.C.1
Chu, C.S.2
Paakko, P.K.3
Banks, T.C.4
Yoshimura, K.5
Ferrans, V.J.6
Chernick, M.S.7
Crystal, R.G.8
-
65
-
-
0034682480
-
Site-specific chemical modification with polyethylene glycol of recombinant immunotoxin anti-Tac(Fv)-' PE38 (LMB-2) improves antitumor activity and reduces animal toxicity and immunogenicity
-
TSUTSUMI, Y., ONDA, M., NAGATA, S., LEE, B., KREITMAN, R.J., and PASTAN, I. (2000). Site-specific chemical modification with polyethylene glycol of recombinant immunotoxin anti-Tac(Fv)-' PE38 (LMB-2) improves antitumor activity and reduces animal toxicity and immunogenicity. Proc. Natl. Acad. Sci. USA. 97, 8548-8553.
-
(2000)
Proc. Natl. Acad. Sci. USA.
, vol.97
, pp. 8548-8553
-
-
Tsutsumi, Y.1
Onda, M.2
Nagata, S.3
Lee, B.4
Kreitman, R.J.5
Pastan, I.6
-
66
-
-
0032490381
-
Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus
-
WAGNER, J.A., REYNOLDS, T., MORAN, M.L., MOSS, R.B., WINE, J.J., FLOTTE, T.R., and GARDNER, P. (1998). Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus. Lancet 351, 1702-1703.
-
(1998)
Lancet
, vol.351
, pp. 1702-1703
-
-
Wagner, J.A.1
Reynolds, T.2
Moran, M.L.3
Moss, R.B.4
Wine, J.J.5
Flotte, T.R.6
Gardner, P.7
-
67
-
-
0033022349
-
Safety and biological efficacy of an adenoassociated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus
-
WAGNER, J.A., MESSNER, A.H., MORAN, M.L., DAIFUKU, R., KOUYAMA, K., DESCH, J.K., MANLEY, S., NORBASH, A.M., CONRAD, C.K., FRIBORG, S., REYNOLDS, T., GUGGINO, W.B., MOSS, R.B., CARTER, B.J., WINE, J.J., FLOTTE, T.R., and GARDNER, P. (1999). Safety and biological efficacy of an adenoassociated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus. Laryngoscope 109, 266-274.
-
(1999)
Laryngoscope
, vol.109
, pp. 266-274
-
-
Wagner, J.A.1
Messner, A.H.2
Moran, M.L.3
Daifuku, R.4
Kouyama, K.5
Desch, J.K.6
Manley, S.7
Norbash, A.M.8
Conrad, C.K.9
Friborg, S.10
Reynolds, T.11
Guggino, W.B.12
Moss, R.B.13
Carter, B.J.14
Wine, J.J.15
Flotte, T.R.16
Gardner, P.17
-
68
-
-
0036377851
-
A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies
-
WAGNER, J.A., NEPOMUCENO, I.B., MESSNER, A.H., MORAN, M.L., BATSON, E.P., DIMICELI, S., BROWN, B.W., DESCH, J.K., NORBASH, A.M., CONRAD, C.K., GUGGINO, W.B., FLOTTE, T.R., WINE, J.J., CARTER, B.J., REYNOLDS, T.C., MOSS, R.B., and GARDNER, P. (2002). 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. 13, 1349-1359.
-
(2002)
Hum. Gene Ther.
, vol.13
, pp. 1349-1359
-
-
Wagner, J.A.1
Nepomuceno, I.B.2
Messner, A.H.3
Moran, M.L.4
Batson, E.P.5
Dimiceli, S.6
Brown, B.W.7
Desch, J.K.8
Norbash, A.M.9
Conrad, C.K.10
Guggino, W.B.11
Flotte, T.R.12
Wine, J.J.13
Carter, B.J.14
Reynolds, T.C.15
Moss, R.B.16
Gardner, P.17
-
69
-
-
0027504119
-
Polyethylene glycol-modified chimeric toxin composed of transforming growth factor a and Pseudomonas exotoxin
-
WANG, Q.C., PAI, L.H., DEBINSKI, W., FITZGERALD, D.J., and PASTAN, I. (1993). Polyethylene glycol-modified chimeric toxin composed of transforming growth factor a and Pseudomonas exotoxin. Cancer Res. 53, 4588-4594.
-
(1993)
Cancer Res.
, vol.53
, pp. 4588-4594
-
-
Wang, Q.C.1
Pai, L.H.2
Debinski, W.3
Fitzgerald, D.J.4
Pastan, I.5
-
70
-
-
0032743351
-
Gene transfer for cystic fibrosis
-
WELSH, M.J. (1999). Gene transfer for cystic fibrosis. J. Clin. Invest. 104, 1265-1266.
-
(1999)
J. Clin. Invest.
, vol.104
, pp. 1265-1266
-
-
Welsh, M.J.1
-
71
-
-
0000026508
-
Cystic fibrosis
-
C. Scriver, A.L. Beaudet, and D. Valle, eds. (McGraw Hill, New York)
-
WELSH, M.J., TSUI, L.-C., BOAT, T.F., and BEAUDET, A.L. (1995). Cystic fibrosis. In The Metabolic and Molecular Basis of Inherited Disease, 7th ed., Vol. 3. C. Scriver, A.L. Beaudet, and D. Valle, eds. (McGraw Hill, New York) pp. 3799-3876.
-
(1995)
The Metabolic and Molecular Basis of Inherited Disease, 7th Ed.
, vol.3
, pp. 3799-3876
-
-
Welsh, M.J.1
Tsui, L.-C.2
Boat, T.F.3
Beaudet, A.L.4
-
72
-
-
19144370024
-
Safety of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA to the lungs of nonhuman primates
-
WILMOTT, R.W., AMIN, R.S., PEREZ, C.R., WERT, S.E., KELLER, G., BOIVIN, G.P., HIRSCH, R., DE INOCENCIO, J., LU, P., REISING, S.F., YEI, S., WHITSETT, J.A., and TRAPNELL, B.C. (1996). Safety of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA to the lungs of nonhuman primates. Hum. Gene Ther. 7, 301-318.
-
(1996)
Hum. Gene Ther.
, vol.7
, pp. 301-318
-
-
Wilmott, R.W.1
Amin, R.S.2
Perez, C.R.3
Wert, S.E.4
Keller, G.5
Boivin, G.P.6
Hirsch, R.7
De Inocencio, J.8
Lu, P.9
Reising, S.F.10
Yei, S.11
Whitsett, J.A.12
Trapnell, B.C.13
-
73
-
-
0033585425
-
Contribution of plasmid DNA to inflammation in the lung after administration of cationic lipid:pDNA complexes
-
YEW, N.S., WANG, K.X., PRZYBYLSKA, M., BAGLEY, R.G., STEDMAN, M., MARSHALL, J., SCHEULE, R.K., and CHENG, S.H. (1999). Contribution of plasmid DNA to inflammation in the lung after administration of cationic lipid:pDNA complexes. Hum. Gene Ther. 10, 223-234.
-
(1999)
Hum. Gene Ther.
, vol.10
, pp. 223-234
-
-
Yew, N.S.1
Wang, K.X.2
Przybylska, M.3
Bagley, R.G.4
Stedman, M.5
Marshall, J.6
Scheule, R.K.7
Cheng, S.H.8
-
74
-
-
13344286312
-
Repeat administration of an adenovirus vector encoding cystic fibrosis transmembrane conductance regulator to the nasal epithelium of patients with cystic fibrosis
-
ZABNER, J., RAMSEY, B.W., MEEKER, D.P., AITKEN, M.L., BALFOUR, R.P., GIBSON, R.L., LAUNSPACH, J., MOSCICKI, R.A., RICHARDS, S.M., STANDAERT, T.A., WILLIAMS-WARREN, J., WADSWORTH, S.C., SMITH, A.E., and WELSH, M.J. (1996). Repeat administration of an adenovirus vector encoding cystic fibrosis transmembrane conductance regulator to the nasal epithelium of patients with cystic fibrosis. J. Clin. Invest. 97, 1504-1511.
-
(1996)
J. Clin. Invest.
, vol.97
, pp. 1504-1511
-
-
Zabner, J.1
Ramsey, B.W.2
Meeker, D.P.3
Aitken, M.L.4
Balfour, R.P.5
Gibson, R.L.6
Launspach, J.7
Moscicki, R.A.8
Richards, S.M.9
Standaert, T.A.10
Williams-Warren, J.11
Wadsworth, S.C.12
Smith, A.E.13
Welsh, M.J.14
-
75
-
-
0030759153
-
Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo
-
ZABNER, J., CHENG, S.H., MEEKER, D., LAUNSPACH, J., BALFOUR, R., PERRICONE, M.A., MORRIS, J.E., MARSHALL, J., FASBENDER, A., SMITH, A.E., and WELSH, M.J. (1997). Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo. J. Clin. Invest. 100, 1529-1537.
-
(1997)
J. Clin. Invest.
, vol.100
, pp. 1529-1537
-
-
Zabner, J.1
Cheng, S.H.2
Meeker, D.3
Launspach, J.4
Balfour, R.5
Perricone, M.A.6
Morris, J.E.7
Marshall, J.8
Fasbender, A.9
Smith, A.E.10
Welsh, M.J.11
-
76
-
-
1242301652
-
Stabilized PLASmin® complexes deliver exogenous genes to the nasal epithelia of cystic fibrosis (CF) knockout mice and confer partial physiological correction of CFTR-mediated chloride transport
-
ZIADY, A.G., OETTE, S., GEDEON, C., PAYNE, J.M., HYATT, S., MUHAMMAD, O., KOWALCZYK, T.H., PASUMARTHY, M.K., COOPER, M.J., MOEN, R.C., and DAVIS, P.B. (2002). Stabilized PLASmin® complexes deliver exogenous genes to the nasal epithelia of cystic fibrosis (CF) knockout mice and confer partial physiological correction of CFTR-mediated chloride transport. Mol. Ther. 5, S435.
-
(2002)
Mol. Ther.
, vol.5
-
-
Ziady, A.G.1
Oette, S.2
Gedeon, C.3
Payne, J.M.4
Hyatt, S.5
Muhammad, O.6
Kowalczyk, T.H.7
Pasumarthy, M.K.8
Cooper, M.J.9
Moen, R.C.10
Davis, P.B.11
-
77
-
-
0347354994
-
Transfection of airway epithelium by stable PEGylated poly-L-lysine DNA nanoparticles in vivo
-
ZIADY, A.G., GEDEON, C.R., MILLER, T., QUAN, W., PAYNE, J.M., HYATT, S.L., FINK, T.L., MUHAMMAD, O., OETTE, S., KOWALCZYK, T., PASUMARTHY, M.K., MOEN, R.C., COOPER, M.J., and DAVIS, P.B. (2003a). Transfection of airway epithelium by stable PEGylated poly-L-lysine DNA nanoparticles in vivo. Mol. Ther. 8, 936-947.
-
(2003)
Mol. Ther.
, vol.8
, pp. 936-947
-
-
Ziady, A.G.1
Gedeon, C.R.2
Miller, T.3
Quan, W.4
Payne, J.M.5
Hyatt, S.L.6
Fink, T.L.7
Muhammad, O.8
Oette, S.9
Kowalczyk, T.10
Pasumarthy, M.K.11
Moen, R.C.12
Cooper, M.J.13
Davis, P.B.14
-
78
-
-
0347917175
-
Minimal toxicity of stabilized compacted DNA nanoparticles in the murine lung
-
ZIADY, A.G., GEDEON, C.R., MUHAMMAD, O., OETTE, S.M., FINK, T.L., QUAN, W., KOWALCZYK, T.H., HYATT, S.L., PEISCHL, A., SENG, J.E., MOEN, R.C., COOPER, M.J., and DAVIS, P.B. (2003b). Minimal toxicity of stabilized compacted DNA nanoparticles in the murine lung. Mol. Ther. 8, 948-956.
-
(2003)
Mol. Ther.
, vol.8
, pp. 948-956
-
-
Ziady, A.G.1
Gedeon, C.R.2
Muhammad, O.3
Oette, S.M.4
Fink, T.L.5
Quan, W.6
Kowalczyk, T.H.7
Hyatt, S.L.8
Peischl, A.9
Seng, J.E.10
Moen, R.C.11
Cooper, M.J.12
Davis, P.B.13
-
79
-
-
4644356629
-
Defining strategies to extend duration of gene expression from targeted compacted DNA vectors
-
ZIADY, A.G., KIM, J., COLLA, J., and DAVIS, P.B. (2004). Defining strategies to extend duration of gene expression from targeted compacted DNA vectors. Gene Ther. 11, 1378-1390.
-
(2004)
Gene Ther.
, vol.11
, pp. 1378-1390
-
-
Ziady, A.G.1
Kim, J.2
Colla, J.3
Davis, P.B.4
-
80
-
-
0033544951
-
A phase I study of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator gene to a lung segment of individuals with cystic fibrosis
-
ZUCKERMAN, J.B., ROBINSON, C.B., MCCOY, K.S., SHELL, R., SFERRA, TJ., CHIRMULE, N., MAGOSIN, S.A., PROPERT, K.J., BROWN-PARR, E.C., HUGHES, J.V., TAZELAAR, J., BAKER, C., GOLDMAN, M.J., and WILSON, J.M. (1999). A phase I study of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator gene to a lung segment of individuals with cystic fibrosis. Hum. Gene Ther. 10, 2973-2985.
-
(1999)
Hum. Gene Ther.
, vol.10
, pp. 2973-2985
-
-
Zuckerman, J.B.1
Robinson, C.B.2
Mccoy, K.S.3
Shell, R.4
Sferra, T.J.5
Chirmule, N.6
Magosin, S.A.7
Propert, K.J.8
Brown-Parr, E.C.9
Hughes, J.V.10
Tazelaar, J.11
Baker, C.12
Goldman, M.J.13
Wilson, J.M.14
|