메뉴 건너뛰기




Volumn 20, Issue 4, 2015, Pages 6237-6253

Model organisms in the fight against muscular dystrophy: Lessons from Drosophila and zebrafish

Author keywords

Animal models; Drosophila; Muscular dystrophies; Pathophysiology; Therapeutic screens; Zebrafish

Indexed keywords

ANIMAL; DISEASE MODEL; DROSOPHILA; DUCHENNE MUSCULAR DYSTROPHY; GENETICS; HUMAN; METABOLISM; MYOTONIC DYSTROPHY; PATHOLOGY; SKELETAL MUSCLE; ZEBRA FISH;

EID: 84928675936     PISSN: None     EISSN: 14203049     Source Type: Journal    
DOI: 10.3390/molecules20046237     Document Type: Review
Times cited : (44)

References (107)
  • 1
    • 0037160782 scopus 로고    scopus 로고
    • The muscular dystrophies
    • Emery, A.E. The muscular dystrophies. Lancet 2002, 359, 687-695.
    • (2002) Lancet , vol.359 , pp. 687-695
    • Emery, A.E.1
  • 2
    • 84874692333 scopus 로고    scopus 로고
    • Muscular dystrophies
    • Mercuri, E.; Muntoni, F. Muscular dystrophies. Lancet 2013, 381, 845-860.
    • (2013) Lancet , vol.381 , pp. 845-860
    • Mercuri, E.1    Muntoni, F.2
  • 3
    • 60549103522 scopus 로고    scopus 로고
    • Other model organisms for sarcomeric muscle diseases
    • Sparrow, J.; Hughes, S.M.; Segalat, L. Other model organisms for sarcomeric muscle diseases. Adv. Exp. Med. Biol. 2008, 642, 192-206.
    • (2008) Adv. Exp. Med. Biol. , vol.642 , pp. 192-206
    • Sparrow, J.1    Hughes, S.M.2    Segalat, L.3
  • 4
    • 0034708613 scopus 로고    scopus 로고
    • A brief history of drosophila's contributions to genome research
    • Rubin, G.M.; Lewis, E.B. A brief history of drosophila's contributions to genome research. Science 2000, 287, 2216-2218.
    • (2000) Science , vol.287 , pp. 2216-2218
    • Rubin, G.M.1    Lewis, E.B.2
  • 5
    • 49649118218 scopus 로고    scopus 로고
    • Drosophila melanogaster and the development of biology in the 20th century
    • Springer: Totowa, NJ, USA
    • Arias, A.M. Drosophila melanogaster and the development of biology in the 20th century. In Methods in Molecular Biology: Drosophila: Methods and Protocols; Springer: Totowa, NJ, USA, 2008; pp. 1-25.
    • (2008) Methods in Molecular Biology: Drosophila: Methods and Protocols , pp. 1-25
    • Arias, A.M.1
  • 7
    • 0034837386 scopus 로고    scopus 로고
    • A systematic analysis of human disease-associated gene sequences in Drosophila melanogaster
    • Reiter, L.T.; Potocki, L.; Chien, S.; Gribskov, M.; Bier, E. A systematic analysis of human disease-associated gene sequences in Drosophila melanogaster. Genome Res. 2001, 11, 1114-1125.
    • (2001) Genome Res. , vol.11 , pp. 1114-1125
    • Reiter, L.T.1    Potocki, L.2    Chien, S.3    Gribskov, M.4    Bier, E.5
  • 8
    • 77949899075 scopus 로고    scopus 로고
    • Flightless flies: Drosophila models of neuromuscular disease
    • Lloyd, T.E.; Taylor, J.P. Flightless flies: Drosophila models of neuromuscular disease. Ann. N. Y. Acad. Sci. 2010, 1184, e1-e20.
    • (2010) Ann. N. Y. Acad. Sci. , vol.1184 , pp. e1-e20
    • Lloyd, T.E.1    Taylor, J.P.2
  • 11
  • 18
    • 84865070369 scopus 로고    scopus 로고
    • A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity
    • Jinek, M.; Chylinski, K.; Fonfara, I.; Hauer, M.; Doudna, J.A.; Charpentier, E. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 2012, 337, 816-821.
    • (2012) Science , vol.337 , pp. 816-821
    • Jinek, M.1    Chylinski, K.2    Fonfara, I.3    Hauer, M.4    Doudna, J.A.5    Charpentier, E.6
  • 19
    • 84919343537 scopus 로고    scopus 로고
    • Precise and efficient genome editing in zebrafish using the CRISPR/Cas9 system
    • Irion, U.; Krauss, J.; Nusslein-Volhard, C. Precise and efficient genome editing in zebrafish using the CRISPR/Cas9 system. Development 2014, 4827-4830.
    • (2014) Development , pp. 4827-4830
    • Irion, U.1    Krauss, J.2    Nusslein-Volhard, C.3
  • 20
    • 84954590035 scopus 로고    scopus 로고
    • Generation of zebrafish models by CRISPR/Cas9 genome editing
    • Neuronal Cell Death; Lossi, L., Merighi, A., Eds. Springer: New York, NY, USA
    • Hruscha, A.; Schmid, B. Generation of zebrafish models by CRISPR/Cas9 genome editing. In Neuronal Cell Death; Lossi, L., Merighi, A., Eds.; Methods in Molecular Biology; Springer: New York, NY, USA, 2015; Series Volume 1254, pp. 341-350.
    • (2015) Methods in Molecular Biology , vol.1254 , pp. 341-350
    • Hruscha, A.1    Schmid, B.2
  • 21
    • 84892437994 scopus 로고    scopus 로고
    • Highly efficient targeted mutagenesis of Drosophila with the CRISPR/Cas9 system
    • Bassett, A.R.; Tibbit, C.; Ponting, C.P.; Liu, J.-L. Highly efficient targeted mutagenesis of Drosophila with the CRISPR/Cas9 system. Cell Rep. 2013, 4, 220-228.
    • (2013) Cell Rep. , vol.4 , pp. 220-228
    • Bassett, A.R.1    Tibbit, C.2    Ponting, C.P.3    Liu, J.-L.4
  • 23
    • 35348917923 scopus 로고    scopus 로고
    • Springer Science & Business Media, New York, NY, USA
    • Sink, H. Muscle Development in Drosophila; Springer Science & Business Media, New York, NY, USA, 2007.
    • (2007) Muscle Development in Drosophila
    • Sink, H.1
  • 24
    • 0032511146 scopus 로고    scopus 로고
    • Myogenesis: A view from Drosophila
    • Baylies, M.K.; Bate, M.; Gomez, M.R. Myogenesis: A view from Drosophila. Cell 1998, 93, 921-927.
    • (1998) Cell , vol.93 , pp. 921-927
    • Baylies, M.K.1    Bate, M.2    Gomez, M.R.3
  • 25
    • 0031768122 scopus 로고    scopus 로고
    • Time course of the development of motor behaviors in the zebrafish embryo
    • Saint-Amant, L.; Drapeau, P. Time course of the development of motor behaviors in the zebrafish embryo. J. Neurobiol. 1998, 37, 622-632.
    • (1998) J. Neurobiol. , vol.37 , pp. 622-632
    • Saint-Amant, L.1    Drapeau, P.2
  • 26
    • 79955749505 scopus 로고    scopus 로고
    • Human disease models in drosophila melanogaster and the role of the fly in therapeutic drug discovery
    • Pandey, U.B.; Nichols, C.D. Human disease models in drosophila melanogaster and the role of the fly in therapeutic drug discovery. Pharmacol. Rev. 2011, 63, 411-436.
    • (2011) Pharmacol. Rev. , vol.63 , pp. 411-436
    • Pandey, U.B.1    Nichols, C.D.2
  • 27
    • 84870661131 scopus 로고    scopus 로고
    • Genetic elevation of Sphingosine 1-phosphate suppresses dystrophic muscle phenotypes in Drosophila
    • Pantoja, M.; Fischer, K.A.; Ieronimakis, N.; Reyes, M.; Ruohola-Baker, H. Genetic elevation of Sphingosine 1-phosphate suppresses dystrophic muscle phenotypes in Drosophila. Development 2013, 140, 136-146.
    • (2013) Development , vol.140 , pp. 136-146
    • Pantoja, M.1    Fischer, K.A.2    Ieronimakis, N.3    Reyes, M.4    Ruohola-Baker, H.5
  • 29
    • 34247186766 scopus 로고    scopus 로고
    • Animal models of human disease: Zebrafish swim into view
    • Lieschke, G.J.; Currie, P.D. Animal models of human disease: Zebrafish swim into view. Nat. Rev. Genet. 2007, 8, 353-367.
    • (2007) Nat. Rev. Genet. , vol.8 , pp. 353-367
    • Lieschke, G.J.1    Currie, P.D.2
  • 30
    • 84906342868 scopus 로고    scopus 로고
    • Recent advances using zebrafish animal models for muscle disease drug discovery
    • Maves, L. Recent advances using zebrafish animal models for muscle disease drug discovery. Expert Opin. Drug Discov. 2014, 9, 1033-1045.
    • (2014) Expert Opin. Drug Discov. , vol.9 , pp. 1033-1045
    • Maves, L.1
  • 31
    • 84863557893 scopus 로고    scopus 로고
    • Hooked! Modeling human disease in zebrafish
    • Santoriello, C.; Zon, L.I. Hooked! Modeling human disease in zebrafish. J. Clin. Investig. 2012, 122, 2337-2343.
    • (2012) J. Clin. Investig. , vol.122 , pp. 2337-2343
    • Santoriello, C.1    Zon, L.I.2
  • 32
    • 84882655566 scopus 로고    scopus 로고
    • Swimming into prominence: The zebrafish as a valuable tool for studying human myopathies and muscular dystrophies
    • Gibbs, E.M.; Horstick, E.J.; Dowling, J.J. Swimming into prominence: The zebrafish as a valuable tool for studying human myopathies and muscular dystrophies. FEBS J. 2013, 280, 4187-4197.
    • (2013) FEBS J. , vol.280 , pp. 4187-4197
    • Gibbs, E.M.1    Horstick, E.J.2    Dowling, J.J.3
  • 36
    • 84880288302 scopus 로고    scopus 로고
    • Novel Drosophila model of myotonic dystrophy type 1: Phenotypic characterization and genome-wide view of altered gene expression
    • Picchio, L.; Plantie, E.; Renaud, Y.; Poovthumkadavil, P.; Jagla, K. Novel Drosophila model of myotonic dystrophy type 1: Phenotypic characterization and genome-wide view of altered gene expression. Hum. Mol. Genet. 2013, 22, 2795-2810.
    • (2013) Hum. Mol. Genet. , vol.22 , pp. 2795-2810
    • Picchio, L.1    Plantie, E.2    Renaud, Y.3    Poovthumkadavil, P.4    Jagla, K.5
  • 38
    • 84892390867 scopus 로고    scopus 로고
    • Transcriptional changes and developmental abnormalities in a zebrafish model of myotonic dystrophy type 1
    • Todd, P.K.; Ackall, F.Y.; Hur, J.; Sharma, K.; Paulson, H.L.; Dowling, J.J. Transcriptional changes and developmental abnormalities in a zebrafish model of myotonic dystrophy type 1. Dis. Model. Mech. 2014, 7, 143-155.
    • (2014) Dis. Model. Mech. , vol.7 , pp. 143-155
    • Todd, P.K.1    Ackall, F.Y.2    Hur, J.3    Sharma, K.4    Paulson, H.L.5    Dowling, J.J.6
  • 39
  • 41
    • 0034792302 scopus 로고    scopus 로고
    • Dystrophin and muscular dystrophy: Past, present, and future
    • O'Brien, K.F.; Kunkel, L.M. Dystrophin and muscular dystrophy: Past, present, and future. Mol. Genet. Metab. 2001, 74, 75-88.
    • (2001) Mol. Genet. Metab. , vol.74 , pp. 75-88
    • O'Brien, K.F.1    Kunkel, L.M.2
  • 43
    • 0031947501 scopus 로고    scopus 로고
    • Dystrophins in vertebrates and invertebrates
    • Roberts, R.G.; Bobrow, M. Dystrophins in vertebrates and invertebrates. Hum. Mol. Genet. 1998, 7, 589-595.
    • (1998) Hum. Mol. Genet. , vol.7 , pp. 589-595
    • Roberts, R.G.1    Bobrow, M.2
  • 44
    • 0027122152 scopus 로고
    • Molecular basis of myotonic dystrophy: Expansion of a trinucleotide (CTG) repeat at the 3′ end of a transcript encoding a protein kinase family member
    • Brook, J.D.; McCurrach, M.E.; Harley, H.G.; Buckler, A.J.; Church, D.; Aburatani, H.; Hunter, K.; Stanton, V.P.; Thirion, J.P.; Hudson, T.; et al. Molecular basis of myotonic dystrophy: Expansion of a trinucleotide (CTG) repeat at the 3′ end of a transcript encoding a protein kinase family member. Cell 1992, 69, 385.
    • (1992) Cell , vol.69 , pp. 385
    • Brook, J.D.1    McCurrach, M.E.2    Harley, H.G.3    Buckler, A.J.4    Church, D.5    Aburatani, H.6    Hunter, K.7    Stanton, V.P.8    Thirion, J.P.9    Hudson, T.10
  • 45
    • 16344382059 scopus 로고    scopus 로고
    • Genetics and molecular pathogenesis of the myotonic dystrophies
    • Day, J.W.; Ranum, L.P.W. Genetics and molecular pathogenesis of the myotonic dystrophies. Curr. Neurol. Neurosci. Rep. 2005, 5, 55-59.
    • (2005) Curr. Neurol. Neurosci. Rep. , vol.5 , pp. 55-59
    • Day, J.W.1    Ranum, L.P.W.2
  • 46
    • 80053568829 scopus 로고    scopus 로고
    • Gain of RNA function in pathological cases: Focus on myotonic dystrophy
    • Klein, A.F.; Gasnier, E.; Furling, D. Gain of RNA function in pathological cases: Focus on myotonic dystrophy. Biochimie 2011, 93, 2006-2012.
    • (2011) Biochimie , vol.93 , pp. 2006-2012
    • Klein, A.F.1    Gasnier, E.2    Furling, D.3
  • 47
    • 84922276167 scopus 로고    scopus 로고
    • Myotonic dystrophies: An update on clinical aspects, genetic, pathology, and molecular pathomechanisms
    • Meola, G.; Cardani, R. Myotonic dystrophies: An update on clinical aspects, genetic, pathology, and molecular pathomechanisms. Biochim. Biophys. Acta BBA-Mol. Basis Dis. 2015, 1852, 594-606.
    • (2015) Biochim. Biophys. Acta BBA-Mol. Basis Dis. , vol.1852 , pp. 594-606
    • Meola, G.1    Cardani, R.2
  • 50
    • 84864449491 scopus 로고    scopus 로고
    • The ever-expanding spectrum of congenital muscular dystrophies
    • Mercuri, E.; Muntoni, F. The ever-expanding spectrum of congenital muscular dystrophies. Ann. Neurol. 2012, 72, 9-17.
    • (2012) Ann. Neurol. , vol.72 , pp. 9-17
    • Mercuri, E.1    Muntoni, F.2
  • 51
    • 79959913312 scopus 로고
    • Congenital muscular dystrophy overview
    • Pagon, R.A., Adam, M.P., Ardinger, H.H., Bird, T.D., Dolan, C.R., Fong, C.-T., Smith, R.J., Stephens, K., Eds.; University of Washington: Seattle, WA, USA
    • Sparks, S.; Quijano-Roy, S.; Harper, A.; Rutkowski, A.; Gordon, E.; Hoffman, E.P.; Pegoraro, E. Congenital muscular dystrophy overview. In GeneReviews(®); Pagon, R.A., Adam, M.P., Ardinger, H.H., Bird, T.D., Dolan, C.R., Fong, C.-T., Smith, R.J., Stephens, K., Eds.; University of Washington: Seattle, WA, USA, 1993.
    • (1993) GeneReviews(®)
    • Sparks, S.1    Quijano-Roy, S.2    Harper, A.3    Rutkowski, A.4    Gordon, E.5    Hoffman, E.P.6    Pegoraro, E.7
  • 52
    • 84876831880 scopus 로고    scopus 로고
    • Congenital muscular dystrophies
    • Dulac, O., Lassonde, M., Sarnat, H., Eds.; Pediatric Neurology Part III; Elsevier: Amsterdam, The Netherlands, Chapter 143
    • Kirschner, J. Congenital muscular dystrophies. In Handbook of Clinical Neurology; Dulac, O., Lassonde, M., Sarnat, H., Eds.; Pediatric Neurology Part III; Elsevier: Amsterdam, The Netherlands, 2013; Volume 113, Chapter 143, pp. 1377-1385.
    • (2013) Handbook of Clinical Neurology , vol.113 , pp. 1377-1385
    • Kirschner, J.1
  • 55
    • 78149433910 scopus 로고    scopus 로고
    • Chronic hypoxia impairs muscle function in the Drosophila model of Duchenne's muscular dystrophy (DMD)
    • Mosqueira, M.; Willmann, G.; Ruohola-Baker, H.; Khurana, T.S. Chronic hypoxia impairs muscle function in the Drosophila model of Duchenne's muscular dystrophy (DMD). PLoS ONE 2010, 5, e13450.
    • (2010) PLoS ONE , vol.5 , pp. e13450
    • Mosqueira, M.1    Willmann, G.2    Ruohola-Baker, H.3    Khurana, T.S.4
  • 57
    • 33646768641 scopus 로고    scopus 로고
    • A Drosophila model of oculopharyngeal muscular dystrophy reveals intrinsic toxicity of PABPN1
    • Chartier, A.; Benoit, B.; Simonelig, M. A Drosophila model of oculopharyngeal muscular dystrophy reveals intrinsic toxicity of PABPN1. EMBO J. 2006, 25, 2253-2262.
    • (2006) EMBO J. , vol.25 , pp. 2253-2262
    • Chartier, A.1    Benoit, B.2    Simonelig, M.3
  • 58
    • 77956601360 scopus 로고    scopus 로고
    • The role of Drosophila Lamin C in muscle function and gene expression
    • Dialynas, G.; Speese, S.; Budnik, V.; Geyer, P.K.; Wallrath, L.L. The role of Drosophila Lamin C in muscle function and gene expression. Dev. Camb. Engl. 2010, 137, 3067-3077.
    • (2010) Dev. Camb. Engl. , vol.137 , pp. 3067-3077
    • Dialynas, G.1    Speese, S.2    Budnik, V.3    Geyer, P.K.4    Wallrath, L.L.5
  • 59
    • 84870058421 scopus 로고    scopus 로고
    • Loss of Drosophila A-type lamin C initially causes tendon abnormality including disintegration of cytoskeleton and nuclear lamina in muscular defects
    • Uchino, R.; Nonaka, Y.; Horigome, T.; Sugiyama, S.; Furukawa, K. Loss of Drosophila A-type lamin C initially causes tendon abnormality including disintegration of cytoskeleton and nuclear lamina in muscular defects. Dev. Biol. 2013, 373, 216-227.
    • (2013) Dev. Biol. , vol.373 , pp. 216-227
    • Uchino, R.1    Nonaka, Y.2    Horigome, T.3    Sugiyama, S.4    Furukawa, K.5
  • 61
    • 0141925699 scopus 로고    scopus 로고
    • The zebrafish as a model for muscular dystrophy and congenital myopathy
    • Bassett, D.I.; Currie, P.D. The zebrafish as a model for muscular dystrophy and congenital myopathy. Hum. Mol. Genet. 2003, 12 Spec No 2, R265-R270.
    • (2003) Hum. Mol. Genet. , vol.12 , pp. R265-R270
    • Bassett, D.I.1    Currie, P.D.2
  • 62
    • 4143130939 scopus 로고    scopus 로고
    • Identification of a zebrafish model of muscular dystrophy
    • Bassett, D.; Currie, P.D. Identification of a zebrafish model of muscular dystrophy. Clin. Exp. Pharmacol. Physiol. 2004, 31, 537-540.
    • (2004) Clin. Exp. Pharmacol. Physiol. , vol.31 , pp. 537-540
    • Bassett, D.1    Currie, P.D.2
  • 63
    • 84870035489 scopus 로고    scopus 로고
    • Zebrafish models flex their muscles to shed light on muscular dystrophies
    • Berger, J.; Currie, P.D. Zebrafish models flex their muscles to shed light on muscular dystrophies. Dis. Model. Mech. 2012, 5, 726-732.
    • (2012) Dis. Model. Mech. , vol.5 , pp. 726-732
    • Berger, J.1    Currie, P.D.2
  • 64
  • 66
    • 41049102573 scopus 로고    scopus 로고
    • Identification of small molecules rescuing fragile X syndrome phenotypes in Drosophila
    • Chang, S.; Bray, S.M.; Li, Z.; Zarnescu, D.C.; He, C.; Jin, P.; Warren, S.T. Identification of small molecules rescuing fragile X syndrome phenotypes in Drosophila. Nat. Chem. Biol. 2008, 4, 256-263.
    • (2008) Nat. Chem. Biol. , vol.4 , pp. 256-263
    • Chang, S.1    Bray, S.M.2    Li, Z.3    Zarnescu, D.C.4    He, C.5    Jin, P.6    Warren, S.T.7
  • 68
    • 84926122695 scopus 로고    scopus 로고
    • Modifications to toxic CUG RNAs induce structural stability, rescue mis-splicing in a myotonic dystrophy cell model and reduce toxicity in a myotonic dystrophy zebrafish model
    • deLorimier, E.; Coonrod, L.A.; Copperman, J.; Taber, A.; Reister, E.E.; Sharma, K.; Todd, P.K.; Guenza, M.G.; Berglund, J.A. Modifications to toxic CUG RNAs induce structural stability, rescue mis-splicing in a myotonic dystrophy cell model and reduce toxicity in a myotonic dystrophy zebrafish model. Nucleic Acids Res. 2014, 42, 12768-12778.
    • (2014) Nucleic Acids Res. , vol.42 , pp. 12768-12778
    • DeLorimier, E.1    Coonrod, L.A.2    Copperman, J.3    Taber, A.4    Reister, E.E.5    Sharma, K.6    Todd, P.K.7    Guenza, M.G.8    Berglund, J.A.9
  • 69
    • 0034730882 scopus 로고    scopus 로고
    • Conservation of components of the dystrophin complex in Drosophila1
    • Greener, M.J.; Roberts, R.G. Conservation of components of the dystrophin complex in Drosophila1. FEBS Lett. 2000, 482, 13-18.
    • (2000) FEBS Lett. , vol.482 , pp. 13-18
    • Greener, M.J.1    Roberts, R.G.2
  • 71
    • 35448947302 scopus 로고    scopus 로고
    • Episodic hypoxia exacerbates respiratory muscle dysfunction in DMDmdx mice
    • Farkas, G.A.; Mccormick, K.M.; Gosselin, L.E. Episodic hypoxia exacerbates respiratory muscle dysfunction in DMDmdx mice. Muscle Nerve 2007, 36, 708-710.
    • (2007) Muscle Nerve , vol.36 , pp. 708-710
    • Farkas, G.A.1    Mccormick, K.M.2    Gosselin, L.E.3
  • 73
    • 43649087291 scopus 로고    scopus 로고
    • Synaptic defects in a drosophila model of congenital muscular dystrophy
    • Wairkar, Y.P.; Fradkin, L.G.; Noordermeer, J.N.; DiAntonio, A. Synaptic defects in a drosophila model of congenital muscular dystrophy. J. Neurosci. 2008, 28, 3781-3789.
    • (2008) J. Neurosci. , vol.28 , pp. 3781-3789
    • Wairkar, Y.P.1    Fradkin, L.G.2    Noordermeer, J.N.3    DiAntonio, A.4
  • 76
    • 0029820310 scopus 로고    scopus 로고
    • Differential expression of dystrophin isoforms in strains of mdx mice with different mutations
    • Im, W.B.; Phelps, S.F.; Copen, E.H.; Adams, E.G.; Slightom, J.L.; Chamberlain, J.S. Differential expression of dystrophin isoforms in strains of mdx mice with different mutations. Hum. Mol. Genet. 1996, 5, 1149-1153.
    • (1996) Hum. Mol. Genet. , vol.5 , pp. 1149-1153
    • Im, W.B.1    Phelps, S.F.2    Copen, E.H.3    Adams, E.G.4    Slightom, J.L.5    Chamberlain, J.S.6
  • 79
    • 33744969831 scopus 로고    scopus 로고
    • Diagnosis and cell-based therapy for Duchenne muscular dystrophy in humans, mice, and zebrafish
    • Kunkel, L.M.; Bachrach, E.; Bennett, R.R.; Guyon, J.; Steffen, L. Diagnosis and cell-based therapy for Duchenne muscular dystrophy in humans, mice, and zebrafish. J. Hum. Genet. 2006, 51, 397-406.
    • (2006) J. Hum. Genet. , vol.51 , pp. 397-406
    • Kunkel, L.M.1    Bachrach, E.2    Bennett, R.R.3    Guyon, J.4    Steffen, L.5
  • 81
    • 0030848338 scopus 로고    scopus 로고
    • Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: A model for Duchenne muscular dystrophy
    • Grady, R.M.; Teng, H.; Nichol, M.C.; Cunningham, J.C.; Wilkinson, R.S.; Sanes, J.R. Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: A model for Duchenne muscular dystrophy. Cell 1997, 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
  • 82
    • 0242468210 scopus 로고    scopus 로고
    • Duchenne's muscular dystrophy: Animal models used to investigate pathogenesis and develop therapeutic strategies
    • Collins, C.A.; Morgan, J.E. Duchenne's muscular dystrophy: Animal models used to investigate pathogenesis and develop therapeutic strategies. Int. J. Exp. Pathol. 2003, 84, 165-172.
    • (2003) Int. J. Exp. Pathol. , vol.84 , pp. 165-172
    • Collins, C.A.1    Morgan, J.E.2
  • 86
    • 84875736063 scopus 로고    scopus 로고
    • Zebrafish based small molecule screens for novel DMD drugs
    • Kawahara, G.; Kunkel, L.M. Zebrafish based small molecule screens for novel DMD drugs. Drug Discov. Today Technol. 2013, 10, e91-e96.
    • (2013) Drug Discov. Today Technol. , vol.10 , pp. e91-e96
    • Kawahara, G.1    Kunkel, L.M.2
  • 89
    • 84901040034 scopus 로고    scopus 로고
    • Muscle dysfunction and structural defects of dystrophin-null sapje mutant zebrafish larvae are rescued by ataluren treatment
    • Li, M.; Andersson-Lendahl, M.; Sejersen, T.; Arner, A. Muscle dysfunction and structural defects of dystrophin-null sapje mutant zebrafish larvae are rescued by ataluren treatment. FASEB J. 2014, 28, 1593-1599.
    • (2014) FASEB J. , vol.28 , pp. 1593-1599
    • Li, M.1    Andersson-Lendahl, M.2    Sejersen, T.3    Arner, A.4
  • 90
    • 82455184945 scopus 로고    scopus 로고
    • Evaluation of exon-skipping strategies for Duchenne muscular dystrophy utilizing dystrophin-deficient zebrafish
    • Berger, J.; Berger, S.; Jacoby, A.S.; Wilton, S.D.; Currie, P.D. Evaluation of exon-skipping strategies for Duchenne muscular dystrophy utilizing dystrophin-deficient zebrafish. J. Cell. Mol. Med. 2011, 15, 2643-2651.
    • (2011) J. Cell. Mol. Med. , vol.15 , pp. 2643-2651
    • Berger, J.1    Berger, S.2    Jacoby, A.S.3    Wilton, S.D.4    Currie, P.D.5
  • 92
    • 84894303874 scopus 로고    scopus 로고
    • High-content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC-independent pathway in myotonic dystrophy cell lines
    • Ketley, A.; Chen, C.Z.; Li, X.; Arya, S.; Robinson, T.E.; Granados-Riveron, J.; Udosen, I.; Morris, G.E.; Holt, I.; Furling, D.; et al. High-content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC-independent pathway in myotonic dystrophy cell lines. Hum. Mol. Genet. 2014, 23, 1551-1562.
    • (2014) Hum. Mol. Genet. , vol.23 , pp. 1551-1562
    • Ketley, A.1    Chen, C.Z.2    Li, X.3    Arya, S.4    Robinson, T.E.5    Granados-Riveron, J.6    Udosen, I.7    Morris, G.E.8    Holt, I.9    Furling, D.10
  • 93
    • 0028886936 scopus 로고
    • Gene therapy-A novel Form of Drug Delivery
    • Blau, H.M.; Springer, M.L. Gene therapy-A novel Form of Drug Delivery. N. Engl. J. Med. 1995, 333, 1204-1207.
    • (1995) N. Engl. J. Med. , vol.333 , pp. 1204-1207
    • Blau, H.M.1    Springer, M.L.2
  • 94
    • 0036796262 scopus 로고    scopus 로고
    • Gene therapy of muscular dystrophy
    • Chamberlain, J.S. Gene therapy of muscular dystrophy. Hum. Mol. Genet. 2002, 11, 2355-2362.
    • (2002) Hum. Mol. Genet. , vol.11 , pp. 2355-2362
    • Chamberlain, J.S.1
  • 96
    • 84882656215 scopus 로고    scopus 로고
    • Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies
    • Benedetti, S.; Hoshiya, H.; Tedesco, F.S. Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies. FEBS J. 2013, 280, 4263-4280.
    • (2013) FEBS J. , vol.280 , pp. 4263-4280
    • Benedetti, S.1    Hoshiya, H.2    Tedesco, F.S.3
  • 97
    • 77951672406 scopus 로고    scopus 로고
    • High-throughput screening and small animal models, where are we?
    • Giacomotto, J.; Ségalat, L. High-throughput screening and small animal models, where are we? Br. J. Pharmacol. 2010, 160, 204-216.
    • (2010) Br. J. Pharmacol. , vol.160 , pp. 204-216
    • Giacomotto, J.1    Ségalat, L.2
  • 98
    • 84869016210 scopus 로고    scopus 로고
    • A novel inhibitor of Amyloid β (Aβ) peptide aggregation from high throughput screening to efficacy in an animal model of alzheimer disease
    • McKoy, A.F.; Chen, J.; Schupbach, T.; Hecht, M.H. A novel inhibitor of Amyloid β (Aβ) peptide aggregation from high throughput screening to efficacy in an animal model of alzheimer disease. J. Biol. Chem. 2012, 287, 38992-39000.
    • (2012) J. Biol. Chem. , vol.287 , pp. 38992-39000
    • McKoy, A.F.1    Chen, J.2    Schupbach, T.3    Hecht, M.H.4
  • 99
    • 84859227688 scopus 로고    scopus 로고
    • Chemical screen reveals small molecules suppressing fragile X premutation rCGG repeat-mediated neurodegeneration in Drosophila
    • Qurashi, A.; Liu, H.; Ray, L.; Nelson, D.L.; Duan, R.; Jin, P. Chemical screen reveals small molecules suppressing fragile X premutation rCGG repeat-mediated neurodegeneration in Drosophila. Hum. Mol. Genet. 2012, 21, 2068-2075.
    • (2012) Hum. Mol. Genet. , vol.21 , pp. 2068-2075
    • Qurashi, A.1    Liu, H.2    Ray, L.3    Nelson, D.L.4    Duan, R.5    Jin, P.6
  • 100
    • 80052333682 scopus 로고    scopus 로고
    • High-content chemical and RNAi screens for suppressors of neurotoxicity in a huntington's disease model
    • Schulte, J.; Sepp, K.J.; Wu, C.; Hong, P.; Littleton, J.T. High-content chemical and RNAi screens for suppressors of neurotoxicity in a huntington's disease model. PLoS ONE 2011, 6, e23841.
    • (2011) PLoS ONE , vol.6 , pp. e23841
    • Schulte, J.1    Sepp, K.J.2    Wu, C.3    Hong, P.4    Littleton, J.T.5
  • 101
    • 79961062490 scopus 로고    scopus 로고
    • In vivo discovery of a peptide that prevents CUG-RNA hairpin formation and reverses RNA toxicity in myotonic dystrophy models
    • García-López, A.; Llamusí, B.; Orzáez, M.; Pérez-Payá, E.; Artero, R.D. In vivo discovery of a peptide that prevents CUG-RNA hairpin formation and reverses RNA toxicity in myotonic dystrophy models. Proc. Natl. Acad. Sci. USA 2011, 108, 11866-11871.
    • (2011) Proc. Natl. Acad. Sci. USA , vol.108 , pp. 11866-11871
    • García-López, A.1    Llamusí, B.2    Orzáez, M.3    Pérez-Payá, E.4    Artero, R.D.5
  • 102
    • 0036244951 scopus 로고    scopus 로고
    • The art and design of genetic screens: Drosophila melanogaster
    • St Johnston, D. The art and design of genetic screens: drosophila melanogaster. Nat. Rev. Genet. 2002, 3, 176-188.
    • (2002) Nat. Rev. Genet. , vol.3 , pp. 176-188
    • St Johnston, D.1
  • 105
    • 79953027747 scopus 로고    scopus 로고
    • Stress and muscular dystrophy: A genetic screen for Dystroglycan and Dystrophin interactors in Drosophila identifies cellular stress response components
    • Kucherenko, M.M.; Marrone, A.K.; Rishko, V.M.; Magliarelli Hde, F.; Shcherbata, H.R. Stress and muscular dystrophy: A genetic screen for Dystroglycan and Dystrophin interactors in Drosophila identifies cellular stress response components. Dev. Biol. 2011, 352, 228-242.
    • (2011) Dev. Biol. , vol.352 , pp. 228-242
    • Kucherenko, M.M.1    Marrone, A.K.2    Rishko, V.M.3    Magliarelli Hde, F.4    Shcherbata, H.R.5
  • 106
    • 84876830225 scopus 로고    scopus 로고
    • Smaug/SAMD4A restores translational activity of CUGBP1 and suppresses CUG-Induced myopathy
    • De Haro, M.; Al-Ramahi, I.; Jones, K.R.; Holth, J.K.; Timchenko, L.T.; Botas, J. Smaug/SAMD4A restores translational activity of CUGBP1 and suppresses CUG-Induced myopathy. PLoS Genet 2013, 9, e1003445.
    • (2013) PLoS Genet , vol.9 , pp. e1003445
    • De Haro, M.1    Al-Ramahi, I.2    Jones, K.R.3    Holth, J.K.4    Timchenko, L.T.5    Botas, J.6
  • 107
    • 84903993789 scopus 로고    scopus 로고
    • Zebrafish models in translational research: Tipping the scales toward advancements in human health
    • Phillips, J.B.; Westerfield, M. Zebrafish models in translational research: Tipping the scales toward advancements in human health. Dis. Model. Mech. 2014, 7, 739-743.
    • (2014) Dis. Model. Mech. , vol.7 , pp. 739-743
    • Phillips, J.B.1    Westerfield, M.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.