-
1
-
-
28544447912
-
The muscular dystrophies: from genes to therapies
-
Lovering RM, Porter NC, Bloch RJ, (2005) The muscular dystrophies: from genes to therapies. Phys Ther 85: 1372-1388.
-
(2005)
Phys Ther
, vol.85
, pp. 1372-1388
-
-
Lovering, R.M.1
Porter, N.C.2
Bloch, R.J.3
-
2
-
-
0036331154
-
Duchenne muscular dystrophy: current knowledge, treatment, and future prospects
-
Biggar WD, Klamut HJ, Demacio PC, Stevens DJ, Ray PN (2002) Duchenne muscular dystrophy: current knowledge, treatment, and future prospects. Clin Orthop Relat Res: 88-106.
-
(2002)
Clin Orthop Relat Res
, pp. 88-106
-
-
Biggar, W.D.1
Klamut, H.J.2
Demacio, P.C.3
Stevens, D.J.4
Ray, P.N.5
-
3
-
-
80052668013
-
The pathology of paralysis with muscular degeneration (paralysie myosclerotique), or paralysis with apparent hypertrophy
-
Duchenne
-
Duchenne (1867) The pathology of paralysis with muscular degeneration (paralysie myosclerotique), or paralysis with apparent hypertrophy. Br Med J 2: 541-542.
-
(1867)
Br Med J
, vol.2
, pp. 541-542
-
-
-
4
-
-
33947377423
-
Muscle diseases: the muscular dystrophies
-
McNally EM, Pytel P, (2007) Muscle diseases: the muscular dystrophies. Annu Rev Pathol 2: 87-109.
-
(2007)
Annu Rev Pathol
, vol.2
, pp. 87-109
-
-
McNally, E.M.1
Pytel, P.2
-
5
-
-
0023718360
-
Recent advances in Duchenne and Becker muscular dystrophy
-
Hyser CL, Mendell JR, (1988) Recent advances in Duchenne and Becker muscular dystrophy. Neurol Clin 6: 429-453.
-
(1988)
Neurol Clin
, vol.6
, pp. 429-453
-
-
Hyser, C.L.1
Mendell, J.R.2
-
6
-
-
78650916689
-
The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy
-
Lu QL, Yokota T, Takeda S, Garcia L, Muntoni F, et al. (2011) The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy. Mol Ther 19: 9-15.
-
(2011)
Mol Ther
, vol.19
, pp. 9-15
-
-
Lu, Q.L.1
Yokota, T.2
Takeda, S.3
Garcia, L.4
Muntoni, F.5
-
7
-
-
79955618736
-
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD
-
Pichavant C, Aartsma-Rus A, Clemens PR, Davies KE, Dickson G, et al. (2011) Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther 19: 830-840.
-
(2011)
Mol Ther
, vol.19
, pp. 830-840
-
-
Pichavant, C.1
Aartsma-Rus, A.2
Clemens, P.R.3
Davies, K.E.4
Dickson, G.5
-
8
-
-
80052513011
-
Restoring dystrophin expression in Duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through
-
Hoffman EP, Bronson A, Levin AA, Takeda S, Yokota T, et al. (2011) Restoring dystrophin expression in Duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through. Am J Pathol 179: 12-22.
-
(2011)
Am J Pathol
, vol.179
, pp. 12-22
-
-
Hoffman, E.P.1
Bronson, A.2
Levin, A.A.3
Takeda, S.4
Yokota, T.5
-
9
-
-
0023614271
-
Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals
-
Koenig M, Hoffman EP, Bertelson CJ, Monaco AP, Feener C, et al. (1987) Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals. Cell 50: 509-517.
-
(1987)
Cell
, vol.50
, pp. 509-517
-
-
Koenig, M.1
Hoffman, E.P.2
Bertelson, C.J.3
Monaco, A.P.4
Feener, C.5
-
10
-
-
0023614188
-
Dystrophin: The protein product of the Duchenne muscular dystrophy locus
-
Hoffman EP Brown RH Jr., Kunkel LM (1987) Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 51: 919-928.
-
(1987)
Cell
, vol.51
, pp. 919-928
-
-
Hoffman, E.P.1
Brown Jr., R.H.2
Kunkel, L.M.3
-
11
-
-
0030025402
-
Expression and regulation of the dystrophin Purkinje promoter in human skeletal muscle, heart, and brain
-
Holder E, Maeda M, Bies RD, (1996) Expression and regulation of the dystrophin Purkinje promoter in human skeletal muscle, heart, and brain. Hum Genet 97: 232-239.
-
(1996)
Hum Genet
, vol.97
, pp. 232-239
-
-
Holder, E.1
Maeda, M.2
Bies, R.D.3
-
12
-
-
0024582731
-
Alternative splicing of human dystrophin mRNA generates isoforms at the carboxy terminus
-
Feener CA, Koenig M, Kunkel LM, (1989) Alternative splicing of human dystrophin mRNA generates isoforms at the carboxy terminus. Nature 338: 509-511.
-
(1989)
Nature
, vol.338
, pp. 509-511
-
-
Feener, C.A.1
Koenig, M.2
Kunkel, L.M.3
-
13
-
-
0029061752
-
A novel dystrophin isoform is required for normal retinal electrophysiology
-
D'Souza VN, Nguyen TM, Morris GE, Karges W, Pillers DA, et al. (1995) A novel dystrophin isoform is required for normal retinal electrophysiology. Hum Mol Genet 4: 837-842.
-
(1995)
Hum Mol Genet
, vol.4
, pp. 837-842
-
-
D'Souza, V.N.1
Nguyen, T.M.2
Morris, G.E.3
Karges, W.4
Pillers, D.A.5
-
14
-
-
0027936780
-
Identification of a novel first exon in the human dystrophin gene and of a new promoter located more than 500 kb upstream of the nearest known promoter
-
Nishio H, Takeshima Y, Narita N, Yanagawa H, Suzuki Y, et al. (1994) Identification of a novel first exon in the human dystrophin gene and of a new promoter located more than 500 kb upstream of the nearest known promoter. J Clin Invest 94: 1037-1042.
-
(1994)
J Clin Invest
, vol.94
, pp. 1037-1042
-
-
Nishio, H.1
Takeshima, Y.2
Narita, N.3
Yanagawa, H.4
Suzuki, Y.5
-
15
-
-
0036087342
-
Function and genetics of dystrophin and dystrophin-related proteins in muscle
-
Blake DJ, Weir A, Newey SE, Davies KE, (2002) Function and genetics of dystrophin and dystrophin-related proteins in muscle. Physiol Rev 82: 291-329.
-
(2002)
Physiol Rev
, vol.82
, pp. 291-329
-
-
Blake, D.J.1
Weir, A.2
Newey, S.E.3
Davies, K.E.4
-
16
-
-
0027290417
-
Identification of a novel T-insertion polymorphism at the DMD locus
-
Muntoni F, Cau M, Congiu R, Congia M, Cao A, et al. (1993) Identification of a novel T-insertion polymorphism at the DMD locus. Hum Genet 92: 103.
-
(1993)
Hum Genet
, vol.92
, pp. 103
-
-
Muntoni, F.1
Cau, M.2
Congiu, R.3
Congia, M.4
Cao, A.5
-
17
-
-
0027265702
-
Brief report: deletion of the dystrophin muscle-promoter region associated with X-linked dilated cardiomyopathy
-
Muntoni F, Cau M, Ganau A, Congiu R, Arvedi G, et al. (1993) Brief report: deletion of the dystrophin muscle-promoter region associated with X-linked dilated cardiomyopathy. N Engl J Med 329: 921-925.
-
(1993)
N Engl J Med
, vol.329
, pp. 921-925
-
-
Muntoni, F.1
Cau, M.2
Ganau, A.3
Congiu, R.4
Arvedi, G.5
-
18
-
-
0037211541
-
The dystrophin lymphocyte promoter revisited: 4.5-megabase intron, or artifact?
-
Wheway JM, Roberts RG, (2003) The dystrophin lymphocyte promoter revisited: 4.5-megabase intron, or artifact? Neuromuscul Disord 13: 17-20.
-
(2003)
Neuromuscul Disord
, vol.13
, pp. 17-20
-
-
Wheway, J.M.1
Roberts, R.G.2
-
19
-
-
0024595610
-
Duchenne muscular dystrophy gene product is not identical in muscle and brain
-
Nudel U, Zuk D, Einat P, Zeelon E, Levy Z, et al. (1989) Duchenne muscular dystrophy gene product is not identical in muscle and brain. Nature 337: 76-78.
-
(1989)
Nature
, vol.337
, pp. 76-78
-
-
Nudel, U.1
Zuk, D.2
Einat, P.3
Zeelon, E.4
Levy, Z.5
-
20
-
-
0022496289
-
Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene
-
Monaco AP, Neve RL, Colletti-Feener C, Bertelson CJ, Kurnit DM, et al. (1986) Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene. Nature 323: 646-650.
-
(1986)
Nature
, vol.323
, pp. 646-650
-
-
Monaco, A.P.1
Neve, R.L.2
Colletti-Feener, C.3
Bertelson, C.J.4
Kurnit, D.M.5
-
21
-
-
0026937998
-
Expression of four alternative dystrophin transcripts in brain regions regulated by different promoters
-
Gorecki DC, Monaco AP, Derry JM, Walker AP, Barnard EA, et al. (1992) Expression of four alternative dystrophin transcripts in brain regions regulated by different promoters. Hum Mol Genet 1: 505-510.
-
(1992)
Hum Mol Genet
, vol.1
, pp. 505-510
-
-
Gorecki, D.C.1
Monaco, A.P.2
Derry, J.M.3
Walker, A.P.4
Barnard, E.A.5
-
22
-
-
0028937525
-
Dp140: a novel 140 kDa CNS transcript from the dystrophin locus
-
Lidov HG, Selig S, Kunkel LM, (1995) Dp140: a novel 140 kDa CNS transcript from the dystrophin locus. Hum Mol Genet 4: 329-335.
-
(1995)
Hum Mol Genet
, vol.4
, pp. 329-335
-
-
Lidov, H.G.1
Selig, S.2
Kunkel, L.M.3
-
23
-
-
0027214837
-
An alternative dystrophin transcript specific to peripheral nerve
-
Byers TJ, Lidov HG, Kunkel LM, (1993) An alternative dystrophin transcript specific to peripheral nerve. Nat Genet 4: 77-81.
-
(1993)
Nat Genet
, vol.4
, pp. 77-81
-
-
Byers, T.J.1
Lidov, H.G.2
Kunkel, L.M.3
-
24
-
-
0026653859
-
A 71-kilodalton protein is a major product of the Duchenne muscular dystrophy gene in brain and other nonmuscle tissues
-
Lederfein D, Levy Z, Augier N, Mornet D, Morris G, et al. (1992) A 71-kilodalton protein is a major product of the Duchenne muscular dystrophy gene in brain and other nonmuscle tissues. Proc Natl Acad Sci U S A 89: 5346-5350.
-
(1992)
Proc Natl Acad Sci U S A
, vol.89
, pp. 5346-5350
-
-
Lederfein, D.1
Levy, Z.2
Augier, N.3
Mornet, D.4
Morris, G.5
-
25
-
-
0029086696
-
Cloning and characterization of alternatively spliced isoforms of Dp71
-
Austin RC, Howard PL, D'Souza VN, Klamut HJ, Ray PN, (1995) Cloning and characterization of alternatively spliced isoforms of Dp71. Hum Mol Genet 4: 1475-1483.
-
(1995)
Hum Mol Genet
, vol.4
, pp. 1475-1483
-
-
Austin, R.C.1
Howard, P.L.2
D'Souza, V.N.3
Klamut, H.J.4
Ray, P.N.5
-
26
-
-
0027314922
-
Apo-dystrophin-3: a 2.2kb transcript from the DMD locus encoding the dystrophin glycoprotein binding site
-
Tinsley JM, Blake DJ, Davies KE, (1993) Apo-dystrophin-3: a 2.2kb transcript from the DMD locus encoding the dystrophin glycoprotein binding site. Hum Mol Genet 2: 521-524.
-
(1993)
Hum Mol Genet
, vol.2
, pp. 521-524
-
-
Tinsley, J.M.1
Blake, D.J.2
Davies, K.E.3
-
27
-
-
0034605070
-
The dystrophin complex forms a mechanically strong link between the sarcolemma and costameric actin
-
Rybakova IN, Patel JR, Ervasti JM, (2000) The dystrophin complex forms a mechanically strong link between the sarcolemma and costameric actin. J Cell Biol 150: 1209-1214.
-
(2000)
J Cell Biol
, vol.150
, pp. 1209-1214
-
-
Rybakova, I.N.1
Patel, J.R.2
Ervasti, J.M.3
-
28
-
-
0033951650
-
Plasma membrane cytoskeleton of muscle: a fine structural analysis
-
Watkins SC, Cullen MJ, Hoffman EP, Billington L, (2000) Plasma membrane cytoskeleton of muscle: a fine structural analysis. Microsc Res Tech 48: 131-141.
-
(2000)
Microsc Res Tech
, vol.48
, pp. 131-141
-
-
Watkins, S.C.1
Cullen, M.J.2
Hoffman, E.P.3
Billington, L.4
-
29
-
-
0026674204
-
An intact cysteine-rich domain is required for dystrophin function
-
Bies RD, Caskey CT, Fenwick R, (1992) An intact cysteine-rich domain is required for dystrophin function. J Clin Invest 90: 666-672.
-
(1992)
J Clin Invest
, vol.90
, pp. 666-672
-
-
Bies, R.D.1
Caskey, C.T.2
Fenwick, R.3
-
30
-
-
24644501292
-
Molecular extensibility of mini-dystrophins and a dystrophin rod construct
-
Bhasin N, Law R, Liao G, Safer D, Ellmer J, et al. (2005) Molecular extensibility of mini-dystrophins and a dystrophin rod construct. J Mol Biol 352: 795-806.
-
(2005)
J Mol Biol
, vol.352
, pp. 795-806
-
-
Bhasin, N.1
Law, R.2
Liao, G.3
Safer, D.4
Ellmer, J.5
-
31
-
-
58449122870
-
A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground
-
Yokota T, Takeda S, Lu QL, Partridge TA, Nakamura A, et al. (2009) A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground. Arch Neurol 66: 32-38.
-
(2009)
Arch Neurol
, vol.66
, pp. 32-38
-
-
Yokota, T.1
Takeda, S.2
Lu, Q.L.3
Partridge, T.A.4
Nakamura, A.5
-
32
-
-
77649263080
-
A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping
-
Walmsley GL, Arechavala-Gomeza V, Fernandez-Fuente M, Burke MM, Nagel N, et al. (2010) A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping. PLoS One 5: e8647.
-
(2010)
PLoS One
, vol.5
-
-
Walmsley, G.L.1
Arechavala-Gomeza, V.2
Fernandez-Fuente, M.3
Burke, M.M.4
Nagel, N.5
-
33
-
-
33644854652
-
Intronic breakpoint definition and transcription analysis in DMD/BMD patients with deletion/duplication at the 5' mutation hot spot of the dystrophin gene
-
Gualandi F, Rimessi P, Trabanelli C, Spitali P, Neri M, et al. (2006) Intronic breakpoint definition and transcription analysis in DMD/BMD patients with deletion/duplication at the 5' mutation hot spot of the dystrophin gene. Gene 370: 26-33.
-
(2006)
Gene
, vol.370
, pp. 26-33
-
-
Gualandi, F.1
Rimessi, P.2
Trabanelli, C.3
Spitali, P.4
Neri, M.5
-
34
-
-
0025738298
-
Genotype-phenotype correlation and germline mosaicism in DMD/BMD patients with deletions of the dystrophin gene
-
Covone AE, Lerone M, Romeo G, (1991) Genotype-phenotype correlation and germline mosaicism in DMD/BMD patients with deletions of the dystrophin gene. Hum Genet 87: 353-360.
-
(1991)
Hum Genet
, vol.87
, pp. 353-360
-
-
Covone, A.E.1
Lerone, M.2
Romeo, G.3
-
35
-
-
0345731966
-
X chromosome-linked muscular dystrophy (mdx) in the mouse
-
Bulfield G, Siller WG, Wight PA, Moore KJ, (1984) X chromosome-linked muscular dystrophy (mdx) in the mouse. Proc Natl Acad Sci U S A 81: 1189-1192.
-
(1984)
Proc Natl Acad Sci U S A
, vol.81
, pp. 1189-1192
-
-
Bulfield, G.1
Siller, W.G.2
Wight, P.A.3
Moore, K.J.4
-
36
-
-
0024353559
-
The molecular basis of muscular dystrophy in the mdx mouse: a point mutation
-
Sicinski P, Geng Y, Ryder-Cook AS, Barnard EA, Darlison MG, et al. (1989) The molecular basis of muscular dystrophy in the mdx mouse: a point mutation. Science 244: 1578-1580.
-
(1989)
Science
, vol.244
, pp. 1578-1580
-
-
Sicinski, P.1
Geng, Y.2
Ryder-Cook, A.S.3
Barnard, E.A.4
Darlison, M.G.5
-
37
-
-
0031577559
-
Targeted disruption of exon 52 in the mouse dystrophin gene induced muscle degeneration similar to that observed in Duchenne muscular dystrophy
-
Araki E, Nakamura K, Nakao K, Kameya S, Kobayashi O, et al. (1997) Targeted disruption of exon 52 in the mouse dystrophin gene induced muscle degeneration similar to that observed in Duchenne muscular dystrophy. Biochem Biophys Res Commun 238: 492-497.
-
(1997)
Biochem Biophys Res Commun
, vol.238
, pp. 492-497
-
-
Araki, E.1
Nakamura, K.2
Nakao, K.3
Kameya, S.4
Kobayashi, O.5
-
38
-
-
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, et al. (2010) In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse. Mol Ther 18: 1995-2005.
-
(2010)
Mol Ther
, vol.18
, pp. 1995-2005
-
-
Aoki, Y.1
Nakamura, A.2
Yokota, T.3
Saito, T.4
Okazawa, H.5
-
39
-
-
84865774967
-
Reprogramming efficiency and quality of induced Pluripotent Stem Cells (iPSCs) generated from muscle-derived fibroblasts of mdx mice at different ages
-
Wang B, Miyagoe-Suzuki Y, Yada E, Ito N, Nishiyama T, et al. (2011) Reprogramming efficiency and quality of induced Pluripotent Stem Cells (iPSCs) generated from muscle-derived fibroblasts of mdx mice at different ages. PLoS Curr 3: RRN1274.
-
(2011)
PLoS Curr
, vol.3
-
-
Wang, B.1
Miyagoe-Suzuki, Y.2
Yada, E.3
Ito, N.4
Nishiyama, T.5
-
40
-
-
33746899641
-
Expansion of revertant fibers in dystrophic mdx muscles reflects activity of muscle precursor cells and serves as an index of muscle regeneration
-
Yokota T, Lu QL, Morgan JE, Davies KE, Fisher R, et al. (2006) Expansion of revertant fibers in dystrophic mdx muscles reflects activity of muscle precursor cells and serves as an index of muscle regeneration. J Cell Sci 119: 2679-2687.
-
(2006)
J Cell Sci
, vol.119
, pp. 2679-2687
-
-
Yokota, T.1
Lu, Q.L.2
Morgan, J.E.3
Davies, K.E.4
Fisher, R.5
-
41
-
-
84865288612
-
Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery
-
Aoki Y, Yokota T, Nagata T, Nakamura A, Tanihata J, et al. (2012) Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery. Proc Natl Acad Sci U S A 109: 13763-13768.
-
(2012)
Proc Natl Acad Sci U S A
, vol.109
, pp. 13763-13768
-
-
Aoki, Y.1
Yokota, T.2
Nagata, T.3
Nakamura, A.4
Tanihata, J.5
-
42
-
-
84865313416
-
Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?
-
Yokota T, Duddy W, Echigoya Y, Kolski H (2012) Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients? Expert Opin Biol Ther.
-
(2012)
Expert Opin Biol Ther
-
-
Yokota, T.1
Duddy, W.2
Echigoya, Y.3
Kolski, H.4
-
43
-
-
0024815723
-
Topography of the Duchenne muscular dystrophy (DMD) gene: FIGE and cDNA analysis of 194 cases reveals 115 deletions and 13 duplications
-
Den Dunnen JT, Grootscholten PM, Bakker E, Blonden LA, Ginjaar HB, et al. (1989) Topography of the Duchenne muscular dystrophy (DMD) gene: FIGE and cDNA analysis of 194 cases reveals 115 deletions and 13 duplications. American Journal of Human Genetics 45: 835-847.
-
(1989)
American Journal of Human Genetics
, vol.45
, pp. 835-847
-
-
Den Dunnen, J.T.1
Grootscholten, P.M.2
Bakker, E.3
Blonden, L.A.4
Ginjaar, H.B.5
-
44
-
-
0025051336
-
Somatic reversion/suppression of the mouse mdx phenotype in vivo
-
Hoffman EP, Morgan JE, Watkins SC, Partridge TA, (1990) Somatic reversion/suppression of the mouse mdx phenotype in vivo. J Neurol Sci 99: 9-25.
-
(1990)
J Neurol Sci
, vol.99
, pp. 9-25
-
-
Hoffman, E.P.1
Morgan, J.E.2
Watkins, S.C.3
Partridge, T.A.4
-
45
-
-
0027203038
-
Dystrophin expression in Duchenne patients with "in-frame" gene deletions
-
Nicholson LV, Bushby KM, Johnson MA, Gardner-Medwin D, Ginjaar IB, (1993) Dystrophin expression in Duchenne patients with "in-frame" gene deletions. Neuropediatrics 24: 93-97.
-
(1993)
Neuropediatrics
, vol.24
, pp. 93-97
-
-
Nicholson, L.V.1
Bushby, K.M.2
Johnson, M.A.3
Gardner-Medwin, D.4
Ginjaar, I.B.5
-
46
-
-
0027487938
-
Exon skipping and translation in patients with frameshift deletions in the dystrophin gene
-
Sherratt TG, Vulliamy T, Dubowitz V, Sewry CA, Strong PN, (1993) Exon skipping and translation in patients with frameshift deletions in the dystrophin gene. American Journal of Human Genetics 53: 1007-1015.
-
(1993)
American Journal of Human Genetics
, vol.53
, pp. 1007-1015
-
-
Sherratt, T.G.1
Vulliamy, T.2
Dubowitz, V.3
Sewry, C.A.4
Strong, P.N.5
-
47
-
-
79960055339
-
Antisense oligo-mediated multiple exon skipping in a dog model of Duchenne muscular dystrophy
-
Yokota T, Hoffman E, Takeda S, (2011) Antisense oligo-mediated multiple exon skipping in a dog model of Duchenne muscular dystrophy. Methods Mol Biol 709: 299-312.
-
(2011)
Methods Mol Biol
, vol.709
, pp. 299-312
-
-
Yokota, T.1
Hoffman, E.2
Takeda, S.3
-
48
-
-
77952010104
-
Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: implication for clinical trials
-
Arechavala-Gomeza V, Kinali M, Feng L, Guglieri M, Edge G, et al. (2010) Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: implication for clinical trials. Neuromuscul Disord 20: 295-301.
-
(2010)
Neuromuscul Disord
, vol.20
, pp. 295-301
-
-
Arechavala-Gomeza, V.1
Kinali, M.2
Feng, L.3
Guglieri, M.4
Edge, G.5
-
49
-
-
0028833771
-
Frameshift deletions of exons 3-7 and revertant fibers in Duchenne muscular dystrophy: mechanisms of dystrophin production
-
Winnard AV, Mendell JR, Prior TW, Florence J, Burghes AH, (1995) Frameshift deletions of exons 3-7 and revertant fibers in Duchenne muscular dystrophy: mechanisms of dystrophin production. American Journal of Human Genetics 56: 158-166.
-
(1995)
American Journal of Human Genetics
, vol.56
, pp. 158-166
-
-
Winnard, A.V.1
Mendell, J.R.2
Prior, T.W.3
Florence, J.4
Burghes, A.H.5
-
50
-
-
0026638903
-
The frequency of revertants in mdx mouse genetic models for Duchenne muscular dystrophy
-
Danko I, Chapman V, Wolff JA, (1992) The frequency of revertants in mdx mouse genetic models for Duchenne muscular dystrophy. Pediatr Res 32: 128-131.
-
(1992)
Pediatr Res
, vol.32
, pp. 128-131
-
-
Danko, I.1
Chapman, V.2
Wolff, J.A.3
-
51
-
-
63449141811
-
Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs
-
Yokota T, Lu QL, Partridge T, Kobayashi M, Nakamura A, et al. (2009) Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs. Ann Neurol 65: 667-676.
-
(2009)
Ann Neurol
, vol.65
, pp. 667-676
-
-
Yokota, T.1
Lu, Q.L.2
Partridge, T.3
Kobayashi, M.4
Nakamura, A.5
-
52
-
-
0034611016
-
Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion
-
Lu QL, Morris GE, Wilton SD, Ly T, Artem'yeva OV, et al. (2000) Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion. J Cell Biol 148: 985-996.
-
(2000)
J Cell Biol
, vol.148
, pp. 985-996
-
-
Lu, Q.L.1
Morris, G.E.2
Wilton, S.D.3
Ly, T.4
Artem'yeva, O.V.5
-
53
-
-
0029119259
-
Dystrophin-positive fibers in Duchenne dystrophy: origin and correlation to clinical course
-
Fanin M, Danieli GA, Cadaldini M, Miorin M, Vitiello L, et al. (1995) Dystrophin-positive fibers in Duchenne dystrophy: origin and correlation to clinical course. Muscle Nerve 18: 1115-1120.
-
(1995)
Muscle Nerve
, vol.18
, pp. 1115-1120
-
-
Fanin, M.1
Danieli, G.A.2
Cadaldini, M.3
Miorin, M.4
Vitiello, L.5
-
54
-
-
0025687705
-
X-irradiation improves mdx mouse muscle as a model of myofiber loss in DMD
-
Wakeford S, Watt DJ, Partridge TA, (1991) X-irradiation improves mdx mouse muscle as a model of myofiber loss in DMD. Muscle Nerve 14: 42-50.
-
(1991)
Muscle Nerve
, vol.14
, pp. 42-50
-
-
Wakeford, S.1
Watt, D.J.2
Partridge, T.A.3
-
55
-
-
0029906168
-
Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene
-
Tinsley JM, Potter AC, Phelps SR, Fisher R, Trickett JI, et al. (1996) Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene. Nature 384: 349-353.
-
(1996)
Nature
, vol.384
, pp. 349-353
-
-
Tinsley, J.M.1
Potter, A.C.2
Phelps, S.R.3
Fisher, R.4
Trickett, J.I.5
-
56
-
-
0036083126
-
Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene
-
Sakamoto M, Yuasa K, Yoshimura M, Yokota T, Ikemoto T, et al. (2002) Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene. Biochem Biophys Res Commun 293: 1265-1272.
-
(2002)
Biochem Biophys Res Commun
, vol.293
, pp. 1265-1272
-
-
Sakamoto, M.1
Yuasa, K.2
Yoshimura, M.3
Yokota, T.4
Ikemoto, T.5
-
57
-
-
0033120705
-
Covert persistence of mdx mouse myopathy is revealed by acute and chronic effects of irradiation
-
Pagel CN, Partridge TA, (1999) Covert persistence of mdx mouse myopathy is revealed by acute and chronic effects of irradiation. Journal of the Neurological Sciences 164: 103-116.
-
(1999)
Journal of the Neurological Sciences
, vol.164
, pp. 103-116
-
-
Pagel, C.N.1
Partridge, T.A.2
-
58
-
-
0019519247
-
Memory of simple learning in young, middle-aged, and aged C57/BL6 mice
-
Fraley SM, Springer AD, (1981) Memory of simple learning in young, middle-aged, and aged C57/BL6 mice. Behav Neural Biol 31: 1-7.
-
(1981)
Behav Neural Biol
, vol.31
, pp. 1-7
-
-
Fraley, S.M.1
Springer, A.D.2
-
59
-
-
0026637764
-
Somatic reversion/suppression in Duchenne muscular dystrophy (DMD): evidence supporting a frame-restoring mechanism in rare dystrophin-positive fibers
-
Klein CJ, Coovert DD, Bulman DE, Ray PN, Mendell JR, et al. (1992) Somatic reversion/suppression in Duchenne muscular dystrophy (DMD): evidence supporting a frame-restoring mechanism in rare dystrophin-positive fibers. American Journal of Human Genetics 50: 950-959.
-
(1992)
American Journal of Human Genetics
, vol.50
, pp. 950-959
-
-
Klein, C.J.1
Coovert, D.D.2
Bulman, D.E.3
Ray, P.N.4
Mendell, J.R.5
-
60
-
-
0032724723
-
Centrally nucleated fibers (CNFs) compensate the fragility of myofibers in mdx mouse
-
Narita S, Yorifuji H, (1999) Centrally nucleated fibers (CNFs) compensate the fragility of myofibers in mdx mouse. Neuroreport 10: 3233-3235.
-
(1999)
Neuroreport
, vol.10
, pp. 3233-3235
-
-
Narita, S.1
Yorifuji, H.2
-
61
-
-
0020058217
-
Five different types of centrally nucleated muscle fibres in man: elemental composition and morphological criteria. A study of myopathies, fetal tissue and muscle spindle
-
Wroblewski R, Edstrom L, Mair WG, (1982) Five different types of centrally nucleated muscle fibres in man: elemental composition and morphological criteria. A study of myopathies, fetal tissue and muscle spindle. J Submicrosc Cytol 14: 377-387.
-
(1982)
J Submicrosc Cytol
, vol.14
, pp. 377-387
-
-
Wroblewski, R.1
Edstrom, L.2
Mair, W.G.3
-
62
-
-
0027385135
-
Age-related changes in replication of myogenic cells in mdx mice: quantitative autoradiographic studies
-
McGeachie JK, Grounds MD, Partridge TA, Morgan JE, (1993) Age-related changes in replication of myogenic cells in mdx mice: quantitative autoradiographic studies. Journal of the Neurological Sciences 119: 169-179.
-
(1993)
Journal of the Neurological Sciences
, vol.119
, pp. 169-179
-
-
McGeachie, J.K.1
Grounds, M.D.2
Partridge, T.A.3
Morgan, J.E.4
-
63
-
-
3042818964
-
Early regeneration of whole skeletal muscle grafts is unaffected by overexpression of IGF-1 in MLC/mIGF-1 transgenic mice
-
Shavlakadze T, Davies M, White JD, Grounds MD, (2004) Early regeneration of whole skeletal muscle grafts is unaffected by overexpression of IGF-1 in MLC/mIGF-1 transgenic mice. J Histochem Cytochem 52: 873-883.
-
(2004)
J Histochem Cytochem
, vol.52
, pp. 873-883
-
-
Shavlakadze, T.1
Davies, M.2
White, J.D.3
Grounds, M.D.4
-
64
-
-
0032599906
-
Computerised dystrophic muscle simulator: prospecting potential therapeutic strategies for muscle dystrophies using a virtual experimental model
-
Garcia L, Peltekian E, Pastoret C, Israeli D, Armande N, et al. (1999) Computerised dystrophic muscle simulator: prospecting potential therapeutic strategies for muscle dystrophies using a virtual experimental model. J Gene Med 1: 43-55.
-
(1999)
J Gene Med
, vol.1
, pp. 43-55
-
-
Garcia, L.1
Peltekian, E.2
Pastoret, C.3
Israeli, D.4
Armande, N.5
-
65
-
-
0035891875
-
Suppression of revertant fibers in mdx mice by expression of a functional dystrophin
-
Crawford GE, Lu QL, Partridge TA, Chamberlain JS, (2001) Suppression of revertant fibers in mdx mice by expression of a functional dystrophin. Hum Mol Genet 10: 2745-2750.
-
(2001)
Hum Mol Genet
, vol.10
, pp. 2745-2750
-
-
Crawford, G.E.1
Lu, Q.L.2
Partridge, T.A.3
Chamberlain, J.S.4
-
66
-
-
33745438706
-
Induction of revertant fibres in the mdx mouse using antisense oligonucleotides
-
Fall AM, Johnsen R, Honeyman K, Iversen P, Fletcher S, et al. (2006) Induction of revertant fibres in the mdx mouse using antisense oligonucleotides. Genet Vaccines Ther 4: 3.
-
(2006)
Genet Vaccines Ther
, vol.4
, pp. 3
-
-
Fall, A.M.1
Johnsen, R.2
Honeyman, K.3
Iversen, P.4
Fletcher, S.5
-
67
-
-
43449113543
-
Optimizing exon skipping therapies for DMD
-
Yokota T, Duddy W, Partridge T, (2007) Optimizing exon skipping therapies for DMD. Acta Myol 26: 179-184.
-
(2007)
Acta Myol
, vol.26
, pp. 179-184
-
-
Yokota, T.1
Duddy, W.2
Partridge, T.3
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