Published in Mol Ther on January 13, 2009
Targeting RNA to treat neuromuscular disease. Nat Rev Drug Discov (2011) 1.89
Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice. Mol Ther (2010) 1.48
Emerging strategies for cell and gene therapy of the muscular dystrophies. Expert Rev Mol Med (2009) 1.29
Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment. Mol Ther (2010) 1.03
Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse. Mol Ther (2010) 0.98
Splicing therapy for neuromuscular disease. Mol Cell Neurosci (2013) 0.95
Design principles for bifunctional targeted oligonucleotide enhancers of splicing. Nucleic Acids Res (2011) 0.91
A prospective study in the rational design of efficient antisense oligonucleotides for exon skipping in the DMD gene. Hum Gene Ther (2012) 0.86
In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophy. PLoS One (2015) 0.83
Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model. J Clin Invest (2014) 0.80
Inclusion of the central exon of parvovirus B19 precursor mRNA is determined by multiple splicing enhancers in both the exon and the downstream intron. J Virol (2010) 0.79
Discovery of Influenza A Virus Sequence Pairs and Their Combinations for Simultaneous Heterosubtypic Targeting that Hedge against Antiviral Resistance. PLoS Comput Biol (2016) 0.75
Peptide nanoparticle delivery of charge-neutral splice-switching morpholino oligonucleotides. Nucleic Acid Ther (2015) 0.75
Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes. PLoS One (2017) 0.75
Expanded sequence dependence of thermodynamic parameters improves prediction of RNA secondary structure. J Mol Biol (1999) 41.48
Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell (1987) 20.27
Predictive identification of exonic splicing enhancers in human genes. Science (2002) 16.86
ESEfinder: A web resource to identify exonic splicing enhancers. Nucleic Acids Res (2003) 12.81
Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med (2007) 7.29
Computational definition of sequence motifs governing constitutive exon splicing. Genes Dev (2004) 4.59
An increased specificity score matrix for the prediction of SF2/ASF-specific exonic splicing enhancers. Hum Mol Genet (2006) 4.56
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat Med (2003) 4.03
Topography of the Duchenne muscular dystrophy (DMD) gene: FIGE and cDNA analysis of 194 cases reveals 115 deletions and 13 duplications. Am J Hum Genet (1989) 3.81
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med (2006) 3.74
RESCUE-ESE identifies candidate exonic splicing enhancers in vertebrate exons. Nucleic Acids Res (2004) 3.70
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci U S A (2001) 2.71
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. Mol Ther (2008) 2.69
Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide. J Gene Med (2006) 2.18
Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum Mol Genet (2003) 2.17
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Ther (2006) 2.00
Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle. Hum Mol Genet (2003) 1.88
Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells. Hum Mol Genet (2001) 1.76
A theoretical approach to select effective antisense oligodeoxyribonucleotides at high statistical probability. Nucleic Acids Res (1999) 1.73
Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy. Neuromuscul Disord (2002) 1.72
Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript. Mol Ther (2007) 1.67
Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle. Hum Gene Ther (2007) 1.63
Effects of RNA secondary structure on cellular antisense activity. Nucleic Acids Res (2000) 1.60
Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides. Mol Ther (2004) 1.46
Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites. Oligonucleotides (2005) 1.42
Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons. Mol Ther (2006) 1.38
The influence of antisense oligonucleotide length on dystrophin exon skipping. Mol Ther (2007) 1.30
Computational searches for splicing signals. Methods (2005) 1.15
Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries. BMC Mol Biol (2007) 1.11
Dynamics of co-transcriptional pre-mRNA folding influences the induction of dystrophin exon skipping by antisense oligonucleotides. PLoS One (2008) 1.09
Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics. Curr Pharm Biotechnol (2004) 1.04
Induced dystrophin exon skipping in human muscle explants. Neuromuscul Disord (2006) 0.98
Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides. Front Biosci (2008) 0.82
Towards a therapeutic inhibition of dystrophin exon 23 splicing in mdx mouse muscle induced by antisense oligoribonucleotides (splicomers): target sequence optimisation using oligonucleotide arrays. J Gene Med (2004) 0.82
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol (2009) 5.35
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. Mol Ther (2011) 2.06
Adenovirus vector vaccination induces expansion of memory CD4 T cells with a mucosal homing phenotype that are readily susceptible to HIV-1. Proc Natl Acad Sci U S A (2009) 1.95
Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice. Mol Ther (2010) 1.48
Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene. Biochem Biophys Res Commun (2002) 1.32
Codon and mRNA sequence optimization of microdystrophin transgenes improves expression and physiological outcome in dystrophic mdx mice following AAV2/8 gene transfer. Mol Ther (2008) 1.24
Poly(A) binding protein nuclear 1 levels affect alternative polyadenylation. Nucleic Acids Res (2012) 1.23
Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice. Hum Gene Ther (2009) 1.19
Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice. Hum Mol Genet (2002) 1.13
Molecular and phenotypic characterization of a mouse model of oculopharyngeal muscular dystrophy reveals severe muscular atrophy restricted to fast glycolytic fibres. Hum Mol Genet (2010) 1.10
Molecular, cellular and physiological investigation of myostatin propeptide-mediated muscle growth in adult mice. Neuromuscul Disord (2009) 1.09
Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases. Nucleic Acids Res (2012) 1.09
Smooth muscle-specific dystrophin expression improves aberrant vasoregulation in mdx mice. Hum Mol Genet (2006) 1.09
Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in. Hum Gene Ther (2013) 1.08
Langerin negative dendritic cells promote potent CD8+ T-cell priming by skin delivery of live adenovirus vaccine microneedle arrays. Proc Natl Acad Sci U S A (2013) 1.05
Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice. Hum Gene Ther (2013) 1.04
Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment. Mol Ther (2010) 1.03
New developments in the use of gene therapy to treat Duchenne muscular dystrophy. Expert Opin Biol Ther (2013) 1.01
Gene therapy for muscular dystrophy: current progress and future prospects. Expert Opin Biol Ther (2009) 1.01
Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Hum Gene Ther (2004) 1.01
Combination of myostatin pathway interference and dystrophin rescue enhances tetanic and specific force in dystrophic mdx mice. Mol Ther (2010) 1.01
RNAi-mediated knockdown of dystrophin expression in adult mice does not lead to overt muscular dystrophy pathology. Hum Mol Genet (2008) 0.99
Long-term systemic administration of unconjugated morpholino oligomers for therapeutic expression of dystrophin by exon skipping in skeletal muscle: implications for cardiac muscle integrity. Nucleic Acid Ther (2011) 0.98
Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog. J Gene Med (2011) 0.95
Adeno-associated virus-8-mediated intravenous transfer of myostatin propeptide leads to systemic functional improvements of slow but not fast muscle. Rejuvenation Res (2009) 0.95
Atrophy, fibrosis, and increased PAX7-positive cells in pharyngeal muscles of oculopharyngeal muscular dystrophy patients. J Neuropathol Exp Neurol (2013) 0.95
Deregulation of the ubiquitin-proteasome system is the predominant molecular pathology in OPMD animal models and patients. Skelet Muscle (2011) 0.94
Genetic therapeutic approaches for Duchenne muscular dystrophy. Hum Gene Ther (2012) 0.91
Current advances in cell therapy strategies for muscular dystrophies. Expert Opin Biol Ther (2011) 0.91
Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids (2012) 0.89
Progressive skeletal muscle weakness in transgenic mice expressing CTG expansions is associated with the activation of the ubiquitin-proteasome pathway. Neuromuscul Disord (2010) 0.87
Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials. Neuromuscul Disord (2010) 0.86
Transcriptomic analysis of dystrophin RNAi knockdown reveals a central role for dystrophin in muscle differentiation and contractile apparatus organization. BMC Genomics (2010) 0.85
Transcription factor rational design improves directed differentiation of human mesenchymal stem cells into skeletal myocytes. Mol Ther (2011) 0.85
Production of non viral DNA vectors. Curr Gene Ther (2010) 0.84
Long-term episomal transgene expression from mitotically stable integration-deficient lentiviral vectors. Hum Gene Ther (2014) 0.84
The Rag2⁻Il2rb⁻Dmd⁻ mouse: a novel dystrophic and immunodeficient model to assess innovating therapeutic strategies for muscular dystrophies. Mol Ther (2013) 0.84
Invited review: Stem cells and muscle diseases: advances in cell therapy strategies. Neuropathol Appl Neurobiol (2015) 0.83
Stable micro-dystrophin gene transfer using an integrating adeno-retroviral hybrid vector ameliorates the dystrophic pathology in mdx mouse muscle. Hum Mol Genet (2002) 0.83
Towards a therapeutic inhibition of dystrophin exon 23 splicing in mdx mouse muscle induced by antisense oligoribonucleotides (splicomers): target sequence optimisation using oligonucleotide arrays. J Gene Med (2004) 0.82
Langerhans cells are more efficiently transduced than dermal dendritic cells by adenovirus vectors expressing either group C or group B fibre protein: implications for mucosal vaccines. Eur J Immunol (2005) 0.82
Human apolipoprotein E expression from mouse skeletal muscle by electrotransfer of nonviral DNA (plasmid) and pseudotyped recombinant adeno-associated virus (AAV2/7). Hum Gene Ther (2008) 0.82
APOE epsilon3 gene transfer attenuates brain damage after experimental stroke. J Cereb Blood Flow Metab (2006) 0.82
Fragmentation of SIV-gag vaccine induces broader T cell responses. PLoS One (2012) 0.81
Codon optimization of the microdystrophin gene for Duchene muscular dystrophy gene therapy. Methods Mol Biol (2011) 0.81
Failure to generate atheroprotective apolipoprotein AI phenotypes using synthetic RNA/DNA oligonucleotides (chimeraplasts). J Gene Med (2003) 0.80
Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD). Curr Gene Ther (2015) 0.80
Highly potent delivery method of gp160 envelope vaccine combining lentivirus-like particles and DNA electrotransfer. J Control Release (2012) 0.79
Apolipoprotein E delivery by peritoneal implantation of encapsulated recombinant cells improves the hyperlipidaemic profile in apoE-deficient mice. Biochim Biophys Acta (2005) 0.79
Novel cationic carotenoid lipids as delivery vectors of antisense oligonucleotides for exon skipping in Duchenne muscular dystrophy. Molecules (2012) 0.78
Bioinformatic and functional optimization of antisense phosphorodiamidate morpholino oligomers (PMOs) for therapeutic modulation of RNA splicing in muscle. Methods Mol Biol (2011) 0.78
Gene repair and mutagenesis mediated by chimeric RNA-DNA oligonucleotides: chimeraplasty for gene therapy and conversion of single nucleotide polymorphisms (SNPs). Biochim Biophys Acta (2002) 0.78
Preliminary evaluation of a self-complementary AAV2/8 vector for hepatic gene transfer of human apoE3 to inhibit atherosclerotic lesion development in apoE-deficient mice. Atherosclerosis (2008) 0.78
Nuclear entrapment and extracellular depletion of PCOLCE is associated with muscle degeneration in oculopharyngeal muscular dystrophy. BMC Neurol (2013) 0.78
Optimizing antisense oligonucleotides using phosphorodiamidate morpholino oligomers. Methods Mol Biol (2012) 0.78
Adeno-associated virus serotypes 7 and 8 outperform serotype 9 in expressing atheroprotective human apoE3 from mouse skeletal muscle. Metabolism (2010) 0.75
Adenovirus-based targeting in myoblasts is hampered by nonhomologous vector integration. Hum Gene Ther (2008) 0.75
Positive in vivo heterologous gene regulation by electric pulses delivery with metallothionein I gene promoter. J Gene Med (2005) 0.75
The future of Duchenne muscular dystrophy gene therapy: shrinking the dystrophin gene. Curr Opin Mol Ther (2002) 0.75
Getting to the heart of things in the field of cardiovascular gene therapy. Hum Gene Ther (2011) 0.75
Efficient liver-directed gene transfer by in situ generation of retroviral vector from adenoviral templates. In Vivo (2009) 0.75
Activity of different vaccine-associated promoter elements in human dendritic cells. Immunol Lett (2007) 0.75