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Review

Recent advances in Duchenne muscular dystrophy

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Pages 141-164 | Published online: 12 Oct 2012

References

  • DubowitzVMuscle disorders in childhoodMajor Probl Clin Pediatr197816iiixiii1282661378
  • EmeryAMuscular dystrophy – the factsNeuromuscul Disord1995565218580734
  • BushbyKFinkelRBirnkrantDJDiagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial managementLancet Neurol201091779319945913
  • MonacoAPNeveRLColletti-FeenerCBertelsonCJKurnitDMKunkelLMIsolation of candidate cDNAs for portions of the Duchenne muscular dystrophy geneNature198632360896466503773991
  • HoffmanEPBrownRHJrKunkelLMDystrophin: the protein product of the Duchenne muscular dystrophy locusCell19875169199283319190
  • KoenigMHoffmanEPBertelsonCJMonacoAPFeenerCKunkelLMComplete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individualsCell19875035095173607877
  • LevineBAMoirAJPatchellVBPerrySVThe interaction of actin with dystrophinFEBS Lett199026311591622185033
  • Ibraghimov-BeskrovnayaOErvastiJMLeveilleCJSlaughterCASernettSWCampbellKPPrimary structure of dystrophin-associated glycoproteins linking dystrophin to the extracellular matrixNature199235563626967021741056
  • DaviesKENowakKJMolecular mechanisms of muscular dystrophies: old and new playersNat Rev Mol Cell Biol200671076277316971897
  • BrenmanJEChaoDSXiaHAldapeKBredtDSNitric oxide synthase complexed with dystrophin and absent from skeletal muscle sarco-lemma in Duchenne muscular dystrophyCell19958257437527545544
  • Sadoulet-PuccioHMRajalaMKunkelLMDystrobrevin and dystrophin: an interaction through coiled-coil motifsProc Natl Acad Sci U S A1997942312413124189356463
  • YangBJungDRafaelJAChamberlainJSCampbellKPIdentification of alpha-syntrophin binding to syntrophin triplet, dystrophin, and utrophinJ Biol Chem199527010497549787890602
  • LynchGSRafaelJAChamberlainJSFaulknerJAContraction-induced injury to single permeabilized muscle fibers from mdx, transgenic mdx, and control miceAm J Physiol Cell Physiol20002794C1290C129411003610
  • Abdel-SalamEAbdel-MeguidIKorraaSSMarkers of degeneration and regeneration in Duchenne muscular dystrophyActa Myol20092839410020476668
  • FoidartMFoidartJMEngelWKCollagen localization in normal and fibrotic human skeletal muscleArch Neurol19813831521577469848
  • MorandiLMoraMGussoniETedeschiSCornelioFDystrophin analysis in Duchenne and Becker muscular dystrophy carriers: correlation with intracellular calcium and albuminAnn Neurol19902856746791979724
  • NetheryDCallahanLAStofanDMatteraRDiMarcoASupinskiGPLA(2) dependence of diaphragm mitochondrial formation of reactive oxygen speciesJ Appl Physiol2000891728010904037
  • McDouallRMDunnMJDubowitzVExpression of class I and class II MHC antigens in neuromuscular diseasesJ Neurol Sci1989892–32132262926449
  • De PaepeBCreusKKMartinJJDe BleeckerJLUpregulation of chemokines and their receptors in Duchenne muscular dystrophy: potential for attenuation of myofiber necrosisMuscle Nerve Epub642012
  • IchimTEAlexandrescuDTSolanoFMesenchymal stem cells as anti-inflammatories: implications for treatment of Duchenne muscular dystrophyCell Immunol20102602758219917503
  • DeSilvaSDrachmanDBMellitsDKunclRWPrednisone treatment in Duchenne muscular dystrophy. Long-term benefitArch Neurol19874488188223632394
  • MoxleyRT3rdAshwalSPandyaSPractice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology SocietyNeurology2005641132015642897
  • WillmannRPossekelSDubach-PowellJMeierTRueggMAMammalian animal models for Duchenne muscular dystrophyNeuromuscul Disord200919424124919217290
  • BulfieldGSillerWGWightPAMooreKJX chromosome-linked muscular dystrophy (mdx) in the mouseProc Natl Acad Sci U S A1984814118911926583703
  • SicinskiPGengYRyder-CookASBarnardEADarlisonMGBarnardPJThe molecular basis of muscular dystrophy in the mdx mouse: a point mutationScience19892444912157815802662404
  • StedmanHHSweeneyHLShragerJBThe mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophyNature199135263355365391865908
  • ChapmanVMMillerDRArmstrongDCaskeyCTRecovery of induced mutations for X chromosome-linked muscular dystrophy in miceProc Natl Acad Sci U S A1989864129212962919177
  • Helderman-van den EndenATStraathofCSAartsma-RusABecker muscular dystrophy patients with deletions around exon 51; a promising outlook for exon skipping therapy in Duchenne patientsNeuromuscul Disord201020425125420153965
  • ArakiENakamuraKNakaoKTargeted disruption of exon 52 in the mouse dystrophin gene induced muscle degeneration similar to that observed in Duchenne muscular dystrophyBiochem Biophys Res Commun199723824924979299538
  • Bremmer-BoutMAartsma-RusAde MeijerEJTargeted exon skipping in transgenic hDMD mice: a model for direct preclinical screening of human-specific antisense oligonucleotidesMol Ther200410223224015294170
  • MegeneyLAKablarBGarrettKAndersonJERudnickiMAMyoD is required for myogenic stem cell function in adult skeletal muscleGenes Dev19961010117311838675005
  • GradyRMGrangeRWLauKSRole for alpha-dystrobrevin in the pathogenesis of dystrophin-dependent muscular dystrophiesNat Cell Biol19991421522010559919
  • RaymackersJMDebaixHColson-Van SchoorMConsequence of parvalbumin deficiency in the mdx mouse: histological, biochemical and mechanical phenotype of a new double mutantNeuromuscul Disord200313537638712798793
  • GuoCWillemMWernerAAbsence of alpha 7 integrin in dystrophin-deficient mice causes a myopathy similar to Duchenne muscular dystrophyHum Mol Genet200615698999816476707
  • ChandrasekharanKYoonJHXuYA human-specific deletion in mouse Cmah increases disease severity in the mdx model of Duchenne muscular dystrophySci Transl Med201024242ra54
  • DeconinckAERafaelJASkinnerJAUtrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophyCell19979047177279288751
  • ZhouLRafael-FortneyJAHuangPHaploinsufficiency of utrophin gene worsens skeletal muscle inflammation and fibrosis in mdx miceJ Neurol Sci20082641–210611117889902
  • SheltonGDEngvallECanine and feline models of human inherited muscle diseasesNeuromuscul Disord200515212713815694134
  • SharpNJKornegayJNVan CampSDAn error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophyGenomics19921311151211577476
  • ShimatsuYKatagiriKFurutaTCanine X-linked muscular dystrophy in Japan (CXMDJ)Exp Anim2003522939712806883
  • WinandNCooperBMolecular characterization of severe Duchenne-type muscular dystrophy in a family of rottweiler dogsProceedings of Molecular Mechanisms of Neuromuscular DiseaseTucsonUniversity of Arizona1994
  • SchatzbergSJOlbyNJBreenMMolecular analysis of a spontaneous dystrophin ‘knockout’ dogNeuromuscul Disord19999528929510407848
  • JonesBRBrennanSMooneyCTMuscular dystrophy with truncated dystrophin in a family of Japanese Spitz dogsJ Neurol Sci2004217214314914706216
  • WalmsleyGLArechavala-GomezaVFernandez-FuenteMA duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skippingPLoS One201051e864720072625
  • CooperBJWinandNJStedmanHThe homologue of the Duchenne locus is defective in X-linked muscular dystrophy of dogsNature198833461781541563290691
  • ShimatsuYYoshimuraMYuasaKMajor clinical and histopathological characteristics of canine X-linked muscular dystrophy in Japan, CXMDJActa Myol200524214515416550932
  • YokotaTLuQLPartridgeTEfficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogsAnn Neurol200965666767619288467
  • Aartsma-RusAJansonAAKamanWEAntisense-induced multiexon skipping for Duchenne muscular dystrophy makes more senseAm J Hum Genet2004741839214681829
  • VosJHvan der Linde-SipmanJSGoedegebuureSADystrophy-like myopathy in the catJ Comp Pathol19869633353413722476
  • KlymiukNThirionCBurkhardtK238 tailored pig model of duchenne muscular dystrophyReprod Fertil Dev2011241231
  • WinandNJEdwardsMPradhanDBerianCACooperBJDeletion of the dystrophin muscle promoter in feline muscular dystrophyNeuromuscul Disord199445–64334457881288
  • PartridgeTModels of dystrophinopathy, pathological mechanisms and assessment of therapiesBrownSCLucyJADystrophin: Gene, Protein and Cell BiologyCambridgeCambridge University Press1997310321
  • MeregalliMFariniAColleoniFCassinelliLTorrenteYThe role of stem cells in muscular dystrophiesCurr Gene Ther201212319220522463740
  • PartridgeTAGroundsMSloperJCEvidence of fusion between host and donor myoblasts in skeletal muscle graftsNature19782735660306308652035
  • MoulyVAamiriAPerieSMyoblast transfer therapy: is there any light at the end of the tunnel?Acta Myol200524212813316550930
  • HuardJRoyRBouchardJPMalouinFRichardsCLTremblayJPHuman myoblast transplantation between immunohistocompatible donors and recipients produces immune reactionsTransplant Proc1992246304930511466052
  • SkukDParadisMGouletMTremblayJPIschemic central necrosis in pockets of transplanted myoblasts in nonhuman primates: implications for cell-transplantation strategiesTransplantation200784101307131518049116
  • PriceFDKurodaKRudnickiMAStem cell based therapies to treat muscular dystrophyBiochim Biophys Acta20071772227228317034994
  • JankowskiRJDeasyBMHuardJMuscle-derived stem cellsGene Ther200291064264712032710
  • CollinsCAOlsenIZammitPSStem cell function, self-renewal, and behavioral heterogeneity of cells from the adult muscle satellite cell nicheCell2005122228930116051152
  • WebsterCBlauHMAccelerated age-related decline in replicative life-span of Duchenne muscular dystrophy myoblasts: implications for cell and gene therapySomat Cell Mol Genet19901665575652267630
  • TorrenteYBelicchiMMarchesiCAutologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patientsCell Transplant200716656357717912948
  • GussoniESoneokaYStricklandCDDystrophin expression in the mdx mouse restored by stem cell transplantationNature1999401675139039410517639
  • MuXXiangGRathboneCRSlow-adhering stem cells derived from injured skeletal muscle have improved regenerative capacityAm J Pathol2011179293194121684246
  • DarabiRGehlbachKBachooRMFunctional skeletal muscle regeneration from differentiating embryonic stem cellsNat Med200814213414318204461
  • JacksonWMNestiLJTuanRSPotential therapeutic applications of muscle-derived mesenchymal stem and progenitor cellsExpert Opin Biol Ther201010450551720218920
  • MaXZhangSZhouJClone-derived human AF-amniotic fluid stem cells are capable of skeletal myogenic differentiation in vitro and in vivoJ Tissue Eng Regen Med20126859861322396316
  • KongKYRenJKrausMFinklesteinSPBrownRHJrHuman umbilical cord blood cells differentiate into muscle in sjl muscular dystrophy miceStem Cells200422698199315536189
  • ClinicalTrials.gov [homepage on the Internet]Clinical Trials for Duchenne muscular dystrophyBethesda, MDUS National Library of Medicine2012 [cited August 8, 2012]. Available from: http://clinicaltrials.gov/ct2/results?term=dmdAccessed August 18, 2012
  • SampaolesiMTorrenteYInnocenziACell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblastsScience2003301563248749212855815
  • DezawaMIshikawaHItokazuYBone marrow stromal cells generate muscle cells and repair muscle degenerationScience2005309573231431716002622
  • Nitahara-KasaharaYHayashita-KinohHOhshima-HosoyamaSLong-term engraftment of multipotent mesenchymal stromal cells that differentiate to form myogenic cells in dogs with Duchenne muscular dystrophyMol Ther201220116817721934652
  • BerrySELiuJChaneyEJKaufmanSJMultipotential mesoangioblast stem cell therapy in the mdx/utrn−/− mouse model for Duchenne muscular dystrophyRegen Med20072327528817511564
  • OptiStem.org [homepage on the Internet]Progress reportsOtpiStem2012 [cited August 8, 2012]. Available from: http://www.optistem.org/progress-reportsAccessed August 18, 2012
  • HarperSQHauserMADelloRussoCModular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophyNat Med20028325326111875496
  • SakamotoMYuasaKYoshimuraMMicro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgeneBiochem Biophys Res Commun200229341265127212054513
  • BowlesDEMcPheeSWLiCPhase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vectorMol Ther201220244345522068425
  • SetoJTRamosJNMuirLChamberlainJSOdomGLGene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectorsCurr Gene Ther201212313915122533379
  • YuasaKYoshimuraMUrasawaNInjection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene productsGene Ther200714171249126017581597
  • KooTMalerbaAAthanasopoulosTDelivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of alpha1-syntrophin and alpha-dystrobrevin in skeletal muscles of mdx miceHum Gene Ther201122111379138821453126
  • KornegayJNLiJBoganJRWidespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogsMol Ther20101881501150820517298
  • OhshimaSShinJHYuasaKTransduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscleMol Ther2009171738018941441
  • MendellJRCampbellKRodino-KlapacLDystrophin immunity in Duchenne’s muscular dystrophyN Engl J Med2010363151429143720925545
  • AcsadiGDicksonGLoveDRHuman dystrophin expression in mdx mice after intramuscular injection of DNA constructsNature199135263388158181881437
  • DankoIFritzJDLatendresseJSHerweijerHSchultzEWolffJADystrophin expression improves myofiber survival in mdx muscle following intramuscular plasmid DNA injectionHum Mol Genet1993212205520618111373
  • DecrouyARenaudJMDavisHLLundeJADicksonGJasminBJMini-dystrophin gene transfer in mdx4cv diaphragm muscle fibers increases sarcolemmal stabilityGene Ther1997454014089274716
  • PichavantCChapdelainePCerriDGBizarioJCTremblayJPElectrotransfer of the full-length dog dystrophin into mouse and dystrophic dog musclesHum Gene Ther201021111591160120553115
  • RomeroNBBraunSBenvenisteOPhase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophyHum Gene Ther200415111065107615610607
  • DuanDMyodys, a full-length dystrophin plasmid vector for Duchenne and Becker muscular dystrophy gene therapyCurr Opin Mol Ther2008101869418228186
  • BachrachELiSPerezALSystemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cellsProc Natl Acad Sci U S A2004101103581358614993597
  • PichavantCChapdelainePCerriDGExpression of dog microdystrophin in mouse and dog muscles by gene therapyMol Ther20101851002100920179674
  • KimuraELiSGregorevicPFallBMChamberlainJSDystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expressionMol Ther201018120621319888194
  • SampaolesiMBlotSD’AntonaGMesoangioblast stem cells ameliorate muscle function in dystrophic dogsNature2006444711957457917108972
  • FengSWChenFCaoJRestoration of muscle fibers and satellite cells after isogenic MSC transplantation with microdystrophin gene deliveryBiochem Biophys Res Commun201241911622321394
  • IkezawaMCaoBQuZDystrophin delivery in dystrophin-deficient DMDmdx skeletal muscle by isogenic muscle-derived stem cell transplantationHum Gene Ther200314161535154614577915
  • MoissetPASkukDAsselinISuccessful transplantation of genetically corrected DMD myoblasts following ex vivo transduction with the dystrophin minigeneBiochem Biophys Res Commun1998247194999636661
  • MoissetPAGagnonYKarpatiGTremblayJPExpression of human dystrophin following the transplantation of genetically modified mdx myoblastsGene Ther1998510134013469930339
  • HoshiyaHKazukiYAbeSA highly stable and nonintegrated human artificial chromosome (HAC) containing the 2.4 Mb entire human dystrophin geneMol Ther200917230931719034264
  • NakahataTAwayaTChangHDerivation of engraftable myogenic precursors from murine ES/iPS cells and generation of disease-specific iPS cells from patients with Duchenne muscular dystrophy (DMD) and other diseasesRinsho Shinkeigaku2010501188921921492
  • KazukiYHiratsukaMTakiguchiMComplete genetic correction of ips cells from Duchenne muscular dystrophyMol Ther201018238639319997091
  • TedescoFSHoshiyaHD’AntonaGStem cell-mediated transfer of a human artificial chromosome ameliorates muscular dystrophySci Transl Med201139696ra78
  • RandoTADisatnikMHZhouLZRescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotidesProc Natl Acad Sci U S A200097105363536810805797
  • BertoniCRandoTADystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotidesHum Gene Ther200213670771811936970
  • BartlettRJStockingerSDenisMMIn vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotideNat Biotechnol200018661562210835598
  • AlexeevVIgouchevaOYoonKSimultaneous targeted alteration of the tyrosinase and c-kit genes by single-stranded oligonucleotidesGene Ther20029241667167512457280
  • BertoniCLauCRandoTARestoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skippingHum Mol Genet200312101087109912719373
  • BertoniCMorrisGERandoTAStrand bias in oligonucleotide- mediated dystrophin gene editingHum Mol Genet200514222123315563511
  • MaguireKSuzukiTDiMatteoDParekh-OlmedoHKmiecEGenetic correction of splice site mutation in purified and enriched myoblasts isolated from mdx5cv miceBMC Mol Biol2009101519236710
  • KoleRSazaniPAntisense effects in the cell nucleus: modification of splicingCurr Opin Mol Ther20013322923411497345
  • BenchaouirRGoyenvalleASplicing modulation mediated by small nuclear RNAs as therapeutic approaches for muscular dystrophiesCurr Gene Ther201212317919122515846
  • DunckleyMGManoharanMVillietPEperonICDicksonGModification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotidesHum Mol Genet199877108310909618164
  • MannCJHoneymanKChengAJAntisense-induced exon skipping and synthesis of dystrophin in the mdx mouseProc Natl Acad Sci U S A2001981424711120883
  • Arechavala-GomezaVAnthonyKMorganJMuntoniFAntisense oligonucleotide-mediated exon skipping for duchenne muscular dystrophy: progress and challengesCurr Gene Ther201212315216022533380
  • LuQLRabinowitzAChenYCSystemic delivery of anti-sense oligoribonucleotide restores dystrophin expression in body-wide skeletal musclesProc Natl Acad Sci U S A2005102119820315608067
  • AokiYNakamuraAYokotaTIn-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouseMol Ther201018111995200520823833
  • YokotaTHoffmanETakedaSAntisense oligo-mediated multiple exon skipping in a dog model of duchenne muscular dystrophyMethods Mol Biol201170929931221194037
  • MitrpantCFletcherSIversenPLWiltonSDBy-passing the nonsense mutation in the 4 CV mouse model of muscular dystrophy by induced exon skippingJ Gene Med2009111465619006096
  • YinHLuQWoodMEffective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx miceMol Ther2008161384517968354
  • Prosensa.eu [homepage on the Internet]Pre-clinical portfolioLeiden, The NetherlandsProsensa; 2012 [cited August 8, 2012]. Available from: http://prosensa.eu/technology-and-products/pre-clinical-portfolioAccessed August 18, 2012
  • van DeutekomJCJansonAAGinjaarIBLocal dystrophin restoration with antisense oligonucleotide PRO051N Engl J Med2007357262677268618160687
  • NeriMTorelliSBrownSDystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the humanNeuromuscul Disord20071711–1291391817826093
  • GoemansNMTuliniusMvan den AkkerJTSystemic administration of PRO051 in Duchenne’s muscular dystrophyN Engl J Med2011364161513152221428760
  • Sareptatherapeutics.com [homepage on the Internet]Duchenne muscular dystrophy: drug candidates/clinical trialsCambridge, MASerepta Therapeutics2012 [cited August 8, 2012]. Available from: http://www.sareptatherapeutics.com/our-programs/rare-diseases/duchenne-muscular-dystrophyAccessed August 18, 2012
  • Arechavala-GomezaVGrahamIRPopplewellLJComparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscleHum Gene Ther200718979881017767400
  • KinaliMArechavala-GomezaVFengLLocal restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept studyLancet Neurol200981091892819713152
  • CirakSArechavala-GomezaVGuglieriMExon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation studyLancet2011378979159560521784508
  • De AngelisFGSthandierOBerarducciBChimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48–50 DMD cellsProc Natl Acad Sci U S A200299149456946112077324
  • BrunCSuterDPauliCU7snRNAs induce correction of mutated dystrophin pre-mRNA by exon skippingCell Mol Life Sci200360355756612737315
  • GoyenvalleAVulinAFougerousseFRescue of dystrophic muscle through U7 snRNA-mediated exon skippingScience200430657021796179915528407
  • DentiMARosaAD’AntonaGChimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx miceHum Gene Ther200617556557416716113
  • GoyenvalleABabbsAWrightJRescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping. Hum Mol GenetMar 132012211125592571
  • GoyenvalleAWrightJBabbsAWilkinsVGarciaLDaviesKEEngineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophyMol Ther20122061212122122354379
  • BishLTSleeperMMForbesSCLong-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skippingMol Ther201220358058922146342
  • BarbashIMCecchiniSFaraneshAZMRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophyGene Ther Epub532012
  • ChapdelainePPichavantCRousseauJPaquesFTremblayJPMeganucleases can restore the reading frame of a mutated dystrophinGene Ther201017784685820393509
  • Aartsma-RusAFokkemaIVerschuurenJTheoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutationsHum Mutat200930329329919156838
  • HeemskerkHde WinterCvan KuikPPreclinical PK and PD studies on 2′-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse modelMol Ther20101861210121720407428
  • Aartsma-RusAvan OmmenGJLess is more: therapeutic exon skipping for Duchenne muscular dystrophyLancet Neurol200981087387519713153
  • ‘t HoenPAde MeijerEJBoerJMGeneration and characterization of transgenic mice with the full-length human DMD geneJ Biol Chem200828395899590718083704
  • MitrpantCAdamsAMMeloniPLMuntoniFFletcherSWiltonSDRational design of antisense oligomers to induce dystrophin exon skippingMol Ther20091781418142619293776
  • O’LearyDASharifOAndersonPIdentification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screeningPLoS One2009412e834820020055
  • MoultonHMCell-penetrating peptides enhance systemic delivery of antisense morpholino oligomersMethods Mol Biol201286740741422454076
  • MalikVRodino-KlapacLRViolletLGentamicin-induced readthrough of stop codons in Duchenne muscular dystrophyAnn Neurol201067677178020517938
  • BushbyKGenetics and the muscular dystrophiesDev Med Child Neurol2000421178078411104352
  • ManuvakhovaMKeelingKBedwellDMAminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation systemRNA2000671044105510917599
  • PalmerEWilhelmJMShermanFPhenotypic suppression of nonsense mutants in yeast by aminoglycoside antibioticsNature19792775692148150366439
  • Barton-DavisERCordierLShoturmaDILelandSESweeneyHLAminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx miceJ Clin Invest1999104437538110449429
  • LoufraniLDubrocaCYouDAbsence of dystrophin in mice reduces NO-dependent vascular function and vascular density: total recovery after a treatment with the aminoglycoside gentamicinArterioscler Thromb Vasc Biol200424467167614751810
  • PolitanoLNigroGNigroVGentamicin administration in Duchenne patients with premature stop codon. Preliminary resultsActa Myol2003221152112966700
  • WagnerKRHamedSHadleyDWGentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutationsAnn Neurol200149670671111409421
  • YoshizawaSFourmyDPuglisiJDStructural origins of gentamicin antibiotic actionEMBO J19981722643764489822590
  • DunantPWalterMCKarpatiGLochmullerHGentamicin fails to increase dystrophin expression in dystrophin-deficient muscleMuscle Nerve200327562462712707984
  • YukiharaMItoKTanoueOEffective drug delivery system for duchenne muscular dystrophy using hybrid liposomes including gentamicin along with reduced toxicityBiol Pharm Bull201134571271621532162
  • A New Approach to Drug Discovery [webpage on the Internet]. Therapeutic areasSouth Plainfield, NJPTC Therapeutics2012 [cited August 8, 2012]. Available from: http://www.ptcbio.com/therapeutic_areasAccessed August 18, 2012
  • WelchEMBartonERZhuoJPTC124 targets genetic disorders caused by nonsense mutationsNature20074477140879117450125
  • ArakawaMShiozukaMNakayamaYNegamycin restores dystrophin expression in skeletal and cardiac muscles of mdx miceJ Biochem2003134575175814688241
  • LoveDRHillDFDicksonGAn autosomal transcript in skeletal muscle with homology to dystrophinNature1989339621955582541343
  • TinsleyJMBlakeDJRocheAPrimary structure of dystrophin-related proteinNature199236064045915931461283
  • KhuranaTSWatkinsSCChafeyPImmunolocalization and developmental expression of dystrophin related protein in skeletal muscleNeuromuscul Disord1991131851941822793
  • FisherRTinsleyJMPhelpsSRNon-toxic ubiquitous over-expression of utrophin in the mdx mouseNeuromuscul Disord200111871372111595513
  • MoorwoodCLozynskaOSuriNNapperADDiamondSLKhuranaTSDrug discovery for Duchenne muscular dystrophy via utrophin promoter activation screeningPLoS One2011610e2616922028826
  • FaircloughRJPerkinsKJDaviesKEPharmacologically targeting the primary defect and downstream pathology in duchenne muscular dystrophyCurr Gene Ther201212320624422571500
  • GramoliniAOBelangerGThompsonJMChakkalakalJVJasminBJIncreased expression of utrophin in a slow vs a fast muscle involves posttranscriptional eventsAm J Physiol Cell Physiol20012814C1300C130911546668
  • SelsbyJTMorineKJPendrakKBartonERSweeneyHLRescue of dystrophic skeletal muscle by PGC-1alpha involves a fast to slow fiber type shift in the mdx mousePLoS One201271e3006322253880
  • HandschinCKobayashiYMChinSSealePCampbellKPSpiegelmanBMPGC-1alpha regulates the neuromuscular junction program and ameliorates Duchenne muscular dystrophyGenes Dev200721777078317403779
  • AngusLMChakkalakalJVMejatACalcineurin-NFAT signaling, together with GABP and peroxisome PGC-1{alpha}, drives utrophin gene expression at the neuromuscular junctionAm J Physiol Cell Physiol20052894C908C91715930144
  • MiuraPChakkalakalJVBoudreaultLPharmacological activation of PPARbeta/delta stimulates utrophin A expression in skeletal muscle fibers and restores sarcolemmal integrity in mature mdx miceHum Mol Genet200918234640464919744959
  • LampenASiehlerSEllerbeckUGottlicherMNauHNew molecular bioassays for the estimation of the teratogenic potency of valproic acid derivatives in vitro: activation of the peroxisomal proliferator-activated receptor (PPARdelta)Toxicol Appl Pharmacol1999160323824910544058
  • Lopez-SorianoJChielliniCMaffeiMGrimaldiPAArgilesJMRoles of skeletal muscle and peroxisome proliferator-activated receptors in the development and treatment of obesityEndocr Rev200627331832916556851
  • GardnerOSDewarBJGravesLMActivation of mitogen-activated protein kinases by peroxisome proliferator-activated receptor ligands: an example of nongenomic signalingMol Pharmacol200568493394116020742
  • BalakumarPKathuriaSSubmaximal PPARgamma activation and endothelial dysfunction: new perspectives for the management of cardiovascular disordersBr J Pharmacol201216671981199222404217
  • LeickLFentzJBiensoRSPGC-1{alpha} is required for AICAR-induced expression of GLUT4 and mitochondrial proteins in mouse skeletal muscleAm J Physiol Endocrinol Metab20102993E456E46520628026
  • LjubicicVKhogaliSRenaudJMJasminBJChronic AMPK stimulation attenuates adaptive signaling in dystrophic skeletal muscleAm J Physiol Cell Physiol20113021C110C12121940670
  • PoldRJensenLSJessenNLong-term AICAR administration and exercise prevents diabetes in ZDF ratsDiabetes200554492893415793229
  • PassanantiCCorbiNOnoriADi CertoMGMatteiETransgenic mice expressing an artificial zinc finger regulator targeting an endogenous geneMethods Mol Biol201064918320620680835
  • TinsleyJMFaircloughRJStorerRDaily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mousePLoS One201165e1918921573153
  • Biomarin Pharmaceutical IncBioMarin and Summit plc sign worldwide licensing agreement for Duchenne muscular dystrophy program [press release]Novato, CABiomarin Pharmaceutical Inc2008722 Available from: http://phx.corporate-ir.net/phoenix.zhtml?c=106657&p=irol-newsArticle&ID=1177621&highlight=Accessed July 22, 2008
  • FaircloughRFSquireSEPotterACIdentification of new chemical compounds which upregulate utrophin for the therapy of Duchenne muscular dystrophyProceedings of the Muscular Dystrophy Campaign UK Neuromuscular Translational Research Conference2012 Mar 22–23Newcastle, UK
  • SchwarzeSRHoAVocero-AkbaniADowdySFIn vivo protein transduction: delivery of a biologically active protein into the mouseScience199928554331569157210477521
  • SonnemannKJHeun-JohnsonHTurnerAJBaltgalvisKALoweDAErvastiJMFunctional substitution by TAT-utrophin in dystrophin-deficient micePLoS Med200965e100008319478831
  • CallJAErvastiJMLoweDATAT-muUtrophin mitigates the pathophysiology of dystrophin and utrophin double-knockout miceJ Appl Physiol2011111120020521565990
  • Retrophin. RE-001: Retrophin’s investigational agent for Duchenne muscular dystrophyNew York, NYRetrophin LLC2011 [cited August 8, 2012]. Available from: http://www.retrophin.com/pipeline.phpAccessed September 25, 2012
  • Tivorsan PharmaceuticalsMDA awards $1 million to Tivorsan Pharmaceuticals for accelerating pivotal pre-clinical work on Tvn-102 as a potential muscular dystrophy treatment [press release]Providence, RITivorsan Pharmaceuticals, Inc201215 Available from: http://www.tivorsan.com/wp-content/uploads/2012/01/PR_MDATivorsan_1M_Final_1_5_12.pdfAccessed January 5, 2012
  • ZanottiSNegriTCappellettiCDecorin and biglycan expression is differentially altered in several muscular dystrophiesBrain2005128Pt 112546255516183658
  • AmentaARYilmazABogdanovichSBiglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx miceProc Natl Acad Sci U S A2010108276276721187385
  • HynesROIntegrins: versatility, modulation, and signaling in cell adhesionCell199269111251555235
  • HodgesBLHayashiYKNonakaIWangWArahataKKaufmanSJAltered expression of the alpha7beta1 integrin in human and murine muscular dystrophiesJ Cell Sci1997110Pt 22287328819427295
  • MayerUSaherGFasslerRAbsence of integrin alpha 7 causes a novel form of muscular dystrophyNat Genet19971733183239354797
  • BurkinDJWallaceGQNicolKJKaufmanDJKaufmanSJEnhanced expression of the alpha 7 beta 1 integrin reduces muscular dystrophy and restores viability in dystrophic miceJ Cell Biol200115261207121811257121
  • LiuJBurkinDJKaufmanSJIncreasing alpha 7 beta 1-integrin promotes muscle cell proliferation, adhesion, and resistance to apoptosis without changing gene expressionAm J Physiol Cell Physiol20082942C627C64018045857
  • GurpurPBLiuJBurkinDJKaufmanSJValproic acid activates the PI3K/Akt/mTOR pathway in muscle and ameliorates pathology in a mouse model of Duchenne muscular dystrophyAm J Pathol20091743999100819179609
  • BodineSCStittTNGonzalezMAkt/mTOR pathway is a crucial regulator of skeletal muscle hypertrophy and can prevent muscle atrophy in vivoNat Cell Biol20013111014101911715023
  • RooneyJEGurpurPBBurkinDJLaminin-111 protein therapy prevents muscle disease in the mdx mouse model for Duchenne muscular dystrophyProc Natl Acad Sci U S A2009106197991799619416897
  • RooneyJEGurpurPBYablonka-ReuveniZBurkinDJLaminin-111 restores regenerative capacity in a mouse model for alpha7 integrin congenital myopathyAm J Pathol2009174125626419074617
  • Coral-VazquezRCohnRDMooreSADisruption of the sarcoglycan-sarcospan complex in vascular smooth muscle: a novel mechanism for cardiomyopathy and muscular dystrophyCell199998446547410481911
  • Prothelia [webpage on the Internet]Therapeutics for muscular dystrophyMilford, MAProthelia Inc2012 [cited August 8, 2012]. Available from: http://www.prothelia.com/livesite/pages/pipelineAccessed September 25, 2012
  • CombinatoRxCombinatoRx and Charley’s Fund/Nash Avery Foundation collaborate to develop novel agents for Duchenne muscular dystrophy [press release]CombinatoRx, Incorporated2007 [cited August 8, 2012]. Available from: http://phx.corporate-ir.net/phoenix.zhtml?c=148036&p=irol-newsArticle&ID=1074719&highlight=Accessed September 25, 2012
  • RandoTAOxidative stress and the pathogenesis of muscular dystrophiesAm J Phys Med Rehabil200281Suppl 11S175S18612409822
  • MorganMJLiuZGCrosstalk of reactive oxygen species and NF-kappaB signalingCell Res201121110311521187859
  • SpurneyCFGuerronADYuQMembrane sealant Poloxamer P188 protects against isoproterenol induced cardiomyopathy in dystrophin deficient miceBMC Cardiovasc Disord2011112021575230
  • TownsendDTurnerIYasudaSChronic administration of membrane sealant prevents severe cardiac injury and ventricular dilatation in dystrophic dogsJ Clin Invest201012041140115020234088
  • TidballJGWehling-HenricksMThe role of free radicals in the pathophysiology of muscular dystrophyJ Appl Physiol200710241677168617095633
  • HankardRMaurasNHammondDHaymondMDarmaunDIs glutamine a ‘conditionally essential’ amino acid in Duchenne muscular dystrophy?Clin Nutr199918636536910634922
  • SalehianBMahabadiVBilasJTaylorWEMaKThe effect of glutamine on prevention of glucocorticoid-induced skeletal muscle atrophy is associated with myostatin suppressionMetabolism20065591239124716919545
  • MokEConstantinBFavreauFl-Glutamine administration reduces oxidized glutathione and MAP kinase signaling in dystrophic muscle of mdx micePediatr Res200863326827318287965
  • HibaouiYReutenauer-PatteJPatthey-VuadensORueggUTDorchiesOMMelatonin improves muscle function of the dystrophic mdx5Cv mouse, a model for Duchenne muscular dystrophyJ Pineal Res201151216317121486366
  • ChahbouniMEscamesGVenegasCMelatonin treatment normalizes plasma pro-inflammatory cytokines and nitrosative/oxidative stress in patients suffering from Duchenne muscular dystrophyJ Pineal Res201048328228920210854
  • SahJFBalasubramanianSEckertRLRorkeEAEpigallocatechin-3-gallate inhibits epidermal growth factor receptor signaling pathway. Evidence for direct inhibition of ERK1/2 and AKT kinasesJ Biol Chem200427913127551276214701854
  • ChenPCWheelerDSMalhotraVOdomsKDenenbergAGWongHRA green tea-derived polyphenol, epigallocatechin-3-gallate, inhibits IkappaB kinase activation and IL-8 gene expression in respiratory epitheliumInflammation200226523324112238566
  • BuyseGMGoemansNvan den HauweMIdebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trialNeuromuscul Disord201121639640521435876
  • AruomaOIHalliwellBHoeyBMButlerJThe antioxidant action of N-acetylcysteine: its reaction with hydrogen peroxide, hydroxyl radical, superoxide, and hypochlorous acidFree Radic Biol Med1989665935972546864
  • WhiteheadNPPhamCGervasioOLAllenDGN-Acetylcysteine ameliorates skeletal muscle pathophysiology in mdx miceJ Physiol200858672003201418258657
  • TerrillJRRadley-CrabbHGGroundsMDArthurPGN-Acetylcysteine treatment of dystrophic mdx mice results in protein thiol modifications and inhibition of exercise induced myofibre necrosisNeuromuscul Disord201122542743422206641
  • SmithRAMurphyMPMitochondria-targeted antioxidants as therapiesDiscov Med2011115710611421356165
  • GehrigSMvan der PoelCSayerTAHsp72 preserves muscle function and slows progression of severe muscular dystrophyNature2012484739439439822495301
  • LemastersJJTheruvathTPZhongZNieminenALMitochondrial calcium and the permeability transition in cell deathBiochim Biophys Acta20091787111395140119576166
  • McGuinnessOYafeiNCostiACromptonMThe presence of two classes of high-affinity cyclosporin A binding sites in mitochondria. Evidence that the minor component is involved in the opening of an inner-membrane Ca(2+)-dependent poreEur J Biochem199019426716792176603
  • MatasJYoungNTBourcier-LucasCIncreased expression and intramitochondrial translocation of cyclophilin-D associates with increased vulnerability of the permeability transition pore to stress-induced opening during compensated ventricular hypertrophyJ Mol Cell Cardiol200946342043019094991
  • De LucaANicoBLiantonioAA multidisciplinary evaluation of the effectiveness of cyclosporine a in dystrophic mdx miceAm J Pathol2005166247748915681831
  • WellerBMassaRKarpatiGCarpenterSGlucocorticoids and immunosuppressants do not change the prevalence of necrosis and regeneration in mdx skeletal musclesMuscle Nerve19911487717741891001
  • SharmaKRMynhierMAMillerRGCyclosporine increases muscular force generation in Duchenne muscular dystrophyNeurology1993433 Pt 15275328450995
  • KirschnerJSchesslJScharaUTreatment of Duchenne muscular dystrophy with ciclosporin A: a randomised, double-blind, placebo-controlled multicentre trialLancet Neurol20109111053105920801085
  • KhairallahMKhairallahRJYoungMESildenafil and cardiomyocyte-specific cGMP signaling prevent cardiomyopathic changes associated with dystrophin deficiencyProc Natl Acad Sci U S A2008105197028703318474859
  • StamlerJSMeissnerGPhysiology of nitric oxide in skeletal musclePhysiol Rev200181120923711152758
  • WehlingMSpencerMJTidballJGA nitric oxide synthase transgene ameliorates muscular dystrophy in mdx miceJ Cell Biol2001155112313111581289
  • BrunelliSScioratiCD’AntonaGNitric oxide release combined with nonsteroidal antiinflammatory activity prevents muscular dystrophy pathology and enhances stem cell therapyProc Natl Acad Sci U S A2007104126426917182743
  • WangGBurczynskiFJHasinoffBBZhangKLuQAndersonJEDevelopment of a nitric oxide-releasing analogue of the muscle relaxant guaifenesin for skeletal muscle satellite cell myogenesisMol Pharm20096389590419317416
  • ScioratiCGalvezBGBrunelliSEx vivo treatment with nitric oxide increases mesoangioblast therapeutic efficacy in muscular dystrophyJ Cell Sci2006119Pt 245114512317158915
  • TakimotoEChampionHCLiMChronic inhibition of cyclic GMP phosphodiesterase 5A prevents and reverses cardiac hypertrophyNat Med200511221422215665834
  • AsaiASahaniNKanekiMOuchiYMartynJAYasuharaSEPrimary role of functional ischemia, quantitative evidence for the two-hit mechanism, and phosphodiesterase-5 inhibitor therapy in mouse muscular dystrophyPLoS One200728e80617726536
  • AdamoCMDaiDFPercivalJMSildenafil reverses cardiac dysfunction in the mdx mouse model of Duchenne muscular dystrophyProc Natl Acad Sci U S A201010744190791908320956307
  • MokELetellierGCuissetJMLack of functional benefit with glutamine versus placebo in Duchenne muscular dystrophy: a randomized crossover trialPLoS One200945e544819421321
  • KumamotoTFujimotoSItoTHorinouchiHUeyamaHTsudaTProteasome expression in the skeletal muscles of patients with muscular dystrophyActa Neuropathol2000100659560211078210
  • TidballJGSpencerMJCalpains and muscular dystrophiesInt J Biochem Cell Biol20003211510661889
  • SawadaHNagahiroKKikukawaYTherapeutic effect of camostat mesilate on Duchenne muscular dystrophy in mdx miceBiol Pharm Bull20032671025102712843632
  • BriguetAErbMCourdier-FruhIEffect of calpain and proteasome inhibition on Ca2+-dependent proteolysis and muscle histopathology in the mdx mouse. FASEB JDec2008221241904200
  • ChildersMKBoganJRBoganDJChronic administration of a leupeptin-derived calpain inhibitor fails to ameliorate severe muscle pathology in a canine model of duchenne muscular dystrophyFront Pharmacol201128922291646
  • BonuccelliGSotgiaFSchubertWProteasome inhibitor (MG-132) treatment of mdx mice rescues the expression and membrane localization of dystrophin and dystrophin-associated proteinsAm J Pathol200316341663167514507673
  • BonuccelliGSotgiaFCapozzaFGazzerroEMinettiCLisantiMPLocalized treatment with a novel FDA-approved proteasome inhibitor blocks the degradation of dystrophin and dystrophin-associated proteins in mdx miceCell Cycle20076101242124817495527
  • GazzerroEAsseretoSBonettoATherapeutic potential of proteasome inhibition in Duchenne and Becker muscular dystrophiesAm J Pathol201017641863187720304949
  • AsseretoSStringaraSSotgiaFPharmacological rescue of the dystrophin-glycoprotein complex in Duchenne and Becker skeletal muscle explants by proteasome inhibitor treatmentAm J Physiol Cell Physiol20062902C577C58216192300
  • AcharyyaSVillaltaSABakkarNInterplay of IKK/NF-kappaB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophyJ Clin Invest2007117488990117380205
  • PorrecaEGuglielmiMDUnciniAHaemostatic abnormalities, cardiac involvement and serum tumor necrosis factor levels in X-linked dystrophic patientsThromb Haemost199981454354610235436
  • WhiteheadNPYeungEWAllenDGMuscle damage in mdx (dystrophic) mice: role of calcium and reactive oxygen speciesClin Exp Pharmacol Physiol200633765766216789936
  • De BosscherKSchmitzMLVanden BergheWPlaisanceSFiersWHaegemanGGlucocorticoid-mediated repression of nuclear factor-kappaB-dependent transcription involves direct interference with transactivationProc Natl Acad Sci U S A1997942513504135099391055
  • PanYChenCShenYCurcumin alleviates dystrophic muscle pathology in mdx miceMol Cells200825453153718460899
  • DurhamWJArbogastSGerkenELiYPReidMBProgressive nuclear factor-kappaB activation resistant to inhibition by contraction and curcumin in mdx miceMuscle Nerve200634329830316718687
  • TangYReayDPSalayMNInhibition of the IKK/NF-kappaB pathway by AAV gene transfer improves muscle regeneration in older mdx miceGene Ther201017121476148320720575
  • PetersonJMKlineWCananBDPeptide-based inhibition of NF-kappaB rescues diaphragm muscle contractile dysfunction in a murine model of Duchenne muscular dystrophyMol Med2011175–650851521267511
  • TilstraJRehmanKKHennonTPlevySEClemensPRobbinsPDProtein transduction: identification, characterization and optimizationBiochem Soc Trans200735Pt 481181517635154
  • ReayDPYangMWatchkoJFSystemic delivery of NEMO binding domain/IKKgamma inhibitory peptide to young mdx mice improves dystrophic skeletal muscle histopathologyNeurobiol Dis201143359860821624467
  • ReayDPNiizawaGAWatchkoJFEffect of NF-kappaB inhibition on AAV9 minidystrophin gene transfer to the mdx mouseMol Med201218146647622231732
  • LeaskAAbrahamDJTGF-beta signaling and the fibrotic responseFASEB J200418781682715117886
  • BernasconiPTorchianaEConfalonieriPExpression of transforming growth factor-beta 1 in dystrophic patient muscles correlates with fibrosis. Pathogenetic role of a fibrogenic cytokineJ Clin Invest1995962113711447635950
  • BurksTNCohnRDRole of TGF-beta signaling in inherited and acquired myopathiesSkelet Muscle2011111921798096
  • BizarioJCCerriDGRodriguesLCImatinib mesylate ameliorates the dystrophic phenotype in exercised mdx miceJ Neuroimmunol20092121–29310119508953
  • HuangPZhaoXSFieldsMRansohoffRMZhouLImatinib attenuates skeletal muscle dystrophy in mdx miceFASEB J20092382539254819289603
  • MorrisCASelsbyJTMorrisLDPendrakKSweeneyHLBowman-Birk inhibitor attenuates dystrophic pathology in mdx miceJ Appl Physiol201010951492149920847128
  • RoffeSHagaiYPinesMHalevyOHalofuginone inhibits Smad3 phosphorylation via the PI3K/Akt and MAPK/ERK pathways in muscle cells: effect on myotube fusionExp Cell Res201031661061106920060825
  • TurgemanTHagaiYHuebnerKPrevention of muscle fibrosis and improvement in muscle performance in the mdx mouse by halofuginoneNeuromuscul Disord2008181185786818672370
  • HuebnerKDJassalDSHalevyOPinesMAndersonJEFunctional resolution of fibrosis in mdx mouse dystrophic heart and skeletal muscle by halofuginoneAm J Physiol Heart Circ Physiol20082944H1550H156118263710
  • GosselinLEWilliamsJEDeeringMBrazeauDKourySMartinezDALocalization and early time course of TGF-beta 1 mRNA expression in dystrophic muscleMuscle Nerve200430564565315389721
  • ShiSHoogaarsWMde GorterDJBMP antagonists enhance myogenic differentiation and ameliorate the dystrophic phenotype in a DMD mouse modelNeurobiol Dis201141235336020940052
  • CohnRDvan ErpCHabashiJPAngiotensin II type 1 receptor blockade attenuates TGF-beta-induced failure of muscle regeneration in multiple myopathic statesNat Med200713220421017237794
  • SpurneyCFSaliAGuerronADLosartan decreases cardiac muscle fibrosis and improves cardiac function in dystrophin-deficient mdx miceJ Cardiovasc Pharmacol Ther2011161879521304057
  • NelsonCAHunterRBQuigleyLAInhibiting TGF-beta activity improves respiratory function in mdx miceAm J Pathol201117862611262121641384
  • Rafael-FortneyJAChimanjiNSSchillKEEarly treatment with lisinopril and spironolactone preserves cardiac and skeletal muscle in Duchenne muscular dystrophy miceCirculation2011124558258821768542
  • McPherronACLawlerAMLeeSJRegulation of skeletal muscle mass in mice by a new TGF-beta superfamily memberNature1997387662883909139826
  • LangleyBThomasMBishopASharmaMGilmourSKambadurRMyostatin inhibits myoblast differentiation by down-regulating MyoD expressionJ Biol Chem200227751498314984012244043
  • WahlMACE-031 clinical trials in Duchenne MD stopped for nowTucson, AZQuest2011 [cited August 8, 2012]. Available from: http://quest.mda.org/news/ace-031-clinical-trials-duchenne-md-stopped-nowAccessed August 18, 2012
  • WagnerKRFleckensteinJLAmatoAAA phase I/IItrial of MYO-029 in adult subjects with muscular dystrophyAnn Neurol200863556157118335515
  • QiaoCLiJZhengHHydrodynamic limb vein injection of adeno-associated virus serotype 8 vector carrying canine myostatin propeptide gene into normal dogs enhances muscle growthHum Gene Ther200920111018828709
  • KangJKMalerbaAPopplewellLFosterKDicksonGAntisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatmentMol Ther201119115916420924365
  • KemaladewiDUHoogaarsWMvan HeiningenSHDual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophyBMC Med Genomics201143621507246
  • Barton-DavisERShoturmaDIMusaroARosenthalNSweeneyHLViral mediated expression of insulin-like growth factor I blocks the aging-related loss of skeletal muscle functionProc Natl Acad Sci U S A1998952615603156079861016
  • GregorevicPPlantDRLeedingKSBachLALynchGSImproved contractile function of the mdx dystrophic mouse diaphragm muscle after insulin-like growth factor-I administrationAm J Pathol200216162263227212466140
  • GehrigSMvan der PoelCHoeflichANaimTLynchGSMetzgerFTherapeutic potential of PEGylated insulin-like growth factor I for skeletal muscle disease evaluated in two murine models of muscular dystrophyGrowth Horm IGF Res2012222697522424862
  • AbmayrSGregorevicPAllenJMChamberlainJSPhenotypic improvement of dystrophic muscles by rAAV/microdystrophin vectors is augmented by Igf1 codeliveryMol Ther200512344145016099410