-
1
-
-
0342275189
-
The scope of gene therapy in humans: Scientific, safety and ethical considerations
-
Karpati G, Lochmuller H. The scope of gene therapy in humans: scientific, safety and ethical considerations. Neuromuscular Disord 1997;7:273-276.
-
(1997)
Neuromuscular Disord
, vol.7
, pp. 273-276
-
-
Karpati, G.1
Lochmuller, H.2
-
2
-
-
0030766408
-
Naked DNA transport and expression in mammalian cells
-
Wolff JA. Naked DNA transport and expression in mammalian cells. Neuromuscular Disord 1997;7:314-318.
-
(1997)
Neuromuscular Disord
, vol.7
, pp. 314-318
-
-
Wolff, J.A.1
-
3
-
-
0031564111
-
Human gene marker/therapy clinical protocols
-
Human gene marker/therapy clinical protocols. Hum Gen Ther 1997; 8:2301-2338.
-
(1997)
Hum Gen Ther
, vol.8
, pp. 2301-2338
-
-
-
4
-
-
0025231388
-
Direct gene transfer into mouse muscle in vivo
-
Wolff JA, Malone RW, Williams P, Chong W, Acsadi G, Jani A, Felger PL. 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
Felger, P.L.7
-
5
-
-
0025887093
-
Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs
-
Acsadi G, Dickson G, Love DRM, Jani A, Walsh FS, Gurusinghe A, Wolff JA, Davies KE. 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
Drm, L.3
Jani, A.4
Walsh, F.S.5
Gurusinghe, A.6
Wolff, J.A.7
Davies, K.E.8
-
6
-
-
0030066219
-
Dystrophin expression in muscles of mdx mice after adenovirus-mediated in vivo gene transfer
-
Acsadi G, Lochmuller H, Jani A, Huard J, Massie B, Prescott S, Simoneau M, Karpati G. Dystrophin expression in muscles of mdx mice after adenovirus-mediated in vivo gene transfer. Hum Gen Ther 1996;7:129-140.
-
(1996)
Hum Gen Ther
, vol.7
, pp. 129-140
-
-
Acsadi, G.1
Lochmuller, H.2
Jani, A.3
Huard, J.4
Massie, B.5
Prescott, S.6
Simoneau, M.7
Karpati, G.8
-
7
-
-
0030870110
-
Use of the dog model for Duchenne muscular dystrophy in gene therapy trials
-
McC Howell, J., Fletcher, S., Kakulas, B.A., O'Hara, M., Lochmuller, H., Karpati, G. Use of the dog model for Duchenne muscular dystrophy in gene therapy trials. Neuromuscular Disord 7;325-328.
-
Neuromuscular Disord
, vol.7
, pp. 325-328
-
-
McC Howell, J.1
Fletcher, S.2
Kakulas, B.A.3
O'Hara, M.4
Lochmuller, H.5
Karpati, G.6
-
8
-
-
0031914607
-
Direct dystrophin and reporter gene transfer into dog muscle in vivo
-
McC Howell J, Fletcher S, O'Hara A, Johnsen R, Lloyd F, Kakulas BA. Direct dystrophin and reporter gene transfer into dog muscle in vivo. Muscle Nerve 1998;21:159-165.
-
(1998)
Muscle Nerve
, vol.21
, pp. 159-165
-
-
McC Howell, J.1
Fletcher, S.2
O'Hara, A.3
Johnsen, R.4
Lloyd, F.5
Kakulas, B.A.6
-
9
-
-
0032549685
-
High-level dystrophin expression after adenovirus-mediated dystrophic minigene transfer to skeletal muscle of dystrophic dog: Prolongation of expression with immunosuppression
-
McC Howell J, Lochmuller H, O'Hara A, Fletcher S, Kakulas BA, Massie B, Nalbantoglu J, Karpati G. High-level dystrophin expression after adenovirus-mediated dystrophic minigene transfer to skeletal muscle of dystrophic dog: prolongation of expression with immunosuppression. Hum Gen Ther 1998;9:629-634.
-
(1998)
Hum Gen Ther
, vol.9
, pp. 629-634
-
-
McC Howell, J.1
Lochmuller, H.2
O'Hara, A.3
Fletcher, S.4
Kakulas, B.A.5
Massie, B.6
Nalbantoglu, J.7
Karpati, G.8
-
10
-
-
0344174589
-
Human trials set for DMD
-
Human trials set for DMD. Quest 1998; 5:9-10.
-
(1998)
Quest
, vol.5
, pp. 9-10
-
-
-
11
-
-
0032077904
-
Implications of maturation for viral gene delivery to skeletal muscle
-
Van Deutekom JCT, Floyd SS, Booth DK, Oligino T, Kirsky D, Marconi P, Glorioso JC, Huard J. Implications of maturation for viral gene delivery to skeletal muscle. Neuromuscular Disord 1998;8:135-148.
-
(1998)
Neuromuscular Disord
, vol.8
, pp. 135-148
-
-
Van Deutekom, J.C.T.1
Floyd, S.S.2
Booth, D.K.3
Oligino, T.4
Kirsky, D.5
Marconi, P.6
Glorioso, J.C.7
Huard, J.8
-
12
-
-
0029741231
-
Transient immunosuppression by FK506 permits a sustained high level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscles of adult dystrophic (mdx) mice
-
Lochmuller H, Petrof BJ, Pari G, Larochelle N, Dodelet V, Wang Q, Allen C, Prescott S, Massie B, Nalbantoglu J, Karpati G. Transient immunosuppression by FK506 permits a sustained high level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscles of adult dystrophic (mdx) mice. Gene Ther 1996;3:706-716.
-
(1996)
Gene Ther
, vol.3
, pp. 706-716
-
-
Lochmuller, H.1
Petrof, B.J.2
Pari, G.3
Larochelle, N.4
Dodelet, V.5
Wang, Q.6
Allen, C.7
Prescott, S.8
Massie, B.9
Nalbantoglu, J.10
Karpati, G.11
-
13
-
-
0030776795
-
Problems and potential for gene therapy in Duchenne muscular dystrophy
-
Kakulas BA. Problems and potential for gene therapy in Duchenne muscular dystrophy. Neuromuscular Disord 1997;7:319-324.
-
(1997)
Neuromuscular Disord
, vol.7
, pp. 319-324
-
-
Kakulas, B.A.1
-
14
-
-
0028915634
-
The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants
-
Huard J, Lochmuller H, Acsadi G, Jani A, Massie B, Karpati G. The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants. Gene Ther 1995;2:107-115.
-
(1995)
Gene Ther
, vol.2
, pp. 107-115
-
-
Huard, J.1
Lochmuller, H.2
Acsadi, G.3
Jani, A.4
Massie, B.5
Karpati, G.6
-
15
-
-
0031883829
-
Production and characterization of improved adenovirus vectors with the E1, E2b and E3 genes deleted
-
Amalfitano A, Hauser MA, Hu H, Serra D, Begy CR, Chamberlain JS. Production and characterization of improved adenovirus vectors with the E1, E2b and E3 genes deleted. J Virol 1998;72:926-933y.
-
(1998)
J Virol
, vol.72
-
-
Amalfitano, A.1
Hauser, M.A.2
Hu, H.3
Serra, D.4
Begy, C.R.5
Chamberlain, J.S.6
-
16
-
-
0030988482
-
-
Ilan Y., Droguett G., Chowdhury N.R., Li Y., Sengupta K., Thummala N.R., Davidson A., Chowdhury J.R., Horwitz M.S. Proc Natl Acad Sci USA, 1997;94:2587-2592.
-
(1997)
Proc Natl Acad Sci USA
, vol.94
, pp. 2587-2592
-
-
Ilan, Y.1
Droguett, G.2
Chowdhury, N.R.3
Li, Y.4
Sengupta, K.5
Thummala, N.R.6
Davidson, A.7
Chowdhury, J.R.8
Horwitz, M.S.9
-
17
-
-
0030850681
-
Strategies to accomplish targeted gene delivery to muscle cells employing tropism-modified adenoviral vectors
-
Douglas JT, Curiel DT. Strategies to accomplish targeted gene delivery to muscle cells employing tropism-modified adenoviral vectors. Neuromuscular Disord 1997;7:284-298.
-
(1997)
Neuromuscular Disord
, vol.7
, pp. 284-298
-
-
Douglas, J.T.1
Curiel, D.T.2
-
18
-
-
0031983639
-
Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia
-
Monahan PE, Samulski RJ, Tazelaar J, Xiao X, Nichols TC, Bellinger DA, Read MS, Walsh CE. 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
-
19
-
-
0030716959
-
Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors
-
Kafri T, Blomer U, Peterson DA, Gage FH, Verma IM. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet 1997;17:314-317.
-
(1997)
Nat Genet
, vol.17
, pp. 314-317
-
-
Kafri, T.1
Blomer, U.2
Peterson, D.A.3
Gage, F.H.4
Verma, I.M.5
-
20
-
-
0031985685
-
Minimal requirement for a lentivirus vector based on human immunodeficiency virus type I
-
Kim VN, Mitrophanous K, Kingsman SM, Kingsman AJ. Minimal requirement for a lentivirus vector based on human immunodeficiency virus type I. J Virol 1998;72:811-816.
-
(1998)
J Virol
, vol.72
, pp. 811-816
-
-
Kim, V.N.1
Mitrophanous, K.2
Kingsman, S.M.3
Kingsman, A.J.4
-
21
-
-
0030794156
-
-
Feero W.G., Li S., Rosenblatt J.D., Sirianni N., Morgan J.E., Partridge T.A., Huang L., Hoffman E.P. Gene Ther 1997;4:664-674.
-
(1997)
Gene Ther
, vol.4
, pp. 664-674
-
-
Feero, W.G.1
Li, S.2
Rosenblatt, J.D.3
Sirianni, N.4
Morgan, J.E.5
Partridge, T.A.6
Huang, L.7
Hoffman, E.P.8
-
22
-
-
0030793767
-
De novo protein design. Fully automated sequence selection
-
Dahlyat BI, Mayo SL. De novo protein design. Fully automated sequence selection. Science 1997;278:82-87.
-
(1997)
Science
, vol.278
, pp. 82-87
-
-
Dahlyat, B.I.1
Mayo, S.L.2
-
23
-
-
0345596435
-
Lipoadenofection-mediated gene delivery to the corneal endothelium: Prospects for modulating graft rejection
-
Arancibia-Carcamo CV, Oral HB, Haskard DO, Larkin DF, George AJ. Lipoadenofection-mediated gene delivery to the corneal endothelium: prospects for modulating graft rejection. Transplantation 1998;65:62-67.
-
(1998)
Transplantation
, vol.65
, pp. 62-67
-
-
Arancibia-Carcamo, C.V.1
Oral, H.B.2
Haskard, D.O.3
Larkin, D.F.4
George, A.J.5
-
24
-
-
0030853159
-
Amelioration of collagen-induced arthritis by CD95 (Apo-1/Fas)-ligand gene transfer
-
Zhang H, Yang Y, Horton JL, Samoilova EB, Judge TA, Turka LA, Wilson JM, Chen Y. Amelioration of collagen-induced arthritis by CD95 (Apo-1/Fas)-ligand gene transfer. J Clin Invest 1997;100:1951-1957.
-
(1997)
J Clin Invest
, vol.100
, pp. 1951-1957
-
-
Zhang, H.1
Yang, Y.2
Horton, J.L.3
Samoilova, E.B.4
Judge, T.A.5
Turka, L.A.6
Wilson, J.M.7
Chen, Y.8
-
25
-
-
0030848338
-
-
Grady R.M., Teng H., Nichol M.C., Cunningham J.C., Wilkinson R.S., Sanes J.R. Cell 1997;90:729-738.
-
(1997)
Cell
, vol.90
, pp. 729-738
-
-
Grady, R.M.1
Teng, H.2
Nichol, M.C.3
Cunningham, J.C.4
Wilkinson, R.S.5
Sanes, J.R.6
-
26
-
-
0030848969
-
Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy
-
Deconinck AE, Rafael JA, Skinner JA, Brown SC, Potter AC, Metzinger L, Watt DJ, Dickson JG, Tinsley JM, Davies KE. Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy. Cell 1997;90:717-727.
-
(1997)
Cell
, vol.90
, pp. 717-727
-
-
Deconinck, A.E.1
Rafael, J.A.2
Skinner, J.A.3
Brown, S.C.4
Potter, A.C.5
Metzinger, L.6
Watt, D.J.7
Dickson, J.G.8
Tinsley, J.M.9
Davies, K.E.10
-
27
-
-
0030723508
-
-
Deconinck N., Tinsley J., De Backer F., Fisher R., Kahn D., Phelps S., Davies K., Gillis J.M. Nat Med 1997;3:1216-1221.
-
(1997)
Nat Med
, vol.3
, pp. 1216-1221
-
-
Deconinck, N.1
Tinsley, J.2
De Backer, F.3
Fisher, R.4
Kahn, D.5
Phelps, S.6
Davies, K.7
Gillis, J.M.8
-
28
-
-
0031023495
-
Utrophin muscles in on the action
-
Karpati G. Utrophin muscles in on the action. Nat Med 1997;3:22-23.
-
(1997)
Nat Med
, vol.3
, pp. 22-23
-
-
Karpati, G.1
-
29
-
-
0032488509
-
Efficient utrophin expression following adenovirus gene transfer in dystrophic muscle
-
Gilbert R, Nalbanoglu J, Tinsley JM, Massie B, Davies KE, Karpati G. Efficient utrophin expression following adenovirus gene transfer in dystrophic muscle. Biochem Biophys Res Commun 1998;242:244-247.
-
(1998)
Biochem Biophys Res Commun
, vol.242
, pp. 244-247
-
-
Gilbert, R.1
Nalbanoglu, J.2
Tinsley, J.M.3
Massie, B.4
Davies, K.E.5
Karpati, G.6
-
30
-
-
0026641794
-
Dystrophin in frame-shift deletion patients with Becker muscular dystrophy
-
Gangopadhyay SB, Sherratt TG, Heckmatt JZ. Dystrophin in frame-shift deletion patients with Becker muscular dystrophy. Hum Genet 1992;51:562-570.
-
(1992)
Hum Genet
, vol.51
, pp. 562-570
-
-
Gangopadhyay, S.B.1
Sherratt, T.G.2
Heckmatt, J.Z.3
-
31
-
-
0345190477
-
Targeted dystrophin gene processing using antisense oligonucleotides
-
Wilton, S., Lloyd, F., Fletcher, S., Kole, R. Targeted dystrophin gene processing using antisense oligonucleotides. Neuromuscular Disord 1998;TO1:264.
-
(1998)
Neuromuscular Disord
, vol.TO1
, pp. 264
-
-
Wilton, S.1
Lloyd, F.2
Fletcher, S.3
Kole, R.4
-
32
-
-
0026703748
-
Prevalence of dystrophin positive fibres in 85 Duchenne muscular patients
-
Fanin M, Danieli GA, Vitiello L, Senter L, Angelini C. Prevalence of dystrophin positive fibres in 85 Duchenne muscular patients. Neuromuscular Disord 1992;2:41-45.
-
(1992)
Neuromuscular Disord
, vol.2
, pp. 41-45
-
-
Fanin, M.1
Danieli, G.A.2
Vitiello, L.3
Senter, L.4
Angelini, C.5
-
33
-
-
0000502268
-
A functional disorder of muscle associated with the absence of phosphorylase
-
Mommaerts WFHM, Illingworth B, Pearson CM, Guillory RJ, Seraydarian K. A functional disorder of muscle associated with the absence of phosphorylase. Proc Natl Acad Sci USA 1959;45:791-797.
-
(1959)
Proc Natl Acad Sci USA
, vol.45
, pp. 791-797
-
-
Mommaerts, W.F.H.M.1
Illingworth, B.2
Pearson, C.M.3
Guillory, R.J.4
Seraydarian, K.5
-
34
-
-
0011242186
-
Hereditary absence of muscle phosphorylase (McArdle's syndrome)
-
Schmid R, Hammaker L. Hereditary absence of muscle phosphorylase (McArdle's syndrome). N Engl J Med 1961;264:223-225.
-
(1961)
N Engl J Med
, vol.264
, pp. 223-225
-
-
Schmid, R.1
Hammaker, L.2
-
35
-
-
0021147082
-
Mitigation of deleterious effects of certain abnormal genes in immature skeletal muscle cells
-
Karpati G. Mitigation of deleterious effects of certain abnormal genes in immature skeletal muscle cells. Trends Neurosci 1984;7:360-361.
-
(1984)
Trends Neurosci
, vol.7
, pp. 360-361
-
-
Karpati, G.1
-
36
-
-
0024064452
-
Studies on the expression and evolution of the glycogen phosphorylase gene family in the rat
-
Crerar MM, Hudson JW, Matthews KE, David ES, Golding GB. Studies on the expression and evolution of the glycogen phosphorylase gene family in the rat. Genome 1988;30:582-590.
-
(1988)
Genome
, vol.30
, pp. 582-590
-
-
Crerar, M.M.1
Hudson, J.W.2
Matthews, K.E.3
David, E.S.4
Golding, G.B.5
-
37
-
-
0030774756
-
A splice-site mutation causing ovine McArdle's disease
-
Tan P, Allen JG, Wilton SD, Akkari PA, Huxtable CR, Laing NG. A splice-site mutation causing ovine McArdle's disease. Neuromuscular Disord 1997;7:336-342.
-
(1997)
Neuromuscular Disord
, vol.7
, pp. 336-342
-
-
Tan, P.1
Allen, J.G.2
Wilton, S.D.3
Akkari, P.A.4
Huxtable, C.R.5
Laing, N.G.6
|