-
1
-
-
0037447517
-
Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients
-
Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, den Dunnen JT, Baas F, van Ommen GJ, van Deutekom JC (2003) Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum Mol Genet 12: 907-914
-
(2003)
Hum Mol Genet
, vol.12
, pp. 907-914
-
-
Aartsma-Rus, A.1
Janson, A.A.2
Kaman, W.E.3
Bremmer-Bout, M.4
Den Dunnen, J.T.5
Baas, F.6
Van Ommen, G.J.7
Van Deutekom, J.C.8
-
2
-
-
32244443828
-
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
-
Alter J, Lou F, Rabinowitz A, Yin H, Rosenfeld J, Wilton SD, Partridge TA, Lu QL (2006) Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 12: 175-177
-
(2006)
Nat Med
, vol.12
, 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
Lu, Q.L.8
-
3
-
-
4344693568
-
Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides
-
Bremmer-Bout M, Aartsma-Rus A, De Meijer EJ, Kaman WE, Janson AA, Vossen RH, van Ommen GJ, den Dunnen JT, van Deutekom JC (2004) Targeted exon skipping in transgenic hDMD mice: a model for direct preclinical screening of human-specific antisense oligonucleotides. Mol Ther 10: 232-240
-
(2004)
Mol Ther
, vol.10
, pp. 232-240
-
-
Bremmer-Bout, M.1
Aartsma-Rus, A.2
De Meijer, E.J.3
Kaman, W.E.4
Janson, A.A.5
Vossen, R.H.6
Van Ommen, G.J.7
Den Dunnen, J.T.8
Van Deutekom, J.C.9
-
4
-
-
77957685944
-
MicroRNAs involved in molecular circuitries relevant for the Duchenne muscular dystrophy pathogenesis are controlled by the dystrophin/nNOS pathway
-
Cacchiarelli D et al (2010) microRNAs involved in molecular circuitries relevant for the Duchenne muscular dystrophy pathogenesis are controlled by the dystrophin/nNOS pathway. Cell Metab 12: 341-351
-
(2010)
Cell Metab
, vol.12
, pp. 341-351
-
-
Cacchiarelli, D.1
-
5
-
-
31744432337
-
The role of microRNA-1 and microRNA-133 in skeletal muscle proliferation and differentiation
-
Chen JF, Mandel EM, Thomson JM, Wu Q, Callis TE, Hammond SM, Conlon FL, Wang DZ (2006) The role of microRNA-1 and microRNA-133 in skeletal muscle proliferation and differentiation. Nat Genet 38: 228-233
-
(2006)
Nat Genet
, vol.38
, pp. 228-233
-
-
Chen, J.F.1
Mandel, E.M.2
Thomson, J.M.3
Wu, Q.4
Callis, T.E.5
Hammond, S.M.6
Conlon, F.L.7
Wang, D.Z.8
-
6
-
-
59649126667
-
The regulatory mechanisms that underlie inappropriate transcription of the myogenic determination gene Myf5 in the central nervous system
-
Daubas P, Crist CG, Bajard L, Relaix F, Pecnard E, Rocancourt D, Buckingham M (2009) The regulatory mechanisms that underlie inappropriate transcription of the myogenic determination gene Myf5 in the central nervous system. Dev Biol 327: 71-82
-
(2009)
Dev Biol
, vol.327
, pp. 71-82
-
-
Daubas, P.1
Crist, C.G.2
Bajard, L.3
Relaix, F.4
Pecnard, E.5
Rocancourt, D.6
Buckingham, M.7
-
7
-
-
0037047111
-
Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a corrected phenotype in D48-50
-
De Angelis FG, Sthandier O, Berarducci B, Toso S, Galluzzi G, Ricci E, Cossu G, Bozzoni I (2002) Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a corrected phenotype in D48-50, DMD cells. Proc Natl Acad Sci USA 99: 9456-9461
-
(2002)
DMD Cells. Proc Natl Acad Sci USA
, vol.99
, pp. 9456-9461
-
-
De Angelis, F.G.1
Sthandier, O.2
Berarducci, B.3
Toso, S.4
Galluzzi, G.5
Ricci, E.6
Cossu, G.7
Bozzoni, I.8
-
8
-
-
3042854926
-
A novel vector for siRNA expression in mammalian cells that relies on polII-dependent transcription
-
Denti MA, Rosa A, Sthandier O, De Angelis FG, Bozzoni I (2004) A novel vector for siRNA expression in mammalian cells that relies on polII-dependent transcription. Mol Ther 10: 191-199
-
(2004)
Mol Ther
, vol.10
, pp. 191-199
-
-
Denti, M.A.1
Rosa, A.2
Sthandier, O.3
De Angelis, F.G.4
Bozzoni, I.5
-
9
-
-
33644857020
-
Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model
-
Denti MA et al (2006) Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model. Proc Natl Acad Sci USA 103: 3758-3763
-
(2006)
Proc Natl Acad Sci USA
, vol.103
, pp. 3758-3763
-
-
Denti, M.A.1
-
10
-
-
46249108952
-
Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice
-
Denti MA, Incitti T, Sthandier O, Nicoletti C, De Angelis FG, Rizzuto E, Auricchio A, Musarò A, Bozzoni I (2008) Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice. Hum Gene Ther 19: 601-608
-
(2008)
Hum Gene Ther
, vol.19
, pp. 601-608
-
-
Denti, M.A.1
Incitti, T.2
Sthandier, O.3
Nicoletti, C.4
De Angelis, F.G.5
Rizzuto, E.6
Auricchio, A.7
Musarò, A.8
Bozzoni, I.9
-
11
-
-
0031800293
-
Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides
-
Dunckley MG, Manoharan M, Villiet P, Eperon IC, Dickson G (1998) Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides. Hum Mol Genet 7: 1083-1090
-
(1998)
Hum Mol Genet
, vol.7
, pp. 1083-1090
-
-
Dunckley, M.G.1
Manoharan, M.2
Villiet, P.3
Eperon, I.C.4
Dickson, G.5
-
12
-
-
36749019110
-
Distinctive patterns of microRNA expression in primary muscular disorders
-
Eisenberg I et al (2007) Distinctive patterns of microRNA expression in primary muscular disorders. Proc Natl Acad Sci USA 104: 17016-17021
-
(2007)
Proc Natl Acad Sci USA
, vol.104
, pp. 17016-17021
-
-
Eisenberg, I.1
-
13
-
-
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 (2006) Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide. J Gene Med 8: 207-216
-
(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
-
14
-
-
58149252151
-
Stable knockdown of microRNA in vivo by lentiviral vectors
-
Gentner B, Schira G, Giustacchini A, Amendola M, Brown BD, Ponzoni M, Naldini L (2009) Stable knockdown of microRNA in vivo by lentiviral vectors. Nat Methods 6: 63-66
-
(2009)
Nat Methods
, vol.6
, pp. 63-66
-
-
Gentner, B.1
Schira, G.2
Giustacchini, A.3
Amendola, M.4
Brown, B.D.5
Ponzoni, M.6
Naldini, L.7
-
15
-
-
10044240371
-
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
-
Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O (2004) Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 306: 1796-1799
-
(2004)
Science
, vol.306
, 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
-
16
-
-
70349668311
-
Common micro-RNA signature in skeletal muscle damage and regeneration induced by Duchenne muscular dystrophy and acute ischemia
-
Greco S et al (2009) Common micro-RNA signature in skeletal muscle damage and regeneration induced by Duchenne muscular dystrophy and acute ischemia. FASEB J 23: 3335-3346
-
(2009)
FASEB J
, vol.23
, pp. 3335-3346
-
-
Greco, S.1
-
17
-
-
77956268227
-
Exon skipping and Duchenne muscular dystrophy therapy: Selection of the most active U1 snRNA-antisense able to induce dystrophin exon 51 skipping
-
Incitti T, De Angelis FG, Cazzella V, Sthandier O, Pinnarò C, Legnini I, Bozzoni I (2010) Exon skipping and Duchenne muscular dystrophy therapy: selection of the most active U1 snRNA-antisense able to induce dystrophin exon 51 skipping. Mol Ther 18: 1675-1682
-
(2010)
Mol Ther
, vol.18
, pp. 1675-1682
-
-
Incitti, T.1
De Angelis, F.G.2
Cazzella, V.3
Sthandier, O.4
Pinnarò, C.5
Legnini, I.6
Bozzoni, I.7
-
18
-
-
33644779735
-
The microRNA miR-181 targets the homeobox protein Hox-A11 during mammalian myoblast differentiation
-
Naguibneva I, Ameyar-Zazoua M, Polesskaya A, Ait-Si-Ali S, Groisman R, Souidi M, Cuvellier S, Harel-Bellan A (2006) The microRNA miR-181 targets the homeobox protein Hox-A11 during mammalian myoblast differentiation. Nat Cell Biol 8: 278-284
-
(2006)
Nat Cell Biol
, vol.8
, pp. 278-284
-
-
Naguibneva, I.1
Ameyar-Zazoua, M.2
Polesskaya, A.3
Ait-Si-Ali, S.4
Groisman, R.5
Souidi, M.6
Cuvellier, S.7
Harel-Bellan, A.8
-
19
-
-
59349089292
-
MiRNA in situ hybridization in formaldehyde and EDC-fixed tissues
-
Pena JT et al (2009) miRNA in situ hybridization in formaldehyde and EDC-fixed tissues. Nat Methods 6: 139-141
-
(2009)
Nat Methods
, vol.6
, pp. 139-141
-
-
Pena, J.T.1
-
20
-
-
0028264320
-
Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy
-
Rando TA, Blau HM (1994) Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy. J Cell Biol 125: 1275-1287
-
(1994)
J Cell Biol
, vol.125
, pp. 1275-1287
-
-
Rando, T.A.1
Blau, H.M.2
-
21
-
-
68849118992
-
The muscle-specific microRNA miR-206 blocks human rhabdomyosarcoma growth in xenotransplanted mice by promoting myogenic differentiation
-
Taulli R, Bersani F, Foglizzo V, Linari A, Vigna E, Ladanyi M, Tuschl T, Ponzetto C (2009) The muscle-specific microRNA miR-206 blocks human rhabdomyosarcoma growth in xenotransplanted mice by promoting myogenic differentiation. J Clin Invest 119: 2366-2378
-
(2009)
J Clin Invest
, vol.119
, pp. 2366-2378
-
-
Taulli, R.1
Bersani, F.2
Foglizzo, V.3
Linari, A.4
Vigna, E.5
Ladanyi, M.6
Tuschl, T.7
Ponzetto, C.8
-
22
-
-
77953554439
-
MiR-31: A crucial overseer of tumor metastasis and other emerging roles
-
Valastyan S, Weinberg RA (2010) miR-31: a crucial overseer of tumor metastasis and other emerging roles. Cell Cycle 9: 2124-2129
-
(2010)
Cell Cycle
, vol.9
, pp. 2124-2129
-
-
Valastyan, S.1
Weinberg, R.A.2
-
23
-
-
37549034298
-
Local dystrophin restoration with antisense oligonucleotide PRO051
-
Van Deutekom JC et al (2007) Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med 357: 2677-2686
-
(2007)
N Engl J Med
, vol.357
, pp. 2677-2686
-
-
Van Deutekom, J.C.1
-
24
-
-
55449100829
-
Toward microRNA-based therapeutics for heart disease: The sense in antisense
-
Van Rooij E, Marshall WS, Olson EN (2008) Toward microRNA-based therapeutics for heart disease: the sense in antisense. Circ Res 103: 919-928
-
(2008)
Circ Res
, vol.103
, pp. 919-928
-
-
Van Rooij, E.1
Marshall, W.S.2
Olson, E.N.3
|