-
1
-
-
77950793623
-
RNA-targeted splice-correction therapy for neuromuscular disease
-
Wood, MJ, Gait, MJ and Yin, H (2010). RNA-targeted splice-correction therapy for neuromuscular disease. Brain 133(Pt 4): 957-972.
-
(2010)
Brain
, vol.133
, Issue.PART 4
, pp. 957-972
-
-
Wood, M.J.1
Gait, M.J.2
Yin, H.3
-
2
-
-
77953017904
-
Toward an oligonucleotide therapy for Duchenne muscular dystrophy: A complex development challenge
-
Wood, MJ (2010). Toward an oligonucleotide therapy for Duchenne muscular dystrophy: a complex development challenge. Sci Transl Med 2: 25ps15.
-
(2010)
Sci Transl Med
, vol.2
-
-
Wood, M.J.1
-
3
-
-
4644227797
-
Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells
-
DOI 10.1038/sj.gt.3302313
-
Aartsma-Rus, A, Kaman, WE, Bremmer-Bout, M, Janson, AA, den Dunnen, JT, van Ommen, GJ et al. (2004). Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells. Gene Ther 11: 1391-1398. (Pubitemid 39263892)
-
(2004)
Gene Therapy
, vol.11
, Issue.18
, pp. 1391-1398
-
-
Aartsma-Rus, A.1
Kaman, W.E.2
Bremmer-Bout, M.3
Janson, A.A.M.4
Den Dunnen, J.T.5
Van Ommen, G.-J.B.6
Van Deutekom, J.C.T.7
-
4
-
-
0037447517
-
Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients
-
DOI 10.1093/hmg/ddg100
-
Aartsma-Rus, A, Janson, AA, Kaman, WE, Bremmer-Bout, M, den Dunnen, JT, Baas, F et al. (2003). Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum Mol Genet 12: 907-914. (Pubitemid 36504033)
-
(2003)
Human Molecular Genetics
, vol.12
, Issue.8
, pp. 907-914
-
-
Aartsma-Rus, A.1
Janson, A.A.M.2
Kaman, W.E.3
Bremmer-Bout, M.4
Den Dunnen, T.J.T.5
Baas, F.6
Van Ommen, G.-J.B.7
Van Deutekom, J.C.T.8
-
5
-
-
33845667857
-
Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides
-
DOI 10.1196/annals.1348.058, Oligonucleotide Therapeutics: First Annual Meeting of the Oligonucleotide Therapeutics Society
-
Aartsma-Rus, A, Janson, AA, Heemskerk, JA, De Winter, CL, Van Ommen, GJ and Van Deutekom, JC (2006). Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides. Ann N Y Acad Sci 1082: 74-76. (Pubitemid 44955452)
-
(2006)
Annals of the New York Academy of Sciences
, vol.1082
, pp. 74-76
-
-
Aartsma-Rus, A.1
Janson, A.A.M.2
Heemskerk, J.A.3
De Winter, C.L.4
Van Ommen, G.-J.B.5
Van Deutekom, J.C.T.6
-
6
-
-
32244443828
-
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
-
DOI 10.1038/nm1345, PII NM1345
-
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. (Pubitemid 43214742)
-
(2006)
Nature Medicine
, vol.12
, Issue.2
, 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
Qi, L.L.8
-
7
-
-
0043133425
-
Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle
-
DOI 10.1093/hmg/ddg196
-
Gebski, BL, Mann, CJ, Fletcher, S and Wilton, SD (2003). Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle. Hum Mol Genet 12: 1801-1811. (Pubitemid 36944135)
-
(2003)
Human Molecular Genetics
, vol.12
, Issue.15
, pp. 1801-1811
-
-
Gebski, B.L.1
Mann, C.J.2
Fletcher, S.3
Wilton, S.D.4
-
8
-
-
0042536463
-
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
-
DOI 10.1038/nm897
-
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. (Pubitemid 37021780)
-
(2003)
Nature Medicine
, vol.9
, Issue.8
, 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
-
9
-
-
0035793047
-
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse
-
DOI 10.1073/pnas.011408598
-
Mann, CJ, Honeyman, K, Cheng, AJ, Ly, T, Lloyd, F, Fletcher, S et al. (2001). Antisenseinduced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci USA 98: 42-47. (Pubitemid 32095860)
-
(2001)
Proceedings of the National Academy of Sciences of the United States of America
, vol.98
, Issue.1
, 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
-
10
-
-
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
-
11
-
-
34248511708
-
Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript
-
DOI 10.1038/sj.mt.6300095, PII 6300095
-
Wilton, SD, Fall, AM, Harding, PL, McClorey, G, Coleman, C and Fletcher, S (2007). Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript. Mol Ther 15: 1288-1296. (Pubitemid 46965325)
-
(2007)
Molecular Therapy
, vol.15
, Issue.7
, pp. 1288-1296
-
-
Wilton, S.D.1
Fall, A.M.2
Harding, P.L.3
McClorey, G.4
Coleman, C.5
Fletcher, S.6
-
12
-
-
37549022222
-
Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice
-
Yin, H, Lu, Q and Wood, M (2008). Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice. Mol Ther 16: 38-45.
-
(2008)
Mol Ther
, vol.16
, pp. 38-45
-
-
Yin, H.1
Lu, Q.2
Wood, M.3
-
13
-
-
57049102809
-
Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function
-
DOI 10.1093/hmg/ddn293
-
Yin, H, Moulton, HM, Seow, Y, Boyd, C, Boutilier, J, Iverson, P et al. (2008). Cellpenetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. Hum Mol Genet 17: 3909-3918. (Pubitemid 352762853)
-
(2008)
Human Molecular Genetics
, vol.17
, Issue.24
, pp. 3909-3918
-
-
Yin, H.1
Moulton, H.M.2
Seow, Y.3
Boyd, C.4
Boutilier, J.5
Iverson, P.6
Wood, M.J.A.7
-
14
-
-
70350697818
-
A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice
-
Yin, H, Moulton, HM, Betts, C, Seow, Y, Boutilier, J, Iverson, PL et al. (2009). A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice. Hum Mol Genet 18: 4405-4414.
-
(2009)
Hum Mol Genet
, vol.18
, pp. 4405-4414
-
-
Yin, H.1
Moulton, H.M.2
Betts, C.3
Seow, Y.4
Boutilier, J.5
Iverson, P.L.6
-
15
-
-
77950898446
-
In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy
-
Wang, Q, Yin, H, Camelliti, P, Betts, C, Moulton, H, Lee, H et al. (2010). In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy. J Gene Med 12: 354-364.
-
(2010)
J Gene Med
, vol.12
, pp. 354-364
-
-
Wang, Q.1
Yin, H.2
Camelliti, P.3
Betts, C.4
Moulton, H.5
Lee, H.6
-
16
-
-
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
-
17
-
-
4043092073
-
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
-
DOI 10.1038/nm1085
-
Gregorevic, P, Blankinship, MJ, Allen, JM, Crawford, RW, Meuse, L, Miller, DG et al. (2004). Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med 10: 828-834. (Pubitemid 39070856)
-
(2004)
Nature Medicine
, vol.10
, Issue.8
, pp. 828-834
-
-
Gregorevic, P.1
Blankinship, M.J.2
Allen, J.M.3
Crawford, R.W.4
Meuse, L.5
Miller, D.G.6
Russell, D.W.7
Chamberlain, J.S.8
-
18
-
-
69949107887
-
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: A single-blind, placebo-controlled, dose-escalation, proof-of-concept study
-
Kinali, M, Arechavala-Gomeza, V, Feng, L, Cirak, S, Hunt, D, Adkin, C et al. (2009). Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol 8: 918-928.
-
(2009)
Lancet Neurol
, vol.8
, pp. 918-928
-
-
Kinali, M.1
Arechavala-Gomeza, V.2
Feng, L.3
Cirak, S.4
Hunt, D.5
Adkin, C.6
-
19
-
-
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
-
20
-
-
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, Li, J 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
Li, J.6
-
21
-
-
77957605909
-
Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO
-
Yin, H, Moulton, HM, Betts, C, Merritt, T, Seow, Y, Ashraf, S et al. (2010). Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO. Mol Ther 18: 1822-1829.
-
(2010)
Mol Ther
, vol.18
, pp. 1822-1829
-
-
Yin, H.1
Moulton, H.M.2
Betts, C.3
Merritt, T.4
Seow, Y.5
Ashraf, S.6
-
22
-
-
56649113066
-
Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle
-
Ivanova, GD, Arzumanov, A, Abes, R, Yin, H, Wood, MJ, Lebleu, B et al. (2008). Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle. Nucleic Acids Res 36: 6418-6428.
-
(2008)
Nucleic Acids Res
, vol.36
, pp. 6418-6428
-
-
Ivanova, G.D.1
Arzumanov, A.2
Abes, R.3
Yin, H.4
Wood, M.J.5
Lebleu, B.6
-
23
-
-
77950518145
-
Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse
-
Yin, H, Betts, C, Saleh, AF, Ivanova, GD, Lee, H, Seow, Y et al. (2010). Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse. Mol Ther 18: 819-827.
-
(2010)
Mol Ther
, vol.18
, pp. 819-827
-
-
Yin, H.1
Betts, C.2
Saleh, A.F.3
Ivanova, G.D.4
Lee, H.5
Seow, Y.6
-
24
-
-
13344277364
-
Interaction of nitric oxide synthase with the postsynaptic density protein PSD-95 and α1-syntrophin mediated by PDZ domains
-
DOI 10.1016/S0092-8674(00)81053-3
-
Brenman, JE, Chao, DS, Gee, SH, McGee, AW, Craven, SE, Santillano, DR et al. (1996). Interaction of nitric oxide synthase with the postsynaptic density protein PSD-95 and alpha1-syntrophin mediated by PDZ domains. Cell 84: 757-767. (Pubitemid 26094205)
-
(1996)
Cell
, vol.84
, Issue.5
, pp. 757-767
-
-
Brenman, J.E.1
Chao, D.S.2
Gee, S.H.3
McGee, A.W.4
Craven, S.E.5
Santillano, D.R.6
Wu, Z.7
Huang, F.8
Xia, H.9
Peters, M.F.10
Froehner, S.C.11
Bredt, D.S.12
-
25
-
-
0034687238
-
Alternative splicing of dystrobrevin regulates the stoichiometry of syntrophin binding to the dystrophin protein complex
-
Newey, SE, Benson, MA, Ponting, CP, Davies, KE and Blake, DJ (2000). Alternative splicing of dystrobrevin regulates the stoichiometry of syntrophin binding to the dystrophin protein complex. Curr Biol 10: 1295-1298.
-
(2000)
Curr Biol
, vol.10
, pp. 1295-1298
-
-
Newey, S.E.1
Benson, M.A.2
Ponting, C.P.3
Davies, K.E.4
Blake, D.J.5
-
26
-
-
0023780812
-
Serum CK, calcium, magnesium, and oxidative phosphorylation in mdx mouse muscular dystrophy
-
Glesby, MJ, Rosenmann, E, Nylen, EG and Wrogemann, K (1988). Serum CK, calcium, magnesium, and oxidative phosphorylation in mdx mouse muscular dystrophy. Muscle Nerve 11: 852-856.
-
(1988)
Muscle Nerve
, vol.11
, pp. 852-856
-
-
Glesby, M.J.1
Rosenmann, E.2
Nylen, E.G.3
Wrogemann, K.4
-
27
-
-
77950658740
-
Heart-specific overexpression of CUGBP1 reproduces functional and molecular abnormalities of myotonic dystrophy type 1
-
Koshelev, M, Sarma, S, Price, RE, Wehrens, XH and Cooper, TA (2010). Heart-specific overexpression of CUGBP1 reproduces functional and molecular abnormalities of myotonic dystrophy type 1. Hum Mol Genet 19: 1066-1075.
-
(2010)
Hum Mol Genet
, vol.19
, pp. 1066-1075
-
-
Koshelev, M.1
Sarma, S.2
Price, R.E.3
Wehrens, X.H.4
Cooper, T.A.5
-
28
-
-
72849117063
-
PKC inhibition ameliorates the cardiac phenotype in a mouse model of myotonic dystrophy type 1
-
Wang, GS, Kuyumcu-Martinez, MN, Sarma, S, Mathur, N, Wehrens, XH and Cooper, TA (2009). PKC inhibition ameliorates the cardiac phenotype in a mouse model of myotonic dystrophy type 1. J Clin Invest 119: 3797-3806.
-
(2009)
J Clin Invest
, vol.119
, pp. 3797-3806
-
-
Wang, G.S.1
Kuyumcu-Martinez, M.N.2
Sarma, S.3
Mathur, N.4
Wehrens, X.H.5
Cooper, T.A.6
-
29
-
-
74349109205
-
Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino
-
Wu, B, Lu, P, Benrashid, E, Malik, S, Ashar, J, Doran, TJ et al. (2010). Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino. Gene Ther 17: 132-140.
-
(2010)
Gene Ther
, vol.17
, pp. 132-140
-
-
Wu, B.1
Lu, P.2
Benrashid, E.3
Malik, S.4
Ashar, J.5
Doran, T.J.6
-
30
-
-
67349230291
-
Microbubble stability is a major determinant of the efficiency of ultrasound and microbubble mediated in vivo gene transfer
-
Alter, J, Sennoga, CA, Lopes, DM, Eckersley, RJ and Wells, DJ (2009). Microbubble stability is a major determinant of the efficiency of ultrasound and microbubble mediated in vivo gene transfer. Ultrasound Med Biol 35: 976-984.
-
(2009)
Ultrasound Med Biol
, vol.35
, pp. 976-984
-
-
Alter, J.1
Sennoga, C.A.2
Lopes, D.M.3
Eckersley, R.J.4
Wells, D.J.5
-
31
-
-
0023790515
-
A rat antibody against a structure functionally related to the mouse T-cell receptor/T3 complex
-
DOI 10.1007/BF00355379
-
Tomonari, K (1988). A rat antibody against a structure functionally related to the mouse T-cell receptor/T3 complex. Immunogenetics 28: 455-458. (Pubitemid 18262880)
-
(1988)
Immunogenetics
, vol.28
, Issue.6
, pp. 455-458
-
-
Tomonari, K.1
-
32
-
-
70450263512
-
Tissue slices from adult mammalian hearts as a model for pharmacological drug testing
-
Bussek, A, Wettwer, E, Christ, T, Lohmann, H, Camelliti, P and Ravens, U (2009). Tissue slices from adult mammalian hearts as a model for pharmacological drug testing. Cell Physiol Biochem 24: 527-536.
-
(2009)
Cell Physiol Biochem
, vol.24
, pp. 527-536
-
-
Bussek, A.1
Wettwer, E.2
Christ, T.3
Lohmann, H.4
Camelliti, P.5
Ravens, U.6
|