-
1
-
-
0021358756
-
Duchenne muscular dystrophy: Pathogenetic aspects and genetic prevention
-
DOI 10.1007/BF00275183
-
Moser H., Duchenne muscular dystrophy: pathogenetic aspects and ge (Pubitemid 14159042)
-
(1984)
Human Genetics
, vol.66
, Issue.1
, pp. 17-40
-
-
Moser, H.1
-
2
-
-
0023614188
-
Dystrophin: The protein product of the Duchenne muscular dystrophy locus
-
Hoffman E. P., Brown R. H. Jr., Kunkel L. M., Dystrophin: the protein product of the Duchenne muscular dystrophy locus Cell 1987 51 6 919 928
-
(1987)
Cell
, vol.51
, Issue.6
, pp. 919-928
-
-
Hoffman, E.P.1
Brown Jr., R.H.2
Kunkel, L.M.3
-
3
-
-
0028914964
-
Three muscular dystrophies: Loss of cytoskeleton-extracellular matrix linkage
-
Campbell K. P., Three muscular dystrophies: loss of cytoskeleton- extracellular matrix linkage Cell 1995 80 5 675 679
-
(1995)
Cell
, vol.80
, Issue.5
, pp. 675-679
-
-
Campbell, K.P.1
-
4
-
-
13444267465
-
Canine and feline models of human inherited muscle diseases
-
DOI 10.1016/j.nmd.2004.10.019, PII S0960896604003116
-
Shelton G. D., Engvall E., Canine and feline models of human inherited muscle diseases Neuromuscular Disorders 2005 15 2 127 138 (Pubitemid 40202739)
-
(2005)
Neuromuscular Disorders
, vol.15
, Issue.2
, pp. 127-138
-
-
Shelton, G.D.1
Engvall, E.2
-
5
-
-
12744268414
-
Effects of stretch-activated channel blockers on [Ca2+]i and muscle damage in the mdx mouse
-
Yeung E. W., Whitehead N. P., Suchyna T. M., Gottlieb P. A., Sachs F., Allen D. G., Effects of stretch-activated channel blockers on [Ca2+]i and muscle damage in the mdx mouse Journal of Physiology 2005 562 2 367 380
-
(2005)
Journal of Physiology
, vol.562
, Issue.2
, pp. 367-380
-
-
Yeung, E.W.1
Whitehead, N.P.2
Suchyna, T.M.3
Gottlieb, P.A.4
Sachs, F.5
Allen, D.G.6
-
6
-
-
0024466501
-
The molecular basis for Duchenne versus becker muscular dystrophy: Correlation of severity with type of deletion
-
Koening M., Beggs A. H., Moyer M., Scherpf S., Heindrich K., Bettecken T., Meng G., Muller C. R., Lindlof M., Kaariainen H., De la Chapelle A., Kiuru A., Savontaus M. L., Gilgenkrantz H., Recan D., Chelly J., Kaplan J. C., Covone A. E., Archidiacono N., The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion American Journal of Human Genetics 1989 45 4 498 506 (Pubitemid 19261437)
-
(1989)
American Journal of Human Genetics
, vol.45
, Issue.4
, pp. 498-506
-
-
Koening, M.1
Beggs, A.H.2
Moyer, M.3
Scherpf, S.4
Heindrich, K.5
Bettecken, T.6
Meng, G.7
Muller, C.R.8
Lindlof, M.9
Kaariainen, H.10
De La Chapelle, A.11
Kiuru, A.12
Savontaus, M.-L.13
Gilgenkrantz, H.14
Recan, D.15
Chelly, J.16
Kaplan, J.-C.17
Covone, A.E.18
Archidiacono, N.19
-
7
-
-
33751430435
-
Building oligonucleotide therapeutics using non-natural chemistries
-
DOI 10.1016/j.cbpa.2006.10.001, PII S1367593106001542
-
Wilson C., Keefe A. D., Building oligonucleotide therapeutics using non-natural chemistries Current Opinion in Chemical Biology 2006 10 6 607 614 (Pubitemid 44821893)
-
(2006)
Current Opinion in Chemical Biology
, vol.10
, Issue.6
, pp. 607-614
-
-
Wilson, C.1
Keefe, A.D.2
-
8
-
-
33746334153
-
Promising nucleic acid analogs and mimics: Characteristic features and applications of PNA, LNA, and morpholino
-
DOI 10.1007/s00253-006-0434-2
-
Karkare S., Bhatnagar D., Promising nucleic acid analogs and mimics: characteristic features and applications of PNA, LNA, and morpholino Applied Microbiology and Biotechnology 2006 71 5 575 586 (Pubitemid 44114786)
-
(2006)
Applied Microbiology and Biotechnology
, vol.71
, Issue.5
, pp. 575-586
-
-
Karkare, S.1
Bhatnagar, D.2
-
9
-
-
7044253593
-
AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype
-
DOI 10.1016/j.ymthe.2004.07.025, PII S1525001604013590
-
Yoshimura M., Sakamoto M., Ikemoto M., Mochizuki Y., Yuasa K., Miyagoe-Suzuki Y., Takeda S., AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype Molecular Therapy 2004 10 5 821 828 (Pubitemid 39419909)
-
(2004)
Molecular Therapy
, vol.10
, Issue.5
, pp. 821-828
-
-
Yoshimura, M.1
Sakamoto, M.2
Ikemoto, M.3
Mochizuki, Y.4
Yuasa, K.5
Miyagoe-Suzuki, Y.6
Takeda, S.7
-
10
-
-
33745894322
-
RAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice
-
DOI 10.1038/nm1439, PII NM1439
-
Gregorevic P., Allen J. M., Minami E., Blankinship M. J., Haraguchi M., Meuse L., Finn E., Adams M. E., Froehner S. C., Murry C. E., Chamberlain J. S., rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice Nature Medicine 2006 12 7 787 789 (Pubitemid 44050067)
-
(2006)
Nature Medicine
, vol.12
, Issue.7
, pp. 787-789
-
-
Gregorevic, P.1
Allen, J.M.2
Minami, E.3
Blankinship, M.J.4
Haraguchi, M.5
Meuse, L.6
Finn, E.7
Adams, M.E.8
Froehner, S.C.9
Murry, C.E.10
Chamberlain, J.S.11
-
11
-
-
0023758480
-
The mdx mouse skeletal muscle myopathy: II. Contractile properties
-
Coulton G. R., Curtin N. A., Morgan J. E., Partridge T. A., The mdx mouse skeletal muscle myopathy: II. Contractile properties Neuropathology and Applied Neurobiology 1988 14 4 299 314
-
(1988)
Neuropathology and Applied Neurobiology
, vol.14
, Issue.4
, pp. 299-314
-
-
Coulton, G.R.1
Curtin, N.A.2
Morgan, J.E.3
Partridge, T.A.4
-
12
-
-
0029820310
-
Differential expression of dystrophin isoforms in strains of mdx mice with different mutations
-
Im W. B., Phelps S. F., Copen E. H., Adams E. G., Slightom J. L., Chamberlain J. S., Differential expression of dystrophin isoforms in strains of mdx mice with different mutations Human Molecular Genetics 1996 5 8 1149 1153 (Pubitemid 26318554)
-
(1996)
Human Molecular Genetics
, vol.5
, Issue.8
, pp. 1149-1153
-
-
Im, W.B.1
Phelps, S.F.2
Copen, E.H.3
Adams, E.G.4
Slightom, J.L.5
Chamberlain, J.S.6
-
13
-
-
0031577559
-
Targeted disruption of exon 52 in the mouse dystrophin gene induced muscle degeneration similar to that observed in duchenne muscular dystrophy
-
DOI 10.1006/bbrc.1997.7328
-
Araki E., Nakamura K., Nakao K., Kameya S., Kobayashi O., Nonaka I., Kobayashi T., Katsuki M., Targeted disruption of exon 52 in the mouse dystrophin gene induced muscle degeneration similar to that observed in duchenne muscular dystrophy Biochemical and Biophysical Research Communications 1997 238 2 492 497 (Pubitemid 27464414)
-
(1997)
Biochemical and Biophysical Research Communications
, vol.238
, Issue.2
, pp. 492-497
-
-
Araki, E.1
Nakamura, K.2
Nakao, K.3
Kameya, S.4
Kobayashi, O.5
Nonaka, I.6
Kobayashi, T.7
Katsuki, M.8
-
14
-
-
0030848969
-
Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy
-
DOI 10.1016/S0092-8674(00)80532-2
-
Deconinck A. E., Rafael J. A., Skinner J. A., Brown S. C., Potter A. C., Metzinger L., Watt D. J., Dickson J. G., Tinsley J. M., Davies K. E., Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy Cell 1997 90 4 717 727 (Pubitemid 27357963)
-
(1997)
Cell
, vol.90
, Issue.4
, 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
-
15
-
-
0345731966
-
X chromosome-linked muscular dystrophy (mdx) in the mouse
-
Bulfield G., Siller W. G., Wight P. A. L., Moore K. J., X chromosome-linked muscular dystrophy (mdx) in the mouse Proceedings of the National Academy of Sciences of the United States of America 1984 81 4 I 1189 1192 (Pubitemid 14171480)
-
(1984)
Proceedings of the National Academy of Sciences of the United States of America
, vol.81
, Issue.I
, pp. 1189-1192
-
-
Bulfield, G.1
Siller, W.G.2
Wight, P.A.L.3
Moore, K.J.4
-
16
-
-
0021713536
-
Muscle development in mdx mutant mice
-
DOI 10.1002/mus.880070903
-
Dangain J., Vrbova G., Muscle development in mdx mutant mice Muscle and Nerve 1984 7 9 700 704 (Pubitemid 15209285)
-
(1984)
Muscle and Nerve
, vol.7
, Issue.9
, pp. 700-704
-
-
Dangain, J.1
Vrbova, G.2
-
17
-
-
0022638233
-
Skeletal muscle pathology in X chromosome-linked muscular dystrophy (mdx) mouse
-
Tanabe Y., Esaki K., Nomura T., Skeletal muscle pathology in X chromosome-linked muscular dystrophy (mdx) mouse Acta Neuropathologica 1986 69 1-2 91 95 (Pubitemid 16146255)
-
(1986)
Acta Neuropathologica
, vol.69
, Issue.1-2
, pp. 91-95
-
-
Tanabe, Y.1
Esaki, K.2
Nomura, T.3
-
18
-
-
0042536463
-
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
-
DOI 10.1038/nm897
-
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 Nature Medicine 2003 9 8 1009 1014 (Pubitemid 37021780)
-
(2003)
Nature Medicine
, vol.9
, Issue.8
, pp. 1009-1014
-
-
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
-
19
-
-
33748316668
-
Therapeutic restoration of dystrophin expression in Duchenne muscular dystrophy
-
DOI 10.1007/s10974-006-9081-6
-
Wells D. J., Therapeutic restoration of dystrophin expression in Duchenne muscular dystrophy Journal of Muscle Research and Cell Motility 2006 27 5-7 387 398 (Pubitemid 44325617)
-
(2006)
Journal of Muscle Research and Cell Motility
, vol.27
, Issue.5-7
, pp. 387-398
-
-
Wells, D.J.1
-
20
-
-
32844460899
-
Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligunucleotide
-
DOI 10.1002/jgm.838
-
Fletcher S., Honeyman K., Fall A. M., Harding P. L., Johnsen R. D., Wilton S. D., Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligunucleotide Journal of Gene Medicine 2006 8 2 207 216 (Pubitemid 43250701)
-
(2006)
Journal of Gene Medicine
, vol.8
, Issue.2
, pp. 207-216
-
-
Fletcher, S.1
Honeyman, K.2
Fall, A.M.3
Harding, P.L.4
Johnsen, R.D.5
Wilton, S.D.6
-
21
-
-
50549093417
-
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice
-
Jearawiriyapaisarn N., Moulton H. M., Buckley B., Roberts J., Sazani P., Fucharoen S., Iversen P. L., Kole R., Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice Molecular Therapy 2008 16 9 1624 1629
-
(2008)
Molecular Therapy
, vol.16
, Issue.9
, pp. 1624-1629
-
-
Jearawiriyapaisarn, N.1
Moulton, H.M.2
Buckley, B.3
Roberts, J.4
Sazani, P.5
Fucharoen, S.6
Iversen, P.L.7
Kole, R.8
-
22
-
-
10044240371
-
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
-
DOI 10.1126/science.1104297
-
Goyenvalle A., Vulin A., Fougerousse F., Leturcq F., Kaplan J. C., Garcia L., Danos O., Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping Science 2004 306 5702 1796 1799 (Pubitemid 39601401)
-
(2004)
Science
, vol.306
, Issue.5702
, pp. 1796-1799
-
-
Goyenvalle, A.1
Vulin, A.2
Fougerousse, F.3
Leturcq, F.4
Kaplan, J.-C.5
Garcia, L.6
Danos, O.7
-
23
-
-
4043092073
-
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
-
DOI 10.1038/nm1085
-
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 Nature Medicine 2004 10 8 828 834 (Pubitemid 39070856)
-
(2004)
Nature Medicine
, vol.10
, Issue.8
, pp. 828-834
-
-
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
-
24
-
-
19644378907
-
Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart
-
DOI 10.1038/nbt1073
-
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 Nature Biotechnology 2005 23 3 321 328 (Pubitemid 41094420)
-
(2005)
Nature Biotechnology
, vol.23
, Issue.3
, 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
-
25
-
-
57749169283
-
Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat
-
Bish L. T., Morine K., Sleeper M. M., Sanmiguel J., Wu DI., Gao G., Wilson J. M., Sweeney H. L., Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat Human Gene Therapy 2008 19 12 1359 1368
-
(2008)
Human Gene Therapy
, vol.19
, Issue.12
, pp. 1359-1368
-
-
Bish, L.T.1
Morine, K.2
Sleeper, M.M.3
Sanmiguel, J.4
Wu, D.I.5
Gao, G.6
Wilson, J.M.7
Sweeney, H.L.8
-
26
-
-
9844224480
-
Dp260 disrupted mice revealed prolonged implicit time of the b-wave in ERG and loss of accumulation of β-dystroglycan in the outer plexiform layer of the retina
-
Kameya S., Araki E., Katsuki M., Mizota A., Adachi E., Nakahara K., Nonaka I., Sakuragi S., Takeda S., Nabeshima YO. I., Dp260 disrupted mice revealed prolonged implicit time of the b-wave in ERG and loss of accumulation of -dystroglycan in the outer plexiform layer of the retina Human Molecular Genetics 1997 6 13 2195 2203 (Pubitemid 27501069)
-
(1997)
Human Molecular Genetics
, vol.6
, Issue.13
, pp. 2195-2203
-
-
Kameya, S.1
Araki, E.2
Katsuki, M.3
Mizota, A.4
Adachi, E.5
Nakahara, K.6
Nonaka, I.7
Sakuragi, S.8
Takeda, S.9
Nabeshima, Y.-I.10
-
27
-
-
61649097962
-
Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
-
Aartsma-Rus A., Fokkema I., Verschuuren J., Ginjaar I., Van Deutekom J., Van Ommen G. J., Den Dunnen J. T., Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations Human Mutation 2009 30 3 293 299
-
(2009)
Human Mutation
, vol.30
, Issue.3
, pp. 293-299
-
-
Aartsma-Rus, A.1
Fokkema, I.2
Verschuuren, J.3
Ginjaar, I.4
Van Deutekom, J.5
Van Ommen, G.J.6
Den Dunnen, J.T.7
-
28
-
-
69949107887
-
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: A single-blind, placebo-controlled, dose-escalation, proof-of-concept study
-
Kinali M., Arechavala-Gomeza V., Feng L., Cirak S., Hunt D., Adkin C., Guglieri M., Ashton E., Abbs S., Nihoyannopoulos P., Garralda M. E., Rutherford M., Mcculley C., Popplewell L., Graham I. R., Dickson G., Wood M. J., Wells D. J., Wilton S. D., Kole R., Straub V., Bushby K., Sewry C., Morgan J. E., Muntoni F., Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study The Lancet Neurology 2009 8 10 918 928
-
(2009)
The Lancet Neurology
, vol.8
, Issue.10
, pp. 918-928
-
-
Kinali, M.1
Arechavala-Gomeza, V.2
Feng, L.3
Cirak, S.4
Hunt, D.5
Adkin, C.6
Guglieri, M.7
Ashton, E.8
Abbs, S.9
Nihoyannopoulos, P.10
Garralda, M.E.11
Rutherford, M.12
McCulley, C.13
Popplewell, L.14
Graham, I.R.15
Dickson, G.16
Wood, M.J.17
Wells, D.J.18
Wilton, S.D.19
Kole, R.20
Straub, V.21
Bushby, K.22
Sewry, C.23
Morgan, J.E.24
Muntoni, F.25
more..
-
29
-
-
77950955983
-
Becker muscular dystrophy patients with deletions around exon 51; A promising outlook for exon skipping therapy in Duchenne patients
-
Helderman-van den Enden A. T. J. M., Straathof C. S. M., Aartsma-Rus A., den Dunnen J. T., Verbist B. M., Bakker E., Verschuuren J. J. G. M., Ginjaar H. B., Becker muscular dystrophy patients with deletions around exon 51; a promising outlook for exon skipping therapy in Duchenne patients Neuromuscular Disorders 2010 20 4 251 254
-
(2010)
Neuromuscular Disorders
, vol.20
, Issue.4
, pp. 251-254
-
-
Helderman-Van Den Enden, A.T.J.M.1
Straathof, C.S.M.2
Aartsma-Rus, A.3
Den Dunnen, J.T.4
Verbist, B.M.5
Bakker, E.6
Verschuuren, J.J.G.M.7
Ginjaar, H.B.8
-
30
-
-
37549034298
-
Local dystrophin restoration with antisense oligonucleotide PRO051
-
van Deutekom J. C., Janson A. A., Ginjaar I. B., Frankhuizen W. S., Aartsma-Rus A., Bremmer-Bout M., Den Dunnen J. T., Koop K., Van Der Kooi A. J., Goemans N. M., De Kimpe S. J., Ekhart P. F., Venneker E. H., Platenburg G. J., Verschuuren J. J., Van Ommen G. J. B., Local dystrophin restoration with antisense oligonucleotide PRO051 The New England Journal of Medicine 2007 357 26 2677 2686
-
(2007)
The New England Journal of Medicine
, vol.357
, Issue.26
, pp. 2677-2686
-
-
Van Deutekom, J.C.1
Janson, A.A.2
Ginjaar, I.B.3
Frankhuizen, W.S.4
Aartsma-Rus, A.5
Bremmer-Bout, M.6
Den Dunnen, J.T.7
Koop, K.8
Van Der Kooi, A.J.9
Goemans, N.M.10
De Kimpe, S.J.11
Ekhart, P.F.12
Venneker, E.H.13
Platenburg, G.J.14
Verschuuren, J.J.15
Van Ommen, G.J.B.16
-
31
-
-
19344369694
-
128th ENMC International Workshop on 'Preclinical optimization and Phase I/II Clinical Trials Using Antisense Oligonucleotides in Duchenne Muscular Dystrophy' 22-24 October 2004, Naarden, The Netherlands
-
DOI 10.1016/j.nmd.2005.02.007, PII S0960896605000659
-
Muntoni F., Bushby K., Van Ommen G., 128th ENMC International Workshop on 'Preclinical optimization and phase I/II clinical trials using antisense oligonucleotides in Duchenne muscular dystrophy' 22-24 October 2004, Naarden, The Netherlands Neuromuscular Disorders 2005 15 6 450 457 (Pubitemid 40720023)
-
(2005)
Neuromuscular Disorders
, vol.15
, Issue.6
, pp. 450-457
-
-
Muntoni, F.1
Bushby, K.2
Van Ommen, G.3
-
32
-
-
78049472917
-
In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse
-
Aoki Y., Nakamura A., Yokota T., Saito T., Okazawa H., Nagata T., Takeda S., In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse Molecular Therapy 2010 18 11 1995 2005
-
(2010)
Molecular Therapy
, vol.18
, Issue.11
, pp. 1995-2005
-
-
Aoki, Y.1
Nakamura, A.2
Yokota, T.3
Saito, T.4
Okazawa, H.5
Nagata, T.6
Takeda, S.7
-
33
-
-
0026538578
-
An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy
-
Sharp N. J. H., Kornegay J. N., van Camp S. D., Herbstreith M. H., Secore S. L., Kettle S., Hung W. Y., Constantinou C. D., Dykstra M. J., Roses A. D., Bartlett R. J., An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy Genomics 1992 13 1 115 121
-
(1992)
Genomics
, vol.13
, Issue.1
, pp. 115-121
-
-
Sharp, N.J.H.1
Kornegay, J.N.2
Van Camp, S.D.3
Herbstreith, M.H.4
Secore, S.L.5
Kettle, S.6
Hung, W.Y.7
Constantinou, C.D.8
Dykstra, M.J.9
Roses, A.D.10
Bartlett, R.J.11
-
34
-
-
0005952293
-
Molecular characterization of severe Duchenne-type muscular dystrophy in a family of Rottwiler dogs
-
Tucson, Ariz, USA Muscular Dystrophy Association
-
Winand N., Pradham D., Cooper B., Molecular characterization of severe Duchenne-type muscular dystrophy in a family of Rottwiler dogs Molecular Mechanism of Neuromuscular Disease 1994 Tucson, Ariz, USA Muscular Dystrophy Association
-
(1994)
Molecular Mechanism of Neuromuscular Disease
-
-
Winand, N.1
Pradham, D.2
Cooper, B.3
-
35
-
-
0032966013
-
Molecular analysis of a spontaneous dystrophin 'knockout' dog
-
DOI 10.1016/S0960-8966(99)00011-5, PII S0960896699000115
-
Schatzberg S. J., Olby N. J., Breen M., Anderson L. V. B., Langford C. F., Dickens H. F., Wilton S. D., Zeiss C. J., Binns M. M., Kornegay J. N., Morris G. E., Sharp N. J. H., Molecular analysis of a spontaneous dystrophin 'knockout' dog Neuromuscular Disorders 1999 9 5 289 295 (Pubitemid 29301708)
-
(1999)
Neuromuscular Disorders
, vol.9
, Issue.5
, pp. 289-295
-
-
Schatzberg, S.J.1
Olby, N.J.2
Breen, M.3
Anderson, L.V.B.4
Langford, C.F.5
Dickens, H.F.6
Wilton, S.D.7
Zeiss, C.J.8
Binns, M.M.9
Kornegay, J.N.10
Morris, G.E.11
Sharp, N.J.H.12
-
36
-
-
77649263080
-
A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping
-
Walmsley G. L., Arechavala-Gomeza V., Fernandez-Fuente M., Burke M. M., Nagel N., Holder A., Stanley R., Chandler K., Marks S. L., Muntoni F., Shelton G. D., Piercy R. J., A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping PloS one 2010 5 1
-
(2010)
PloS One
, vol.5
, Issue.1
-
-
Walmsley, G.L.1
Arechavala-Gomeza, V.2
Fernandez-Fuente, M.3
Burke, M.M.4
Nagel, N.5
Holder, A.6
Stanley, R.7
Chandler, K.8
Marks, S.L.9
Muntoni, F.10
Shelton, G.D.11
Piercy, R.J.12
-
37
-
-
0023883621
-
The homologue of the Duchenne locus is defective in X-linked muscular dystrophy of dogs
-
Cooper B. J., Winand N. J., Stedman H., Valentine B. A., Hoffman E. P., Kunkel L. M., Scot M. O., Fischbeck K. H., Kornegay J. N., Avery R. J., Williams J. R., Schmickel R. D., Sylvester J. E., The homologue of the Duchenne locus is defective in X-linked muscular dystrophy of dogs Nature 1988 334 6178 154 156
-
(1988)
Nature
, vol.334
, Issue.6178
, pp. 154-156
-
-
Cooper, B.J.1
Winand, N.J.2
Stedman, H.3
Valentine, B.A.4
Hoffman, E.P.5
Kunkel, L.M.6
Scot, M.O.7
Fischbeck, K.H.8
Kornegay, J.N.9
Avery, R.J.10
Williams, J.R.11
Schmickel, R.D.12
Sylvester, J.E.13
-
38
-
-
0024263367
-
Canine X-linked muscular dystrophy. An animal model of Duchenne muscular dystrophy: Clinical studies
-
DOI 10.1016/0022-510X(88)90206-7
-
Valentine B. A., Cooper B. J., De Lahunta A., O'Quinn R., Blue J. T., Canine X-linked muscular dystrophy. An animal model of Duchenne muscular dystrophy: clinical studies Journal of the Neurological Sciences 1988 88 1-3 69 81 (Pubitemid 19016584)
-
(1988)
Journal of the Neurological Sciences
, vol.88
, Issue.1-3
, pp. 69-81
-
-
Valentine, B.A.1
Cooper, B.J.2
De Lahunta, A.3
O'Quinn, R.4
Blue, J.T.5
-
39
-
-
0036485777
-
Muscle lesions associated with dystrophin deficiency in neonatal golden retriever puppies
-
Nguyen F., Cherel Y., Guigand L., Goubault-Leroux I., Wyers M., Muscle lesions associated with dystrophin deficiency in neonatal golden retriever puppies Journal of Comparative Pathology 2002 126 2-3 100 108
-
(2002)
Journal of Comparative Pathology
, vol.126
, Issue.23
, pp. 100-108
-
-
Nguyen, F.1
Cherel, Y.2
Guigand, L.3
Goubault-Leroux, I.4
Wyers, M.5
-
40
-
-
0025755512
-
Duchenne's cardiomyopathy in a canine model: Electrocardiographic and echocardiographic studies
-
Moise N. S., Valentine B. A., Brown C. A., Erb H. N., Beck K. A., Cooper B. J., Gilmour R. F., Duchenne's cardiomyopathy in a canine model: electrocardiographic and echocardiographic studies Journal of the American College of Cardiology 1991 17 3 812 820
-
(1991)
Journal of the American College of Cardiology
, vol.17
, Issue.3
, pp. 812-820
-
-
Moise, N.S.1
Valentine, B.A.2
Brown, C.A.3
Erb, H.N.4
Beck, K.A.5
Cooper, B.J.6
Gilmour, R.F.7
-
41
-
-
0014056078
-
The distinctive electrocardiogram of Duchenne's progressive muscular dystrophy. An electrocardiographic-pathologic correlative study
-
Perloff J. K., Roberts W. C., de Leon A. C. Jr., O'Doherty D., The distinctive electrocardiogram of Duchenne's progressive muscular dystrophy. An electrocardiographic-pathologic correlative study The American Journal of Medicine 1967 42 2 179 188
-
(1967)
The American Journal of Medicine
, vol.42
, Issue.2
, pp. 179-188
-
-
Perloff, J.K.1
Roberts, W.C.2
De Leon Jr., A.C.3
O'Doherty, D.4
-
42
-
-
0030870110
-
Use of the dog model for Duchenne muscular dystrophy in gene therapy trials
-
DOI 10.1016/S0960-8966(97)00057-6, PII S0960896697000576
-
Howell J. M., 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 Disorders 1997 7 5 325 328 (Pubitemid 27375837)
-
(1997)
Neuromuscular Disorders
, vol.7
, Issue.5
, pp. 325-328
-
-
Howell, J.McC.1
Fletcher, S.2
Kakulas, B.A.3
O'Hara, M.4
Lochmuller, H.5
Karpati, G.6
-
43
-
-
0032549685
-
High-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscle of dystrophic dogs: Prolongation of expression with immunosuppression
-
Howell J. M., Lochmller H., O'Hara A., Fletcher S., Kakulas B. A., Massie B., Nalbantoglu J., Karpati G., High-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscle of dystrophic dogs: prolongation of expression with immunosuppression Human Gene Therapy 1998 9 5 629 634 (Pubitemid 28200619)
-
(1998)
Human Gene Therapy
, vol.9
, Issue.5
, pp. 629-634
-
-
Howell, J.M.1
Lochmuller, H.2
O'Hara, A.3
Fletcher, S.4
Kakulas, B.A.5
Massie, B.6
Nalbantoglu, J.7
Karpati, G.8
-
44
-
-
77955177089
-
Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs
-
Kornegay J. N., Li J., Bogan J. R., Bogan D. J., Chen C., Zheng H., Wang B., Qiao C., Howard J. F., Xiao X., Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs Molecular Therapy 2010 19 8 1501 1508
-
(2010)
Molecular Therapy
, vol.19
, Issue.8
, pp. 1501-1508
-
-
Kornegay, J.N.1
Li, J.2
Bogan, J.R.3
Bogan, D.J.4
Chen, C.5
Zheng, H.6
Wang, B.7
Qiao, C.8
Howard, J.F.9
Xiao, X.10
-
45
-
-
0034086776
-
In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide
-
DOI 10.1038/76448
-
Bartlett R. J., Stockinger S., Denis M. M., Bartlett W. T., Inverardi L., Le T. T., Man N. T., 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 Nature Biotechnology 2000 18 6 615 622 (Pubitemid 30415294)
-
(2000)
Nature Biotechnology
, vol.18
, Issue.6
, 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
Man, N.T.7
Morris, G.E.8
Bogan, D.J.9
Metcalf-Bogan, J.10
Kornegay, J.N.11
-
46
-
-
33745479703
-
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
-
DOI 10.1038/sj.gt.3302800, PII 3302800
-
McClorey G., Moulton H. M., Iversen P. L., Fletcher S., Wilton S. D., Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD Gene Therapy 2006 13 19 1373 1381 (Pubitemid 44420632)
-
(2006)
Gene Therapy
, vol.13
, Issue.19
, pp. 1373-1381
-
-
McClorey, G.1
Moulton, H.M.2
Iversen, P.L.3
Fletcher, S.4
Wilton, S.D.5
-
47
-
-
0038117843
-
J)
-
DOI 10.1538/expanim.52.93
-
Shimatsu Y., Katagiri K., Furuta T., Nakura M., Tanioka Y., Yuasa K., Tomohiro M., Kornegay J. N., Nonaka I., Takeda S., Canine X-linked muscular dystrophy in Japan (CXMD) Experimental Animals 2003 52 2 93 97 (Pubitemid 41349972)
-
(2003)
Experimental Animals
, vol.52
, Issue.2
, pp. 93-97
-
-
Shimatsu, Y.1
Katagiri, K.2
Furuta, T.3
Nakura, M.4
Tanioka, Y.5
Yuasa, K.6
Tomohiro, M.7
Kornegay, J.N.8
Nonaka, I.9
Takeda, S.10
-
48
-
-
33344470361
-
J
-
Shimatsu Y., Yoshimura M., Yuasa K., Urasawa N., Tomohiro M., Nakura M., Tanigawa M., Nakamura A., Takeda S., Major clinical and histopathological characteristics of canine X-linked muscular dystrophy in Japan, CXMD Acta Myologica 2005 24 2 145 154 (Pubitemid 43287352)
-
(2005)
Acta Myologica
, vol.24
, Issue.2
, pp. 145-154
-
-
Shimatsu, Y.1
Yoshimura, M.2
Yuasa, K.3
Urasawa, N.4
Tomohiro, M.5
Nakura, M.6
Tanigawa, M.7
Nakamura, A.8
Takeda, S.9
-
49
-
-
33845675071
-
Cardiac involvement in Beagle-based canine X-linked muscular dystrophy in Japan (CXMD): Electrocardiographic, echocardiographic, and morphologic studies
-
Yugeta N., Urasawa N., Fujii Y., Yoshimura M., Yuasa K., Wada M. R., Nakura M., Shimatsu Y., Tomohiro M., Takahashi A., Machida N., Wakao Y., Nakamura A., Takeda S., Cardiac involvement in Beagle-based canine X-linked muscular dystrophy in Japan (CXMD): electrocardiographic, echocardiographic, and morphologic studies BMC Cardiovascular Disorders 2006 6, article 47
-
(2006)
BMC Cardiovascular Disorders
, vol.647
-
-
Yugeta, N.1
Urasawa, N.2
Fujii, Y.3
Yoshimura, M.4
Yuasa, K.5
Wada, M.R.6
Nakura, M.7
Shimatsu, Y.8
Tomohiro, M.9
Takahashi, A.10
MacHida, N.11
Wakao, Y.12
Nakamura, A.13
Takeda, S.14
-
50
-
-
43449114823
-
Selective vacuolar degeneration in dystrophin-deficient canine Purkinje fibers despite preservation of dystrophin-associated proteins with overexpression of Dp71
-
DOI 10.1161/CIRCULATIONAHA.107.739326, PII 0000301720080513000006
-
Urasawa N., Wada M. R., Machida N., Yuasa K., Shimatsu Y., Wakao Y., Yuasa S., Sano T., Nonaka I., Nakamura A., Takeda S., Selective vacuolar degeneration in dystrophin-deficient canine Purkinje fibers despite preservation of dystrophin-associated proteins with overexpression of Dp71 Circulation 2008 117 19 2437 2448 (Pubitemid 351670450)
-
(2008)
Circulation
, vol.117
, Issue.19
, pp. 2437-2448
-
-
Urasawa, N.1
Wada, M.R.2
Machida, N.3
Yuasa, K.4
Shimatsu, Y.5
Wakao, Y.6
Yuasa, S.7
Sano, T.8
Nonaka, I.9
Nakamura, A.10
Takeda, S.11
-
51
-
-
63449141811
-
Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs
-
Yokota T., Lu Q. L., Partridge T., Kobayashi M., Nakamura A., Takeda S., Hoffman E., Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs Annals of Neurology 2009 65 6 667 676
-
(2009)
Annals of Neurology
, vol.65
, Issue.6
, pp. 667-676
-
-
Yokota, T.1
Lu, Q.L.2
Partridge, T.3
Kobayashi, M.4
Nakamura, A.5
Takeda, S.6
Hoffman, E.7
-
52
-
-
77957913755
-
Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient
-
Saito T., Nakamura A., Aoki Y., Yokota T., Okada T., Osawa M., Takeda S., Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient PLoS One 2010 5 8
-
(2010)
PLoS One
, vol.5
, Issue.8
-
-
Saito, T.1
Nakamura, A.2
Aoki, Y.3
Yokota, T.4
Okada, T.5
Osawa, M.6
Takeda, S.7
-
53
-
-
34548138958
-
Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products
-
DOI 10.1038/sj.gt.3302984, PII 3302984
-
Yuasa K., Yoshimura M., Urasawa N., Ohshima S., Howell J. M., Nakamura A., Hijikata T., Miyagoe-Suzuki Y., Takeda S., Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products Gene Therapy 2007 14 17 1249 1260 (Pubitemid 47299950)
-
(2007)
Gene Therapy
, vol.14
, Issue.17
, pp. 1249-1260
-
-
Yuasa, K.1
Yoshimura, M.2
Urasawa, N.3
Ohshima, S.4
Howell, J.M.5
Nakamura, A.6
Hijikata, T.7
Miyagoe-Suzuki, Y.8
Takeda, S.9
-
54
-
-
58149259952
-
Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle
-
Ohshima S., Shin J. H., Yuasa K., Nishiyama A., Kira J., Okada T., Takeda S., Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle Molecular Therapy 2009 17 1 73 80
-
(2009)
Molecular Therapy
, vol.17
, Issue.1
, pp. 73-80
-
-
Ohshima, S.1
Shin, J.H.2
Yuasa, K.3
Nishiyama, A.4
Kira, J.5
Okada, T.6
Takeda, S.7
-
55
-
-
32244443828
-
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
-
DOI 10.1038/nm1345, PII NM1345
-
Alter J., Lou F., Rabinowitz A., Yin H., Rosenfeld J., Wilton S. D., Partridge T. A., Qi L. L., Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology Nature Medicine 2006 12 2 175 177 (Pubitemid 43214742)
-
(2006)
Nature Medicine
, vol.12
, Issue.2
, pp. 175-177
-
-
Alter, J.1
Lou, F.2
Rabinowitz, A.3
Yin, H.4
Rosenfeld, J.5
Wilton, S.D.6
Partridge, T.A.7
Qi, L.L.8
-
56
-
-
0347003516
-
Antisense-Induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More Sense
-
DOI 10.1086/381039
-
Aartsma-Rus A., Janson A. A. M., Kaman W. E., Bremmer-Bout M., Van Ommen G. J. B., Den Dunnen J. T., Van Deutekom J. C. T., Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense American Journal of Human Genetics 2004 74 1 83 92 (Pubitemid 38085240)
-
(2004)
American Journal of Human Genetics
, vol.74
, Issue.1
, pp. 83-92
-
-
Aartsma-Rus, A.1
Janson, A.A.M.2
Kaman, W.E.3
Bremmer-Bout, M.4
Van Ommen, G.-J.B.5
Den Dunnen, J.T.6
Van Deutekom, J.C.T.7
-
57
-
-
34848904544
-
Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle
-
DOI 10.1089/hum.2006.061
-
Arechavala-Gomeza V., Graham I. R., Popplewell L. J., Adams A. M., Aartsma-Rus A., Kinali M., Morgan J. E., Van Deutekom J. C., Wilton S. D., Dickson G., Muntoni F., Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle Human Gene Therapy 2007 18 9 798 810 (Pubitemid 47502846)
-
(2007)
Human Gene Therapy
, vol.18
, Issue.9
, pp. 798-810
-
-
Arechavala-Gomeza, V.1
Graham, I.R.2
Popplewell, L.J.3
Adams, A.M.4
Aartsma-Rus, A.5
Kinali, M.6
Morgan, J.E.7
Van Deutekom, J.C.8
Wilton, S.D.9
Dickson, G.10
Muntoni, F.11
-
58
-
-
67651243772
-
Exon-skipping therapy for Duchenne muscular dystrophy
-
Nakamura A., Takeda S., Exon-skipping therapy for Duchenne muscular dystrophy Neuropathology 2009 29 4 494 501
-
(2009)
Neuropathology
, vol.29
, Issue.4
, pp. 494-501
-
-
Nakamura, A.1
Takeda, S.2
-
60
-
-
0024346882
-
Feline muscular dystrophy with dystrophin deficiency
-
Carpenter J. L., Hoffmann E. P., Romanul F. C. A., Kunkel L. M., Rosales R. K., Ma N. S. F., Dasbach J. J., Rae J. F., Moore F. M., McAfee M. B., Pearce L. K., Feline muscular dystrophy with dystrophin deficiency The American Journal of Pathology 1989 135 5 909 919 (Pubitemid 20009501)
-
(1989)
American Journal of Pathology
, vol.135
, Issue.5
, pp. 909-919
-
-
Carpenter, J.L.1
Hoffmann, E.P.2
Romanul, F.C.A.3
Kunkel, L.M.4
Rosales, R.K.5
Ma, N.S.F.6
Dasbach, J.J.7
Rae, J.F.8
Moore, F.M.9
McAfee, M.B.10
Pearce, L.K.11
-
61
-
-
0026734511
-
Dystrophin deficiency causes lethal muscle hypertrophy in cats
-
Gaschen F. P., Hoffman E. P., Gorospe J. R. M., Uhl E. W., Senior D. R., Cardinet G. H., Pearce L. K., Dystrophin deficiency causes lethal muscle hypertrophy in cats Journal of the Neurological Sciences 1992 110 1-2 149 159
-
(1992)
Journal of the Neurological Sciences
, vol.110
, Issue.12
, pp. 149-159
-
-
Gaschen, F.P.1
Hoffman, E.P.2
Gorospe, J.R.M.3
Uhl, E.W.4
Senior, D.R.5
Cardinet, G.H.6
Pearce, L.K.7
-
62
-
-
0027940280
-
Deletion of the dystrophin muscle promoter in feline muscular dystrophy
-
DOI 10.1016/0960-8966(94)90082-5
-
Winand N. J., Edwards M., Pradhan D., Berian C. A., Cooper B. J., Deletion of the dystrophin muscle promoter in feline muscular dystrophy Neuromuscular Disorders 1994 4 5-6 433 445 (Pubitemid 24331530
-
(1994)
Neuromuscular Disorders
, vol.4
, Issue.5-6
, pp. 433-445
-
-
Winand, N.J.1
Edwards, M.2
Pradhan, D.3
Berian, C.A.4
Cooper, B.J.5
|