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Volumn 15, Issue 9, 2007, Pages 1587-1592

Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CELL PENETRATING PEPTIDE; DYSTROPHIN; PHOSHORODIAMIDATE MORPHOLINO OLIGONUCLEOTIDE; UNCLASSIFIED DRUG;

EID: 34547691961     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/sj.mt.6300245     Document Type: Article
Times cited : (141)

References (48)
  • 1
    • 26844494867 scopus 로고    scopus 로고
    • RNA splicing manipulation: Strategies to modify gene expression for a variety of therapeutic outcomes
    • Wilton, SD and Fletcher, S (2005). RNA splicing manipulation: Strategies to modify gene expression for a variety of therapeutic outcomes. Curr Gene Ther 5: 467-483.
    • (2005) Curr Gene Ther , vol.5 , pp. 467-483
    • Wilton, S.D.1    Fletcher, S.2
  • 2
    • 19344362163 scopus 로고    scopus 로고
    • Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: Where are we now?
    • Wilton, SD and Fletcher, S (2005). Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: Where are we now? Neuromuscul Disord 15: 399-402.
    • (2005) Neuromuscul Disord , vol.15 , pp. 399-402
    • Wilton, S.D.1    Fletcher, S.2
  • 3
    • 18844442551 scopus 로고    scopus 로고
    • Gene therapy: The 'pro-sense' approach to Duchenne muscular dystrophy
    • van Deutekom, JC (2005). Gene therapy: The 'pro-sense' approach to Duchenne muscular dystrophy. Eur J Hum Genet 13: 518-519.
    • (2005) Eur J Hum Genet , vol.13 , pp. 518-519
    • van Deutekom, J.C.1
  • 4
    • 0141594934 scopus 로고    scopus 로고
    • Advances in Duchenne muscular dystrophy gene therapy
    • van Deutekom, JC and van Ommen, GJ (2003). Advances in Duchenne muscular dystrophy gene therapy. Nat Rev Genet 4: 774-783.
    • (2003) Nat Rev Genet , vol.4 , pp. 774-783
    • van Deutekom, J.C.1    van Ommen, G.J.2
  • 5
    • 0024527173 scopus 로고
    • Clinical and molecular studies in Duchenne muscular dystrophy
    • Emery, AE (1989). Clinical and molecular studies in Duchenne muscular dystrophy. Prog Clin Biol Res 306: 15-28.
    • (1989) Prog Clin Biol Res , vol.306 , pp. 15-28
    • Emery, A.E.1
  • 6
    • 0036227796 scopus 로고    scopus 로고
    • Muscular dystrophy into the new millennium
    • Emery, AE (2002). Muscular dystrophy into the new millennium. Neuromuscul Disord 12: 343-349.
    • (2002) Neuromuscul Disord , vol.12 , pp. 343-349
    • Emery, A.E.1
  • 7
    • 0024815723 scopus 로고
    • Topography of the Duchenne muscular dystrophy (DMD) gene: FIGE and cDNA analysis of 194 cases reveals 115 deletions and 13 duplications
    • Den Dunnen, JT, Grootscholten, PM, Bakker, E, Blonden, LA, Ginjaar, HB, Wapenaar, MC et al. (1989). Topography of the Duchenne muscular dystrophy (DMD) gene: FIGE and cDNA analysis of 194 cases reveals 115 deletions and 13 duplications. Am J Hum Genet 45: 835-847.
    • (1989) Am J Hum Genet , vol.45 , pp. 835-847
    • Den Dunnen, J.T.1    Grootscholten, P.M.2    Bakker, E.3    Blonden, L.A.4    Ginjaar, H.B.5    Wapenaar, M.C.6
  • 8
    • 0344420060 scopus 로고    scopus 로고
    • Dystrophin and mutations: One gene, several proteins, multiple phenotypes
    • Muntoni, F, Torelli, S and Ferlini, A (2003). Dystrophin and mutations: one gene, several proteins, multiple phenotypes. Lancet Neurol 2: 731-740.
    • (2003) Lancet Neurol , vol.2 , pp. 731-740
    • Muntoni, F.1    Torelli, S.2    Ferlini, A.3
  • 9
    • 0035094729 scopus 로고    scopus 로고
    • Novel dystrophin mutations revealed by analysis of dystrophin mRNA: Alternative splicing suppresses the phenotypic effect of a nonsense mutation
    • Fajkusova, L, Lukas, Z, Tvrdikova, M, Kuhrova, V, Hajek, J and Fajkus, J (2001). Novel dystrophin mutations revealed by analysis of dystrophin mRNA: Alternative splicing suppresses the phenotypic effect of a nonsense mutation. Neuromuscul Disord 11: 133-138.
    • (2001) Neuromuscul Disord , vol.11 , pp. 133-138
    • Fajkusova, L.1    Lukas, Z.2    Tvrdikova, M.3    Kuhrova, V.4    Hajek, J.5    Fajkus, J.6
  • 10
    • 0028303798 scopus 로고
    • Searching for the 1 in 2,400,000: A review of dystrophin gene point mutations
    • Roberts, RG, Gardner, RJ and Bobrow, M (1994). Searching for the 1 in 2,400,000: A review of dystrophin gene point mutations. Hum Mutat 4: 1-11.
    • (1994) Hum Mutat , vol.4 , pp. 1-11
    • Roberts, R.G.1    Gardner, R.J.2    Bobrow, M.3
  • 11
    • 0023718118 scopus 로고
    • An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus
    • Monaco, AP, Bertelson, CJ, Liechti-Gallati, S, Moser, H and 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
  • 12
    • 0028347196 scopus 로고
    • Clinical variability in Becker muscular dystrophy. Genetic, biochemical and immunohistochemical correlates
    • Comi, GP, Prelle, A, Bresolin, N, Moggio, M, Bardoni, A, Gallanti, A et al. (1994). Clinical variability in Becker muscular dystrophy. Genetic, biochemical and immunohistochemical correlates. Brain 117 (Pt 1): 1-14.
    • (1994) Brain , vol.117 , Issue.PART 1 , pp. 1-14
    • Comi, G.P.1    Prelle, A.2    Bresolin, N.3    Moggio, M.4    Bardoni, A.5    Gallanti, A.6
  • 14
    • 0025647598 scopus 로고
    • Effect of dystrophin gene deletions on mRNA levels and processing in Duchenne and Becker muscular dystrophies
    • Chelly, J, Gilgenkrantz, H, Lambert, M, Hamard, G, Chafey, P, Recan, D et al. (1990). Effect of dystrophin gene deletions on mRNA levels and processing in Duchenne and Becker muscular dystrophies. Cell 63: 1239-1248.
    • (1990) Cell , vol.63 , pp. 1239-1248
    • Chelly, J.1    Gilgenkrantz, H.2    Lambert, M.3    Hamard, G.4    Chafey, P.5    Recan, D.6
  • 16
    • 0031722730 scopus 로고    scopus 로고
    • 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
  • 17
    • 0034611016 scopus 로고    scopus 로고
    • 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
  • 18
    • 0030767225 scopus 로고    scopus 로고
    • Revertant fibres: A possible genetic therapy for Duchenne muscular dystrophy?
    • Wilton, SD, Dye, DE, Blechynden, LM and Laing, NG (1997). Revertant fibres: A possible genetic therapy for Duchenne muscular dystrophy? Neuromuscul Disord 7: 329-335.
    • (1997) Neuromuscul Disord , vol.7 , pp. 329-335
    • Wilton, S.D.1    Dye, D.E.2    Blechynden, L.M.3    Laing, N.G.4
  • 19
    • 0027487938 scopus 로고
    • 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
  • 20
    • 0028910144 scopus 로고
    • Characterization of revertant muscle fibers in Duchenne muscular dystrophy, using exon-specific monoclonal antibodies against dystrophin
    • Thanh, LT, Nguyen, TM, Helliwell, TR and Morris, GE (1995). Characterization of revertant muscle fibers in Duchenne muscular dystrophy, using exon-specific monoclonal antibodies against dystrophin. Am J Hum Genet 56: 725-731.
    • (1995) Am J Hum Genet , vol.56 , pp. 725-731
    • Thanh, L.T.1    Nguyen, T.M.2    Helliwell, T.R.3    Morris, G.E.4
  • 21
    • 0027183926 scopus 로고
    • Immunohistological evidence for second or somatic mutations as the underlying cause of dystrophin expression by isolated fibres in Xp21 muscular dystrophy of Duchenne-type severity
    • Wallgren-Pettersson, C, Jasani, B, Rosser, LG, Lazarou, LP, Nicholson, LV and Clarke, A (1993). Immunohistological evidence for second or somatic mutations as the underlying cause of dystrophin expression by isolated fibres in Xp21 muscular dystrophy of Duchenne-type severity. J Neurol Sci 118: 56-63.
    • (1993) J Neurol Sci , vol.118 , pp. 56-63
    • Wallgren-Pettersson, C.1    Jasani, B.2    Rosser, L.G.3    Lazarou, L.P.4    Nicholson, L.V.5    Clarke, A.6
  • 22
    • 4644227797 scopus 로고    scopus 로고
    • Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells
    • 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.
    • (2004) Gene Ther , vol.11 , pp. 1391-1398
    • Aartsma-Rus, A.1    Kaman, W.E.2    Bremmer-Bout, M.3    Janson, A.A.4    den Dunnen, J.T.5    van Ommen, G.J.6
  • 23
    • 32844460899 scopus 로고    scopus 로고
    • Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotice
    • Fletcher, S, Honeyman, K, Fall, AM, Harding, PL, Johnsen, RD and Wilton, SD (2006). Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotice. 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
  • 25
    • 0043133425 scopus 로고    scopus 로고
    • Morpholino antisense oligonucleotice induced dystrophin exon 23 skipping in mdx mouse muscle
    • Gebski, BL, Mann, CJ, Fletcher, S and Wilton, SD (2003). Morpholino antisense oligonucleotice induced dystrophin exon 23 skipping in mdx mouse muscle. Hum Mol Genet 12: 1801-1811.
    • (2003) Hum Mol Genet , vol.12 , pp. 1801-1811
    • Gebski, B.L.1    Mann, C.J.2    Fletcher, S.3    Wilton, S.D.4
  • 26
  • 27
    • 32244443828 scopus 로고    scopus 로고
    • 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
  • 29
    • 0013181060 scopus 로고    scopus 로고
    • Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy
    • Mann, CJ, Honeyman, K, McClorey, G, Fletcher, S and Wilton, SD (2002). Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy. J Gene Med 4: 644-654.
    • (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
  • 30
    • 1542344795 scopus 로고    scopus 로고
    • Therapeutics for Duchenne muscular dystrophy: Current approaches and future directions
    • Bogdanovich, S, Perkins, KJ, Krag, TO and Khurana, TS (2004). Therapeutics for Duchenne muscular dystrophy: Current approaches and future directions. J Mol Med 82: 102-115.
    • (2004) J Mol Med , vol.82 , pp. 102-115
    • Bogdanovich, S.1    Perkins, K.J.2    Krag, T.O.3    Khurana, T.S.4
  • 32
    • 0036824144 scopus 로고    scopus 로고
    • Steroids in Duchenne muscular dystrophy: From clinical trials to genomic research
    • Muntoni, F, Fisher, I, Morgan, JE and Abraham, D (2002). Steroids in Duchenne muscular dystrophy: From clinical trials to genomic research. Neuromuscul Disord 12 (Suppl. 1): S162-S165.
    • (2002) Neuromuscul Disord , vol.12 , Issue.SUPPL. 1
    • Muntoni, F.1    Fisher, I.2    Morgan, J.E.3    Abraham, D.4
  • 33
    • 0036895043 scopus 로고    scopus 로고
    • Survival in Duchenne muscular dystrophy: Improvements in life expectancy since 1967 and the impact of home nocturnal ventilation
    • Eagle, M, Baudouin, SV, Chandler, C, Giddings, DR, Bullock, R and Bushby, K (2002). Survival in Duchenne muscular dystrophy: Improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. Neuromuscul Disord 12: 926-929.
    • (2002) Neuromuscul Disord , vol.12 , pp. 926-929
    • Eagle, M.1    Baudouin, S.V.2    Chandler, C.3    Giddings, D.R.4    Bullock, R.5    Bushby, K.6
  • 35
    • 0037447517 scopus 로고    scopus 로고
    • 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 et al. (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
  • 36
    • 0042536463 scopus 로고    scopus 로고
    • 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
  • 37
    • 11844256373 scopus 로고    scopus 로고
    • Systemic delivery of antisense oligoribonucleotice 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 oligoribonucleotice 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
  • 38
    • 0037479844 scopus 로고    scopus 로고
    • Efficacy of antisense morpholino oligomer targeted to c-myc in prostate cancer xenograft murine model and a phase I safety study in humans
    • Iversen, PL, Arora, V, Acker, AJ, Mason, DH and Devi, GR (2003). Efficacy of antisense morpholino oligomer targeted to c-myc in prostate cancer xenograft murine model and a phase I safety study in humans. Clin Cancer Res 9: 2510-2519.
    • (2003) Clin Cancer Res , vol.9 , pp. 2510-2519
    • Iversen, P.L.1    Arora, V.2    Acker, A.J.3    Mason, D.H.4    Devi, G.R.5
  • 39
    • 0034939942 scopus 로고    scopus 로고
    • Phosphorodiamidate morpholino oligomers: Favorable properties for sequence-specific gene inactivation
    • Iversen, PL (2001). Phosphorodiamidate morpholino oligomers: Favorable properties for sequence-specific gene inactivation. Curr Opin Mol Ther 3: 235-238.
    • (2001) Curr Opin Mol Ther , vol.3 , pp. 235-238
    • Iversen, P.L.1
  • 40
    • 0032699477 scopus 로고    scopus 로고
    • Morpholino antisense oligomers: The case for an RNase H-independent structural type
    • Summerton, J (1999). Morpholino antisense oligomers: The case for an RNase H-independent structural type. Biochim Biophys Acta 1489 141-158.
    • (1999) Biochim Biophys Acta , vol.1489 , pp. 141-158
    • Summerton, J.1
  • 45
    • 33847036106 scopus 로고    scopus 로고
    • Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptice allows efficient splicing correction in the absence of endosomolytic agents
    • Abes, S, Moulton, HM, Clair, P, Prevot, F, Youngblood, DS, Wu, RP et al. (2006). Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptice 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, F.4    Youngblood, D.S.5    Wu, R.P.6
  • 46
    • 33745479703 scopus 로고    scopus 로고
    • Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
    • McClorey, G, Moulton, HM, Iversen, PL, Fletcher, S and Wilton, SD (2006). Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Ther 13: 1373-1381.
    • (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


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.