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Original Research

Cationic polyelectrolyte-mediated delivery of antisense morpholino oligonucleotides for exon-skipping in vitro and in mdx mice

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Pages 5635-5646 | Published online: 03 Sep 2015

References

  • HoffmanEPBrownRHJrKunkelLMDystrophin: the protein product of the Duchenne muscular dystrophy locusCell1987519199283319190
  • KoenigMBeggsAHMoyerMThe molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletionAm J Hum Genet1989454985062491009
  • WuBMoultonHMIversenPLEffective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomerProc Natl Acad Sci U S A2008105148141481918806224
  • YinHMoultonHMSeowYCell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and functionHum Mol Genet2008173909391818784278
  • WuBLuPBenrashidEDose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholinoGene Ther20101713214019759562
  • WuBCloerCShabanMMoultonHLuPJLuQLLong-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholinoAm J Pathol201218139240022683468
  • van DeutekomJCJansonAAGinjaarIBLocal dystrophin restoration with antisense oligonucleotide PRO051N Engl J Med20073572677268618160687
  • 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 Neurol2009891892819713152
  • AmantanaAMoultonHMCateMLPharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugateBioconjug Chem2007181325133117583927
  • WuBLiYMorcosPADoranTJLuPLuQLOcta-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx miceMol Ther20091786487119277018
  • GoemansNMTuliniusMvan den AkkerJTSystemic administration of PRO051 in Duchenne’s muscular dystrophyN Engl J Med20113641513152221428760
  • MendellJRRodino-KlapacLRSahenkZEteplirsen for the treatment of Duchenne muscular dystrophyAnn Neurol20137463764723907995
  • 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 studyLancet201137859560521784508
  • FletcherSHoneymanKFallAMHardingPLJohnsenRDWiltonSDDystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotideJ Gene Med2006820721616285002
  • MalerbaASharpPSGrahamIRChronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx miceMol Ther20111934535421102560
  • SummertonJWellerDMorpholino antisense oligomers design, preparation, and propertiesAntisense Nucleic Acid Drug Dev199771871959212909
  • YanoJSmythGENew antisense strategies: chemical synthesis of RNA oligomersAdv Polym Sci2012249148
  • HuYWuBZillmerAGuanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivoMol Ther20101881281820087314
  • WangMXWuBTuckerJDLuPCloerCLuQLEvaluation of Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked polyethylenimine as antisense morpholino oligomer delivery vehicle in cell culture and dystrophic mdx MiceHum Gene Ther20142541942724405395
  • WangMXWuBLuPCloerCTuckerJDLuQLPolyethylenimine modified pluronics (PCMs) improve morpholino oligomer delivery in cell culture and dystrophic mdx miceMol Ther20132121021623164938
  • LiverpoolTBStapperMThe scaling behaviour of screened polyelectrolytesEurophys Lett199740485490
  • DobryninAVColbyRHRubinsteinMScaling theory of polyelectrolyte solutionsMacromolecules19952818591871
  • JaegerWHahnMLieskeAZimmermannABrandFPolymerization of water soluble cationic vinyl monomersMacromol Symp199611195106
  • DautzenbergHGornitzEJaegerWSynthesis and characterization of poly(diallyldimethylammonium chloride) in a broad range of molecular weightMacromol Chem Phys199819915611571
  • DautzenbergHPolyelectrolyte complex formation: Role of a double hydrophilic polymerMacromol Chem Phys200020117651773
  • CornettaKAndersonWFProtamine sulfate as an effective alternative to polybrene in retroviral-mediated gene-transfer: implications for human gene therapyJ Virol Methods1989231871942786000
  • SorgiFLBhattacharyaSHuangLProtamine sulfate enhances lipid-mediated gene transferGene Ther199749619689349433
  • SazaniPKangSHMaierMANuclear antisense effects of neutral, anionic and cationic oligonucleotide analogsNucleic Acids Res2001293965397411574678
  • BalhornRThe protamine family of sperm nuclear proteinsGenome Biol2007822717903313
  • LauferSDRestleTPeptide-mediated cellular delivery of oligonucleotide-based therapeutics in vitro: quantitative evaluation of overall efficacy employing easy to handle reporter systemsCurr Pharm Des2008143637365519075740
  • MaitaniYHattoriYOligoarginine-PEG-lipid particles for gene deliveryExpert Opin Drug Deliv200961065107719637981
  • BarronLGKathleenBMeyerBFrancisCSzokaFCJrEffects of complement depletion on the pharmacokinetics and gene delivery mediated by cationic lipid-DNA complexesHum Gene Ther199893153239508049
  • SakuraiFNishiokaTSaitoHInteraction between DNA-cationic liposome complexes and erythrocytes is an important factor in systemic gene transfer via the intravenous route in mice: the role of the natural helper lipidGene Ther2001867768611406762
  • HoshinoYNakamotoMMiuraYControl of protein-binding kinetics on synthetic polymer nanoparticles by tuning flexibility and inducing conformation changes of polymer chainsJ Am Chem Soc2012134152091521222946923
  • NakamotoMHoshinoYMiuraYEffect of physical properties of nanogel particles on the kinetic constants of multipoint protein recognition processBiomacromolecules20141554154724359155