Published in Nucleic Acids Res on October 08, 2008
Targeting RNA to treat neuromuscular disease. Nat Rev Drug Discov (2011) 1.89
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
Cellular uptake and intracellular trafficking of antisense and siRNA oligonucleotides. Bioconjug Chem (2011) 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
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Therapeutic approaches to muscular dystrophy. Hum Mol Genet (2011) 1.30
Emerging strategies for cell and gene therapy of the muscular dystrophies. Expert Rev Mol Med (2009) 1.29
Cell-penetrating peptides as versatile vehicles for oligonucleotide delivery. Mol Ther (2012) 1.13
Nanoparticles deliver triplex-forming PNAs for site-specific genomic recombination in CD34+ human hematopoietic progenitors. Mol Ther (2010) 1.13
A chemical view of oligonucleotides for exon skipping and related drug applications. Nucleic Acid Ther (2013) 1.00
Chemical structure requirements and cellular targeting of microRNA-122 by peptide nucleic acids anti-miRs. Nucleic Acids Res (2011) 1.00
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
The small molecule Retro-1 enhances the pharmacological actions of antisense and splice switching oligonucleotides. Nucleic Acids Res (2013) 0.97
Loop and backbone modifications of peptide nucleic acid improve g-quadruplex binding selectivity. J Am Chem Soc (2009) 0.96
The chemistry and biology of oligonucleotide conjugates. Acc Chem Res (2012) 0.95
Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA. Mol Ther Nucleic Acids (2012) 0.95
Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice. Sci Rep (2015) 0.94
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Antisense therapy in neurology. J Pers Med (2013) 0.87
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Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells. Nucleic Acids Res (2013) 0.86
Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells. Front Physiol (2014) 0.86
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Parallel synthesis of cell-penetrating peptide conjugates of PMO toward exon skipping enhancement in Duchenne muscular dystrophy. Nucleic Acid Ther (2014) 0.83
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Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy. Nucleic Acids Res (2014) 0.81
Parallel synthesis and splicing redirection activity of cell-penetrating peptide conjugate libraries of a PNA cargo. Org Biomol Chem (2013) 0.80
Delivery of antisense peptide nucleic acids to cells by conjugation with small arginine-rich cell-penetrating peptide (R/W)9. PLoS One (2014) 0.80
Effective dystrophin restoration by a novel muscle-homing peptide-morpholino conjugate in dystrophin-deficient mdx mice. Mol Ther (2014) 0.79
Building Cell Selectivity into CPP-Mediated Strategies. Pharmaceuticals (Basel) (2010) 0.78
Antisense mediated exon skipping therapy for duchenne muscular dystrophy (DMD). Artif DNA PNA XNA (2011) 0.78
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Prednisolone treatment does not interfere with 2'-O-methyl phosphorothioate antisense-mediated exon skipping in Duchenne muscular dystrophy. Hum Gene Ther (2012) 0.77
Dendritic Guanidines as Efficient Analogues of Cell Penetrating Peptides. Pharmaceuticals (Basel) (2010) 0.77
Modulation of mdm2 pre-mRNA splicing by 9-aminoacridine-PNA (peptide nucleic acid) conjugates targeting intron-exon junctions. BMC Cancer (2010) 0.76
Probing of miniPEGγ-PNA-DNA Hybrid Duplex Stability with AFM Force Spectroscopy. Biochemistry (2016) 0.76
ClickIn: a flexible protocol for quantifying mitochondrial uptake of nucleobase derivatives. Interface Focus (2017) 0.75
Therapeutic Potential of Tricyclo-DNA antisense oligonucleotides. J Neuromuscul Dis (2016) 0.75
Electroporation Enhanced Effect of Dystrophin Splice Switching PNA Oligomers in Normal and Dystrophic Muscle. Mol Ther Nucleic Acids (2015) 0.75
An amphipathic trans-acting phosphorothioate DNA element delivers uncharged PNA and PMO nucleic acid sequences in mammalian cells. Curr Protoc Nucleic Acid Chem (2016) 0.75
Insights on chiral, backbone modified peptide nucleic acids: Properties and biological activity. Artif DNA PNA XNA (2016) 0.75
Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents. Appl Clin Genet (2011) 0.75
An amphipathic trans-acting phosphorothioate RNA element delivers an uncharged phosphorodiamidate morpholino sequence in mdx mouse myotubes. RSC Adv (2017) 0.75
Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med (2007) 7.29
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Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med (2006) 3.74
Antisense technologies. Improvement through novel chemical modifications. Eur J Biochem (2003) 3.67
Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci U S A (2004) 3.34
Cellular uptake of arginine-rich peptides: roles for macropinocytosis and actin rearrangement. Mol Ther (2004) 2.82
Cationic TAT peptide transduction domain enters cells by macropinocytosis. J Control Release (2005) 2.74
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
Cellular uptake of unconjugated TAT peptide involves clathrin-dependent endocytosis and heparan sulfate receptors. J Biol Chem (2005) 2.46
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
Cell penetrating peptides: intracellular pathways and pharmaceutical perspectives. Pharm Res (2007) 2.08
Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides. Hum Mol Genet (1998) 2.07
Calcium ions effectively enhance the effect of antisense peptide nucleic acids conjugated to cationic tat and oligoarginine peptides. Chem Biol (2005) 1.85
Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications. RNA (2007) 1.78
Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy. Neuromuscul Disord (2002) 1.72
Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents. J Control Release (2006) 1.70
Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development. Biochemistry (1998) 1.66
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
Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse. Mol Ther (2007) 1.57
Cell-penetrating-peptide-based delivery of oligonucleotides: an overview. Biochem Soc Trans (2007) 1.49
Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice. Mol Ther (2007) 1.48
Cell penetrating peptide conjugates of steric block oligonucleotides. Adv Drug Deliv Rev (2007) 1.47
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
Endosome trapping limits the efficiency of splicing correction by PNA-oligolysine conjugates. J Control Release (2005) 1.38
Cell-dependent differential cellular uptake of PNA, peptides, and PNA-peptide conjugates. Antisense Nucleic Acid Drug Dev (2002) 1.32
The influence of antisense oligonucleotide length on dystrophin exon skipping. Mol Ther (2007) 1.30
Evaluation of cell-penetrating peptides (CPPs) as vehicles for intracellular delivery of antisense peptide nucleic acid (PNA). Bioconjug Chem (2006) 1.29
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
Peptide-mediated cellular delivery of antisense oligonucleotides and their analogues. Cell Mol Life Sci (2003) 1.15
Induction of splice correction by cell-penetrating peptide nucleic acids. J Gene Med (2006) 1.14
Peptide-based delivery of nucleic acids: design, mechanism of uptake and applications to splice-correcting oligonucleotides. Biochem Soc Trans (2007) 1.09
Intracellular uptake and inhibition of gene expression by PNAs and PNA-peptide conjugates. Biochemistry (2004) 1.05
Peptide conjugates of oligonucleotides: synthesis and applications. Chem Rev (2006) 1.03
Structural requirements for cellular uptake and antisense activity of peptide nucleic acids conjugated with various peptides. Biochemistry (2006) 1.02
Peptide-oligonucleotide conjugates for the delivery of antisense and siRNA. Curr Opin Mol Ther (2005) 0.97
Modification of alternative splicing by antisense therapeutics. Oligonucleotides (2004) 0.94
Disulfide conjugation of peptides to oligonucleotides and their analogs. Curr Protoc Nucleic Acid Chem (2006) 0.93
Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes. Nat Biotechnol (2011) 9.68
Cell-penetrating peptides. A reevaluation of the mechanism of cellular uptake. J Biol Chem (2002) 6.01
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
Extracellular vesicles: biology and emerging therapeutic opportunities. Nat Rev Drug Discov (2013) 4.35
Tat peptide-mediated cellular delivery: back to basics. Adv Drug Deliv Rev (2005) 2.70
Cellular uptake of unconjugated TAT peptide involves clathrin-dependent endocytosis and heparan sulfate receptors. J Biol Chem (2005) 2.46
Lysosomal dysfunction increases exosome-mediated alpha-synuclein release and transmission. Neurobiol Dis (2011) 2.32
Exosomes and microvesicles: extracellular vesicles for genetic information transfer and gene therapy. Hum Mol Genet (2012) 2.13
miR-122 targeting with LNA/2'-O-methyl oligonucleotide mixmers, peptide nucleic acids (PNA), and PNA-peptide conjugates. RNA (2007) 1.90
Tsc1 (hamartin) confers neuroprotection against ischemia by inducing autophagy. Nat Med (2013) 1.89
Targeting RNA to treat neuromuscular disease. Nat Rev Drug Discov (2011) 1.89
Exosome-mediated delivery of siRNA in vitro and in vivo. Nat Protoc (2012) 1.70
Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents. J Control Release (2006) 1.70
Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. Hum Mol Genet (2008) 1.69
Local RNA target structure influences siRNA efficacy: a systematic global analysis. J Mol Biol (2005) 1.54
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
Different mechanisms for cellular internalization of the HIV-1 Tat-derived cell penetrating peptide and recombinant proteins fused to Tat. Eur J Biochem (2002) 1.52
Cell penetrating peptide conjugates of steric block oligonucleotides. Adv Drug Deliv Rev (2007) 1.47
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
Systemic delivery of BH4 anti-apoptotic peptide using CPPs prevents cardiac ischemia-reperfusion injuries in vivo. J Control Release (2011) 1.43
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
RNA-targeted splice-correction therapy for neuromuscular disease. Brain (2010) 1.36
Design of a peptide-based vector, PepFect6, for efficient delivery of siRNA in cell culture and systemically in vivo. Nucleic Acids Res (2011) 1.35
A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice. Hum Mol Genet (2009) 1.34
Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice. Mol Ther (2011) 1.32
Genetic therapies for RNA mis-splicing diseases. Trends Genet (2011) 1.31
Cell penetrating peptides: overview and applications to the delivery of oligonucleotides. Cell Mol Life Sci (2009) 1.31
Ribonuclease L proteolysis in peripheral blood mononuclear cells of chronic fatigue syndrome patients. J Biol Chem (2002) 1.31
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
RNA targeting with peptide conjugates of oligonucleotides, siRNA and PNA. Blood Cells Mol Dis (2006) 1.22
Electroporation-induced siRNA precipitation obscures the efficiency of siRNA loading into extracellular vesicles. J Control Release (2013) 1.21
Microvesicles and exosomes: opportunities for cell-derived membrane vesicles in drug delivery. J Control Release (2011) 1.21
RNase L levels in peripheral blood mononuclear cells: 37-kilodalton/83-kilodalton isoform ratio is a potential test for chronic fatigue syndrome. Clin Diagn Lab Immunol (2003) 1.21
Localization of double-stranded small interfering RNA to cytoplasmic processing bodies is Ago2 dependent and results in up-regulation of GW182 and Argonaute-2. Mol Biol Cell (2008) 1.20
The biogenesis and characterization of mammalian microRNAs of mirtron origin. Nucleic Acids Res (2011) 1.20
Novel RNA-based strategies for therapeutic gene silencing. Mol Ther (2010) 1.20
Diaphragm rescue alone prevents heart dysfunction in dystrophic mice. Hum Mol Genet (2010) 1.19
Exosomes for targeted siRNA delivery across biological barriers. Adv Drug Deliv Rev (2012) 1.17
DNA cage delivery to mammalian cells. ACS Nano (2011) 1.13
MALDI-TOF mass spectral analysis of siRNA degradation in serum confirms an RNAse A-like activity. Mol Biosyst (2006) 1.10
Exosomes and the emerging field of exosome-based gene therapy. Curr Gene Ther (2012) 1.10
Insights into the cellular trafficking of splice redirecting oligonucleotides complexed with chemically modified cell-penetrating peptides. J Control Release (2011) 1.09
Extracellular microRNAs are dynamic non-vesicular biomarkers of muscle turnover. Nucleic Acids Res (2013) 1.05
Structural requirements for cellular uptake and antisense activity of peptide nucleic acids conjugated with various peptides. Biochemistry (2006) 1.02
MicroRNA fate upon targeting with anti-miRNA oligonucleotides as revealed by an improved Northern-blot-based method for miRNA detection. RNA (2011) 1.02
RNA splicing: disease and therapy. Brief Funct Genomics (2011) 1.02
Delivery of nucleic acids with a stearylated (RxR)4 peptide using a non-covalent co-incubation strategy. J Control Release (2009) 1.01
A chemical view of oligonucleotides for exon skipping and related drug applications. Nucleic Acid Ther (2013) 1.00
Delivery of steric block morpholino oligomers by (R-X-R)4 peptides: structure-activity studies. Nucleic Acids Res (2008) 1.00
Alpha-synuclein release by neurons activates the inflammatory response in a microglial cell line. Neurosci Res (2011) 1.00
Chemical structure requirements and cellular targeting of microRNA-122 by peptide nucleic acids anti-miRs. Nucleic Acids Res (2011) 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
Design of RNAi hairpins for mutation-specific silencing of ataxin-7 and correction of a SCA7 phenotype. PLoS One (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
Short non-coding RNA biology and neurodegenerative disorders: novel disease targets and therapeutics. Hum Mol Genet (2009) 0.97
The acute inflammatory response to intranigral α-synuclein differs significantly from intranigral lipopolysaccharide and is exacerbated by peripheral inflammation. J Neuroinflammation (2011) 0.97
The CRTC1-SIK1 pathway regulates entrainment of the circadian clock. Cell (2013) 0.97
Cellular uptake and intracellular fate of antisense oligonucleotides. Curr Opin Mol Ther (2003) 0.96
Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice. Hum Mol Genet (2013) 0.95
Splicing therapy for neuromuscular disease. Mol Cell Neurosci (2013) 0.95
Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA. Mol Ther Nucleic Acids (2012) 0.95
Variability of the RNase L isoform ratio (37 kiloDaltons/83 kiloDaltons) in diagnosis of chronic fatigue syndrome. Clin Diagn Lab Immunol (2005) 0.93
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
Mirtrons, an emerging class of atypical miRNA. Wiley Interdiscip Rev RNA (2012) 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
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
Silencing of Parkinson's disease-associated genes with artificial mirtron mimics of miR-1224. Nucleic Acids Res (2012) 0.90
2'-O-methyl-RNA hairpins generate loop-loop complexes and selectively inhibit HIV-1 Tat-mediated transcription. Biochemistry (2002) 0.90
Pyrenemethyl ara-uridine-2'-carbamate: a strong interstrand excimer in the major groove of a DNA duplex. Chembiochem (2003) 0.90