-
1
-
-
0023572154
-
Subcellular fractionation of dystrophin to the triads of skeletal muscle
-
Hoffman EP, Knudson CM, Campbell KP etal. Subcellular fractionation of dystrophin to the triads of skeletal muscle. Nature 1987; 330: 754-758.
-
(1987)
Nature
, vol.330
, pp. 754-758
-
-
Hoffman, E.P.1
Knudson, C.M.2
Campbell, K.P.3
-
2
-
-
0023614188
-
Dystrophin: The protein product of the Duchenne muscular dystrophy locus
-
Hoffman EP, Brown RH, Jr, Kunkel LM. Dystrophin: The protein product of the Duchenne muscular dystrophy locus. Cell 1987; 51: 919-928.
-
(1987)
Cell
, vol.51
, pp. 919-928
-
-
Hoffman, E.P.1
Brown Jr, R.H.2
Kunkel, L.M.3
-
3
-
-
0027460658
-
Dystrophin protects the sarcolemma from stresses developed during muscle contraction
-
Petrof BJ, Shrager JB, Stedman HH etal. Dystrophin protects the sarcolemma from stresses developed during muscle contraction. Proc Natl Acad Sci U S A 1993; 90: 3710-3714.
-
(1993)
Proc Natl Acad Sci U S A
, vol.90
, pp. 3710-3714
-
-
Petrof, B.J.1
Shrager, J.B.2
Stedman, H.H.3
-
4
-
-
0347915657
-
Loss of dystrophin causes aberrant mechanotransduction in skeletal muscle fibers
-
Kumar A, Khandelwal N, Malya R etal. Loss of dystrophin causes aberrant mechanotransduction in skeletal muscle fibers. FASEB J 2004; 18: 102-113.
-
(2004)
FASEB J
, vol.18
, pp. 102-113
-
-
Kumar, A.1
Khandelwal, N.2
Malya, R.3
-
5
-
-
0028929061
-
Mechanical function of dystrophin in muscle cells
-
Pasternak C, Wong S, Elson EL. Mechanical function of dystrophin in muscle cells. J Cell Biol 1995; 128: 355-361.
-
(1995)
J Cell Biol
, vol.128
, pp. 355-361
-
-
Pasternak, C.1
Wong, S.2
Elson, E.L.3
-
6
-
-
77954613481
-
Stem cell therapies to treat muscular dystrophy: Progress to date
-
Meregalli M, Farini A, Parolini D etal. Stem cell therapies to treat muscular dystrophy: Progress to date. BioDrugs 2010; 24: 237-247.
-
(2010)
BioDrugs
, vol.24
, pp. 237-247
-
-
Meregalli, M.1
Farini, A.2
Parolini, D.3
-
7
-
-
84869079595
-
Myoblasts derived from normal hESCs and dystrophic hiPSCs efficiently fuse with existing muscle fibers following transplantation
-
Goudenege S, Lebel C, Huot NB etal. Myoblasts derived from normal hESCs and dystrophic hiPSCs efficiently fuse with existing muscle fibers following transplantation. Mol Ther 2012; 20: 2153-2167.
-
(2012)
Mol Ther
, vol.20
, pp. 2153-2167
-
-
Goudenege, S.1
Lebel, C.2
Huot, N.B.3
-
8
-
-
69749102716
-
Effects of prednisolone on skeletal muscle contractility in mdx mice
-
Baltgalvis KA, Call JA, Nikas JB etal. Effects of prednisolone on skeletal muscle contractility in mdx mice. Muscle Nerve 2009; 40: 443-454.
-
(2009)
Muscle Nerve
, vol.40
, pp. 443-454
-
-
Baltgalvis, K.A.1
Call, J.A.2
Nikas, J.B.3
-
9
-
-
33845990550
-
Weekly oral prednisolone improves survival and strength in male mdx mice
-
Keeling RM, Golumbek PT, Streif EM etal. Weekly oral prednisolone improves survival and strength in male mdx mice. Muscle Nerve 2007; 35: 43-48.
-
(2007)
Muscle Nerve
, vol.35
, pp. 43-48
-
-
Keeling, R.M.1
Golumbek, P.T.2
Streif, E.M.3
-
10
-
-
50649083054
-
Adeno-associated virus serotype-9 microdystrophin gene therapy ameliorates electrocardiographic abnormalities in mdx mice
-
Bostick B, Yue Y, Lai Y etal. Adeno-associated virus serotype-9 microdystrophin gene therapy ameliorates electrocardiographic abnormalities in mdx mice. Hum Gene Ther 2008; 19: 851-856.
-
(2008)
Hum Gene Ther
, vol.19
, pp. 851-856
-
-
Bostick, B.1
Yue, Y.2
Lai, Y.3
-
11
-
-
7044253593
-
AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype
-
Yoshimura M, Sakamoto M, Ikemoto M etal. AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype. Mol Ther 2004; 10: 821-828.
-
(2004)
Mol Ther
, vol.10
, pp. 821-828
-
-
Yoshimura, M.1
Sakamoto, M.2
Ikemoto, M.3
-
12
-
-
32244443828
-
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
-
Alter J, Lou F, Rabinowitz A etal. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 2006; 12: 175-177.
-
(2006)
Nat Med
, vol.12
, pp. 175-177
-
-
Alter, J.1
Lou, F.2
Rabinowitz, A.3
-
13
-
-
80052911776
-
An octaguanidine-morpholino oligo conjugate improves muscle function of mdx mice
-
Widrick JJ, Jiang S, Choi SJ etal. An octaguanidine-morpholino oligo conjugate improves muscle function of mdx mice. Muscle Nerve 2011; 44: 563-570.
-
(2011)
Muscle Nerve
, vol.44
, pp. 563-570
-
-
Widrick, J.J.1
Jiang, S.2
Choi, S.J.3
-
14
-
-
54449095504
-
Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer
-
Wu B, Moulton HM, Iversen PL etal. Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer. Proc Natl Acad Sci U S A 2008; 105: 14814-14819.
-
(2008)
Proc Natl Acad Sci U S A
, vol.105
, pp. 14814-14819
-
-
Wu, B.1
Moulton, H.M.2
Iversen, P.L.3
-
15
-
-
67349137953
-
Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice
-
Wu B, Li Y, Morcos PA etal. Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice. Mol Ther 2009; 17: 864-871.
-
(2009)
Mol Ther
, vol.17
, pp. 864-871
-
-
Wu, B.1
Li, Y.2
Morcos, P.A.3
-
16
-
-
84870344270
-
Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs
-
Yokota T, Nakamura A, Nagata T etal. Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs. Nucleic Acid Ther 2012; 22: 306-315.
-
(2012)
Nucleic Acid Ther
, vol.22
, pp. 306-315
-
-
Yokota, T.1
Nakamura, A.2
Nagata, T.3
-
17
-
-
84869086454
-
Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping
-
Vulin A, Barthelemy I, Goyenvalle A etal. Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping. Mol Ther 2012; 20: 2120-2133.
-
(2012)
Mol Ther
, vol.20
, pp. 2120-2133
-
-
Vulin, A.1
Barthelemy, I.2
Goyenvalle, A.3
-
18
-
-
80051690306
-
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study
-
Cirak S, Arechavala-Gomeza V, Guglieri M etal. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study. Lancet 2011; 378: 595-605.
-
(2011)
Lancet
, vol.378
, pp. 595-605
-
-
Cirak, S.1
Arechavala-Gomeza, V.2
Guglieri, M.3
-
19
-
-
67650391305
-
Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice
-
Malerba A, Thorogood FC, Dickson G etal. Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice. Hum Gene Ther 2009; 20: 955-965.
-
(2009)
Hum Gene Ther
, vol.20
, pp. 955-965
-
-
Malerba, A.1
Thorogood, F.C.2
Dickson, G.3
-
20
-
-
11844256373
-
Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles
-
Lu QL, Rabinowitz A, Chen YC etal. Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci U S A 2005; 102: 198-203.
-
(2005)
Proc Natl Acad Sci U S A
, vol.102
, pp. 198-203
-
-
Lu, Q.L.1
Rabinowitz, A.2
Chen, Y.C.3
-
21
-
-
84858797261
-
Multiple exon skipping strategies to by-pass dystrophin mutations
-
Adkin CF, Meloni PL, Fletcher S etal. Multiple exon skipping strategies to by-pass dystrophin mutations. Neuromuscul Disord 2012; 22: 297-305.
-
(2012)
Neuromuscul Disord
, vol.22
, pp. 297-305
-
-
Adkin, C.F.1
Meloni, P.L.2
Fletcher, S.3
-
22
-
-
79954570149
-
Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy
-
Kemaladewi DU, Hoogaars WM, van Heiningen SH etal. Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy. BMC Med Genomics 2011; 4: 36.
-
(2011)
BMC Med Genomics
, vol.4
, pp. 36
-
-
Kemaladewi, D.U.1
Hoogaars, W.M.2
van Heiningen, S.H.3
-
23
-
-
84861915467
-
Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy
-
Goyenvalle A, Wright J, Babbs A etal. Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy. Mol Ther 2012; 20: 1212-1221.
-
(2012)
Mol Ther
, vol.20
, pp. 1212-1221
-
-
Goyenvalle, A.1
Wright, J.2
Babbs, A.3
-
24
-
-
84859834957
-
U1 snRNA as an effective vector for stable expression of antisense molecules and for the inhibition of the splicing reaction
-
Martone J, De Angelis FG, Bozzoni I. U1 snRNA as an effective vector for stable expression of antisense molecules and for the inhibition of the splicing reaction. Methods Mol Biol 2012; 867: 239-257.
-
(2012)
Methods Mol Biol
, vol.867
, pp. 239-257
-
-
Martone, J.1
De Angelis, F.G.2
Bozzoni, I.3
-
25
-
-
84878653844
-
Cell penetrating peptide delivery of splice directing oligonucleotides as a treatment for Duchenne muscular dystrophy
-
Betts CA, Wood MJ. Cell penetrating peptide delivery of splice directing oligonucleotides as a treatment for Duchenne muscular dystrophy. Curr Pharm Des 2013; 19: 2948-2962.
-
(2013)
Curr Pharm Des
, vol.19
, pp. 2948-2962
-
-
Betts, C.A.1
Wood, M.J.2
-
26
-
-
84859816424
-
Cell-penetrating peptides enhance systemic delivery of antisense morpholino oligomers
-
Moulton HM. Cell-penetrating peptides enhance systemic delivery of antisense morpholino oligomers. Methods Mol Biol 2012; 867: 407-414.
-
(2012)
Methods Mol Biol
, vol.867
, pp. 407-414
-
-
Moulton, H.M.1
-
27
-
-
34547167416
-
Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate
-
Amantana A, Moulton HM, Cate ML etal. Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate. Bioconjug Chem 2007; 18: 1325-1331.
-
(2007)
Bioconjug Chem
, vol.18
, pp. 1325-1331
-
-
Amantana, A.1
Moulton, H.M.2
Cate, M.L.3
-
28
-
-
0029153067
-
Expression of transforming growth factor-beta 1 in dystrophic patient muscles correlates with fibrosis. Pathogenetic role of a fibrogenic cytokine
-
Bernasconi P, Torchiana E, Confalonieri P etal. Expression of transforming growth factor-beta 1 in dystrophic patient muscles correlates with fibrosis. Pathogenetic role of a fibrogenic cytokine. J Clin Invest 1995; 96: 1137-1144.
-
(1995)
J Clin Invest
, vol.96
, pp. 1137-1144
-
-
Bernasconi, P.1
Torchiana, E.2
Confalonieri, P.3
-
30
-
-
0024580644
-
Regulation of skeletal muscle satellite cell proliferation and differentiation by transforming growth factor-beta, insulin-like growth factor I, and fibroblast growth factor
-
Allen RE, Boxhorn LK. Regulation of skeletal muscle satellite cell proliferation and differentiation by transforming growth factor-beta, insulin-like growth factor I, and fibroblast growth factor. J Cell Physiol 1989; 138: 311-315.
-
(1989)
J Cell Physiol
, vol.138
, pp. 311-315
-
-
Allen, R.E.1
Boxhorn, L.K.2
-
31
-
-
77952477337
-
Regulatory factors and cell populations involved in skeletal muscle regeneration
-
Ten Broek RW, Grefte S, Von den Hoff JW. Regulatory factors and cell populations involved in skeletal muscle regeneration. J Cell Physiol 2010; 224: 7-16.
-
(2010)
J Cell Physiol
, vol.224
, pp. 7-16
-
-
Ten Broek, R.W.1
Grefte, S.2
Von den Hoff, J.W.3
-
32
-
-
33846946114
-
Angiotensin II type 1 receptor blockade attenuates TGF-beta-induced failure of muscle regeneration in multiple myopathic states
-
Cohn RD, van Erp C, Habashi JP etal. Angiotensin II type 1 receptor blockade attenuates TGF-beta-induced failure of muscle regeneration in multiple myopathic states. Nat Med 2007; 13: 204-210.
-
(2007)
Nat Med
, vol.13
, pp. 204-210
-
-
Cohn, R.D.1
van Erp, C.2
Habashi, J.P.3
-
33
-
-
33846989762
-
ACE inhibitor bulks up muscle
-
Chamberlain JS. ACE inhibitor bulks up muscle. Nat Med 2007; 13: 125-126.
-
(2007)
Nat Med
, vol.13
, pp. 125-126
-
-
Chamberlain, J.S.1
-
34
-
-
79955882466
-
Losartan restores skeletal muscle remodeling and protects against disuse atrophy in sarcopenia
-
82ra37
-
Burks TN, Andres-Mateos E, Marx R etal. Losartan restores skeletal muscle remodeling and protects against disuse atrophy in sarcopenia. Sci Transl Med 2011; 3: 82ra37.
-
(2011)
Sci Transl Med
, vol.3
-
-
Burks, T.N.1
Andres-Mateos, E.2
Marx, R.3
-
35
-
-
84871605026
-
Losartan improves adipose tissue-derived stem cell niche by inhibiting transforming growth factor-beta and fibrosis in skeletal muscle injury
-
Park JK, Ki MR, Lee EM etal. Losartan improves adipose tissue-derived stem cell niche by inhibiting transforming growth factor-beta and fibrosis in skeletal muscle injury. Cell Transplant 2012; 21: 2407-2424.
-
(2012)
Cell Transplant
, vol.21
, pp. 2407-2424
-
-
Park, J.K.1
Ki, M.R.2
Lee, E.M.3
-
36
-
-
0035793047
-
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse
-
Mann CJ, Honeyman K, Cheng AJ etal. 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
-
37
-
-
33748307493
-
Exercise improves the success of myoblast transplantation in mdx mice
-
Bouchentouf M, Benabdallah BF, Mills P etal. Exercise improves the success of myoblast transplantation in mdx mice. Neuromuscul Disord 2006; 16: 518-529.
-
(2006)
Neuromuscul Disord
, vol.16
, pp. 518-529
-
-
Bouchentouf, M.1
Benabdallah, B.F.2
Mills, P.3
-
38
-
-
0017184389
-
A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding
-
Bradford MM. A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding. Anal Biochem 1976; 72: 248-254.
-
(1976)
Anal Biochem
, vol.72
, pp. 248-254
-
-
Bradford, M.M.1
-
39
-
-
34447129588
-
Co-detection of GFP and dystrophin in skeletal muscle tissue sections
-
Liadaki K, Luth ES, Kunkell LM. Co-detection of GFP and dystrophin in skeletal muscle tissue sections. Biotechniques 2007; 42: 699-700.
-
(2007)
Biotechniques
, vol.42
, pp. 699-700
-
-
Liadaki, K.1
Luth, E.S.2
Kunkell, L.M.3
-
40
-
-
65349121206
-
In vivo comparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping
-
Heemskerk HA, de Winter CL, de Kimpe SJ etal. In vivo comparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping. J Gene Med 2009; 11: 257-266.
-
(2009)
J Gene Med
, vol.11
, pp. 257-266
-
-
Heemskerk, H.A.1
de Winter, C.L.2
de Kimpe, S.J.3
-
41
-
-
0033804899
-
Regulation of muscle regulatory factors by DNA-binding, interacting proteins, and post-transcriptional modifications
-
Puri PL, Sartorelli V. Regulation of muscle regulatory factors by DNA-binding, interacting proteins, and post-transcriptional modifications. J Cell Physiol 2000; 185: 155-173.
-
(2000)
J Cell Physiol
, vol.185
, pp. 155-173
-
-
Puri, P.L.1
Sartorelli, V.2
-
42
-
-
34548157008
-
Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo
-
Moulton HM, Fletcher S, Neuman BW etal. Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo. Biochem Soc Trans 2007; 35: 826-828.
-
(2007)
Biochem Soc Trans
, vol.35
, pp. 826-828
-
-
Moulton, H.M.1
Fletcher, S.2
Neuman, B.W.3
-
43
-
-
77958152873
-
Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy
-
Moulton HM, Moulton JD. Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy. Biochim Biophys Acta 2010; 1798: 2296-2303.
-
(2010)
Biochim Biophys Acta
, vol.1798
, pp. 2296-2303
-
-
Moulton, H.M.1
Moulton, J.D.2
-
44
-
-
0031442922
-
Muscle fibers of mdx mice are more vulnerable to exercise than those of normal mice
-
Brussee V, Tardif F, Tremblay JP. Muscle fibers of mdx mice are more vulnerable to exercise than those of normal mice. Neuromuscul Disord 1997; 7: 487-492.
-
(1997)
Neuromuscul Disord
, vol.7
, pp. 487-492
-
-
Brussee, V.1
Tardif, F.2
Tremblay, J.P.3
-
45
-
-
0027301717
-
The effects of endurance exercise on dystrophic mdx mice. II. Contractile properties of skinned muscle fibres
-
Lynch GS, Hayes A, Lam MH etal. The effects of endurance exercise on dystrophic mdx mice. II. Contractile properties of skinned muscle fibres. Proc Biol Sci 1993; 253: 27-33.
-
(1993)
Proc Biol Sci
, vol.253
, pp. 27-33
-
-
Lynch, G.S.1
Hayes, A.2
Lam, M.H.3
-
46
-
-
0031051395
-
Contractile properties of clenbuterol-treated mdx muscle are enhanced by low-intensity swimming
-
Hayes A, Williams DA. Contractile properties of clenbuterol-treated mdx muscle are enhanced by low-intensity swimming. J Appl Physiol 1997; 82: 435-439.
-
(1997)
J Appl Physiol
, vol.82
, pp. 435-439
-
-
Hayes, A.1
Williams, D.A.2
-
47
-
-
84857126451
-
The effects of low levels of dystrophin on mouse muscle function and pathology
-
van Putten M, Hulsker M, Nadarajah VD etal. The effects of low levels of dystrophin on mouse muscle function and pathology. PLoS ONE 2012; 7: e31937.
-
(2012)
PLoS ONE
, vol.7
-
-
van Putten, M.1
Hulsker, M.2
Nadarajah, V.D.3
|