Published in Hum Gene Ther on August 01, 2011
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Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice. Mol Ther (2010) 1.48
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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
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Smooth muscle-specific dystrophin expression improves aberrant vasoregulation in mdx mice. Hum Mol Genet (2006) 1.09
Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases. Nucleic Acids Res (2012) 1.09
Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in. Hum Gene Ther (2013) 1.08
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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
Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene. Mol Ther (2009) 0.98
Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog. J Gene Med (2011) 0.95
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Deregulation of the ubiquitin-proteasome system is the predominant molecular pathology in OPMD animal models and patients. Skelet Muscle (2011) 0.94
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Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials. Neuromuscul Disord (2010) 0.86
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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
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
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
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
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
APOE epsilon3 gene transfer attenuates brain damage after experimental stroke. J Cereb Blood Flow Metab (2006) 0.82
Codon optimization of the microdystrophin gene for Duchene muscular dystrophy gene therapy. Methods Mol Biol (2011) 0.81
Fragmentation of SIV-gag vaccine induces broader T cell responses. PLoS One (2012) 0.81
Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD). Curr Gene Ther (2015) 0.80
Failure to generate atheroprotective apolipoprotein AI phenotypes using synthetic RNA/DNA oligonucleotides (chimeraplasts). J Gene Med (2003) 0.80
Apolipoprotein E delivery by peritoneal implantation of encapsulated recombinant cells improves the hyperlipidaemic profile in apoE-deficient mice. Biochim Biophys Acta (2005) 0.79
Highly potent delivery method of gp160 envelope vaccine combining lentivirus-like particles and DNA electrotransfer. J Control Release (2012) 0.79
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Bioinformatic and functional optimization of antisense phosphorodiamidate morpholino oligomers (PMOs) for therapeutic modulation of RNA splicing in muscle. Methods Mol Biol (2011) 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
Novel cationic carotenoid lipids as delivery vectors of antisense oligonucleotides for exon skipping in Duchenne muscular dystrophy. Molecules (2012) 0.78
Optimizing antisense oligonucleotides using phosphorodiamidate morpholino oligomers. Methods Mol Biol (2012) 0.78
Nuclear entrapment and extracellular depletion of PCOLCE is associated with muscle degeneration in oculopharyngeal muscular dystrophy. BMC Neurol (2013) 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
The future of Duchenne muscular dystrophy gene therapy: shrinking the dystrophin gene. Curr Opin Mol Ther (2002) 0.75
Activity of different vaccine-associated promoter elements in human dendritic cells. Immunol Lett (2007) 0.75
Efficient liver-directed gene transfer by in situ generation of retroviral vector from adenoviral templates. In Vivo (2009) 0.75