-
1
-
-
38449100768
-
Small RNAs: Delivering the future
-
Blow, N (2007). Small RNAs: Delivering the future. Nature 450 1117-1120.
-
(2007)
Nature
, vol.450
, pp. 1117-1120
-
-
Blow, N.1
-
2
-
-
34447114618
-
Transvascular delivery of small interfering RNA to the central nervous system
-
Kumar, P, Wu, H, McBride, JL, Jung, KE, Kim, MH, Davidson, BL et al. (2007). Transvascular delivery of small interfering RNA to the central nervous system. Nature 448: 39-43.
-
(2007)
Nature
, vol.448
, pp. 39-43
-
-
Kumar, P.1
Wu, H.2
McBride, J.L.3
Jung, K.E.4
Kim, M.H.5
Davidson, B.L.6
-
3
-
-
0027487938
-
Exon skipping and translation in patients with frameshift deletions in the dystrophin gene
-
Sherratt, TG, Vulliamy, T, Dubowitz, V, Sewry, CA and Strong, PN (1993). Exon skipping and translation in patients with frameshift deletions in the dystrophin gene. Am J Hum Genet 53: 1007-1015.
-
(1993)
Am J Hum Genet
, vol.53
, pp. 1007-1015
-
-
Sherratt, T.G.1
Vulliamy, T.2
Dubowitz, V.3
Sewry, C.A.4
Strong, P.N.5
-
4
-
-
0031800293
-
Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides
-
Dunckley, MG, Manoharan, M, Villiet, P, Eperon, IC and 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
-
5
-
-
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 et al. (2001). Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci USA 98: 42-47.
-
(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
-
6
-
-
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 et al. (2003). Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat Med 9: 1009-1014.
-
(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
-
7
-
-
0036824033
-
Screening for antisense modulation of dystrophin pre-mRNA splicing
-
Dickson, G, Hill, V and Graham, IR (2002). Screening for antisense modulation of dystrophin pre-mRNA splicing. Neuromuscul Disord 12 (suppl. 1): S67-S70.
-
(2002)
Neuromuscul Disord
, vol.12
, Issue.SUPPL. 1
-
-
Dickson, G.1
Hill, V.2
Graham, I.R.3
-
8
-
-
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 and van Deutekom, JC (2002). Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy. Neuromuscul Disord 12 (suppl. 1): S71-S77.
-
(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
-
9
-
-
0025159208
-
Very mild muscular dystrophy associated with the deletion of 46% of dystrophin
-
England, SB, Nicholson, LV, Johnson, MA, Forrest, SM, Love, DR, Zubrzycka-Gaarn, EE et al. (1990). Very mild muscular dystrophy associated with the deletion of 46% of dystrophin. Nature 343 180-182.
-
(1990)
Nature
, vol.343
, pp. 180-182
-
-
England, S.B.1
Nicholson, L.V.2
Johnson, M.A.3
Forrest, S.M.4
Love, D.R.5
Zubrzycka-Gaarn, E.E.6
-
10
-
-
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, Suzuki, M 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
Suzuki, M.6
-
11
-
-
0027207248
-
Characterization of translational frame exception patients in Duchenne/Becker muscular dystrophy
-
Winnard, AV, Klein, CJ, Coovert, DD, Prior, T, Papp, A, Snyder, P et al. (1993). Characterization of translational frame exception patients in Duchenne/Becker muscular dystrophy. Hum Mol Genet 2 737-744.
-
(1993)
Hum Mol Genet
, vol.2
, pp. 737-744
-
-
Winnard, A.V.1
Klein, C.J.2
Coovert, D.D.3
Prior, T.4
Papp, A.5
Snyder, P.6
-
12
-
-
0031722730
-
Giant dystrophin deletion associated with congenital cataract and mild muscular dystrophy
-
Mirabella, M, Galluzzi, G, Manfredi, G, Bertini, E, Ricci, E, De Leo, R et al. (1998). Giant dystrophin deletion associated with congenital cataract and mild muscular dystrophy. Neurology 51 592-595.
-
(1998)
Neurology
, vol.51
, pp. 592-595
-
-
Mirabella, M.1
Galluzzi, G.2
Manfredi, G.3
Bertini, E.4
Ricci, E.5
De Leo, R.6
-
13
-
-
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, Strong, P 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
Strong, P.6
-
14
-
-
37549034298
-
Local dystrophin restoration with antisense oligonucleotide PRO051
-
van Deutekom, JC, Janson, AA, Ginjaar, IB, Frankhuizen, WS, Aartsma-Rus, A, Bremmer-Bout, M 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
Janson, A.A.2
Ginjaar, I.B.3
Frankhuizen, W.S.4
Aartsma-Rus, A.5
Bremmer-Bout, M.6
-
15
-
-
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 et al. (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
-
16
-
-
34249099687
-
Managing Duchenne muscular dystrophy-the additive effect of spinal surgery and home nocturnal ventilation in improving survival
-
Eagle, M, Bourke, J, Bullock, R, Gibson, M, Mehta, J, Giddings, D et al. (2007). Managing Duchenne muscular dystrophy-the additive effect of spinal surgery and home nocturnal ventilation in improving survival. Neuromuscul Disord 17: 470-475.
-
(2007)
Neuromuscul Disord
, vol.17
, pp. 470-475
-
-
Eagle, M.1
Bourke, J.2
Bullock, R.3
Gibson, M.4
Mehta, J.5
Giddings, D.6
-
17
-
-
33846288380
-
Current treatment of adult Duchenne muscular dystrophy
-
Wagner, KR, Lechtzin, N and Judge, DP (2007). Current treatment of adult Duchenne muscular dystrophy. Biochim Biophys Acta 1772: 229-237.
-
(2007)
Biochim Biophys Acta
, vol.1772
, pp. 229-237
-
-
Wagner, K.R.1
Lechtzin, N.2
Judge, D.P.3
-
18
-
-
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 et al. (2007). Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse. Mol Ther 15: 587-1592.
-
(2007)
Mol Ther
, vol.15
, pp. 587-1592
-
-
Fletcher, S.1
Honeyman, K.2
Fall, A.M.3
Harding, P.L.4
Johnsen, R.D.5
Steinhaus, J.P.6
-
19
-
-
54449095504
-
Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer
-
Wu, B, Moulton, HM, Iversen, PL, Jiang, J, Li, J, Spurney, CF et al. (2008). Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer. Proc Natl Acad Sci USA 105: 14814-14819.
-
(2008)
Proc Natl Acad Sci USA
, vol.105
, pp. 14814-14819
-
-
Wu, B.1
Moulton, H.M.2
Iversen, P.L.3
Jiang, J.4
Li, J.5
Spurney, C.F.6
-
20
-
-
57049102809
-
Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function
-
Yin, H, Moulton, HM, Seow, Y, Boyd, C, Boutilier, J, Iverson, P et al. (2008). Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. Hum Mol Genet 17: 3909-3918.
-
(2008)
Hum Mol Genet
, vol.17
, pp. 3909-3918
-
-
Yin, H.1
Moulton, H.M.2
Seow, Y.3
Boyd, C.4
Boutilier, J.5
Iverson, P.6
-
21
-
-
50549093417
-
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice
-
Jearawiriyapaisarn, N, Moulton, HM, Buckley, B, Roberts, J, Sazani, P, Fucharoen, S et al. (2008). Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. Mol Ther 16: 1624-1629.
-
(2008)
Mol Ther
, vol.16
, pp. 1624-1629
-
-
Jearawiriyapaisarn, N.1
Moulton, H.M.2
Buckley, B.3
Roberts, J.4
Sazani, P.5
Fucharoen, S.6
-
22
-
-
0037172801
-
Translocation of branched-chain arginine peptides through cell membranes: Flexibility in the spatial disposition of positive charges in membrane-permeable peptides
-
Futaki, S, Nakase, I, Suzuki, T, Youjun, Z and Sugiura, Y (2002). Translocation of branched-chain arginine peptides through cell membranes: Flexibility in the spatial disposition of positive charges in membrane-permeable peptides. Biochemistry 41: 7925-7930.
-
(2002)
Biochemistry
, vol.41
, pp. 7925-7930
-
-
Futaki, S.1
Nakase, I.2
Suzuki, T.3
Youjun, Z.4
Sugiura, Y.5
-
23
-
-
0025051336
-
Somatic reversion/suppression of the mouse mdx phenotype in vivo
-
Hoffman, EP, Morgan, JE, Watkins, SC and 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
-
24
-
-
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 and Barnard, PJ (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
Barnard, P.J.6
-
25
-
-
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 et al. (2005). Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci USA 102: 198-203.
-
(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
-
26
-
-
0037733977
-
Antisense technologies. Improvement through novel chemical modifications
-
Kurreck, J (2003). Antisense technologies. Improvement through novel chemical modifications. Eur J Biochem 270: 1628-1644.
-
(2003)
Eur J Biochem
, vol.270
, pp. 1628-1644
-
-
Kurreck, J.1
-
27
-
-
0030862707
-
Morpholino antisense oligomers: Design, preparation, and properties
-
Summerton, J and Weller, D (1997). Morpholino antisense oligomers: design, preparation, and properties. Antisense Nucleic Acid Drug Dev 7: 187-195.
-
(1997)
Antisense Nucleic Acid Drug Dev
, vol.7
, pp. 187-195
-
-
Summerton, J.1
Weller, D.2
-
28
-
-
33847036106
-
Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents
-
Abes, S, Moulton, HM, Clair, P, Prevot, P, Youngblood, DS, Wu, RP et al. (2006). Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents. J Control Release 116: 304-313.
-
(2006)
J Control Release
, vol.116
, pp. 304-313
-
-
Abes, S.1
Moulton, H.M.2
Clair, P.3
Prevot, P.4
Youngblood, D.S.5
Wu, R.P.6
-
29
-
-
47749133787
-
Design and synthesis of the dendritic molecular transporter that achieves efficient in vivo delivery of morpholino antisense oligo
-
Li, YF and Morcos, PA (2008). Design and synthesis of the dendritic molecular transporter that achieves efficient in vivo delivery of morpholino antisense oligo. Bioconjug Chem 19: 1464-1470.
-
(2008)
Bioconjug Chem
, vol.19
, pp. 1464-1470
-
-
Li, Y.F.1
Morcos, P.A.2
|