-
1
-
-
0025231388
-
Direct gene transfer into mouse muscle in vivo
-
Wolff, J.A., Malone, R.W., Williams, P., Chong, W., Acsadi, G., Jani, A., Felgner, P.L. Direct gene transfer into mouse muscle in vivo. Science 1990, 247: 1465-1468.
-
(1990)
Science
, vol.247
, pp. 1465-1468
-
-
Wolff, J.A.1
Malone, R.W.2
Williams, P.3
Chong, W.4
Acsadi, G.5
Jani, A.6
Felgner, P.L.7
-
2
-
-
0028500191
-
Cultured human myoblasts and myotubes show markedly different transducibility by replication-defective adenovirus recombinants
-
Acsadi, G., Jani, A., Huard, J., Blaschuk, K., Massie, B., Holland, P., Lochmuller, H., Karpati, G. Cultured human myoblasts and myotubes show markedly different transducibility by replication-defective adenovirus recombinants. Gene Ther. 1994, 1: 338-340.
-
(1994)
Gene Ther.
, vol.1
, pp. 338-340
-
-
Acsadi, G.1
Jani, A.2
Huard, J.3
Blaschuk, K.4
Massie, B.5
Holland, P.6
Lochmuller, H.7
Karpati, G.8
-
3
-
-
0028346944
-
A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity
-
Acsadi, G., Jani, A., Massie, B., Simoneau, M., Holland, P., Blaschuk, K., Karpati, G. A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity. Hum. Mol. Genet. 1994, 3: 579-584.
-
(1994)
Hum. Mol. Genet.
, vol.3
, pp. 579-584
-
-
Acsadi, G.1
Jani, A.2
Massie, B.3
Simoneau, M.4
Holland, P.5
Blaschuk, K.6
Karpati, G.7
-
4
-
-
0033541587
-
Expression of the primary coxsackie and adenovirus receptor is downregulated during skeletal muscle maturation and limits the efficacy of adenovirus-mediated gene delivery to muscle cells
-
Nalbantoglu, J., Pari, G., Karpati, G., Holland, P.C. Expression of the primary coxsackie and adenovirus receptor is downregulated during skeletal muscle maturation and limits the efficacy of adenovirus-mediated gene delivery to muscle cells. Hum. Gene Ther. 1999, 10: 1009-1019.
-
(1999)
Hum. Gene Ther.
, vol.10
, pp. 1009-1019
-
-
Nalbantoglu, J.1
Pari, G.2
Karpati, G.3
Holland, P.C.4
-
5
-
-
0026576154
-
Adenovirus as an expression vector in muscle cells in vivo
-
Quanti, B., Perricaudet, L.D., Tajbakhsh, S., Mandel, J.L. Adenovirus as an expression vector in muscle cells in vivo. Proc. Natl. Acad. Sci. U. S. A. 1992, 89: 2581-2584.
-
(1992)
Proc. Natl. Acad. Sci. U. S. A.
, vol.89
, pp. 2581-2584
-
-
Quanti, B.1
Perricaudet, L.D.2
Tajbakhsh, S.3
Mandel, J.L.4
-
6
-
-
0028807696
-
FK506 immunosuppression to control the immune reactions triggered by first-generation adenovirus-mediated gene transfer
-
Vilquin, J.T., Guerette, B., Kinoshita, I., Roy, B., Goulet, M., Gravel, C., Roy, R., Tremblay, J.P. FK506 immunosuppression to control the immune reactions triggered by first-generation adenovirus-mediated gene transfer. Hum. Gene Ther. 1995, 6: 1391-1401.
-
(1995)
Hum. Gene Ther.
, vol.6
, pp. 1391-1401
-
-
Vilquin, J.T.1
Guerette, B.2
Kinoshita, I.3
Roy, B.4
Goulet, M.5
Gravel, C.6
Roy, R.7
Tremblay, J.P.8
-
7
-
-
0031883829
-
Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted
-
Amalfitano, A., Hauser, M.A., Hu, H., Serra, D., Begy, C.R., Chamberlain, J.S. Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted. J. Virol. 1998, 72: 926-933.
-
(1998)
J. Virol.
, vol.72
, pp. 926-933
-
-
Amalfitano, A.1
Hauser, M.A.2
Hu, H.3
Serra, D.4
Begy, C.R.5
Chamberlain, J.S.6
-
8
-
-
0029943155
-
A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase
-
Kochanek, S., Clemens, P.R., Mitani, K., Chen, H.H., Chan, S., Caskey, C.T. A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase. Proc. Natl. Acad. Sci. U. S. A. 1996, 93: 5731-5736.
-
(1996)
Proc. Natl. Acad. Sci. U. S. A.
, vol.93
, pp. 5731-5736
-
-
Kochanek, S.1
Clemens, P.R.2
Mitani, K.3
Chen, H.H.4
Chan, S.5
Caskey, C.T.6
-
9
-
-
0031049699
-
Persistence in muscle of an adenoviral vector that lacks all viral genes
-
Chen, H.H., Mack, L.M., Kelly, R., Ontell, M., Kochanek, S., Clemens, P.R. Persistence in muscle of an adenoviral vector that lacks all viral genes. Proc. Natl. Acad. Sci. U. S. A. 1997, 94: 1645-1650.
-
(1997)
Proc. Natl. Acad. Sci. U. S. A.
, vol.94
, pp. 1645-1650
-
-
Chen, H.H.1
Mack, L.M.2
Kelly, R.3
Ontell, M.4
Kochanek, S.5
Clemens, P.R.6
-
10
-
-
0037372398
-
Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors
-
Chuah, M.K., Schiedner, G., Thorrez, L., Brown, B., Johnston, M., Gillijns, V., Hertel, S., van Rooijen, N., Lillicrap, D., Collen, D., VandenDriessche, T., Kochanek, S. Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors. Blood 2003, 101: 1734-1743.
-
(2003)
Blood
, vol.101
, pp. 1734-1743
-
-
Chuah, M.K.1
Schiedner, G.2
Thorrez, L.3
Brown, B.4
Johnston, M.5
Gillijns, V.6
Hertel, S.7
van Rooijen, N.8
Lillicrap, D.9
Collen, D.10
VandenDriessche, T.11
Kochanek, S.12
-
11
-
-
0033540620
-
DNA from both high-capacity and first-generation adenoviral vectors remains intact in skeletal muscle
-
Chen, H.H., Mack, L.M., Choi, S.Y., Ontell., M., Kochanek, S., Clemens, P.R. DNA from both high-capacity and first-generation adenoviral vectors remains intact in skeletal muscle. Hum. Gene Ther. 1999, 10: 365-373.
-
(1999)
Hum. Gene Ther.
, vol.10
, pp. 365-373
-
-
Chen, H.H.1
Mack, L.M.2
Choi, S.Y.3
Ontell, M.4
Kochanek, S.5
Clemens, P.R.6
-
12
-
-
6844257545
-
Ex vivo gene transfer using adenovirus-mediated full-length dystrophin delivery to dystrophic muscles
-
Floyd, S.S. Jr., Clemens, P.R., Ontell, M.R., Kochanek, S., Day, C.S., Yang, J., Hauschka, S.D., Balkir, L., Morgan, J., Moreland, M.S., Feero, G.W., Epperly, M., Huard, J. Ex vivo gene transfer using adenovirus-mediated full-length dystrophin delivery to dystrophic muscles. Gene Ther. 1998, 5: 19-30.
-
(1998)
Gene Ther.
, vol.5
, pp. 19-30
-
-
Floyd Jr., S.S.1
Clemens, P.R.2
Ontell, M.R.3
Kochanek, S.4
Day, C.S.5
Yang, J.6
Hauschka, S.D.7
Balkir, L.8
Morgan, J.9
Moreland, M.S.10
Feero, G.W.11
Epperly, M.12
Huard, J.13
-
13
-
-
0029857641
-
In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes
-
Clemens, P.R., Kochanek, S., Sunada, Y., Chan, S., Chen, H.H., Campbell, K.P., Caskey, C.T. In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes. Gene Ther. 1996, 3: 965-972.
-
(1996)
Gene Ther.
, vol.3
, pp. 965-972
-
-
Clemens, P.R.1
Kochanek, S.2
Sunada, Y.3
Chan, S.4
Chen, H.H.5
Campbell, K.P.6
Caskey, C.T.7
-
14
-
-
11844269203
-
Comparison of high-capacity and first-generation adenoviral vector gene delivery to murine muscle in utero
-
Bilbao, R., Reay, D.P., Wu, E., Zheng, H., Biermann, V., Kochanek, S., Clemens, P.R. Comparison of high-capacity and first-generation adenoviral vector gene delivery to murine muscle in utero. Gene Ther. 2005, 12: 39-47.
-
(2005)
Gene Ther.
, vol.12
, pp. 39-47
-
-
Bilbao, R.1
Reay, D.P.2
Wu, E.3
Zheng, H.4
Biermann, V.5
Kochanek, S.6
Clemens, P.R.7
-
15
-
-
0036790998
-
Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin
-
DelloRusso, C., Scott, J.M., Hartigan-O'Connor, D., Salvatori, G., Barjot, C., Robinson, A.S., Crawford, R.W., Brooks, S.V., Chamberlain, J.S. Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin. Proc. Natl. Acad. Sci. U. S. A. 2002, 99: 12979-12984.
-
(2002)
Proc. Natl. Acad. Sci. U. S. A.
, vol.99
, pp. 12979-12984
-
-
DelloRusso, C.1
Scott, J.M.2
Hartigan-O'Connor, D.3
Salvatori, G.4
Barjot, C.5
Robinson, A.S.6
Crawford, R.W.7
Brooks, S.V.8
Chamberlain, J.S.9
-
16
-
-
0038344798
-
Prolonged dystrophin expression and functional correction of mdx mouse muscle following gene transfer with a helper-dependent (gutted) adenovirus-encoding murine dystrophin
-
Gilbert, R., Dudley, R.W., Liu, A.B., Petrof, B.J., Nalbantoglu, J., Karpati, G. Prolonged dystrophin expression and functional correction of mdx mouse muscle following gene transfer with a helper-dependent (gutted) adenovirus-encoding murine dystrophin. Hum. Mol. Genet. 2003, 12: 1287-1299.
-
(2003)
Hum. Mol. Genet.
, vol.12
, pp. 1287-1299
-
-
Gilbert, R.1
Dudley, R.W.2
Liu, A.B.3
Petrof, B.J.4
Nalbantoglu, J.5
Karpati, G.6
-
17
-
-
0041806537
-
Gene transfer establishes primacy of striated vs. smooth muscle sarcoglycan complex in limb-girdle muscular dystrophy
-
Durbeej, M., Sawatzki, S.M., Barresi, R., Schmainda, K.M., Allamand, V., Michele, D.E., Campbell, K.P. Gene transfer establishes primacy of striated vs. smooth muscle sarcoglycan complex in limb-girdle muscular dystrophy. Proc. Natl. Acad. Sci. U. S. A. 2003.
-
(2003)
Proc. Natl. Acad. Sci. U. S. A.
-
-
Durbeej, M.1
Sawatzki, S.M.2
Barresi, R.3
Schmainda, K.M.4
Allamand, V.5
Michele, D.E.6
Campbell, K.P.7
-
18
-
-
0033843869
-
Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice
-
Allamand, V., Donahue, K.M., Straub, V., Davisson, R.L., Davidson, B.L., Campbell, K.P. Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice. Gene Ther. 2000, 7: 1385-1391.
-
(2000)
Gene Ther.
, vol.7
, pp. 1385-1391
-
-
Allamand, V.1
Donahue, K.M.2
Straub, V.3
Davisson, R.L.4
Davidson, B.L.5
Campbell, K.P.6
-
19
-
-
0034610364
-
Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
-
Wang, B., Li, J., Xiao, X. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc. Natl. Acad. Sci. U. S. A. 2000, 97: 13714-13719.
-
(2000)
Proc. Natl. Acad. Sci. U. S. A.
, vol.97
, pp. 13714-13719
-
-
Wang, B.1
Li, J.2
Xiao, X.3
-
20
-
-
12744269885
-
Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury
-
Liu, M., Yue, Y., Harper, S.Q., Grange, R.W., Chamberlain, J.S., Duan, D. Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Mol. Ther. 2006, 11: 245-256.
-
(2006)
Mol. Ther.
, vol.11
, pp. 245-256
-
-
Liu, M.1
Yue, Y.2
Harper, S.Q.3
Grange, R.W.4
Chamberlain, J.S.5
Duan, D.6
-
21
-
-
4043092073
-
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
-
Gregorevic, P., Blankinship, M.J., Allen, J.M., Crawford, R.W., Meuse, L., Miller, D.G., Russell, D.W., Chamberlain, J.S. Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat. Med. 2004.
-
(2004)
Nat. Med.
-
-
Gregorevic, P.1
Blankinship, M.J.2
Allen, J.M.3
Crawford, R.W.4
Meuse, L.5
Miller, D.G.6
Russell, D.W.7
Chamberlain, J.S.8
-
22
-
-
0032924996
-
rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy
-
Li, J., Dressman, D., Tsao, Y.P., Sakamoto, A., Hoffman, E.P., Xiao, X. rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy. Gene Ther. 1999, 6: 74-82.
-
(1999)
Gene Ther.
, vol.6
, pp. 74-82
-
-
Li, J.1
Dressman, D.2
Tsao, Y.P.3
Sakamoto, A.4
Hoffman, E.P.5
Xiao, X.6
-
23
-
-
0034138226
-
Rescue of skeletal muscles of gamma-sarcoglycan-deficient mice with adeno-associated virus-mediated gene transfer
-
Cordier, L., Hack, A.A., Scott, M.O., Barton-Davis, E.R., Gao, G., Wilson, J.M., McNally, E.M., Sweeney, H.L. Rescue of skeletal muscles of gamma-sarcoglycan-deficient mice with adeno-associated virus-mediated gene transfer. Mol. Ther. 2000, 1: 119-129.
-
(2000)
Mol. Ther.
, vol.1
, pp. 119-129
-
-
Cordier, L.1
Hack, A.A.2
Scott, M.O.3
Barton-Davis, E.R.4
Gao, G.5
Wilson, J.M.6
McNally, E.M.7
Sweeney, H.L.8
-
24
-
-
0032907636
-
Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector
-
Greelish, J.P., Su, L.T., Lankford, E.B., Burkman, J.M., Chen, H., Konig, S.K., Mercier, I.M., Desjardins, P.R., Mitchell, M.A., Zheng, X.G., Leferovich, J., Gao, G.P., Balice-Gordon, R.J., Wilson, J.M., Stedman, H.H. Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector. Nat. Med. 1999, 5: 439-443.
-
(1999)
Nat. Med.
, vol.5
, pp. 439-443
-
-
Greelish, J.P.1
Su, L.T.2
Lankford, E.B.3
Burkman, J.M.4
Chen, H.5
Konig, S.K.6
Mercier, I.M.7
Desjardins, P.R.8
Mitchell, M.A.9
Zheng, X.G.10
Leferovich, J.11
Gao, G.P.12
Balice-Gordon, R.J.13
Wilson, J.M.14
Stedman, H.H.15
-
25
-
-
0034050904
-
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
-
Kay, M.A., Manno, C.S., Ragni, M.V., Larson, P.J., Couto, L.B., McClelland, A., Glader, B., Chew, A.J., Tai, S.J., Herzog, R.W., Arruda, V., Johnson, F., Scallan, C., Skarsgard, E., Flake, A.W., High, K.A. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat. Genet. 2000, 24: 257-261.
-
(2000)
Nat. Genet.
, vol.24
, pp. 257-261
-
-
Kay, M.A.1
Manno, C.S.2
Ragni, M.V.3
Larson, P.J.4
Couto, L.B.5
McClelland, A.6
Glader, B.7
Chew, A.J.8
Tai, S.J.9
Herzog, R.W.10
Arruda, V.11
Johnson, F.12
Scallan, C.13
Skarsgard, E.14
Flake, A.W.15
High, K.A.16
-
26
-
-
0035042012
-
Gene therapy for hemophilia B: AAV-mediated transfer of the gene for coagulation factor IX to human muscle
-
Larson, P.J. and High, K.A. Gene therapy for hemophilia B: AAV-mediated transfer of the gene for coagulation factor IX to human muscle. Adv. Exp. Med. Biol. 2001, 489: 45-57.
-
(2001)
Adv. Exp. Med. Biol.
, vol.489
, pp. 45-57
-
-
Larson, P.J.1
High, K.A.2
-
27
-
-
0031983639
-
Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia
-
Monahan, P.E., Samulski, R.J., Tazelaar, J., Xiao, X., Nichols, T.C., Bellinger, D.A., Read, M.S., Walsh, C.E. Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Ther. 1998, 5: 40-49.
-
(1998)
Gene Ther.
, vol.5
, pp. 40-49
-
-
Monahan, P.E.1
Samulski, R.J.2
Tazelaar, J.3
Xiao, X.4
Nichols, T.C.5
Bellinger, D.A.6
Read, M.S.7
Walsh, C.E.8
-
28
-
-
0042528664
-
Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model
-
Kaspar, B.K., Llado, J., Sherkat, N., Rothstein, J.D., Gage, F.H. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 2003, 301: 839-842.
-
(2003)
Science
, vol.301
, pp. 839-842
-
-
Kaspar, B.K.1
Llado, J.2
Sherkat, N.3
Rothstein, J.D.4
Gage, F.H.5
-
29
-
-
0031723577
-
Adenovirus-mediated transfer of the neurotrophin-3 gene into skeletal muscle of pmn mice: Therapeutic effects and mechanisms of action
-
Haase, G., Pettmann, B., Vigne, E., Castelnau-Ptakhine, L., Schmalbruch, H., Kahn, A. Adenovirus-mediated transfer of the neurotrophin-3 gene into skeletal muscle of pmn mice: therapeutic effects and mechanisms of action. J. Neurol. Sci. 1998, 160 Suppl 1: S97-105.
-
(1998)
J. Neurol. Sci.
, vol.160
, Issue.SUPPL. 1
-
-
Haase, G.1
Pettmann, B.2
Vigne, E.3
Castelnau-Ptakhine, L.4
Schmalbruch, H.5
Kahn, A.6
-
30
-
-
0030451214
-
Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein
-
Kessler, P.D., Podsakoff, G.M., Chen, X., McQuiston, S.A., Colosi, P.C., Matelis, L.A., Kurtzman, G.J., Byrne, B.J. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc. Natl. Acad. Sci. U. S. A. 1996, 93: 14082-14087.
-
(1996)
Proc. Natl. Acad. Sci. U. S. A.
, vol.93
, pp. 14082-14087
-
-
Kessler, P.D.1
Podsakoff, G.M.2
Chen, X.3
McQuiston, S.A.4
Colosi, P.C.5
Matelis, L.A.6
Kurtzman, G.J.7
Byrne, B.J.8
-
31
-
-
0035444173
-
Protective effects of cardiotrophin-1 adenoviral gene transfer on neuromuscular degeneration in transgeic ALS mice
-
Bordet, T., Lesbordes, J.C., Rouhani, S., Castelnau-Ptakhine, L., Schmalbruch, H., Haase, G., Kahn, A. Protective effects of cardiotrophin-1 adenoviral gene transfer on neuromuscular degeneration in transgeic ALS mice. Hum. Mol. Genet. 2001, 10: 1925-1933.
-
(2001)
Hum. Mol. Genet.
, vol.10
, pp. 1925-1933
-
-
Bordet, T.1
Lesbordes, J.C.2
Rouhani, S.3
Castelnau-Ptakhine, L.4
Schmalbruch, H.5
Haase, G.6
Kahn, A.7
-
32
-
-
0032699093
-
Adenoviral cardiotrophin-1 gene transfer protects pmn mice from progressive motor neuronopathy
-
Bordet, T., Schmalbruch, H., Pettmann, B., Hagege, A., Castelnau-Ptakhine, L., Kahn, A., Haase, G. Adenoviral cardiotrophin-1 gene transfer protects pmn mice from progressive motor neuronopathy. J. Clin. Invest. 1999, 104: 1077-1085.
-
(1999)
J. Clin. Invest.
, vol.104
, pp. 1077-1085
-
-
Bordet, T.1
Schmalbruch, H.2
Pettmann, B.3
Hagege, A.4
Castelnau-Ptakhine, L.5
Kahn, A.6
Haase, G.7
-
33
-
-
0036646739
-
In vivo electrotransfer of the cardiotrophin-1 gene into skeletal muscle slows down progression of motor neuron degeneration in pmn mice
-
Lesbordes, J.C., Bordet, T., Haase, G., Castelnau-Ptakhine, L., Rouhani, S., Gilgenkrantz, H., Kahn, A. In vivo electrotransfer of the cardiotrophin-1 gene into skeletal muscle slows down progression of motor neuron degeneration in pmn mice. Hum. Mol. Genet. 2002, 11: 1615-1625.
-
(2002)
Hum. Mol. Genet.
, vol.11
, pp. 1615-1625
-
-
Lesbordes, J.C.1
Bordet, T.2
Haase, G.3
Castelnau-Ptakhine, L.4
Rouhani, S.5
Gilgenkrantz, H.6
Kahn, A.7
-
34
-
-
0037669048
-
Therapeutic benefits of cardiotrophin-1 gene transfer in a mouse model of spinal muscular atrophy
-
Lesbordes, J.C., Cifuentes-Diaz, C., Miroglio, A., Joshi, V., Bordet, T., Kahn, A., Melki, J. Therapeutic benefits of cardiotrophin-1 gene transfer in a mouse model of spinal muscular atrophy. Hum. Mol. Genet. 2003, 12: 1233-1239.
-
(2003)
Hum. Mol. Genet.
, vol.12
, pp. 1233-1239
-
-
Lesbordes, J.C.1
Cifuentes-Diaz, C.2
Miroglio, A.3
Joshi, V.4
Bordet, T.5
Kahn, A.6
Melki, J.7
-
35
-
-
17444387438
-
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
-
Herzog, R.W., Yang, E.Y., Couto, L.B., Hagstrom, J.N., Elwell, D., Fields, P.A., Burton, M., Bellinger, D.A., Read, M.S., Brinkhous, K.M., Podsakoff, G.M., Nichols, T.C., Kurtzman, G.J., High, K.A. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat. Med. 1999, 5: 56-63.
-
(1999)
Nat. Med.
, vol.5
, pp. 56-63
-
-
Herzog, R.W.1
Yang, E.Y.2
Couto, L.B.3
Hagstrom, J.N.4
Elwell, D.5
Fields, P.A.6
Burton, M.7
Bellinger, D.A.8
Read, M.S.9
Brinkhous, K.M.10
Podsakoff, G.M.11
Nichols, T.C.12
Kurtzman, G.J.13
High, K.A.14
-
36
-
-
0034849318
-
Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation
-
Herzog, R.W., Mount, J.D., Arruda, V.R., High, K.A., Lothrop, C.D., Jr. Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation. Mol. Ther. 2001, 4: 192-200.
-
(2001)
Mol. Ther.
, vol.4
, pp. 192-200
-
-
Herzog, R.W.1
Mount, J.D.2
Arruda, V.R.3
High, K.A.4
Lothrop Jr., C.D.5
-
37
-
-
0036196808
-
AAV-mediated VEGF gene transfer into skeletal muscle stimulates angiogenesis and improves blood flow in a rat hindlimb ischemia model
-
Shimpo, M., Ikeda, U., Maeda, Y., Takahashi, M., Miyashita, H., Mizukami, H., Urabe, M., Kume, A., Takizawa, T., Shibuya, M., Ozawa, K., Shimada, K. AAV-mediated VEGF gene transfer into skeletal muscle stimulates angiogenesis and improves blood flow in a rat hindlimb ischemia model. Cardiovasc. Res. 2002, 53: 993-1001.
-
(2002)
Cardiovasc. Res.
, vol.53
, pp. 993-1001
-
-
Shimpo, M.1
Ikeda, U.2
Maeda, Y.3
Takahashi, M.4
Miyashita, H.5
Mizukami, H.6
Urabe, M.7
Kume, A.8
Takizawa, T.9
Shibuya, M.10
Ozawa, K.11
Shimada, K.12
-
38
-
-
19644378907
-
Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart
-
Wang, Z., Zhu, T., Qiao, C., Zhou, L., Wang, B., Zhang, J., Chen, C., Li, J., Xiao, X. Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat. Biotechnol. 2005, 23: 321-328.
-
(2005)
Nat. Biotechnol.
, vol.23
, pp. 321-328
-
-
Wang, Z.1
Zhu, T.2
Qiao, C.3
Zhou, L.4
Wang, B.5
Zhang, J.6
Chen, C.7
Li, J.8
Xiao, X.9
-
39
-
-
0032853699
-
Immune responses to adenovirus and adeno-associated virus in humans
-
Chirmule, N., Propert, K., Magosin, S., Qian, Y., Qian, R., Wilson, J. Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther. 1999, 6: 1574-1583.
-
(1999)
Gene Ther.
, vol.6
, pp. 1574-1583
-
-
Chirmule, N.1
Propert, K.2
Magosin, S.3
Qian, Y.4
Qian, R.5
Wilson, J.6
-
40
-
-
0036892308
-
Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product
-
Yuasa, K., Sakamoto, M., Miyagoe-Suzuki, Y., Tanouchi, A., Yamamoto, H., Li, J., Chamberlain, J.S., Xiao, X., Takeda, S. Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product. Gene Ther. 2002, 9: 1576-1588.
-
(2002)
Gene Ther.
, vol.9
, pp. 1576-1588
-
-
Yuasa, K.1
Sakamoto, M.2
Miyagoe-Suzuki, Y.3
Tanouchi, A.4
Yamamoto, H.5
Li, J.6
Chamberlain, J.S.7
Xiao, X.8
Takeda, S.9
-
41
-
-
0033977047
-
Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and non-human primate muscle
-
Chirmule, N., Xiao, W., Truneh, A., Schnell, M.A., Hughes, J.V., Zoltick, P., Wilson, J.M. Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and non-human primate muscle. J. Virol. 2000, 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
-
42
-
-
0037056026
-
Improved cellular and Immoral immune responses against Mycobacterium tuberculosis antigens after intramuscular DNA immunisation combined with muscle electroporation
-
Tollefsen, S., Tjelle, T., Schneider, J., Harboe, M., Wiker, H., Hewinson, G., Huygen, K., Mathiesen, I. Improved cellular and Immoral immune responses against Mycobacterium tuberculosis antigens after intramuscular DNA immunisation combined with muscle electroporation. Vaccine 2002, 20: 3370-3378.
-
(2002)
Vaccine
, vol.20
, pp. 3370-3378
-
-
Tollefsen, S.1
Tjelle, T.2
Schneider, J.3
Harboe, M.4
Wiker, H.5
Hewinson, G.6
Huygen, K.7
Mathiesen, I.8
-
43
-
-
0026570486
-
Retroviral-mediated transfer of a dystrophin minigene into mdx mouse myoblasts in vitro
-
Dunckley, M.G., Love, D.R., Davies, K.E., Walsh, F.S., Morris, G.E., Dickson, G. Retroviral-mediated transfer of a dystrophin minigene into mdx mouse myoblasts in vitro. FEBS Lett. 1992, 296: 128-134.
-
(1992)
FEBS Lett.
, vol.296
, pp. 128-134
-
-
Dunckley, M.G.1
Love, D.R.2
Davies, K.E.3
Walsh, F.S.4
Morris, G.E.5
Dickson, G.6
-
44
-
-
0027174251
-
Direct retroviral-mediated transfer of a dystrophin minigene into mdx mouse muscle in vivo
-
Dunckley, M.G., Wells, D.J., Walsh, F.S., Dickson, G. Direct retroviral-mediated transfer of a dystrophin minigene into mdx mouse muscle in vivo. Hum. Mol. Genet. 1993, 2: 717-723.
-
(1993)
Hum. Mol. Genet.
, vol.2
, pp. 717-723
-
-
Dunckley, M.G.1
Wells, D.J.2
Walsh, F.S.3
Dickson, G.4
-
45
-
-
0030755548
-
Genetic correction of dystrophin deficiency and skeletal muscle remodeling in adult MDX mouse via transplantation of retroviral producer cells
-
Fassati, A., Wells, D.J., Sgro Serpente, P.A., Walsh, F.S., Brown, S.C., Strong, P.N., Dickson, G. Genetic correction of dystrophin deficiency and skeletal muscle remodeling in adult MDX mouse via transplantation of retroviral producer cells. J. Clin. Invest 1997, 100: 620-628.
-
(1997)
J. Clin. Invest
, vol.100
, pp. 620-628
-
-
Fassati, A.1
Wells, D.J.2
Sgro Serpente, P.A.3
Walsh, F.S.4
Brown, S.C.5
Strong, P.N.6
Dickson, G.7
-
46
-
-
0242382779
-
Dystrophin delivery in dystrophin-deficient DMDmdx skeletal muscle by isogenic muscle-derived stem cell transplantation
-
Ikezawa, M., Cao, B., Qu, Z., Peng, H., Xiao, X., Pruchnic, R., Kimura, S., Miike, T., Huard, J. Dystrophin delivery in dystrophin-deficient DMDmdx skeletal muscle by isogenic muscle-derived stem cell transplantation. Hum. Gene Ther. 2003, 14: 1535-1546.
-
(2003)
Hum. Gene Ther.
, vol.14
, pp. 1535-1546
-
-
Ikezawa, M.1
Cao, B.2
Qu, Z.3
Peng, H.4
Xiao, X.5
Pruchnic, R.6
Kimura, S.7
Miike, T.8
Huard, J.9
-
47
-
-
0031743608
-
Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
-
Zufferey, R., Dull, T., Mandel, R.J., Bukovsky, A., Quiroz, D., Naldini, L., Trono, D. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 1998, 72: 9873-9880.
-
(1998)
J. Virol.
, vol.72
, pp. 9873-9880
-
-
Zufferey, R.1
Dull, T.2
Mandel, R.J.3
Bukovsky, A.4
Quiroz, D.5
Naldini, L.6
Trono, D.7
-
48
-
-
0035437193
-
Lentivirus administration to rat muscle provides efficient sustained expression of erythropoietin
-
Seppen, J., Barry, S.C., Harder, B., Osborne, W.R. Lentivirus administration to rat muscle provides efficient sustained expression of erythropoietin. Blood 2001, 98: 594-596.
-
(2001)
Blood
, vol.98
, pp. 594-596
-
-
Seppen, J.1
Barry, S.C.2
Harder, B.3
Osborne, W.R.4
-
49
-
-
1542513708
-
Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells
-
Bachrach, E., Li, S., Perez, A.L., Schienda, J., Liadaki, K., Volinski, J., Flint, A., Chamberlain, J., Kunkel, L.M. Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells. Proc. Natl. Acad. Sci. U. S. A. 2004, 101: 3581-3586.
-
(2004)
Proc. Natl. Acad. Sci. U. S. A.
, vol.101
, pp. 3581-3586
-
-
Bachrach, E.1
Li, S.2
Perez, A.L.3
Schienda, J.4
Liadaki, K.5
Volinski, J.6
Flint, A.7
Chamberlain, J.8
Kunkel, L.M.9
-
50
-
-
0346035738
-
Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes
-
MacKenzie, T.C., Kobinger, G.P., Kootstra, N.A., Radu, A., Sena-Esteves, M., Bouchard, S., Wilson, J.M., Verma, I.M., Flake, A.W. Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes. Mol. Ther. 2002, 6: 349-358.
-
(2002)
Mol. Ther.
, vol.6
, pp. 349-358
-
-
MacKenzie, T.C.1
Kobinger, G.P.2
Kootstra, N.A.3
Radu, A.4
Sena-Esteves, M.5
Bouchard, S.6
Wilson, J.M.7
Verma, I.M.8
Flake, A.W.9
-
51
-
-
12744274695
-
Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors
-
MacKenzie, T.C., Kobinger, G.P., Louboutin, J.P., Radu, A., Javazon, E.H., Sena-Esteves, M., Wilson, J.M., Flake, A.W. Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors. J. Gene Med. 2005, 7: 50-58.
-
(2005)
J. Gene Med.
, vol.7
, pp. 50-58
-
-
MacKenzie, T.C.1
Kobinger, G.P.2
Louboutin, J.P.3
Radu, A.4
Javazon, E.H.5
Sena-Esteves, M.6
Wilson, J.M.7
Flake, A.W.8
-
52
-
-
0035157991
-
Post-mitotic, differentiated myotubes efficiently produce retroviral vector from hybrid adeno-retrovirus templates
-
Roberts, M.L., Athanasopoulos, T., Pohlschmidt, M., Duisit, G., Cosset, F.L., Dickson, G. Post-mitotic, differentiated myotubes efficiently produce retroviral vector from hybrid adeno-retrovirus templates. Gene Ther. 2001, 8: 1580-1586.
-
(2001)
Gene Ther.
, vol.8
, pp. 1580-1586
-
-
Roberts, M.L.1
Athanasopoulos, T.2
Pohlschmidt, M.3
Duisit, G.4
Cosset, F.L.5
Dickson, G.6
-
53
-
-
0037098953
-
Stable micro-dystrophin gene transfer using an integrating adeno-retroviral hybrid vector ameliorates the dystrophic pathology in mdx mouse muscle
-
Roberts, M.L., Wells, D.J., Graham, I.R., Fabb, S.A., Hill, V.J., Duisit, G., Yuasa, K., Takeda, S., Cosset, F.L., Dickson, G. Stable micro-dystrophin gene transfer using an integrating adeno-retroviral hybrid vector ameliorates the dystrophic pathology in mdx mouse muscle. Hum. Mol. Genet. 2002, 11: 1719-1730.
-
(2002)
Hum. Mol. Genet.
, vol.11
, pp. 1719-1730
-
-
Roberts, M.L.1
Wells, D.J.2
Graham, I.R.3
Fabb, S.A.4
Hill, V.J.5
Duisit, G.6
Yuasa, K.7
Takeda, S.8
Cosset, F.L.9
Dickson, G.10
-
54
-
-
13944271703
-
Transfer of the full-length dystrophin-coding sequence into muscle cells by a dual high-capacity hybrid viral vector with site-specific integration ability
-
Goncalves, M.A., van Nierop, G.P., Tijssen, M.R., Lefesvre, P., Knaan-Shanzer, S., van, d.V., I, van Bekkum, D.W., Valerio, D., de Vries, A.A. Transfer of the full-length dystrophin-coding sequence into muscle cells by a dual high-capacity hybrid viral vector with site-specific integration ability. J. Virol. 2005, 79: 3146-3162.
-
(2005)
J. Virol.
, vol.79
, pp. 3146-3162
-
-
Goncalves, M.A.1
van Nierop, G.P.2
Tijssen, M.R.3
Lefesvre, P.4
Knaan-Shanzer, S.5
van, I.D.V.6
van Bekkum, D.W.7
Valerio, D.8
de Vries, A.A.9
-
55
-
-
0031052263
-
Isolation of a common receptor for Coxsackie B viruses and adenoviruses 2 and 5
-
Bergelson, J.M., Cunningham, J.A., Droguett, G., Kurt-Jones, E.A., Krithivas, A., Hong, J.S., Horwitz, M.S., Crowell, R.L., Finberg, R.W. Isolation of a common receptor for Coxsackie B viruses and adenoviruses 2 and 5. Science 1997, 275: 1320-1323.
-
(1997)
Science
, vol.275
, pp. 1320-1323
-
-
Bergelson, J.M.1
Cunningham, J.A.2
Droguett, G.3
Kurt-Jones, E.A.4
Krithivas, A.5
Hong, J.S.6
Horwitz, M.S.7
Crowell, R.L.8
Finberg, R.W.9
-
56
-
-
0035050152
-
Muscle-specific overexpression of the adenovirus primary receptor CAR overcomes low efficiency of gene transfer to mature skeletal muscle
-
Nalbantoglu, J., Larochelle, N., Wolf, E., Karpati, G., Lochmuller, H., Holland, P.C. Muscle-specific overexpression of the adenovirus primary receptor CAR overcomes low efficiency of gene transfer to mature skeletal muscle. J. Virol. 2001, 75: 4276-4282.
-
(2001)
J. Virol.
, vol.75
, pp. 4276-4282
-
-
Nalbantoglu, J.1
Larochelle, N.2
Wolf, E.3
Karpati, G.4
Lochmuller, H.5
Holland, P.C.6
-
57
-
-
0035188994
-
Forced myofiber regeneration promotes dystrophin gene transfer and improved muscle function despite advanced disease in old dystrophic mice
-
Guibinga, G.H., Ebihara, S., Nalbantoglu, J., Holland, P., Karpati, G., Petrof, B.J. Forced myofiber regeneration promotes dystrophin gene transfer and improved muscle function despite advanced disease in old dystrophic mice. Mol. Ther. 2001, 4: 499-507.
-
(2001)
Mol. Ther.
, vol.4
, pp. 499-507
-
-
Guibinga, G.H.1
Ebihara, S.2
Nalbantoglu, J.3
Holland, P.4
Karpati, G.5
Petrof, B.J.6
-
58
-
-
0029858682
-
Adenovirus targeted to heparan-containing receptors increases its gene delivery efficiency to multiple cell types
-
Wickham, T.J., Roelvink, P.W., Brough, D.E., Kovesdi, I. Adenovirus targeted to heparan-containing receptors increases its gene delivery efficiency to multiple cell types. Nat. Biotechnol. 1996, 14: 1570-1573.
-
(1996)
Nat. Biotechnol.
, vol.14
, pp. 1570-1573
-
-
Wickham, T.J.1
Roelvink, P.W.2
Brough, D.E.3
Kovesdi, I.4
-
59
-
-
0033166881
-
Polylysine modification of adenoviral fiber protein enhances muscle cell transduction
-
Bouri, K., Feero, W.G., Myerburg, M.M., Wickham, T.J., Kovesdi, I., Hoffman, E.P., Clemens, P.R. Polylysine modification of adenoviral fiber protein enhances muscle cell transduction. Hum. Gene Ther. 1999, 10: 1633-1640.
-
(1999)
Hum. Gene Ther.
, vol.10
, pp. 1633-1640
-
-
Bouri, K.1
Feero, W.G.2
Myerburg, M.M.3
Wickham, T.J.4
Kovesdi, I.5
Hoffman, E.P.6
Clemens, P.R.7
-
60
-
-
0031592755
-
Comparison of various expression plasmids for the induction of immune response by DNA immunization
-
Lee, A.H, Suh, Y.S., Sung, J.H., Yang, S.H., Sung, Y.C. Comparison of various expression plasmids for the induction of immune response by DNA immunization. Mol. Cells 1997, 7: 495-501.
-
(1997)
Mol. Cells
, vol.7
, pp. 495-501
-
-
Lee, A.H.1
Suh, Y.S.2
Sung, J.H.3
Yang, S.H.4
Sung, Y.C.5
-
61
-
-
0028864849
-
DNA-mediated immunization in mice induces a potent MHC class I-restricted cytotoxic T lymphocyte response to the hepatitis B envelope protein
-
Davis, H.L., Schirmbeck, R., Reimann, J., Whalen, R.G. DNA-mediated immunization in mice induces a potent MHC class I-restricted cytotoxic T lymphocyte response to the hepatitis B envelope protein. Hum. Gene Ther. 1995, 6: 1447-1456.
-
(1995)
Hum. Gene Ther.
, vol.6
, pp. 1447-1456
-
-
Davis, H.L.1
Schirmbeck, R.2
Reimann, J.3
Whalen, R.G.4
-
62
-
-
0030174678
-
DNA-mediated immunization to hepatitis B surface antigen: Longevity of primary response and effect of boost
-
Davis, H.L., Mancini, M., Michel, M.L., Whalen, R.G. DNA-mediated immunization to hepatitis B surface antigen: longevity of primary response and effect of boost. Vaccine 1996, 14: 910-915.
-
(1996)
Vaccine
, vol.14
, pp. 910-915
-
-
Davis, H.L.1
Mancini, M.2
Michel, M.L.3
Whalen, R.G.4
-
63
-
-
0031916435
-
Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity
-
Schiedner, G., Morral, N., Parks, R.J., Wu, Y., Koopmans, S.C., Langston, C., Graham, F.L., Beaudet, A.L., Kochanek, S. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat. Genet. 1998, 18: 180-183.
-
(1998)
Nat. Genet.
, vol.18
, pp. 180-183
-
-
Schiedner, G.1
Morral, N.2
Parks, R.J.3
Wu, Y.4
Koopmans, S.C.5
Langston, C.6
Graham, F.L.7
Beaudet, A.L.8
Kochanek, S.9
-
64
-
-
0036088754
-
The short MCK1350 promoter/enhancer allows for sufficient dystrophin expression in skeletal muscles of mdx mice
-
Larochelle, N., Oualikene, W., Dunant, P., Massie, B., Karpati, G., Nalbantoglu, J., Lochmuller, H. The short MCK1350 promoter/enhancer allows for sufficient dystrophin expression in skeletal muscles of mdx mice. Biochem. Biophys. Res. Commun. 2002, 292: 626-631.
-
(2002)
Biochem. Biophys. Res. Commun.
, vol.292
, pp. 626-631
-
-
Larochelle, N.1
Oualikene, W.2
Dunant, P.3
Massie, B.4
Karpati, G.5
Nalbantoglu, J.6
Lochmuller, H.7
-
65
-
-
0035915778
-
Muscle-specific promoters may be necessary for Adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies
-
Cordier, L., Gao, G.P., Hack, A.A., McNally, E.M., Wilson, J.M., Chirmule, N., Sweeney, H.L. Muscle-specific promoters may be necessary for Adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies. Hum. Gene Ther. 2001, 12: 205-215.
-
(2001)
Hum. Gene Ther.
, vol.12
, pp. 205-215
-
-
Cordier, L.1
Gao, G.P.2
Hack, A.A.3
McNally, E.M.4
Wilson, J.M.5
Chirmule, N.6
Sweeney, H.L.7
-
66
-
-
0035713273
-
Designing gene therapy vectors: Avoiding immune responses by using tissue-specific promoters
-
Weeratna, R.D., Wu, T., Efler, S.M., Zhang, L., Davis, H.L. Designing gene therapy vectors: avoiding immune responses by using tissue-specific promoters. Gene Ther. 2001, 8: 1872-1878.
-
(2001)
Gene Ther.
, vol.8
, pp. 1872-1878
-
-
Weeratna, R.D.1
Wu, T.2
Efler, S.M.3
Zhang, L.4
Davis, H.L.5
-
67
-
-
0345829919
-
Systemic delivery of a high-capacity adenoviral vector expressing mouse CTLA4Ig improves skeletal muscle gene therapy
-
Jiang, Z., Feingold, E., Kochanek, S., Clemens, P.R. Systemic delivery of a high-capacity adenoviral vector expressing mouse CTLA4Ig improves skeletal muscle gene therapy. Mol. Ther. 2002, 6: 369-376.
-
(2002)
Mol. Ther.
, vol.6
, pp. 369-376
-
-
Jiang, Z.1
Feingold, E.2
Kochanek, S.3
Clemens, P.R.4
-
68
-
-
0142089747
-
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
-
Raper, S.E., Chirmule, N., Lee, F.S., Wivel, N.A., Bagg, A., Gao, G.P., Wilson, J.M., Batshaw, M.L. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol. Genet. Metab 2003, 80: 148-158.
-
(2003)
Mol. Genet. Metab
, vol.80
, pp. 148-158
-
-
Raper, S.E.1
Chirmule, N.2
Lee, F.S.3
Wivel, N.A.4
Bagg, A.5
Gao, G.P.6
Wilson, J.M.7
Batshaw, M.L.8
-
69
-
-
0034724857
-
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
-
Cavazzana-Calvo, M., Hacein-Bey, S., de Saint, B.G., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J.L., Bousso, P., Deist, F.L., Fischer, A. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000, 288: 669-672.
-
(2000)
Science
, vol.288
, pp. 669-672
-
-
Cavazzana-Calvo, M.1
Hacein-Bey, S.2
de Saint, B.G.3
Gross, F.4
Yvon, E.5
Nusbaum, P.6
Selz, F.7
Hue, C.8
Certain, S.9
Casanova, J.L.10
Bousso, P.11
Deist, F.L.12
Fischer, A.13
-
70
-
-
0037020112
-
Clinical research. Gene therapy a suspect in leukemia-like disease
-
Marshall, E. Clinical research. Gene therapy a suspect in leukemia-like disease. Science 2002, 298: 34-35.
-
(2002)
Science
, vol.298
, pp. 34-35
-
-
Marshall, E.1
-
71
-
-
0025887093
-
Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs
-
Acsadi, G., Dickson, G., Love, D.R., Jani, A., Walsh, F.S., Gurusinghe, A., Wolff, J.A., Davies, K.E. Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs. Nature 1991, 352: 815-818.
-
(1991)
Nature
, vol.352
, pp. 815-818
-
-
Acsadi, G.1
Dickson, G.2
Love, D.R.3
Jani, A.4
Walsh, F.S.5
Gurusinghe, A.6
Wolff, J.A.7
Davies, K.E.8
-
72
-
-
0029892249
-
Targeted gene correction of episomal DNA in mammalian cells mediated by a chimeric RNA.DNA oligonucleotide
-
Yoon, K., Cole-Strauss, A., Kmiec, E.B. Targeted gene correction of episomal DNA in mammalian cells mediated by a chimeric RNA.DNA oligonucleotide. Proc. Natl. Acad. Sci. U. S. A. 1996, 93: 2071-2076.
-
(1996)
Proc. Natl. Acad. Sci. U. S. A.
, vol.93
, pp. 2071-2076
-
-
Yoon, K.1
Cole-Strauss, A.2
Kmiec, E.B.3
-
73
-
-
0030913556
-
Targeted nucleotide exchange in the alkaline phosphatase gene of HuH-7 cells mediated by a chimeric RNA/DNA oligonucleotide
-
Kren, B.T., Cole-Strauss, A., Kmiec, E.B., Steer, C.J. Targeted nucleotide exchange in the alkaline phosphatase gene of HuH-7 cells mediated by a chimeric RNA/DNA oligonucleotide. Hepatology 1997, 25: 1462-1468.
-
(1997)
Hepatology
, vol.25
, pp. 1462-1468
-
-
Kren, B.T.1
Cole-Strauss, A.2
Kmiec, E.B.3
Steer, C.J.4
-
74
-
-
0034624974
-
Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides
-
Rando, T.A., Disatnik, M.H., Zhou, L.Z. Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides. Proc. Natl. Acad. Sci. U. S. A. 2000, 97: 5363-5368.
-
(2000)
Proc. Natl. Acad. Sci. U. S. A.
, vol.97
, pp. 5363-5368
-
-
Rando, T.A.1
Disatnik, M.H.2
Zhou, L.Z.3
-
75
-
-
0036211418
-
Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides
-
Bertoni C and Rando, T.A. Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides. Hum. Gene Ther. 2002, 13: 707-718.
-
(2002)
Hum. Gene Ther.
, vol.13
, pp. 707-718
-
-
Bertoni, C.1
Rando, T.A.2
-
76
-
-
0034086776
-
In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide
-
Bartlett, R.J., Stockinger, S., Denis, M.M., Bartlett, W.T., Inverardi, L., Le, T.T., thi, M.N., Morris, G.E., Bogan, D.J., Metcalf-Bogan, J., Kornegay, J.N. In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide. Nat. Biotechnol. 2000, 18: 615-622.
-
(2000)
Nat. Biotechnol.
, vol.18
, pp. 615-622
-
-
Bartlett, R.J.1
Stockinger, S.2
Denis, M.M.3
Bartlett, W.T.4
Inverardi, L.5
Le, T.T.6
Thi, M.N.7
Morris, G.E.8
Bogan, D.J.9
Metcalf-Bogan, J.10
Kornegay, J.N.11
-
77
-
-
0031796816
-
Targeted replacement of normal and mutant CFTR sequences in human airway epithelial cells using DNA fragments
-
Goncz, K.K., Kunzelmann, K., Xu, Z., Gruenert, D.C. Targeted replacement of normal and mutant CFTR sequences in human airway epithelial cells using DNA fragments. Hum. Mol. Genet. 1998, 7: 1913-1919.
-
(1998)
Hum. Mol. Genet.
, vol.7
, pp. 1913-1919
-
-
Goncz, K.K.1
Kunzelmann, K.2
Xu, Z.3
Gruenert, D.C.4
-
78
-
-
0036268053
-
Application of SFHR to gene therapy of monogenic disorders
-
Goncz, K.K., Prokopishyn, N.L., Chow, B.L., Davis, B.R., Gruenert, D.C. Application of SFHR to gene therapy of monogenic disorders. Gene Ther. 2002, 9: 691-694.
-
(2002)
Gene Ther.
, vol.9
, pp. 691-694
-
-
Goncz, K.K.1
Prokopishyn, N.L.2
Chow, B.L.3
Davis, B.R.4
Gruenert, D.C.5
-
79
-
-
0034743499
-
Expression of DeltaF508 CFTR in normal mouse lung after site-specific modification of CFTR sequences by SFHR
-
Goncz, K.K., Colosimo, A., Dallapiccola, B., Gagne, L., Hong, K., Novelli, G., Papahadjopoulos, D., Sawa, T., Schreier, H., Wiener-Kronish, J., Xu, Z., Gruenert, D.C. Expression of DeltaF508 CFTR in normal mouse lung after site-specific modification of CFTR sequences by SFHR. Gene Ther. 2001, 8: 961-965.
-
(2001)
Gene Ther.
, vol.8
, pp. 961-965
-
-
Goncz, K.K.1
Colosimo, A.2
Dallapiccola, B.3
Gagne, L.4
Hong, K.5
Novelli, G.6
Papahadjopoulos, D.7
Sawa, T.8
Schreier, H.9
Wiener-Kronish, J.10
Xu, Z.11
Gruenert, D.C.12
-
80
-
-
0033987325
-
Site-directed alteration of genomic DNA by small-fragment homologous replacement
-
Goncz, K.K. and Gruenert, D.C. Site-directed alteration of genomic DNA by small-fragment homologous replacement. Methods Mol. Biol. 2000, 133: 85-99.
-
(2000)
Methods Mol. Biol.
, vol.133
, pp. 85-99
-
-
Goncz, K.K.1
Gruenert, D.C.2
-
81
-
-
0036268304
-
Targeted gene correction in the mdx mouse using short DNA fragments: Towards application with bone marrow-derived cells for autologous remodeling of dystrophic muscle
-
Kapsa, R.M., Quigley, A.F., Vadolas, J., Steeper, K., Ioannou, P.A., Byrne, E., Kornberg, A.J. Targeted gene correction in the mdx mouse using short DNA fragments: towards application with bone marrow-derived cells for autologous remodeling of dystrophic muscle. Gene Ther. 2002, 9: 695-699.
-
(2002)
Gene Ther.
, vol.9
, pp. 695-699
-
-
Kapsa, R.M.1
Quigley, A.F.2
Vadolas, J.3
Steeper, K.4
Ioannou, P.A.5
Byrne, E.6
Kornberg, A.J.7
-
82
-
-
23744496714
-
DNA electroporation in vivo targets mature fibres in dystrophic mdx muscle
-
Wong, S.H., Lowes. K.N., Quigley, A.F., Marotta, R., Kita, M., Byrne, E., Kornberg, A.J., Cook, M.J., Kapsa, R.M. DNA electroporation in vivo targets mature fibres in dystrophic mdx muscle. Neuromuscul Disord. 2005, 15: 630-641.
-
(2005)
Neuromuscul Disord.
, vol.15
, pp. 630-641
-
-
Wong, S.H.1
Lowes, K.N.2
Quigley, A.F.3
Marotta, R.4
Kita, M.5
Byrne, E.6
Kornberg, A.J.7
Cook, M.J.8
Kapsa, R.M.9
-
83
-
-
0030582315
-
Induction of exon skipping of the dystrophin transcript in lymphoblastoid cells by transfecting an antisense oligodeoxynucleotide complementary to an exon recognition sequence
-
Pramono, Z.A., Takeshima, Y., Alimsardjono, H., Ishii, A., Takeda, S., Matsuo, M. Induction of exon skipping of the dystrophin transcript in lymphoblastoid cells by transfecting an antisense oligodeoxynucleotide complementary to an exon recognition sequence. Biochem. Biophys. Res. Commun. 1996, 226: 445-449.
-
(1996)
Biochem. Biophys. Res. Commun.
, vol.226
, pp. 445-449
-
-
Pramono, Z.A.1
Takeshima, Y.2
Alimsardjono, H.3
Ishii, A.4
Takeda, S.5
Matsuo, M.6
-
84
-
-
0035793047
-
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse
-
Mann, C.J., Honeyman, K., Cheng, A.J., Ly, T., Lloyd, F., Fletcher, S., Morgan, J.E., Partridge, T.A., Wilton, S.D. Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc. Natl. Acad. Sci. U. S. A. 2001, 98: 42-47.
-
(2001)
Proc. Natl. Acad. Sci. U. S. A.
, vol.98
, pp. 42-47
-
-
Mann, C.J.1
Honeyman, K.2
Cheng, A.J.3
Ly, T.4
Lloyd, F.5
Fletcher, S.6
Morgan, J.E.7
Partridge, T.A.8
Wilton, S.D.9
-
85
-
-
0035878539
-
Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells
-
van Deutekom, J.C., Bremmer-Bout, M., Janson, A.A., Ginjaar, I.B., Baas, F., den Dunnen, J.T., van Ommen, G.J. Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells. Hum. Mol. Genet. 2001, 10: 1547-1554.
-
(2001)
Hum. Mol. Genet.
, vol.10
, pp. 1547-1554
-
-
van Deutekom, J.C.1
Bremmer-Bout, M.2
Janson, A.A.3
Ginjaar, I.B.4
Baas, F.5
den Dunnen, J.T.6
van Ommen, G.J.7
-
86
-
-
0036823504
-
Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy
-
Aartsma-Rus, A., Bremmer-Bout, M., Janson, A.A., den Dunnen, J.T., van Ommen, G.J., van Deutekom, J.C. Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy. Neuromuscul. Disord. 2002, 12 Suppl 1: S71-S77.
-
(2002)
Neuromuscul. Disord.
, vol.12
, Issue.SUPPL. 1
-
-
Aartsma-Rus, A.1
Bremmer-Bout, M.2
Janson, A.A.3
den Dunnen, J.T.4
van Ommen, G.J.5
van Deutekom, J.C.6
-
87
-
-
0037047111
-
Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells
-
De Angelis, F.G., Sthandier, O., Berarducci, B., Toso, S., Galluzzi, G., Ricci, E., Cossu, G., Bozzoni, I. Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells. Proc. Natl. Acad. Sci. U. S. A. 2002, 99: 9456-9461.
-
(2002)
Proc. Natl. Acad. Sci. U. S. A.
, vol.99
, pp. 9456-9461
-
-
De Angelis, F.G.1
Sthandier, O.2
Berarducci, B.3
Toso, S.4
Galluzzi, G.5
Ricci, E.6
Cossu, G.7
Bozzoni, I.8
-
88
-
-
0013181060
-
Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy
-
Mann, C.J., Honeyman, K., McClorey, G., Fletcher, S., Wilton, S.D. Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy. J. Gene Med. 2002, 4: 644-654.
-
(2002)
J. Gene Med.
, vol.4
, pp. 644-654
-
-
Mann, C.J.1
Honeyman, K.2
McClorey, G.3
Fletcher, S.4
Wilton, S.D.5
-
89
-
-
0037447517
-
Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients
-
Aartsma-Rus, A., Janson, A.A., Kaman, W.E., Bremmer-Bout, M., den Dunnen, J.T., Baas, F., van Ommen, G.J., van Deutekom, J.C. Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum. Mol. Genet. 2003, 12: 907-914.
-
(2003)
Hum. Mol. Genet.
, vol.12
, pp. 907-914
-
-
Aartsma-Rus, A.1
Janson, A.A.2
Kaman, W.E.3
Bremmer-Bout, M.4
den Dunnen, J.T.5
Baas, F.6
van Ommen, G.J.7
van Deutekom, J.C.8
-
90
-
-
0042536463
-
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
-
Lu, Q.L., Mann, C.J., Lou, F., Bou-Gharios, G., Morris, G.E., Xue, S.A., Fletcher, S., Partridge, T.A., Wilton, S.D. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat. Med. 2003.
-
(2003)
Nat. Med.
-
-
Lu, Q.L.1
Mann, C.J.2
Lou, F.3
Bou-Gharios, G.4
Morris, G.E.5
Xue, S.A.6
Fletcher, S.7
Partridge, T.A.8
Wilton, S.D.9
-
91
-
-
0028989346
-
Pharmacokinetics of adenoviral vector-mediated gene delivery to vascular smooth muscle cells: Modulation by poloxamer 407 and implications for cardiovascular gene therapy
-
March, K.L., Madison, J.E., Trapnell, B.C. Pharmacokinetics of adenoviral vector-mediated gene delivery to vascular smooth muscle cells: modulation by poloxamer 407 and implications for cardiovascular gene therapy. Hum. Gene Ther. 1995, 6: 41-53.
-
(1995)
Hum. Gene Ther.
, vol.6
, pp. 41-53
-
-
March, K.L.1
Madison, J.E.2
Trapnell, B.C.3
-
92
-
-
0037148596
-
Pluronic block copolymers: Novel functional molecules for gene therapy
-
Kabanov, A.V., Lemieux, P., Vinogradov, S., Alakhov, V. Pluronic block copolymers: novel functional molecules for gene therapy. Adv. Drug Deliv. Rev. 2002, 54: 223-233.
-
(2002)
Adv. Drug Deliv. Rev.
, vol.54
, pp. 223-233
-
-
Kabanov, A.V.1
Lemieux, P.2
Vinogradov, S.3
Alakhov, V.4
-
93
-
-
0026638985
-
Age and sex influence expression of plasmid DNA directly injected into mouse skeletal muscle
-
Wells, D.J. and Goldspink, G. Age and sex influence expression of plasmid DNA directly injected into mouse skeletal muscle. FEBS Lett. 1992, 306: 203-205.
-
(1992)
FEBS Lett.
, vol.306
, pp. 203-205
-
-
Wells, D.J.1
Goldspink, G.2
-
94
-
-
0034152996
-
Hypothesis: Naked plasmid DNA is taken up by cells in vivo by a receptor-mediated process
-
Budker, V., Budker, T., Zhang, G., Subbotin, V., Loomis, A., Wolff, J.A. Hypothesis: naked plasmid DNA is taken up by cells in vivo by a receptor-mediated process. J. Gene Med. 2000, 2: 76-88.
-
(2000)
J. Gene Med.
, vol.2
, pp. 76-88
-
-
Budker, V.1
Budker, T.2
Zhang, G.3
Subbotin, V.4
Loomis, A.5
Wolff, J.A.6
-
95
-
-
0345570537
-
Characterization of oligonucleotide transport into living cells
-
Loke, S.L., Stein, C.A., Zhang, X.H., Mori, K., Nakanishi, M., Subasinghe, C., Cohen, J.S., Neckers, L.M. Characterization of oligonucleotide transport into living cells. Proc. Natl. Acad. Sci. U. S. A. 1989, 86: 3474-3478.
-
(1989)
Proc. Natl. Acad. Sci. U. S. A.
, vol.86
, pp. 3474-3478
-
-
Loke, S.L.1
Stein, C.A.2
Zhang, X.H.3
Mori, K.4
Nakanishi, M.5
Subasinghe, C.6
Cohen, J.S.7
Neckers, L.M.8
-
96
-
-
0026922764
-
Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle
-
Wolff, J.A., Ludtke, J.J., Acsadi, G., Williams, P., Jani, A. Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum. Mol. Genet. 1992, 1: 363-369.
-
(1992)
Hum. Mol. Genet.
, vol.1
, pp. 363-369
-
-
Wolff, J.A.1
Ludtke, J.J.2
Acsadi, G.3
Williams, P.4
Jani, A.5
-
97
-
-
0026545612
-
Potocytosis: Sequestration and transport of small molecules by caveolae
-
Anderson, R.G., Kamen, B.A., Rothberg, K.G., Lacey, S.W. Potocytosis: sequestration and transport of small molecules by caveolae. Science 1992, 255: 410-411.
-
(1992)
Science
, vol.255
, pp. 410-411
-
-
Anderson, R.G.1
Kamen, B.A.2
Rothberg, K.G.3
Lacey, S.W.4
-
98
-
-
0032496393
-
Caveolin-3 is not an integral component of the dystrophin glycoprotein complex
-
Crosbie, R.H., Yamada, H., Venzke, D.P., Lisanti, M.P., Campbell, K.P. Caveolin-3 is not an integral component of the dystrophin glycoprotein complex. FEBS Lett. 1998, 427: 279-282.
-
(1998)
FEBS Lett.
, vol.427
, pp. 279-282
-
-
Crosbie, R.H.1
Yamada, H.2
Venzke, D.P.3
Lisanti, M.P.4
Campbell, K.P.5
-
99
-
-
0033755733
-
Caveolae: An alternative membrane transport compartment
-
Gumbleton, M., Abulrob, A.G., Campbell, L. Caveolae: an alternative membrane transport compartment. Pharm. Res. 2000, 17: 1035-1048.
-
(2000)
Pharm. Res.
, vol.17
, pp. 1035-1048
-
-
Gumbleton, M.1
Abulrob, A.G.2
Campbell, L.3
-
100
-
-
15844401780
-
Expression of caveolin-3 in skeletal, cardiac, and smooth muscle cells. Caveolin-3 is a component of the sarcolemma and co-fractionates with dystrophin and dystrophin-associated glycoproteins
-
Song, K.S., Scherer, P.E., Tang, Z., Okamoto, T., Li, S., Chafel, M., Chu, C., Kohtz, D.S., Lisanti, M.P. Expression of caveolin-3 in skeletal, cardiac, and smooth muscle cells. Caveolin-3 is a component of the sarcolemma and co-fractionates with dystrophin and dystrophin-associated glycoproteins. J. Biol. Chem. 1996, 271: 15160-15165.
-
(1996)
J. Biol. Chem.
, vol.271
, pp. 15160-15165
-
-
Song, K.S.1
Scherer, P.E.2
Tang, Z.3
Okamoto, T.4
Li, S.5
Chafel, M.6
Chu, C.7
Kohtz, D.S.8
Lisanti, M.P.9
-
101
-
-
0000855817
-
Fine Structure of Blood Capillaries
-
Palade, G.E. Fine Structure of Blood Capillaries. J. Appl. Phys. 1953, 24: 1424-1436.
-
(1953)
J. Appl. Phys.
, vol.24
, pp. 1424-1436
-
-
Palade, G.E.1
-
102
-
-
77049234363
-
The fine structure of the gall bladder epithelium of the mouse
-
Yamada, E. The fine structure of the gall bladder epithelium of the mouse. J. Biophys. Biochem. Cytol. 1955, 1: 445-458.
-
(1955)
J. Biophys. Biochem. Cytol.
, vol.1
, pp. 445-458
-
-
Yamada, E.1
-
103
-
-
0026499749
-
The sequence of human caveolin reveals identity with VIP21, a component of transport vesicles
-
Glenney, J.R. Jr. The sequence of human caveolin reveals identity with VIP21, a component of transport vesicles. FEBS Lett. 1992, 314: 45-48.
-
(1992)
FEBS Lett.
, vol.314
, pp. 45-48
-
-
Glenney Jr., J.R.1
-
104
-
-
0026559095
-
Caveolin, a protein component of caveolae membrane coats
-
Rothberg, K.G., Heuser, J.E., Donzell, W.C., Ying, Y.S., Glenney, J.R., Anderson, R.G. Caveolin, a protein component of caveolae membrane coats. Cell 1992, 68: 673-682.
-
(1992)
Cell
, vol.68
, pp. 673-682
-
-
Rothberg, K.G.1
Heuser, J.E.2
Donzell, W.C.3
Ying, Y.S.4
Glenney, J.R.5
Anderson, R.G.6
-
105
-
-
0030060941
-
Molecular cloning of caveolin-3, a novel member of the caveolin gene family expressed predominantly in muscle
-
Tang, Z., Scherer, P.E., Okamoto, T., Song, K., Chu, C., Kohtz, D.S., Nishimoto, I., Lodish, H.F., Lisanti, M.P. Molecular cloning of caveolin-3, a novel member of the caveolin gene family expressed predominantly in muscle. J. Biol. Chem. 1996, 271: 2255-2261.
-
(1996)
J. Biol. Chem.
, vol.271
, pp. 2255-2261
-
-
Tang, Z.1
Scherer, P.E.2
Okamoto, T.3
Song, K.4
Chu, C.5
Kohtz, D.S.6
Nishimoto, I.7
Lodish, H.F.8
Lisanti, M.P.9
-
106
-
-
0030561979
-
M-caveolin, a muscle-specific caveolin-related protein
-
Way, M. and Parton, R.G. M-caveolin, a muscle-specific caveolin-related protein. FEBS Lett. 1995, 376: 108-112.
-
(1995)
FEBS Lett.
, vol.376
, pp. 108-112
-
-
Way, M.1
Parton, R.G.2
-
107
-
-
0031030664
-
Caveolin-3 associates with developing T-tubules during muscle differentiation
-
Parton, R.G., Way, M., Zorzi, N., Stang, E. Caveolin-3 associates with developing T-tubules during muscle differentiation. J. Cell Biol. 1997, 136: 137-154.
-
(1997)
J. Cell Biol.
, vol.136
, pp. 137-154
-
-
Parton, R.G.1
Way, M.2
Zorzi, N.3
Stang, E.4
-
108
-
-
0027070497
-
Expression of naked plasmids by cultured myotubes and entry of plasmids into T tubules and caveolae of mammalian skeletal muscle
-
Wolff, J.A., Dowty, M.E., Jiao, S., Repetto, G., Berg, R.K., Ludtke, J.J., Williams, P., Slautterback, D.B. Expression of naked plasmids by cultured myotubes and entry of plasmids into T tubules and caveolae of mammalian skeletal muscle. J. Cell Sci. 1992, 103 (Pt 4): 1249-1259.
-
(1992)
J. Cell Sci.
, vol.103
, Issue.PART 4
, pp. 1249-1259
-
-
Wolff, J.A.1
Dowty, M.E.2
Jiao, S.3
Repetto, G.4
Berg, R.K.5
Ludtke, J.J.6
Williams, P.7
Slautterback, D.B.8
-
109
-
-
0028956615
-
Binding, uptake, and intracellular trafficking of phosphorothioate-modified oligodeoxynucleotides
-
Beltinger, C., Saragovi, H.U., Smith, R.M., LeSauteur, L., Shah, N., DeDionisio, L., Christensen, L., Raible, A., Jarett, L., Gewirtz, A.M. Binding, uptake, and intracellular trafficking of phosphorothioate-modified oligodeoxynucleotides. J. Clin. Invest 1995, 95: 1814-1823.
-
(1995)
J. Clin. Invest
, vol.95
, pp. 1814-1823
-
-
Beltinger, C.1
Saragovi, H.U.2
Smith, R.M.3
LeSauteur, L.4
Shah, N.5
DeDionisio, L.6
Christensen, L.7
Raible, A.8
Jarett, L.9
Gewirtz, A.M.10
-
110
-
-
0029875289
-
Characterization of plasmid DNA transfer into mouse skeletal muscle: Evaluation of uptake mechanism, expression and secretion of gene products into blood
-
Levy, M.Y., Barron, L.G., Meyer, K.B., Szoka, F.C., Jr. Characterization of plasmid DNA transfer into mouse skeletal muscle: evaluation of uptake mechanism, expression and secretion of gene products into blood. Gene Ther. 1996, 3: 201-211.
-
(1996)
Gene Ther.
, vol.3
, pp. 201-211
-
-
Levy, M.Y.1
Barron, L.G.2
Meyer, K.B.3
Szoka Jr., F.C.4
-
111
-
-
0034760639
-
Slow accumulation of plasmid in muscle cells: Supporting evidence for a mechanism of DNA uptake by receptor-mediated endocytosis
-
Satkauskas, S., Bureau, M.F., Mahfoudi, A., Mir, L.M. Slow accumulation of plasmid in muscle cells: supporting evidence for a mechanism of DNA uptake by receptor-mediated endocytosis. Mol. Ther. 2001, 4: 317-323.
-
(2001)
Mol. Ther.
, vol.4
, pp. 317-323
-
-
Satkauskas, S.1
Bureau, M.F.2
Mahfoudi, A.3
Mir, L.M.4
-
112
-
-
0032571392
-
Polyethylenimine but not cationic lipids promotes transgene delivery to the nucleus in mammalian cells
-
Pollard, H., Remy, J.S., Loussouam, G., Demolombe, S., Behr, J.P., Escande, D. Polyethylenimine but not cationic lipids promotes transgene delivery to the nucleus in mammalian cells. J. Biol. Chem. 1998, 273: 7507-7511.
-
(1998)
J. Biol. Chem.
, vol.273
, pp. 7507-7511
-
-
Pollard, H.1
Remy, J.S.2
Loussouam, G.3
Demolombe, S.4
Behr, J.P.5
Escande, D.6
-
113
-
-
1642443376
-
Quantitative Three-Dimensional Analysis of the Intracellular Trafficking of Plasmid DNA Transfected by a Nonviral Gene Delivery System Using Confocal Laser Scanning Microscopy
-
Akita, H., Ito, R., Khalil, A.I., Futaki, S., Harashima, H. Quantitative Three-Dimensional Analysis of the Intracellular Trafficking of Plasmid DNA Transfected by a Nonviral Gene Delivery System Using Confocal Laser Scanning Microscopy. Mol. Ther. 2004, 9: 443-451.
-
(2004)
Mol. Ther.
, vol.9
, pp. 443-451
-
-
Akita, H.1
Ito, R.2
Khalil, A.I.3
Futaki, S.4
Harashima, H.5
-
114
-
-
0034717044
-
Gatekeepers of the Nucleus
-
Wente, S.R. Gatekeepers of the Nucleus. Science 2000, 288: 1374-1377.
-
(2000)
Science
, vol.288
, pp. 1374-1377
-
-
Wente, S.R.1
-
115
-
-
0033572896
-
Sequence requirements for plasmid nuclear import
-
Dean, D.A., Dean, B.S., Muller, S., Smith, L.C. Sequence requirements for plasmid nuclear import. Exp. Cell Res. 1999, 253: 713-722.
-
(1999)
Exp. Cell Res.
, vol.253
, pp. 713-722
-
-
Dean, D.A.1
Dean, B.S.2
Muller, S.3
Smith, L.C.4
-
116
-
-
0031079644
-
Import of plasmid DNA into the nucleus is sequence specific
-
Dean, D.A. Import of plasmid DNA into the nucleus is sequence specific. Exp. Cell Res. 1997, 230: 293-302.
-
(1997)
Exp. Cell Res.
, vol.230
, pp. 293-302
-
-
Dean, D.A.1
-
117
-
-
0000976047
-
Amino terminus of the yeast GAL4 gene product is sufficient for nuclear localization
-
Silver, P.A., Keegan, L.P., Ptashime, M. Amino terminus of the yeast GAL4 gene product is sufficient for nuclear localization. Proc. Natl. Acad. Sci. U. S. A. 1984, 81: 5951-5955.
-
(1984)
Proc. Natl. Acad. Sci. U. S. A.
, vol.81
, pp. 5951-5955
-
-
Silver, P.A.1
Keegan, L.P.2
Ptashime, M.3
-
118
-
-
0033847140
-
Hyaluronidase enhances recombinant adeno-associated virus (rAAV)-mediated gene transfer in the rat skeletal muscle
-
Favre, D., Cherel, Y., Provost, N., Blouin, V., Ferry, N., Moullier, P., Salvetti, A. Hyaluronidase enhances recombinant adeno-associated virus (rAAV)-mediated gene transfer in the rat skeletal muscle. Gene Ther. 2000, 7: 1417-1420.
-
(2000)
Gene Ther.
, vol.7
, pp. 1417-1420
-
-
Favre, D.1
Cherel, Y.2
Provost, N.3
Blouin, V.4
Ferry, N.5
Moullier, P.6
Salvetti, A.7
-
119
-
-
0034893580
-
Optimisation of electrotransfer of plasmid into skeletal muscle by pretreatment with hyaluronidase - Increased expression with reduced muscle damage
-
McMahon, J.M., Signori, E., Wells, K.E., Fazio, V.M., Wells, D.J. Optimisation of electrotransfer of plasmid into skeletal muscle by pretreatment with hyaluronidase -- increased expression with reduced muscle damage. Gene Ther. 2001, 8: 1264-1270.
-
(2001)
Gene Ther.
, vol.8
, pp. 1264-1270
-
-
McMahon, J.M.1
Signori, E.2
Wells, K.E.3
Fazio, V.M.4
Wells, D.J.5
-
120
-
-
0036207351
-
Hyaluronidase increases electrogene transfer efficiency in skeletal muscle
-
Mennuni, C., Calvaruso, F., Zampaglione, I., Rizzuto, G., Rinaudo, D., Dammassa, E., Ciliberto, G., Fattori, E., La Monica, N. Hyaluronidase increases electrogene transfer efficiency in skeletal muscle. Hum. Gene Ther. 2002, 13: 355-365.
-
(2002)
Hum. Gene Ther.
, vol.13
, pp. 355-365
-
-
Mennuni, C.1
Calvaruso, F.2
Zampaglione, I.3
Rizzuto, G.4
Rinaudo, D.5
Dammassa, E.6
Ciliberto, G.7
Fattori, E.8
La Monica, N.9
-
121
-
-
0031753867
-
Gene transfer into muscle by electroporation in vivo
-
Aihara, H. and Miyazaki, J. Gene transfer into muscle by electroporation in vivo. Nat. Biotechnol. 1998, 16: 867-870.
-
(1998)
Nat. Biotechnol.
, vol.16
, pp. 867-870
-
-
Aihara, H.1
Miyazaki, J.2
-
122
-
-
0034787753
-
Electrotransfer of naked DNA in the skeletal muscles of animal models of muscular dystrophies
-
Vilquin, J.T., Kennel, P.F., Paturneau-Jouas, M., Chapdelaine, P., Boissel, N., Delaere, P., Tremblay, J.P., Scherman, D., Fiszman, M.Y., Schwartz, K. Electrotransfer of naked DNA in the skeletal muscles of animal models of muscular dystrophies. Gene Ther. 2001, 8: 1097-1107.
-
(2001)
Gene Ther.
, vol.8
, pp. 1097-1107
-
-
Vilquin, J.T.1
Kennel, P.F.2
Paturneau-Jouas, M.3
Chapdelaine, P.4
Boissel, N.5
Delaere, P.6
Tremblay, J.P.7
Scherman, D.8
Fiszman, M.Y.9
Schwartz, K.10
-
123
-
-
0037348637
-
High-efficiency plasmid gene transfer into dystrophic muscle
-
Gollins, H., McMahon, J., Wells, K.E., Wells, D.J. High-efficiency plasmid gene transfer into dystrophic muscle. Gene Ther. 2003, 10: 504-512.
-
(2003)
Gene Ther.
, vol.10
, pp. 504-512
-
-
Gollins, H.1
McMahon, J.2
Wells, K.E.3
Wells, D.J.4
-
124
-
-
0034194049
-
Importance of association between permeabilization and electrophoretic forces for intramuscular DNA electrotransfer
-
Bureau, M.F., Gehl, J., Deleuze, V., Mir, L.M., Scherman, D. Importance of association between permeabilization and electrophoretic forces for intramuscular DNA electrotransfer. Biochim. Biophys. Acta 2000, 1474: 353-359.
-
(2000)
Biochim. Biophys. Acta
, vol.1474
, pp. 353-359
-
-
Bureau, M.F.1
Gehl, J.2
Deleuze, V.3
Mir, L.M.4
Scherman, D.5
-
125
-
-
13044257108
-
High-efficiency gene transfer into skeletal muscle mediated by electric pulses
-
Mir, L.M., Bureau, M.F., Gehl, J., Rangara, R., Rouy, D., Caillaud, J.M., Delaere, P., Branellec, D., Schwartz, B., Scherman, D. High-efficiency gene transfer into skeletal muscle mediated by electric pulses. Proc. Natl. Acad. Sci. U. S. A. 1999, 96: 4262-4267.
-
(1999)
Proc. Natl. Acad. Sci. U. S. A.
, vol.96
, pp. 4262-4267
-
-
Mir, L.M.1
Bureau, M.F.2
Gehl, J.3
Rangara, R.4
Rouy, D.5
Caillaud, J.M.6
Delaere, P.7
Branellec, D.8
Schwartz, B.9
Scherman, D.10
-
126
-
-
0037309533
-
Full-length dystrophin cDNA transfer into skeletal muscle of adult mdx mice by electroporation
-
Murakami, T., Nishi, T., Kimura, E., Goto, T., Maeda, Y., Ushio, Y., Uchino, M., Sunada, Y. Full-length dystrophin cDNA transfer into skeletal muscle of adult mdx mice by electroporation. Muscle Nerve 2003, 27: 237-241.
-
(2003)
Muscle Nerve
, vol.27
, pp. 237-241
-
-
Murakami, T.1
Nishi, T.2
Kimura, E.3
Goto, T.4
Maeda, Y.5
Ushio, Y.6
Uchino, M.7
Sunada, Y.8
-
127
-
-
0031935395
-
The efficient expression of intravascularly delivered DNA in rat muscle
-
Budker, V., Zhang, G., Danko, I., Williams, P., Wolff, J. The efficient expression of intravascularly delivered DNA in rat muscle. Gene Ther. 1998, 5: 272-276.
-
(1998)
Gene Ther.
, vol.5
, pp. 272-276
-
-
Budker, V.1
Zhang, G.2
Danko, I.3
Williams, P.4
Wolff, J.5
-
128
-
-
0035111572
-
Efficient expression of naked dna delivered intraarterially to limb muscles of nonhuman primates
-
Zhang, G., Budker, V., Williams, P., Subbotin, V., Wolff, J.A. Efficient expression of naked dna delivered intraarterially to limb muscles of nonhuman primates. Hum. Gene Ther. 2001, 12: 427-438.
-
(2001)
Hum. Gene Ther.
, vol.12
, pp. 427-438
-
-
Zhang, G.1
Budker, V.2
Williams, P.3
Subbotin, V.4
Wolff, J.A.5
-
129
-
-
4143091589
-
Intraarterial delivery of naked plasmid DNA expressing full-length mouse dystrophin in the mdx mouse model of duchenne muscular dystrophy
-
Zhang, G., Ludtke, J.J., Thioudellet, C., Kleinpeter, P., Antoniou, M., Herweijer, H., Braun, S., Wolff, J.A. Intraarterial delivery of naked plasmid DNA expressing full-length mouse dystrophin in the mdx mouse model of duchenne muscular dystrophy. Hum. Gene Ther. 2004, 15: 770-782.
-
(2004)
Hum. Gene Ther.
, vol.15
, pp. 770-782
-
-
Zhang, G.1
Ludtke, J.J.2
Thioudellet, C.3
Kleinpeter, P.4
Antoniou, M.5
Herweijer, H.6
Braun, S.7
Wolff, J.A.8
-
130
-
-
0035064802
-
Effects of liposome-mediated gene transfer of VEGF in ischemic rat gracilis muscle
-
Neumeister, M.W., Song, Y.H., Mowlavi, A., Suchy, H., Mathur, A. Effects of liposome-mediated gene transfer of VEGF in ischemic rat gracilis muscle. microsurgery 2001, 21: 58-62.
-
(2001)
Microsurgery
, vol.21
, pp. 58-62
-
-
Neumeister, M.W.1
Song, Y.H.2
Mowlavi, A.3
Suchy, H.4
Mathur, A.5
-
131
-
-
0036401268
-
A nonionic amphiphile agent promotes gene delivery in vivo to skeletal and cardiac muscles
-
Pitard, B., Pollard, H., Agbulut, O., Lambert, O., Vilquin, J.T., Cherel, Y., Abadie, J., Samuel, J.L., Rigaud, J.L., Menoret, S., Anegon, I., Escande, D. A nonionic amphiphile agent promotes gene delivery in vivo to skeletal and cardiac muscles. Hum. Gene Ther. 2002, 13: 1767-1775.
-
(2002)
Hum. Gene Ther.
, vol.13
, pp. 1767-1775
-
-
Pitard, B.1
Pollard, H.2
Agbulut, O.3
Lambert, O.4
Vilquin, J.T.5
Cherel, Y.6
Abadie, J.7
Samuel, J.L.8
Rigaud, J.L.9
Menoret, S.10
Anegon, I.11
Escande, D.12
-
132
-
-
0037366338
-
Gene delivery with in-situ crosslinking polymer networks generates long-term systemic protein expression
-
Roy, K., Wang, D., Hedley, M.L., Barman, S.P. Gene delivery with in-situ crosslinking polymer networks generates long-term systemic protein expression. Mol. Ther. 2003, 7: 401-408.
-
(2003)
Mol. Ther.
, vol.7
, pp. 401-408
-
-
Roy, K.1
Wang, D.2
Hedley, M.L.3
Barman, S.P.4
-
133
-
-
0037836968
-
Enhanced adjuvanticity of interleukin-2 plasmid DNA administered in polyethylenimine complexes
-
Oh, Y.K., Park, J.S., Kang, M.J., Ko, J.J., Kim, J.M., Kim, C.K. Enhanced adjuvanticity of interleukin-2 plasmid DNA administered in polyethylenimine complexes. Vaccine 2003, 21: 2837-2843.
-
(2003)
Vaccine
, vol.21
, pp. 2837-2843
-
-
Oh, Y.K.1
Park, J.S.2
Kang, M.J.3
Ko, J.J.4
Kim, J.M.5
Kim, C.K.6
-
134
-
-
0035064802
-
Effects of Liposome-Mediated gene Transfer of VEGF in Ischemic Rat Gracilis Muscle
-
Neumeister, M.W., Song, Y.H., Mowlavi, A., Suchy, H., Mathur, A. Effects of Liposome-Mediated gene Transfer of VEGF in Ischemic Rat Gracilis Muscle. Microsurgery 2003, 21: 58-62.
-
(2003)
Microsurgery
, vol.21
, pp. 58-62
-
-
Neumeister, M.W.1
Song, Y.H.2
Mowlavi, A.3
Suchy, H.4
Mathur, A.5
-
135
-
-
0033959448
-
Effective suicide gene therapy in vivo by EBV-based plasmid vector coupled with polyamidoamine dendrimer
-
Maruyama-Tabata, H., Harada, Y., Matsumura, T., Satoh, E., Cui, F., Iwai, M., Kita, M., Hibi, S., Imanishi, J., Sawada, T., Mazda, O. Effective suicide gene therapy in vivo by EBV-based plasmid vector coupled with polyamidoamine dendrimer. Gene Ther. 2000, 7: 53-60.
-
(2000)
Gene Ther.
, vol.7
, pp. 53-60
-
-
Maruyama-Tabata, H.1
Harada, Y.2
Matsumura, T.3
Satoh, E.4
Cui, F.5
Iwai, M.6
Kita, M.7
Hibi, S.8
Imanishi, J.9
Sawada, T.10
Mazda, O.11
-
136
-
-
0038725718
-
Co-encapsulation of two plasmids in chitosan microspheres as a non-viral gene delivery vehicle
-
Ozbas-Turan, S., Aral, C., Kabasakal, L., Keyer-Uysal, M., Akbuga, J. Co-encapsulation of two plasmids in chitosan microspheres as a non-viral gene delivery vehicle. J. Pharm. Pharm. Sci. 2003, 6: 27-32.
-
(2003)
J. Pharm. Pharm. Sci.
, vol.6
, pp. 27-32
-
-
Ozbas-Turan, S.1
Aral, C.2
Kabasakal, L.3
Keyer-Uysal, M.4
Akbuga, J.5
-
137
-
-
0038512159
-
Effect of Pluronic-block copolymers on the reduction of serum-mediated inhibition of gene transfer of polyethyleneimine-DNA complexes
-
Kuo, J.H. Effect of Pluronic-block copolymers on the reduction of serum-mediated inhibition of gene transfer of polyethyleneimine-DNA complexes. Biotechnol. Appl. Biochem. 2003, 37: 267-271.
-
(2003)
Biotechnol. Appl. Biochem.
, vol.37
, pp. 267-271
-
-
Kuo, J.H.1
-
138
-
-
0028219485
-
Inhibition of vascular smooth muscle cell proliferation in vitro and in vivo by c-myc antisense oligodeoxynucleotides
-
Bennett, M.R., Anglin, S., McEwan, J.R., Jagoe, R., Newby, A.C., Evan, G.I. Inhibition of vascular smooth muscle cell proliferation in vitro and in vivo by c-myc antisense oligodeoxynucleotides. J. Clin. Invest 1994, 93: 820-828.
-
(1994)
J. Clin. Invest
, vol.93
, pp. 820-828
-
-
Bennett, M.R.1
Anglin, S.2
McEwan, J.R.3
Jagoe, R.4
Newby, A.C.5
Evan, G.I.6
-
139
-
-
0032838651
-
Antisense oligonucleotides to c-fos and c-jun inhibit intimal thickening in a rat vein graft model
-
Suggs, W.D., Olson, S.C., Madnani, D., Patel, S., Veith, F.J., Mandani, D. Antisense oligonucleotides to c-fos and c-jun inhibit intimal thickening in a rat vein graft model. Surgery 1999, 126: 443-449.
-
(1999)
Surgery
, vol.126
, pp. 443-449
-
-
Suggs, W.D.1
Olson, S.C.2
Madnani, D.3
Patel, S.4
Veith, F.J.5
Mandani, D.6
-
140
-
-
0034119142
-
Block and graft copolymers and NanoGel copolymer networks for DNA delivery into cell
-
Lemieux, P., Vinogradov, S.V., Gebhart, C.L., Guerin, N., Paradis, G., Nguyen, H.K., Ochietti, B., Suzdaltseva, Y.G., Bartakova, E.V., Bronich, T.K., St Pierre, Y., Alakhov, V.Y., Kabanov, A.V. Block and graft copolymers and NanoGel copolymer networks for DNA delivery into cell. J. Drug Target 2000, 8: 91-105.
-
(2000)
J. Drug Target
, vol.8
, pp. 91-105
-
-
Lemieux, P.1
Vinogradov, S.V.2
Gebhart, C.L.3
Guerin, N.4
Paradis, G.5
Nguyen, H.K.6
Ochietti, B.7
Suzdaltseva, Y.G.8
Bartakova, E.V.9
Bronich, T.K.10
St Pierre, Y.11
Alakhov, V.Y.12
Kabanov, A.V.13
-
141
-
-
16544377151
-
Negatively charged self-assembling DNA/poloxamine nanospheres for in vivo gene transfer
-
Pitard, B., Bello-Roufai, M., Lambert, O., Richard, P., Desigaux, L., Fernandes, S., Lanctin, C., Pollard, H., Zeghal, M., Rescan, P.Y., Escande, D. Negatively charged self-assembling DNA/poloxamine nanospheres for in vivo gene transfer. Nucleic Acids Res. 2004, 32: e159-.
-
(2004)
Nucleic Acids Res.
, vol.32
-
-
Pitard, B.1
Bello-Roufai, M.2
Lambert, O.3
Richard, P.4
Desigaux, L.5
Fernandes, S.6
Lanctin, C.7
Pollard, H.8
Zeghal, M.9
Rescan, P.Y.10
Escande, D.11
-
142
-
-
0034689207
-
Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: Alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered
-
Stedman, H., Wilson, J.M., Finke, R., Kleckner, A.L., Mendell, J. Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors. Hum. Gene Ther. 2000, 11: 777-790.
-
(2000)
Hum. Gene Ther.
, vol.11
, pp. 777-790
-
-
Stedman, H.1
Wilson, J.M.2
Finke, R.3
Kleckner, A.L.4
Mendell, J.5
-
143
-
-
0036823512
-
Current protocol of a research phase I clinical trial of full-length dystrophin plasmid DNA in Duchenne/Becker muscular dystrophies. Part III. Ethical considerations
-
Fardeau, M. Current protocol of a research phase I clinical trial of full-length dystrophin plasmid DNA in Duchenne/Becker muscular dystrophies. Part III. Ethical considerations. Neuromuscular Disorders 2002, 12(Suppl): S52-.
-
(2002)
Neuromuscular Disorders
, vol.12
, Issue.SUPPL.
-
-
Fardeau, M.1
-
144
-
-
0036824011
-
Current protocol of a research phase I clinical trial of full-length dystrophin plasmid DNA in Duchenne/Becker muscular dystrophies. Part II: Clinical protocol
-
Romero, N.B., Benveniste, O., Payan, C., Braun, S., Squiban, P., Herson, S., Fardeau, M. Current protocol of a research phase I clinical trial of full-length dystrophin plasmid DNA in Duchenne/Becker muscular dystrophies. Part II: clinical protocol. Neuromuscul. Disord. 2002, 12 Suppl 1: S45-S48.
-
(2002)
Neuromuscul. Disord.
, vol.12
, Issue.SUPPL. 1
-
-
Romero, N.B.1
Benveniste, O.2
Payan, C.3
Braun, S.4
Squiban, P.5
Herson, S.6
Fardeau, M.7
-
145
-
-
0036823752
-
Current protocol of a research phase I clinical trial of full-length dystrophin plasmid DNA in Duchenne/Becker muscular dystrophies. Part I: Rationale
-
Thioudellet, C., Blot, S., Squiban, P., Fardeau, M., Braun, S. Current protocol of a research phase I clinical trial of full-length dystrophin plasmid DNA in Duchenne/Becker muscular dystrophies. Part I: rationale. Neuromuscul. Disord. 2002, 12 Suppl 1: S49-S51.
-
(2002)
Neuromuscul. Disord.
, vol.12
, Issue.SUPPL. 1
-
-
Thioudellet, C.1
Blot, S.2
Squiban, P.3
Fardeau, M.4
Braun, S.5
-
146
-
-
19344362163
-
Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: Where are we now?
-
Wilton, S.D. and Fletcher, S. Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: Where are we now? Neuromuscul. Disord. 2005, 15: 399-402.
-
(2005)
Neuromuscul. Disord.
, vol.15
, pp. 399-402
-
-
Wilton, S.D.1
Fletcher, S.2
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