메뉴 건너뛰기




Volumn 96, Issue 11, 2011, Pages 1101-1113

Progress in therapy for Duchenne muscular dystrophy

Author keywords

[No Author keywords available]

Indexed keywords

AMINOGLYCOSIDE ANTIBIOTIC AGENT; DYSTROPHIN ASSOCIATED PROTEIN COMPLEX; GENTAMICIN; UTROPHIN;

EID: 80055064588     PISSN: 09580670     EISSN: 1469445X     Source Type: Journal    
DOI: 10.1113/expphysiol.2010.053025     Document Type: Article
Times cited : (59)

References (136)
  • 4
    • 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 HF, 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.F.4    Rosenfeld, J.5    Wilton, S.D.6    Partridge, T.A.7    Lu, Q.L.8
  • 5
    • 0033544850 scopus 로고    scopus 로고
    • Utrophin lacks the rod domain actin binding activity of dystrophin
    • Amann KJ, Guo AW & Ervasti JM (1999). Utrophin lacks the rod domain actin binding activity of dystrophin. J Biol Chem 274, 35375-35380.
    • (1999) J Biol Chem , vol.274 , pp. 35375-35380
    • Amann, K.J.1    Guo, A.W.2    Ervasti, J.M.3
  • 6
    • 0032561199 scopus 로고    scopus 로고
    • A cluster of basic repeats in the dystrophin rod domain binds F-actin through an electrostatic interaction
    • Amann KJ, Renley BA & Ervasti JM (1998). A cluster of basic repeats in the dystrophin rod domain binds F-actin through an electrostatic interaction. J Biol Chem 273, 28419-28423.
    • (1998) J Biol Chem , vol.273 , pp. 28419-28423
    • Amann, K.J.1    Renley, B.A.2    Ervasti, J.M.3
  • 10
    • 80055046458 scopus 로고    scopus 로고
    • Utrophin upregulation and microRNAs - two avenues of Duchenne muscular dystrophy therapy research. D.Phil. Thesis, Department of Physiology, Anatomy and Genetics, Oxford University, Oxford, UK.
    • Bareja A (2011). Utrophin upregulation and microRNAs - two avenues of Duchenne muscular dystrophy therapy research. D.Phil. Thesis, Department of Physiology, Anatomy and Genetics, Oxford University, Oxford, UK.
    • (2011)
    • Bareja, A.1
  • 11
    • 33645739391 scopus 로고    scopus 로고
    • Treatment of the heart in Duchenne muscular dystrophy
    • Baxter P (2006). Treatment of the heart in Duchenne muscular dystrophy. Dev Med Child Neurol 48, 163.
    • (2006) Dev Med Child Neurol , vol.48 , pp. 163
    • Baxter, P.1
  • 13
    • 1942489186 scopus 로고    scopus 로고
    • Isolation and enrichment of skeletal muscle progenitor cells from mouse bone marrow
    • Bhagavati S & Xu W (2004). Isolation and enrichment of skeletal muscle progenitor cells from mouse bone marrow. Biochem Biophys Res Commun 318, 119-124.
    • (2004) Biochem Biophys Res Commun , vol.318 , pp. 119-124
    • Bhagavati, S.1    Xu, W.2
  • 14
    • 1542344795 scopus 로고    scopus 로고
    • Therapeutics for Duchenne muscular dystrophy: current approaches and future directions
    • Bogdanovich S, Perkins KJ, Krag TO & 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
  • 15
    • 0033598694 scopus 로고    scopus 로고
    • A second promoter provides an alternative target for therapeutic up-regulation of utrophin in Duchenne muscular dystrophy
    • Burton EA, Tinsley JM, Holzfeind PJ, Rodrigues NR & Davies KE (1999). A second promoter provides an alternative target for therapeutic up-regulation of utrophin in Duchenne muscular dystrophy. Proc Natl Acad Sci USA 96, 14025-14030.
    • (1999) Proc Natl Acad Sci USA , vol.96 , pp. 14025-14030
    • Burton, E.A.1    Tinsley, J.M.2    Holzfeind, P.J.3    Rodrigues, N.R.4    Davies, K.E.5
  • 16
    • 71549172816 scopus 로고    scopus 로고
    • Interventions for muscular dystrophy: molecular medicines entering the clinic
    • Bushby K, Lochmuller H, Lynn S & Straub V (2009). Interventions for muscular dystrophy: molecular medicines entering the clinic. Lancet 374, 1849-1856.
    • (2009) Lancet , vol.374 , pp. 1849-1856
    • Bushby, K.1    Lochmuller, H.2    Lynn, S.3    Straub, V.4
  • 18
    • 0010728863 scopus 로고    scopus 로고
    • Dystrophin levels required for genetic correction of Duchenne muscular dystrophy
    • Chamberlain JS (1997). Dystrophin levels required for genetic correction of Duchenne muscular dystrophy. Basic and Applied Myology 7, 255-257.
    • (1997) Basic and Applied Myology , vol.7 , pp. 255-257
    • Chamberlain, J.S.1
  • 20
    • 0034638834 scopus 로고    scopus 로고
    • Expression profiling in the muscular dystrophies: identification of novel aspects of molecular pathophysiology
    • Chen YW, Zhao P, Borup R & Hoffman EP (2000). Expression profiling in the muscular dystrophies: identification of novel aspects of molecular pathophysiology. J Cell Biol 151, 1321-1336.
    • (2000) J Cell Biol , vol.151 , pp. 1321-1336
    • Chen, Y.W.1    Zhao, P.2    Borup, R.3    Hoffman, E.P.4
  • 21
    • 0027255549 scopus 로고
    • Dystrophin-related protein, utrophin, in normal and dystrophic human fetal skeletal muscle
    • Clerk A, Morris GE, Dubowitz V, Davies KE & Sewry CA (1993). Dystrophin-related protein, utrophin, in normal and dystrophic human fetal skeletal muscle. Histochem J 25, 554-561.
    • (1993) Histochem J , vol.25 , pp. 554-561
    • Clerk, A.1    Morris, G.E.2    Dubowitz, V.3    Davies, K.E.4    Sewry, C.A.5
  • 22
    • 0034810216 scopus 로고    scopus 로고
    • Three mouse models of muscular dystrophy: the natural history of strength and fatigue in dystrophin-, dystrophin/utrophin-, and laminin α2-deficient mice
    • Connolly AM, Keeling RM, Mehta S, Pestronk A & Sanes JR (2001). Three mouse models of muscular dystrophy: the natural history of strength and fatigue in dystrophin-, dystrophin/utrophin-, and laminin α2-deficient mice. Neuromuscul Disord 11, 703-712.
    • (2001) Neuromuscul Disord , vol.11 , pp. 703-712
    • Connolly, A.M.1    Keeling, R.M.2    Mehta, S.3    Pestronk, A.4    Sanes, J.R.5
  • 23
    • 0036893708 scopus 로고    scopus 로고
    • High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy
    • Connolly AM, Schierbecker J, Renna R & Florence J (2002). High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy. Neuromuscul Disord 12, 917-925.
    • (2002) Neuromuscul Disord , vol.12 , pp. 917-925
    • Connolly, A.M.1    Schierbecker, J.2    Renna, R.3    Florence, J.4
  • 24
    • 0034089616 scopus 로고    scopus 로고
    • The artificial zinc finger coding gene 'Jazz' binds the utrophin promoter and activates transcription
    • Corbi N, Libri V, Fanciulli M, Tinsley JM, Davies KE & Passananti C (2000). The artificial zinc finger coding gene 'Jazz' binds the utrophin promoter and activates transcription. Gene Ther 7, 1076-1083.
    • (2000) Gene Ther , vol.7 , pp. 1076-1083
    • Corbi, N.1    Libri, V.2    Fanciulli, M.3    Tinsley, J.M.4    Davies, K.E.5    Passananti, C.6
  • 25
    • 0030981051 scopus 로고    scopus 로고
    • Dystrophies and heart disease
    • Cox GF & Kunkel LM (1997). Dystrophies and heart disease. Curr Opin Cardiol 12, 329-343.
    • (1997) Curr Opin Cardiol , vol.12 , pp. 329-343
    • Cox, G.F.1    Kunkel, L.M.2
  • 26
    • 33749015734 scopus 로고    scopus 로고
    • Molecular mechanisms of muscular dystrophies: old and new players
    • Davies KE & Nowak KJ (2006). Molecular mechanisms of muscular dystrophies: old and new players. Nat Rev Mol Cell Biol 7, 762-773.
    • (2006) Nat Rev Mol Cell Biol , vol.7 , pp. 762-773
    • Davies, K.E.1    Nowak, K.J.2
  • 30
    • 77957562387 scopus 로고    scopus 로고
    • CT-GalNAc transferase overexpression in adult mice is associated with extrasynaptic utrophin in skeletal muscle fibres
    • Durko M, Allen C, Nalbantoglu J & Karpati G (2010). CT-GalNAc transferase overexpression in adult mice is associated with extrasynaptic utrophin in skeletal muscle fibres. J Muscle Res Cell Motil 31, 181-193.
    • (2010) J Muscle Res Cell Motil , vol.31 , pp. 181-193
    • Durko, M.1    Allen, C.2    Nalbantoglu, J.3    Karpati, G.4
  • 31
    • 0036432223 scopus 로고    scopus 로고
    • The urokinase plasminogen activator: an interesting way to improve myoblast migration following their transplantation
    • El Fahime E, Mills P, Lafreniere JF, Torrente Y & Tremblay JP (2002). The urokinase plasminogen activator: an interesting way to improve myoblast migration following their transplantation. Exp Cell Res 280, 169-178.
    • (2002) Exp Cell Res , vol.280 , pp. 169-178
    • El Fahime, E.1    Mills, P.2    Lafreniere, J.F.3    Torrente, Y.4    Tremblay, J.P.5
  • 32
    • 0034255965 scopus 로고    scopus 로고
    • In vivo migration of transplanted myoblasts requires matrix metalloproteinase activity
    • El Fahime E, Torrente Y, Caron NJ, Bresolin MD & Tremblay JP (2000). In vivo migration of transplanted myoblasts requires matrix metalloproteinase activity. Exp Cell Res 258, 279-287.
    • (2000) Exp Cell Res , vol.258 , pp. 279-287
    • El Fahime, E.1    Torrente, Y.2    Caron, N.J.3    Bresolin, M.D.4    Tremblay, J.P.5
  • 37
    • 0029881026 scopus 로고    scopus 로고
    • A potential alternative strategy for myoblast transfer therapy: the use of sliced muscle grafts
    • Fan Y, Beilharz MW & Grounds MD (1996). A potential alternative strategy for myoblast transfer therapy: the use of sliced muscle grafts. Cell Transplant 5, 421-429.
    • (1996) Cell Transplant , vol.5 , pp. 421-429
    • Fan, Y.1    Beilharz, M.W.2    Grounds, M.D.3
  • 38
    • 77956311645 scopus 로고    scopus 로고
    • Read-through strategies for suppression of nonsense mutations in Duchenne/Becker muscular dystrophy: aminoglycosides and ataluren (PTC124)
    • Finkel RS (2010). Read-through strategies for suppression of nonsense mutations in Duchenne/Becker muscular dystrophy: aminoglycosides and ataluren (PTC124). J Child Neurol 25, 1158-1164.
    • (2010) J Child Neurol , vol.25 , pp. 1158-1164
    • Finkel, R.S.1
  • 40
    • 65649133138 scopus 로고    scopus 로고
    • RhoA leads to up-regulation and relocalization of utrophin in muscle fibers
    • Gauthier-Rouviere C & Bonet-Kerrache A (2009). RhoA leads to up-regulation and relocalization of utrophin in muscle fibers. Biochem Biophys Res Commun 384, 322-328.
    • (2009) Biochem Biophys Res Commun , vol.384 , pp. 322-328
    • Gauthier-Rouviere, C.1    Bonet-Kerrache, A.2
  • 43
    • 79955836028 scopus 로고    scopus 로고
    • 24 week follow-up data from a phase I/IIa extension study of PRO051/GSK2402968 in subjects with Duchenne muscular dystrophy
    • Goemans NM, Tulinius M, Buyse G, Wilson R, de Kimpe R, van Deutekom JCT & Campion G (2010). 24 week follow-up data from a phase I/IIa extension study of PRO051/GSK2402968 in subjects with Duchenne muscular dystrophy. Neuromusc Disord 20, 639.
    • (2010) Neuromusc Disord , vol.20 , pp. 639
    • Goemans, N.M.1    Tulinius, M.2    Buyse, G.3    Wilson, R.4    de Kimpe, R.5    van Deutekom, J.C.T.6    Campion, G.7
  • 46
    • 74149093605 scopus 로고    scopus 로고
    • Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping
    • Goyenvalle A, Babbs A, Powell D, Kole R, Fletcher S, Wilton SD & Davies KE (2010). Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping. Mol Ther 18, 198-205.
    • (2010) Mol Ther , vol.18 , pp. 198-205
    • Goyenvalle, A.1    Babbs, A.2    Powell, D.3    Kole, R.4    Fletcher, S.5    Wilton, S.D.6    Davies, K.E.7
  • 47
    • 80055038015 scopus 로고    scopus 로고
    • Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy
    • Goyenvalle A & Davies KE (2011a). Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy. Skelet Muscle 1, 8.
    • (2011) Skelet Muscle , vol.1 , pp. 8
    • Goyenvalle, A.1    Davies, K.E.2
  • 48
    • 79960052376 scopus 로고    scopus 로고
    • Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy
    • Goyenvalle A & Davies KE (2011b). Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy. Methods Mol Biol 709, 179-196.
    • (2011) Methods Mol Biol , vol.709 , pp. 179-196
    • Goyenvalle, A.1    Davies, K.E.2
  • 49
    • 0030848338 scopus 로고    scopus 로고
    • Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy
    • Grady RM, Teng H, Nichol MC, Cunningham JC, Wilkinson RS & Sanes JR (1997). Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy. Cell 90, 729-738.
    • (1997) Cell , vol.90 , pp. 729-738
    • Grady, R.M.1    Teng, H.2    Nichol, M.C.3    Cunningham, J.C.4    Wilkinson, R.S.5    Sanes, J.R.6
  • 51
    • 77954658532 scopus 로고    scopus 로고
    • Molecular treatments in Duchenne muscular dystrophy
    • Guglieri M & Bushby K (2010). Molecular treatments in Duchenne muscular dystrophy. Curr Opin Pharmacol 10, 331-337.
    • (2010) Curr Opin Pharmacol , vol.10 , pp. 331-337
    • Guglieri, M.1    Bushby, K.2
  • 52
    • 0026560260 scopus 로고
    • Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation
    • Gussoni E, Pavlath GK, Lanctot AM, Sharma KR, Miller RG, Steinman L & Blau HM (1992). Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation. Nature 356, 435-438.
    • (1992) Nature , vol.356 , pp. 435-438
    • Gussoni, E.1    Pavlath, G.K.2    Lanctot, A.M.3    Sharma, K.R.4    Miller, R.G.5    Steinman, L.6    Blau, H.M.7
  • 53
    • 78349258144 scopus 로고    scopus 로고
    • Prevention of muscle aging by myofiber-associated satellite cell transplantation
    • Hall JK, Banks GB, Chamberlain JS & Olwin BB (2011). Prevention of muscle aging by myofiber-associated satellite cell transplantation. Sci Transl Med 2, 57ra83.
    • (2011) Sci Transl Med , vol.2
    • Hall, J.K.1    Banks, G.B.2    Chamberlain, J.S.3    Olwin, B.B.4
  • 54
    • 0033617473 scopus 로고    scopus 로고
    • Unexpected modes of PDZ domain scaffolding revealed by structure of nNOS-syntrophin complex
    • Hillier BJ, Christopherson KS, Prehoda KE, Bredt DS & Lim WA (1999). Unexpected modes of PDZ domain scaffolding revealed by structure of nNOS-syntrophin complex. Science 284, 812-815.
    • (1999) Science , vol.284 , pp. 812-815
    • Hillier, B.J.1    Christopherson, K.S.2    Prehoda, K.E.3    Bredt, D.S.4    Lim, W.A.5
  • 55
    • 0036778387 scopus 로고    scopus 로고
    • Cyclosporine blocks muscle differentiation by inducing oxidative stress and inhibiting the peptidylprolyl-cis-trans-isomerase activity of cyclophilin A: cyclophilin A protects myoblasts from cyclosporine-induced cytotoxicity
    • Hong F, Lee J, Song JW, Lee SJ, Ahn H, Cho JJ, Ha J & Kim SS (2002). Cyclosporine blocks muscle differentiation by inducing oxidative stress and inhibiting the peptidylprolyl-cis-trans-isomerase activity of cyclophilin A: cyclophilin A protects myoblasts from cyclosporine-induced cytotoxicity. FASEB J 16, 1633-1635.
    • (2002) FASEB J , vol.16 , pp. 1633-1635
    • Hong, F.1    Lee, J.2    Song, J.W.3    Lee, S.J.4    Ahn, H.5    Cho, J.J.6    Ha, J.7    Kim, S.S.8
  • 57
    • 1842429177 scopus 로고    scopus 로고
    • ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to β-dystroglycan
    • Ishikawa-Sakurai M, Yoshida M, Imamura M, Davies KE & Ozawa E (2004). ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to β-dystroglycan. Hum Mol Genet 13, 693-702.
    • (2004) Hum Mol Genet , vol.13 , pp. 693-702
    • Ishikawa-Sakurai, M.1    Yoshida, M.2    Imamura, M.3    Davies, K.E.4    Ozawa, E.5
  • 59
    • 74249109987 scopus 로고    scopus 로고
    • Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers
    • Jearawiriyapaisarn N, Moulton HM, Sazani P, Kole R & Willis MS (2010). Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers. Cardiovasc Res 85, 444-453.
    • (2010) Cardiovasc Res , vol.85 , pp. 444-453
    • Jearawiriyapaisarn, N.1    Moulton, H.M.2    Sazani, P.3    Kole, R.4    Willis, M.S.5
  • 60
    • 0028805790 scopus 로고
    • Identification and characterization of the dystrophin anchoring site on β-dystroglycan
    • Jung D, Yang B, Meyer J, Chamberlain JS & Campbell KP (1995). Identification and characterization of the dystrophin anchoring site on β-dystroglycan. J Biol Chem 270, 27305-27310.
    • (1995) J Biol Chem , vol.270 , pp. 27305-27310
    • Jung, D.1    Yang, B.2    Meyer, J.3    Chamberlain, J.S.4    Campbell, K.P.5
  • 61
    • 0033593119 scopus 로고    scopus 로고
    • α1-Syntrophin gene disruption results in the absence of neuronal-type nitric-oxide synthase at the sarcolemma but does not induce muscle degeneration
    • Kameya S, Miyagoe Y, Nonaka I, Ikemoto T, Endo M, Hanaoka K, Nabeshima Y & Takeda S (1999). α1-Syntrophin gene disruption results in the absence of neuronal-type nitric-oxide synthase at the sarcolemma but does not induce muscle degeneration. J Biol Chem 274, 2193-2200.
    • (1999) J Biol Chem , vol.274 , pp. 2193-2200
    • Kameya, S.1    Miyagoe, Y.2    Nonaka, I.3    Ikemoto, T.4    Endo, M.5    Hanaoka, K.6    Nabeshima, Y.7    Takeda, S.8
  • 64
    • 0038725634 scopus 로고    scopus 로고
    • Pharmacological strategies for muscular dystrophy
    • Khurana TS & Davies KE (2003). Pharmacological strategies for muscular dystrophy. Nat Rev Drug Discov 2, 379-390.
    • (2003) Nat Rev Drug Discov , vol.2 , pp. 379-390
    • Khurana, T.S.1    Davies, K.E.2
  • 66
    • 33745183090 scopus 로고    scopus 로고
    • Naturally occurring utrophin correlates with disease severity in Duchenne muscular dystrophy
    • Kleopa KA, Drousiotou A, Mavrikiou E, Ormiston A & Kyriakides T (2006). Naturally occurring utrophin correlates with disease severity in Duchenne muscular dystrophy. Hum Mol Genet 15, 1623-1628.
    • (2006) Hum Mol Genet , vol.15 , pp. 1623-1628
    • Kleopa, K.A.1    Drousiotou, A.2    Mavrikiou, E.3    Ormiston, A.4    Kyriakides, T.5
  • 67
    • 0023614271 scopus 로고
    • Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals
    • Koenig M, Hoffman EP, Bertelson CJ, Monaco AP, Feener C & Kunkel LM (1987). Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals. Cell 50, 509-517.
    • (1987) Cell , vol.50 , pp. 509-517
    • Koenig, M.1    Hoffman, E.P.2    Bertelson, C.J.3    Monaco, A.P.4    Feener, C.5    Kunkel, L.M.6
  • 68
    • 0023904860 scopus 로고
    • The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein
    • Koenig M, Monaco AP & Kunkel LM (1988). The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein. Cell 53, 219-228.
    • (1988) Cell , vol.53 , pp. 219-228
    • Koenig, M.1    Monaco, A.P.2    Kunkel, L.M.3
  • 70
    • 65649111197 scopus 로고    scopus 로고
    • Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy
    • Lai Y, Thomas GD, Yue Y, Yang HT, Li D, Long C, Judge L, Bostick B, Chamberlain JS, Terjung RL & Duan D (2009). Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy. J Clin Invest 119, 624-635.
    • (2009) J Clin Invest , vol.119 , pp. 624-635
    • Lai, Y.1    Thomas, G.D.2    Yue, Y.3    Yang, H.T.4    Li, D.5    Long, C.6    Judge, L.7    Bostick, B.8    Chamberlain, J.S.9    Terjung, R.L.10    Duan, D.11
  • 75
    • 0027385135 scopus 로고
    • Age-related changes in replication of myogenic cells in mdx mice: quantitative autoradiographic studies
    • McGeachie JK, Grounds MD, Partridge TA & Morgan JE (1993). Age-related changes in replication of myogenic cells in mdx mice: quantitative autoradiographic studies. J Neurol Sci 119, 169-179.
    • (1993) J Neurol Sci , vol.119 , pp. 169-179
    • McGeachie, J.K.1    Grounds, M.D.2    Partridge, T.A.3    Morgan, J.E.4
  • 77
    • 0033929810 scopus 로고    scopus 로고
    • Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system
    • Manuvakhova M, Keeling K & Bedwell DM (2000). Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system. RNA 6, 1044-1055.
    • (2000) RNA , vol.6 , pp. 1044-1055
    • Manuvakhova, M.1    Keeling, K.2    Bedwell, D.M.3
  • 78
    • 73349127499 scopus 로고    scopus 로고
    • Diagnosis and new treatments in muscular dystrophies
    • Manzur AY & Muntoni F (2009). Diagnosis and new treatments in muscular dystrophies. Postgrad Med J 85, 622-630.
    • (2009) Postgrad Med J , vol.85 , pp. 622-630
    • Manzur, A.Y.1    Muntoni, F.2
  • 82
    • 70449388874 scopus 로고    scopus 로고
    • Pharmacological activation of PPARβ/δ stimulates utrophin A expression in skeletal muscle fibers and restores sarcolemmal integrity in mature mdx mice
    • Miura P, Chakkalakal JV, Boudreault L, Belanger G, Hebert RL, Renaud JM & Jasmin BJ (2009). Pharmacological activation of PPARβ/δ stimulates utrophin A expression in skeletal muscle fibers and restores sarcolemmal integrity in mature mdx mice. Hum Mol Genet 18, 4640-4649.
    • (2009) Hum Mol Genet , vol.18 , pp. 4640-4649
    • Miura, P.1    Chakkalakal, J.V.2    Boudreault, L.3    Belanger, G.4    Hebert, R.L.5    Renaud, J.M.6    Jasmin, B.J.7
  • 83
    • 0027358450 scopus 로고
    • Reciprocal expression of dystrophin and utrophin in muscles of Duchenne muscular dystrophy patients, female DMD-carriers and control subjects
    • Mizuno Y, Nonaka I, Hirai S & Ozawa E (1993). Reciprocal expression of dystrophin and utrophin in muscles of Duchenne muscular dystrophy patients, female DMD-carriers and control subjects. J Neurol Sci 119, 43-52.
    • (1993) J Neurol Sci , vol.119 , pp. 43-52
    • Mizuno, Y.1    Nonaka, I.2    Hirai, S.3    Ozawa, E.4
  • 84
    • 0026928720 scopus 로고
    • Cell transplantation and gene therapy in muscular dystrophy
    • Morgan JE & Partridge TA (1992). Cell transplantation and gene therapy in muscular dystrophy. Bioessays 14, 641-645.
    • (1992) Bioessays , vol.14 , pp. 641-645
    • Morgan, J.E.1    Partridge, T.A.2
  • 85
    • 0028280007 scopus 로고
    • Myogenic cell lines derived from transgenic mice carrying a thermolabile T antigen: a model system for the derivation of tissue-specific and mutation-specific cell lines
    • Morgan JE, Beauchamp JR, Pagel CN, Peckham M, Ataliotis P, Jat PS, Noble MD, Farmer K & Partridge TA (1994). Myogenic cell lines derived from transgenic mice carrying a thermolabile T antigen: a model system for the derivation of tissue-specific and mutation-specific cell lines. Dev Biol 162, 486-498.
    • (1994) Dev Biol , vol.162 , pp. 486-498
    • Morgan, J.E.1    Beauchamp, J.R.2    Pagel, C.N.3    Peckham, M.4    Ataliotis, P.5    Jat, P.S.6    Noble, M.D.7    Farmer, K.8    Partridge, T.A.9
  • 87
    • 77958152873 scopus 로고    scopus 로고
    • Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy
    • Moulton HM & Moulton JD (2010). Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy. Biochim Biophys Acta 1798, 2296-2303.
    • (2010) Biochim Biophys Acta , vol.1798 , pp. 2296-2303
    • Moulton, H.M.1    Moulton, J.D.2
  • 88
    • 0026011430 scopus 로고
    • Invited review: myoblast transfer: a possible therapy for inherited myopathies?
    • Partridge TA (1991). Invited review: myoblast transfer: a possible therapy for inherited myopathies? Muscle Nerve 14, 197-212.
    • (1991) Muscle Nerve , vol.14 , pp. 197-212
    • Partridge, T.A.1
  • 89
    • 0024540879 scopus 로고
    • Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts
    • Partridge TA, Morgan JE, Coulton GR, Hoffman EP & Kunkel LM (1989). Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts. Nature 337, 176-179.
    • (1989) Nature , vol.337 , pp. 176-179
    • Partridge, T.A.1    Morgan, J.E.2    Coulton, G.R.3    Hoffman, E.P.4    Kunkel, L.M.5
  • 90
    • 79952195167 scopus 로고    scopus 로고
    • The survival of myoblasts after intramuscular transplantation is improved when fewer cells are injected
    • Pellegrini KL & Beilharz MW (2011). The survival of myoblasts after intramuscular transplantation is improved when fewer cells are injected. Transplantation 91, 522-526.
    • (2011) Transplantation , vol.91 , pp. 522-526
    • Pellegrini, K.L.1    Beilharz, M.W.2
  • 91
    • 0035584042 scopus 로고    scopus 로고
    • The role of basal and myogenic factors in the transcriptional activation of utrophin promoter A: implications for therapeutic up-regulation in Duchenne muscular dystrophy
    • Perkins KJ, Burton EA & Davies KE (2001). The role of basal and myogenic factors in the transcriptional activation of utrophin promoter A: implications for therapeutic up-regulation in Duchenne muscular dystrophy. Nucleic Acids Res 29, 4843-4850.
    • (2001) Nucleic Acids Res , vol.29 , pp. 4843-4850
    • Perkins, K.J.1    Burton, E.A.2    Davies, K.E.3
  • 95
    • 0027745279 scopus 로고
    • Dystrophin and dystrophin-related protein (utrophin) distribution in normal and dystrophin-deficient skeletal muscles
    • Pons F, Nicholson LV, Robert A, Voit T & Leger JJ (1993). Dystrophin and dystrophin-related protein (utrophin) distribution in normal and dystrophin-deficient skeletal muscles. Neuromuscul Disord 3, 507-514.
    • (1993) Neuromuscul Disord , vol.3 , pp. 507-514
    • Pons, F.1    Nicholson, L.V.2    Robert, A.3    Voit, T.4    Leger, J.J.5
  • 96
    • 33750815428 scopus 로고    scopus 로고
    • Overactivity of exercise-sensitive cation channels and their impaired modulation by IGF-1 in mdx native muscle fibers: beneficial effect of pentoxifylline
    • Rolland JF, De Luca A, Burdi R, Andreetta F, Confalonieri P & Conte Camerino D (2006). Overactivity of exercise-sensitive cation channels and their impaired modulation by IGF-1 in mdx native muscle fibers: beneficial effect of pentoxifylline. Neurobiol Dis 24, 466-474.
    • (2006) Neurobiol Dis , vol.24 , pp. 466-474
    • Rolland, J.F.1    De Luca, A.2    Burdi, R.3    Andreetta, F.4    Confalonieri, P.5    Conte Camerino, D.6
  • 97
    • 20744456279 scopus 로고    scopus 로고
    • Identification of spectrin-like repeats required for high affinity utrophin-actin interaction
    • Rybakova IN & Ervasti JM (2005). Identification of spectrin-like repeats required for high affinity utrophin-actin interaction. J Biol Chem 280, 23018-23023.
    • (2005) J Biol Chem , vol.280 , pp. 23018-23023
    • Rybakova, I.N.1    Ervasti, J.M.2
  • 98
    • 0034605070 scopus 로고    scopus 로고
    • The dystrophin complex forms a mechanically strong link between the sarcolemma and costameric actin
    • Rybakova IN, Patel JR & Ervasti JM (2000). The dystrophin complex forms a mechanically strong link between the sarcolemma and costameric actin. J Cell Biol 150, 1209-1214.
    • (2000) J Cell Biol , vol.150 , pp. 1209-1214
    • Rybakova, I.N.1    Patel, J.R.2    Ervasti, J.M.3
  • 99
    • 0030775377 scopus 로고    scopus 로고
    • Dystrobrevin and dystrophin: an interaction through coiled-coil motifs
    • Sadoulet-Puccio HM, Rajala M & Kunkel LM (1997). Dystrobrevin and dystrophin: an interaction through coiled-coil motifs. Proc Natl Acad Sci USA 94, 12413-12418.
    • (1997) Proc Natl Acad Sci USA , vol.94 , pp. 12413-12418
    • Sadoulet-Puccio, H.M.1    Rajala, M.2    Kunkel, L.M.3
  • 101
    • 0034610326 scopus 로고    scopus 로고
    • Functional muscle ischemia in neuronal nitric oxide synthase-deficient skeletal muscle of children with Duchenne muscular dystrophy
    • Sander M, Chavoshan B, Harris SA, Iannaccone ST, Stull JT, Thomas GD & Victor RG (2000). Functional muscle ischemia in neuronal nitric oxide synthase-deficient skeletal muscle of children with Duchenne muscular dystrophy. Proc Natl Acad Sci USA 97, 13818-13823.
    • (2000) Proc Natl Acad Sci USA , vol.97 , pp. 13818-13823
    • Sander, M.1    Chavoshan, B.2    Harris, S.A.3    Iannaccone, S.T.4    Stull, J.T.5    Thomas, G.D.6    Victor, R.G.7
  • 103
    • 79955808677 scopus 로고    scopus 로고
    • Current progress and preliminary results with the systemic administration trial of AVI-4658, a novel phosphorodiamidate morpholino oligomer (PMO) skipping dystrophin exon 51 in Duchenne muscular dystrophy (DMD)
    • Shrewsbury SB, Cirak S, Guglieri M, Bushby K & Muntoni F (2010). Current progress and preliminary results with the systemic administration trial of AVI-4658, a novel phosphorodiamidate morpholino oligomer (PMO) skipping dystrophin exon 51 in Duchenne muscular dystrophy (DMD). In Neuromuscular Disorders 20, 639-640.
    • (2010) Neuromuscular Disorders , vol.20 , pp. 639-640
    • Shrewsbury, S.B.1    Cirak, S.2    Guglieri, M.3    Bushby, K.4    Muntoni, F.5
  • 104
    • 0036255397 scopus 로고    scopus 로고
    • Efficacy of myoblast transplantation in nonhuman primates following simple intramuscular cell injections: toward defining strategies applicable to humans
    • Skuk D, Goulet M, Roy B & Tremblay JP (2002). Efficacy of myoblast transplantation in nonhuman primates following simple intramuscular cell injections: toward defining strategies applicable to humans. Exp Neurol 175, 112-126.
    • (2002) Exp Neurol , vol.175 , pp. 112-126
    • Skuk, D.1    Goulet, M.2    Roy, B.3    Tremblay, J.P.4
  • 105
    • 0035035323 scopus 로고    scopus 로고
    • Absence of desmin slightly prolongs myoblast proliferation and delays fusion in vivo in regenerating grafts of skeletal muscle
    • Smythe GM, Davies MJ, Paulin D & Grounds MD (2001). Absence of desmin slightly prolongs myoblast proliferation and delays fusion in vivo in regenerating grafts of skeletal muscle. Cell Tissue Res 304, 287-294.
    • (2001) Cell Tissue Res , vol.304 , pp. 287-294
    • Smythe, G.M.1    Davies, M.J.2    Paulin, D.3    Grounds, M.D.4
  • 106
    • 0033842201 scopus 로고    scopus 로고
    • Exposure to tissue culture conditions can adversely affect myoblast behavior in vivo in whole muscle grafts: implications for myoblast transfer therapy
    • Smythe GM & Grounds MD (2000). Exposure to tissue culture conditions can adversely affect myoblast behavior in vivo in whole muscle grafts: implications for myoblast transfer therapy. Cell Transplant 9, 379-393.
    • (2000) Cell Transplant , vol.9 , pp. 379-393
    • Smythe, G.M.1    Grounds, M.D.2
  • 107
    • 0035879188 scopus 로고    scopus 로고
    • Absence of MyoD increases donor myoblast migration into host muscle
    • Smythe GM & Grounds MD (2001). Absence of MyoD increases donor myoblast migration into host muscle. Exp Cell Res 267, 267-274.
    • (2001) Exp Cell Res , vol.267 , pp. 267-274
    • Smythe, G.M.1    Grounds, M.D.2
  • 109
    • 0030218963 scopus 로고    scopus 로고
    • Calpain translocation during muscle fiber necrosis and regeneration in dystrophin-deficient mice
    • Spencer MJ & Tidball JG (1996). Calpain translocation during muscle fiber necrosis and regeneration in dystrophin-deficient mice. Exp Cell Res 226, 264-272.
    • (1996) Exp Cell Res , vol.226 , pp. 264-272
    • Spencer, M.J.1    Tidball, J.G.2
  • 112
    • 0032411144 scopus 로고    scopus 로고
    • Impaired metabolic modulation of α-adrenergic vasoconstriction in dystrophin-deficient skeletal muscle
    • Thomas GD, Sander M, Lau KS, Huang PL, Stull JT & Victor RG (1998). Impaired metabolic modulation of α-adrenergic vasoconstriction in dystrophin-deficient skeletal muscle. Proc Natl Acad Sci USA 95, 15090-15095.
    • (1998) Proc Natl Acad Sci USA , vol.95 , pp. 15090-15095
    • Thomas, G.D.1    Sander, M.2    Lau, K.S.3    Huang, P.L.4    Stull, J.T.5    Victor, R.G.6
  • 116
    • 0029906168 scopus 로고    scopus 로고
    • Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene
    • Tinsley JM, Potter AC, Phelps SR, Fisher R, Trickett JI & Davies KE (1996). Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene. Nature 384, 349-353.
    • (1996) Nature , vol.384 , pp. 349-353
    • Tinsley, J.M.1    Potter, A.C.2    Phelps, S.R.3    Fisher, R.4    Trickett, J.I.5    Davies, K.E.6
  • 117
    • 0032916127 scopus 로고    scopus 로고
    • Solution structure of the extended neuronal nitric oxide synthase PDZ domain complexed with an associated peptide
    • Tochio H, Zhang Q, Mandal P, Li M & Zhang M (1999). Solution structure of the extended neuronal nitric oxide synthase PDZ domain complexed with an associated peptide. Nat Struct Biol 6, 417-421.
    • (1999) Nat Struct Biol , vol.6 , pp. 417-421
    • Tochio, H.1    Zhang, Q.2    Mandal, P.3    Li, M.4    Zhang, M.5
  • 118
    • 0028103785 scopus 로고
    • Expression of dystrophin-associated glycoproteins during human fetal muscle development: a preliminary immunocytochemical study
    • Tome FM, Matsumura K, Chevallay M, Campbell KP & Fardeau M (1994). Expression of dystrophin-associated glycoproteins during human fetal muscle development: a preliminary immunocytochemical study. Neuromuscul Disord 4, 343-348.
    • (1994) Neuromuscul Disord , vol.4 , pp. 343-348
    • Tome, F.M.1    Matsumura, K.2    Chevallay, M.3    Campbell, K.P.4    Fardeau, M.5
  • 120
    • 0033775811 scopus 로고    scopus 로고
    • Intramuscular migration of myoblasts transplanted after muscle pretreatment with metalloproteinases
    • Torrente Y, El Fahime E, Caron NJ, Bresolin N & Tremblay JP (2000). Intramuscular migration of myoblasts transplanted after muscle pretreatment with metalloproteinases. Cell Transplant 9, 539-549.
    • (2000) Cell Transplant , vol.9 , pp. 539-549
    • Torrente, Y.1    El Fahime, E.2    Caron, N.J.3    Bresolin, N.4    Tremblay, J.P.5
  • 122
    • 0027166930 scopus 로고
    • Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy
    • Tremblay JP, Malouin F, Roy R, Huard J, Bouchard JP, Satoh A & Richards CL (1993b). Results of a triple blind clinical study of myoblast transplantations without immunosuppressive treatment in young boys with Duchenne muscular dystrophy. Cell Transplant 2, 99-112.
    • (1993) Cell Transplant , vol.2 , pp. 99-112
    • Tremblay, J.P.1    Malouin, F.2    Roy, R.3    Huard, J.4    Bouchard, J.P.5    Satoh, A.6    Richards, C.L.7
  • 123
    • 0028577672 scopus 로고
    • Myoblast allotransplantation in mice: degree of success varies depending on the efficacy of various immunosuppressive treatments
    • Vilquin JT, Asselin I, Guerette B, Kinoshita I, Lille S, Roy R & Tremblay JP (1994). Myoblast allotransplantation in mice: degree of success varies depending on the efficacy of various immunosuppressive treatments. Transplant Proc 26, 3372-3373.
    • (1994) Transplant Proc , vol.26 , pp. 3372-3373
    • Vilquin, J.T.1    Asselin, I.2    Guerette, B.3    Kinoshita, I.4    Lille, S.5    Roy, R.6    Tremblay, J.P.7
  • 124
    • 23444439648 scopus 로고    scopus 로고
    • [Pharmacological treatments for Duchenne and Becker dystrophies]
    • Article in French.]
    • Voisin V & de la Porte S (2005). [Pharmacological treatments for Duchenne and Becker dystrophies]. [Article in French.] J Soc Biol 199, 17-28.
    • (2005) J Soc Biol , vol.199 , pp. 17-28
    • Voisin, V.1    de la Porte, S.2
  • 125
    • 0020448517 scopus 로고
    • Incorporation of donor muscle precursor cells into an area of muscle regeneration in the host mouse
    • Watt DJ, Lambert K, Morgan JE, Partridge TA & Sloper JC (1982). Incorporation of donor muscle precursor cells into an area of muscle regeneration in the host mouse. J Neurol Sci 57, 319-331.
    • (1982) J Neurol Sci , vol.57 , pp. 319-331
    • Watt, D.J.1    Lambert, K.2    Morgan, J.E.3    Partridge, T.A.4    Sloper, J.C.5
  • 126
    • 0037160098 scopus 로고    scopus 로고
    • A- and B-utrophin have different expression patterns and are differentially up-regulated in mdx muscle
    • Weir AP, Burton EA, Harrod G & Davies KE (2002). A- and B-utrophin have different expression patterns and are differentially up-regulated in mdx muscle. J Biol Chem 277, 45285-45290.
    • (2002) J Biol Chem , vol.277 , pp. 45285-45290
    • Weir, A.P.1    Burton, E.A.2    Harrod, G.3    Davies, K.E.4
  • 130
    • 74349109205 scopus 로고    scopus 로고
    • 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 & Lu QL (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    Lu, Q.L.7
  • 131
    • 0028986593 scopus 로고
    • Identification of α-syntrophin binding to syntrophin triplet, dystrophin, and utrophin
    • Yang B, Jung D, Rafael JA, Chamberlain JS & Campbell KP (1995). Identification of α-syntrophin binding to syntrophin triplet, dystrophin, and utrophin. J Biol Chem 270, 4975-4978.
    • (1995) J Biol Chem , vol.270 , pp. 4975-4978
    • Yang, B.1    Jung, D.2    Rafael, J.A.3    Chamberlain, J.S.4    Campbell, K.P.5
  • 132
    • 0032848317 scopus 로고    scopus 로고
    • Clinical characteristics of aged Becker muscular dystrophy patients with onset after 30 years
    • Yazaki M, Yoshida K, Nakamura A, Koyama J, Nanba T, Ohori N & Ikeda S (1999). Clinical characteristics of aged Becker muscular dystrophy patients with onset after 30 years. Eur Neurol 42, 145-149.
    • (1999) Eur Neurol , vol.42 , pp. 145-149
    • Yazaki, M.1    Yoshida, K.2    Nakamura, A.3    Koyama, J.4    Nanba, T.5    Ohori, N.6    Ikeda, S.7
  • 133
    • 37549022222 scopus 로고    scopus 로고
    • Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice
    • Yin H, Lu Q & Wood M (2008a). 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
  • 134
    • 57049102809 scopus 로고    scopus 로고
    • 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 & Wood MJ (2008b). 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    Wood, M.J.7
  • 135
    • 70350697818 scopus 로고    scopus 로고
    • 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 & Wood MJ (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    Wood, M.J.7
  • 136
    • 77955472624 scopus 로고    scopus 로고
    • Targeting fibrosis in Duchenne muscular dystrophy
    • Zhou L & Lu H (2010). Targeting fibrosis in Duchenne muscular dystrophy. J Neuropathol Exp Neurol 69, 771-776.
    • (2010) J Neuropathol Exp Neurol , vol.69 , pp. 771-776
    • Zhou, L.1    Lu, H.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.