-
1
-
-
0023718118
-
An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus
-
Monaco AP, Bertelson CJ, Liechti-Gallati S, Moser H, Kunkel LM (1988) An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics 2:90-95
-
(1988)
Genomics
, vol.2
, pp. 90-95
-
-
Monaco, A.P.1
Bertelson, C.J.2
Liechti-Gallati, S.3
Moser, H.4
Kunkel, L.M.5
-
2
-
-
38449095231
-
Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides
-
DOI 10.2741/2697
-
Bertoni C (2008) Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides. Front Biosci 13:517-527 (Pubitemid 351599762)
-
(2008)
Frontiers in Bioscience
, vol.13
, Issue.2
, pp. 517-527
-
-
Bertoni, C.1
-
3
-
-
67650369591
-
Gene therapy for muscular dystrophy: Current progress and future prospects
-
Trollet C, Athanasopoulos T, Popplewell L, Malerba A, Dickson G (2009) Gene therapy for muscular dystrophy: current progress and future prospects. Expert Opin Biol Ther 9: 849-866
-
(2009)
Expert Opin Biol Ther
, vol.9
, pp. 849-866
-
-
Trollet, C.1
Athanasopoulos, T.2
Popplewell, L.3
Malerba, A.4
Dickson, G.5
-
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 Dennen 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
-
-
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 et al (2001) Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells. Hum Mol Genet 10: 1547-1554 (Pubitemid 32734306)
-
(2001)
Human Molecular Genetics
, vol.10
, Issue.15
, pp. 1547-1554
-
-
Van Deutekom, J.C.T.1
Bremmer-Bout, M.2
Janson, A.A.M.3
Ginjaar, I.B.4
Baas, F.5
Den Dunnen, J.T.6
Van Ommen, G.-J.B.7
-
6
-
-
34848904544
-
Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle
-
DOI 10.1089/hum.2006.061
-
Arechavala-Gomeza V, Graham IR, Popplewell LJ, Adams AM, Aartsma-Rus A, Kinali M et al (2007) Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle. Hum Gene Ther 18: 798-810 (Pubitemid 47502846)
-
(2007)
Human Gene Therapy
, vol.18
, Issue.9
, pp. 798-810
-
-
Arechavala-Gomeza, V.1
Graham, I.R.2
Popplewell, L.J.3
Adams, A.M.4
Aartsma-Rus, A.5
Kinali, M.6
Morgan, J.E.7
Van Deutekom, J.C.8
Wilton, S.D.9
Dickson, G.10
Muntoni, F.11
-
7
-
-
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
-
8
-
-
0347003516
-
Antisense-induced multiexon skipping for duchenne muscular dystrophy makes more sense
-
DOI 10.1086/381039
-
Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, van Ommen GJ, den Dunnen JT et al (2004) Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense. Am J Hum Genet 74:83-92 (Pubitemid 38085240)
-
(2004)
American Journal of Human Genetics
, vol.74
, Issue.1
, pp. 83-92
-
-
Aartsma-Rus, A.1
Janson, A.A.M.2
Kaman, W.E.3
Bremmer-Bout, M.4
Van Ommen, G.-J.B.5
Den Dunnen, J.T.6
Van Deutekom, J.C.T.7
-
10
-
-
4344693568
-
Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides
-
DOI 10.1016/j.ymthe.2004.05.031, PII S1525001604002096
-
Bremmer-Bout M, Aartsma-Rus A, de Meijer EJ, Kaman WE, Janson AA, Vossen RH et al (2004) Targeted exon skipping in transgenic hDMD mice: a model for direct preclinical screening of human-specific antisense oligonucleotides. Mol Ther 10:232-240 (Pubitemid 39117869)
-
(2004)
Molecular Therapy
, vol.10
, Issue.2
, pp. 232-240
-
-
Bremmer-Bout, M.1
Aartsma-Rus, A.2
De Meijer, E.J.3
Kaman, W.E.4
Janson, A.A.M.5
Vossen, R.H.A.M.6
Van Ommen, G.-J.B.7
Den Dunnen, J.T.8
Van Deutekom, J.C.T.9
-
11
-
-
13644267068
-
Towards a therapeutic inhibition of dystrophin exon 23 splicing in mdx mouse muscle induced by antisense oligoribonucleotides (splicomers): Target sequence optimisation using oligonucleotide arrays
-
DOI 10.1002/jgm.603
-
Graham IR, Hill VJ, Manoharan M, Inamati GB, Dickson G (2004) Towards a therapeutic inhibition of dystrophin exon 23 splicing in mdx mouse muscle induced by antisense oligoribonucleotides (splicomers): target sequence optimisation using oligonucleotide arrays. J Gene Med 6:1149-1158 (Pubitemid 40228127)
-
(2004)
Journal of Gene Medicine
, vol.6
, Issue.10
, pp. 1149-1158
-
-
Graham, I.R.1
Hill, V.J.2
Manoharan, M.3
Inamati, G.B.4
Dickson, G.5
-
12
-
-
11844256373
-
Systemic delivery of antisense oligoribonucleotide restorers dystrophin expression in body-wide skeletal muscles
-
DOI 10.1073/pnas.0406700102
-
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 (Pubitemid 40094452)
-
(2005)
Proceedings of the National Academy of Sciences of the United States of America
, vol.102
, Issue.1
, pp. 198-203
-
-
Qi, L.L.1
Rabinowitz, A.2
Yun, C.C.3
Yokota, T.4
Yin, H.5
Alter, J.6
Jadoon, A.7
Bou-Gharios, G.8
Partridge, T.9
-
13
-
-
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
-
14
-
-
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
-
15
-
-
37549034298
-
Local antisense dystrophin restoration with antisense oligonucleotide PRO051
-
van Deutekom JC, Janson AA, Ginjaar IB, Franzhuzen WS, Aartsma-Rus A, Bremmer-Bout M et al (2007) Local antisense dystrophin restoration with antisense oligonucleotide PRO051. N Eng J Med 357:2677-2687
-
(2007)
N Eng J Med
, vol.357
, pp. 2677-2687
-
-
Van Deutekom, J.C.1
Janson, A.A.2
Ginjaar, I.B.3
Franzhuzen, W.S.4
Aartsma-Rus, A.5
Bremmer-Bout, M.6
-
16
-
-
69949107887
-
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: A single-blind, placebocontrolled, 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, placebocontrolled, dose-escalation, proof-of-concept study. Lancet Neurol 8(10):918-928
-
(2009)
Lancet Neurol
, vol.8
, Issue.10
, pp. 918-928
-
-
Kinali, M.1
Arechavala-Gomeza, V.2
Feng, L.3
Cirak, S.4
Hunt, D.5
Adkin, C.6
-
17
-
-
4844225667
-
Neutrally charged phosphorodiamidate Morpholino antisense oligomers: Uptake, efficacy and pharmacokinetics
-
DOI 10.2174/1389201043376706
-
Arora V, Devi GR, Iversen PL (2004) Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics. Curr Pharm Biotechnol 5:431-439 (Pubitemid 39317610)
-
(2004)
Current Pharmaceutical Biotechnology
, vol.5
, Issue.5
, pp. 431-439
-
-
Arora, V.1
Devi, G.R.2
Iversen, P.L.3
-
18
-
-
65349121206
-
In vivo comparison of 2'O-methyl-PS and morpholino antisense oligonucleotides for DMD exon skipping
-
Heemskerk HA, de Winter CL, de Kimpe SJ, van Kuik-Romeijn P, Heuvelmans N, Platenburg GJ et al (2009) In vivo comparison of 2'O-methyl-PS and morpholino antisense oligonucleotides for DMD exon skipping. J Gene Med 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
Van Kuik-Romeijn, P.4
Heuvelmans, N.5
Platenburg, G.J.6
-
19
-
-
0043133425
-
Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle
-
DOI 10.1093/hmg/ddg196
-
Gebski BL, Mann CJ, Fletcher S, 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
-
20
-
-
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
-
21
-
-
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, Graham IR (2009) Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice. Hum Gene Ther 20(9):955-965
-
(2009)
Hum Gene Ther
, vol.20
, Issue.9
, pp. 955-965
-
-
Malerba, A.1
Thorogood, F.C.2
Dickson, G.3
Graham, I.R.4
-
22
-
-
33748566255
-
Induced dystrophin exon skipping in human muscle explants
-
DOI 10.1016/j.nmd.2006.05.017, PII S096089660600174X
-
McClorey G, Fall AM, Moulton HM, Iversen PL, Rasko JE, Ryan M et al (2006) Induced dystrophin exon skipping in human muscle explants. Neuromuscul Disord 16:583-590 (Pubitemid 44374319)
-
(2006)
Neuromuscular Disorders
, vol.16
, Issue.9-10
, pp. 583-590
-
-
McClorey, G.1
Fall, A.M.2
Moulton, H.M.3
Iversen, P.L.4
Rasko, J.E.5
Ryan, M.6
Fletcher, S.7
Wilton, S.D.8
-
23
-
-
33745479703
-
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
-
DOI 10.1038/sj.gt.3302800, PII 3302800
-
McClorey G, Moulton HM, Iversen PL, Fletcher S, Wilton SD (2006) Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Ther 13:1373-1381 (Pubitemid 44420632)
-
(2006)
Gene Therapy
, vol.13
, Issue.19
, pp. 1373-1381
-
-
McClorey, G.1
Moulton, H.M.2
Iversen, P.L.3
Fletcher, S.4
Wilton, S.D.5
-
24
-
-
61649097962
-
Theoretic applicability of antisensemediated exon skipping for Duchenne muscular dystrophy mutations
-
Aartsma-Rus A, Fokkema I, Verschuuren J, Ginjaar I, van Deutekom J, van Ommen GJ et al (2009) Theoretic applicability of antisensemediated exon skipping for Duchenne muscular dystrophy mutations. Hum Mutat 30:292-299
-
(2009)
Hum Mutat
, vol.30
, pp. 292-299
-
-
Aartsma-Rus, A.1
Fokkema, I.2
Verschuuren, J.3
Ginjaar, I.4
Van Deutekom, J.5
Van Ommen, G.J.6
-
25
-
-
30844436415
-
Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: Indication for steric hindrance of SR protein binding sites
-
DOI 10.1089/oli.2005.15.284
-
Aartsma-Rus A, De Winter CL, Janson AAM, Kaman WE, van Ommen G-JB, Den Dunnen JT et al (2005) Functional analysis of 114 exoninternal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites. Oligonucleotides 15: 284-297 (Pubitemid 43108225)
-
(2005)
Oligonucleotides
, vol.15
, Issue.4
, pp. 284-297
-
-
Aartsma-Rus, A.1
De Winter, C.L.2
Janson, A.A.M.3
Kaman, W.E.4
Van Ommen, G.-J.B.5
Den Dunnen, J.T.6
Van Deutekom, J.C.T.7
-
26
-
-
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, 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
-
27
-
-
61649127296
-
Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms
-
Aartsma-Rus A, van Vliet L, Hirschi M, Janson AA, Heemskerk H, de Winter CL et al (2009) Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms. Mol Ther 17:548-553
-
(2009)
Mol Ther
, vol.17
, pp. 548-553
-
-
Aartsma-Rus, A.1
Van Vliet, L.2
Hirschi, M.3
Janson, A.A.4
Heemskerk, H.5
De Winter, C.L.6
-
28
-
-
61649109478
-
Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene
-
Popplewell LJ, Trollet C, Dickson G, Graham IR (2009) Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene. Mol Ther 17:554-561
-
(2009)
Mol Ther
, vol.17
, pp. 554-561
-
-
Popplewell, L.J.1
Trollet, C.2
Dickson, G.3
Graham, I.R.4
|