Published in Mol Ther Nucleic Acids on December 18, 2012
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Targeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor. Mol Ther Nucleic Acids (2014) 0.79
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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
miR-122 targeting with LNA/2'-O-methyl oligonucleotide mixmers, peptide nucleic acids (PNA), and PNA-peptide conjugates. RNA (2007) 1.90
Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript. Mol Ther (2007) 1.67
Expression analysis in multiple muscle groups and serum reveals complexity in the microRNA transcriptome of the mdx mouse with implications for therapy. Mol Ther Nucleic Acids (2012) 1.53
Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle. Nucleic Acids Res (2008) 1.52
Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice. Mol Ther (2010) 1.48
Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy. J Gene Med (2002) 1.48
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Cell-penetrating peptide conjugates of peptide nucleic acids (PNA) as inhibitors of HIV-1 Tat-dependent trans-activation in cells. Nucleic Acids Res (2005) 1.44
Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment. Mol Ther Nucleic Acids (2012) 1.41
Efficient inhibition of miR-155 function in vivo by peptide nucleic acids. Nucleic Acids Res (2010) 1.41
Modulating the expression of disease genes with RNA-based therapy. PLoS Genet (2007) 1.38
Endosome trapping limits the efficiency of splicing correction by PNA-oligolysine conjugates. J Control Release (2005) 1.38
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Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene. Biochem Biophys Res Commun (2002) 1.32
Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice. Mol Ther (2011) 1.32
Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide. Nucleic Acids Res (2007) 1.28
Synthesis, cellular uptake and HIV-1 Tat-dependent trans-activation inhibition activity of oligonucleotide analogues disulphide-conjugated to cell-penetrating peptides. Nucleic Acids Res (2005) 1.27
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
β-catenin as a potential key target for tumor suppression. Int J Cancer (2011) 1.23
RNA targeting with peptide conjugates of oligonucleotides, siRNA and PNA. Blood Cells Mol Dis (2006) 1.22
Diaphragm rescue alone prevents heart dysfunction in dystrophic mice. Hum Mol Genet (2010) 1.19
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
MALDI-TOF mass spectral analysis of siRNA degradation in serum confirms an RNAse A-like activity. Mol Biosyst (2006) 1.10
Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO. Mol Ther (2010) 1.10
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
Extracellular microRNAs are dynamic non-vesicular biomarkers of muscle turnover. Nucleic Acids Res (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
MicroRNA fate upon targeting with anti-miRNA oligonucleotides as revealed by an improved Northern-blot-based method for miRNA detection. RNA (2011) 1.02
New developments in the use of gene therapy to treat Duchenne muscular dystrophy. Expert Opin Biol Ther (2013) 1.01
Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Hum Gene Ther (2004) 1.01
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A chemical view of oligonucleotides for exon skipping and related drug applications. Nucleic Acid Ther (2013) 1.00
Synthesis of peptide-oligonucleotide conjugates with single and multiple peptides attached to 2'-aldehydes through thiazolidine, oxime, and hydrazine linkages. Bioconjug Chem (2002) 1.00
Chemical structure requirements and cellular targeting of microRNA-122 by peptide nucleic acids anti-miRs. Nucleic Acids Res (2011) 1.00
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
Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene. Mol Ther (2009) 0.98
Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse. Mol Ther (2010) 0.98
PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation. Nucleic Acids Res (2011) 0.97
Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog. J Gene Med (2011) 0.95
Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA. Mol Ther Nucleic Acids (2012) 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
Deregulation of the ubiquitin-proteasome system is the predominant molecular pathology in OPMD animal models and patients. Skelet Muscle (2011) 0.94
Disulfide conjugation of peptides to oligonucleotides and their analogs. Curr Protoc Nucleic Acid Chem (2006) 0.93
In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy. J Gene Med (2010) 0.93
A structure-activity study of the inhibition of HIV-1 Tat-dependent trans-activation by mixmer 2'-O-methyl oligoribonucleotides containing locked nucleic acid (LNA), alpha-L-LNA, or 2'-thio-LNA residues. Oligonucleotides (2003) 0.92
A peptide-based dendrimer that enhances the splice-redirecting activity of PNA conjugates in cells. Bioconjug Chem (2009) 0.92
Cell-penetrating peptides do not cross mitochondrial membranes even when conjugated to a lipophilic cation: evidence against direct passage through phospholipid bilayers. Biochem J (2004) 0.91
Genetic therapeutic approaches for Duchenne muscular dystrophy. Hum Gene Ther (2012) 0.91
Loop-loop interaction of HIV-1 TAR RNA with N3'-->P5' deoxyphosphoramidate aptamers inhibits in vitro Tat-mediated transcription. Proc Natl Acad Sci U S A (2002) 0.91
Pyrenemethyl ara-uridine-2'-carbamate: a strong interstrand excimer in the major groove of a DNA duplex. Chembiochem (2003) 0.90
2'-O-methyl-RNA hairpins generate loop-loop complexes and selectively inhibit HIV-1 Tat-mediated transcription. Biochemistry (2002) 0.90
Small RNA-Mediated Epigenetic Myostatin Silencing. Mol Ther Nucleic Acids (2012) 0.89
Lipoplex and peptide-based strategies for the delivery of steric-block oligonucleotides. Int J Pharm (2007) 0.89
Synthesis and biological activity of phosphonoacetate- and thiophosphonoacetate-modified 2'-O-methyl oligoribonucleotides. Org Biomol Chem (2011) 0.89
Targeting the HIV-1 RNA leader sequence with synthetic oligonucleotides and siRNA: chemistry and cell delivery. Biochim Biophys Acta (2005) 0.88
Exploiting cell surface thiols to enhance cellular uptake. Trends Biotechnol (2012) 0.88
Overview of alternative oligonucleotide chemistries for exon skipping. Methods Mol Biol (2012) 0.88
Potent and sustained cellular inhibition of miR-122 by lysine-derivatized peptide nucleic acids (PNA) and phosphorothioate locked nucleic acid (LNA)/2'-O-methyl (OMe) mixmer anti-miRs in the absence of transfection agents. Artif DNA PNA XNA (2012) 0.87
Synthesis and splice-redirecting activity of branched, arginine-rich peptide dendrimer conjugates of peptide nucleic acid oligonucleotides. Bioconjug Chem (2010) 0.87
Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells. Nucleic Acids Res (2013) 0.86
Use of carbonyl group addition--elimination reactions for synthesis of nucleic acid conjugates. Bioconjug Chem (2005) 0.86
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
Antiviral activity of steric-block oligonucleotides targeting the HIV-1 trans-activation response and packaging signal stem-loop RNAs. Nucleosides Nucleotides Nucleic Acids (2005) 0.85
Increased RNAi is related to intracellular release of siRNA via a covalently attached signal peptide. RNA (2009) 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
A bi-functional siRNA construct induces RNA interference and also primes PCR amplification for its own quantification. Nucleic Acids Res (2005) 0.84