-
1
-
-
0036823504
-
Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy
-
Aartsma-Rus A, Bremmer-Bout M, Janson AA, den Dunnen JT, van Ommen GJ, van Deutekom JC. Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy. Neuromuscul Disord 12, Suppl 1: S71-S77, 2002.
-
(2002)
Neuromuscul Disord
, vol.12
, Issue.SUPPL. 1
-
-
Aartsma-Rus, A.1
Bremmer-Bout, M.2
Janson, A.A.3
den Dunnen, J.T.4
van Ommen, G.J.5
van Deutekom, J.C.6
-
2
-
-
1242353110
-
Stimulation of calcineurin signaling attenuates the dystrophic pathology in mdx mice
-
Chakkalakal JV, Harrison MA, Carbonetto S, Chin E, Michel RN, Jasmin BJ. Stimulation of calcineurin signaling attenuates the dystrophic pathology in mdx mice. Hum Mol Genet 13: 379-388, 2004.
-
(2004)
Hum Mol Genet
, vol.13
, pp. 379-388
-
-
Chakkalakal, J.V.1
Harrison, M.A.2
Carbonetto, S.3
Chin, E.4
Michel, R.N.5
Jasmin, B.J.6
-
3
-
-
0036537529
-
Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa microdystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice
-
Fabb SA, Wells DJ, Serpente P, Dickson G. Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa microdystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice. Hum Mol Genet 11: 733-741, 2002.
-
(2002)
Hum Mol Genet
, vol.11
, pp. 733-741
-
-
Fabb, S.A.1
Wells, D.J.2
Serpente, P.3
Dickson, G.4
-
4
-
-
34547691961
-
Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse
-
Fletcher S, Honeyman K, Fall AM, Harding PL, Johnsen RD, Steinhaus JP, Moulton HM, Iversen PL, Wilton SD. Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse. Mol Ther 15: 1587-1592, 2007.
-
(2007)
Mol Ther
, vol.15
, pp. 1587-1592
-
-
Fletcher, S.1
Honeyman, K.2
Fall, A.M.3
Harding, P.L.4
Johnsen, R.D.5
Steinhaus, J.P.6
Moulton, H.M.7
Iversen, P.L.8
Wilton, S.D.9
-
5
-
-
32844460899
-
Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide
-
Fletcher S, Honeyman K, Fall AM, Harding PL, Johnsen RD, Wilton SD. Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide. J Gene Med 8: 207-216, 2006.
-
(2006)
J Gene Med
, vol.8
, 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
-
6
-
-
0033562175
-
Alphaactinin-2 is a new component of the dystrophin-glycoprotein complex
-
Hance JE, Fu SY, Watkins SC, Beggs AH, Michalak M. Alphaactinin-2 is a new component of the dystrophin-glycoprotein complex. Arch Biochem Biophys 365: 216-222, 1999.
-
(1999)
Arch Biochem Biophys
, vol.365
, pp. 216-222
-
-
Hance, J.E.1
Fu, S.Y.2
Watkins, S.C.3
Beggs, A.H.4
Michalak, M.5
-
7
-
-
0030272647
-
Dystroglycan: An extracellular matrix receptor linked to the cytoskeleton
-
Henry MD, Campbell KP. Dystroglycan: an extracellular matrix receptor linked to the cytoskeleton. Curr Opin Cell Biol 8: 625-631, 1996.
-
(1996)
Curr Opin Cell Biol
, vol.8
, pp. 625-631
-
-
Henry, M.D.1
Campbell, K.P.2
-
8
-
-
0024466501
-
The molecular basis for Duchenne versus Becker muscular dystrophy: Correlation of severity with type of deletion
-
Koenig M, Beggs AH, Moyer M, Scherpf S, Heindrich K, Bettecken T, Meng G, Muller CR, Lindlof M, Kaariainen H, et al. The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion Am J Hum Genet 45: 498-506, 1989.
-
(1989)
Am J Hum Genet
, vol.45
, pp. 498-506
-
-
Koenig, 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
-
9
-
-
0037336186
-
Mechanical stress activates the nuclear factorkappaB pathway in skeletal muscle fibers: A possible role in Duchenne muscular dystrophy
-
Kumar A, Boriek AM. Mechanical stress activates the nuclear factorkappaB pathway in skeletal muscle fibers: a possible role in Duchenne muscular dystrophy. FASEB J 17: 386-396, 2003.
-
(2003)
FASEB J
, vol.17
, pp. 386-396
-
-
Kumar, A.1
Boriek, A.M.2
-
10
-
-
7044272470
-
Progression of kyphosis in mdx mice
-
Laws N, Hoey A. Progression of kyphosis in mdx mice. J Appl Physiol 97: 1970-1977, 2004.
-
(2004)
J Appl Physiol
, vol.97
, pp. 1970-1977
-
-
Laws, N.1
Hoey, A.2
-
11
-
-
0029001953
-
Phenotype of dystrophinopathy in old mdx mice
-
Lefaucheur JP, Pastoret C, Sebille A. Phenotype of dystrophinopathy in old mdx mice. Anat Rec 242: 70-76, 1995.
-
(1995)
Anat Rec
, vol.242
, pp. 70-76
-
-
Lefaucheur, J.P.1
Pastoret, C.2
Sebille, A.3
-
12
-
-
0042536463
-
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
-
Lu QL, Mann CJ, Lou F, Bou-Gharios G, Morris GE, Xue SA, Fletcher S, Partridge TA, Wilton SD. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat Med 9: 1009-1014, 2003.
-
(2003)
Nat Med
, vol.9
, 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
-
13
-
-
11844256373
-
Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles
-
Lu QL, Rabinowitz A, Chen YC, Yokota T, Yin H, Alter J, Jadoon A, Bou-Gharios G, Partridge T. Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci USA 102: 198-203, 2005.
-
(2005)
Proc Natl Acad Sci USA
, vol.102
, pp. 198-203
-
-
Lu, Q.L.1
Rabinowitz, A.2
Chen, Y.C.3
Yokota, T.4
Yin, H.5
Alter, J.6
Jadoon, A.7
Bou-Gharios, G.8
Partridge, T.9
-
14
-
-
0035793047
-
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse
-
Mann CJ, Honeyman K, Cheng AJ, Ly T, Lloyd F, Fletcher S, Morgan JE, Partridge TA, Wilton SD. Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci USA 98: 42-47, 2001.
-
(2001)
Proc Natl Acad Sci USA
, vol.98
, pp. 42-47
-
-
Mann, C.J.1
Honeyman, K.2
Cheng, A.J.3
Ly, T.4
Lloyd, F.5
Fletcher, S.6
Morgan, J.E.7
Partridge, T.A.8
Wilton, S.D.9
-
15
-
-
0013181060
-
Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy
-
Mann CJ, Honeyman K, McClorey G, Fletcher S, Wilton SD. Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy. J Gene Med 4: 644-654, 2002.
-
(2002)
J Gene Med
, vol.4
, pp. 644-654
-
-
Mann, C.J.1
Honeyman, K.2
McClorey, G.3
Fletcher, S.4
Wilton, S.D.5
-
16
-
-
33745479703
-
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
-
McClorey G, Moulton HM, Iversen PL, Fletcher S, Wilton SD. Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Ther 13: 1373-1381, 2006.
-
(2006)
Gene Ther
, vol.13
, pp. 1373-1381
-
-
McClorey, G.1
Moulton, H.M.2
Iversen, P.L.3
Fletcher, S.4
Wilton, S.D.5
-
17
-
-
0029124967
-
Age-related differences in regeneration of dystrophic (mdx) and normal muscle in the mouse
-
Pastoret C, Sebille A. Age-related differences in regeneration of dystrophic (mdx) and normal muscle in the mouse. Muscle Nerve 18: 1147-1154, 1995.
-
(1995)
Muscle Nerve
, vol.18
, pp. 1147-1154
-
-
Pastoret, C.1
Sebille, A.2
-
18
-
-
0026419948
-
The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy
-
Stedman HH, Sweeney HL, Shrager JB, Maguire HC, Panettieri RA, Petrof B, Narusawa M, Leferovich JM, Sladky JT, Kelly AM. The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy. Nature 352: 536-539, 1991.
-
(1991)
Nature
, vol.352
, pp. 536-539
-
-
Stedman, H.H.1
Sweeney, H.L.2
Shrager, J.B.3
Maguire, H.C.4
Panettieri, R.A.5
Petrof, B.6
Narusawa, M.7
Leferovich, J.M.8
Sladky, J.T.9
Kelly, A.M.10
-
19
-
-
0035878539
-
Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells
-
van Deutekom JC, Bremmer-Bout M, Janson AA, Ginjaar IB, Baas F, den Dunnen JT, van Ommen GJ. Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells. Hum Mol Genet 10: 1547-1554, 2001.
-
(2001)
Hum Mol Genet
, vol.10
, pp. 1547-1554
-
-
van Deutekom, J.C.1
Bremmer-Bout, M.2
Janson, A.A.3
Ginjaar, I.B.4
Baas, F.5
den Dunnen, J.T.6
van Ommen, G.J.7
-
20
-
-
33644811885
-
Enhancing cGMP in experimental progressive renal fibrosis: Soluble guanylate cyclase stimulation vs. phosphodiesterase inhibition
-
Wang Y, Kramer S, Loof T, Martini S, Kron S, Kawachi H, Shimizu F, Neumayer HH, Peters H. Enhancing cGMP in experimental progressive renal fibrosis: soluble guanylate cyclase stimulation vs. phosphodiesterase inhibition. Am J Physiol Renal Physiol 290: F167-F176, 2006.
-
(2006)
Am J Physiol Renal Physiol
, vol.290
-
-
Wang, Y.1
Kramer, S.2
Loof, T.3
Martini, S.4
Kron, S.5
Kawachi, H.6
Shimizu, F.7
Neumayer, H.H.8
Peters, H.9
-
21
-
-
0037211804
-
Relocalization of neuronal nitric oxide synthase (nNOS) as a marker for complete restoration of the dystrophin associated protein complex in skeletal muscle
-
Wells KE, Torelli S, Lu Q, Brown SC, Partridge T, Muntoni F, Wells DJ. Relocalization of neuronal nitric oxide synthase (nNOS) as a marker for complete restoration of the dystrophin associated protein complex in skeletal muscle. Neuromuscul Disord 13: 21-31, 2003.
-
(2003)
Neuromuscul Disord
, vol.13
, pp. 21-31
-
-
Wells, K.E.1
Torelli, S.2
Lu, Q.3
Brown, S.C.4
Partridge, T.5
Muntoni, F.6
Wells, D.J.7
-
22
-
-
0033044501
-
Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides
-
Wilton SD, Lloyd F, Carville K, Fletcher S, Honeyman K, Agrawal S, Kole R. Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides. Neuromuscul Disord 9: 330-338, 1999.
-
(1999)
Neuromuscul Disord
, vol.9
, pp. 330-338
-
-
Wilton, S.D.1
Lloyd, F.2
Carville, K.3
Fletcher, S.4
Honeyman, K.5
Agrawal, S.6
Kole, R.7
-
23
-
-
7044253593
-
AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype
-
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. Mol Ther 10: 821-828, 2004.
-
(2004)
Mol Ther
, vol.10
, 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
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