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An overview of the clinical application of antisense oligonucleotides for RNA-targeting therapies

Current opinion in pharmacology, 2015-10, Vol.24, p.52-58

2015

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Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment

Nucleic acid therapeutics, 2019-02, Vol.29 (1), p.1

2019

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An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides

Nature communications, 2022-07, Vol.13 (1), p.4036-4036, Article 4036

2022

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Modulating the expression of disease genes with RNA-based therapy

PLoS genetics, 2007-06, Vol.3 (6), p.e109-e109

2007

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Cell-Penetrating Peptides to Enhance Delivery of Oligonucleotide-Based Therapeutics

Biomedicines, 2018-05, Vol.6 (2), p.51

2018

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Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies

Human molecular genetics, 2015-09, Vol.24 (17), p.4916-4932

2015

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Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration

Human molecular genetics, 2015-12, Vol.24 (23), p.6756-6768

2015

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Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles

Human molecular genetics, 2016-08, Vol.25 (16), p.3555-3563

2016

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Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy

PloS one, 2018-06, Vol.13 (6), p.e0198897-e0198897

2018

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Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics

Scientific reports, 2015-11, Vol.5 (1), p.17014-17014, Article 17014

2015

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Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice

Scientific reports, 2015-03, Vol.5 (1), p.8986-8986, Article 8986

2015

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Uniform sarcolemmal dystrophin expression is required to prevent extracellular microRNA release and improve dystrophic pathology

Journal of cachexia, sarcopenia and muscle, 2020-04, Vol.11 (2), p.578-593

2020

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Expression Analysis in Multiple Muscle Groups and Serum Reveals Complexity in the MicroRNA Transcriptome of the mdx Mouse with Implications for Therapy

Molecular therapy. Nucleic acids, 2012, Vol.1 (8), p.e39-e39, Article e39

2012

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Fine Tuning of Phosphorothioate Inclusion in 2′-O-Methyl Oligonucleotides Contributes to Specific Cell Targeting for Splice-Switching Modulation

Frontiers in physiology, 2021-10, Vol.12, p.689179-689179

2021

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Splice-Switching Therapy for Spinal Muscular Atrophy

Genes, 2017-06, Vol.8 (6), p.161

2017

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Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy

Scientific reports, 2015-06, Vol.5 (1), p.11632, Article 11632

2015

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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

Molecular therapy. Nucleic acids, 2012-12, Vol.1 (12), p.e62-e62, Article e62

2012

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Small RNA-Mediated Epigenetic Myostatin Silencing

Molecular therapy. Nucleic acids, 2012, Vol.1 (5), p.e23-e23, Article e23

2012

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Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides

Molecular therapy. Nucleic acids, 2014, Vol.3 (11), p.e212-e212, Article e212

2014

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How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse

Human molecular genetics, 2015-08, Vol.24 (15), p.4225-4237

2015

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Engineered extracellular vesicle decoy receptor-mediated modulation of the IL6 trans-signalling pathway in muscle

Biomaterials, 2021-01, Vol.266, p.120435-120435, Article 120435

2021

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Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides

Nano letters, 2015-07, Vol.15 (7), p.4364-4373

2015

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Extracellular microRNAs are dynamic non-vesicular biomarkers of muscle turnover

Nucleic acids research, 2013-11, Vol.41 (20), p.9500

2013

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Control of backbone chemistry and chirality boost oligonucleotide splice switching activity

Nucleic acids research, 2022-06, Vol.50 (10), p.5443

2022

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Antisense Oligonucleotide-induced Exon Skipping Across the Human Dystrophin Gene Transcript

Molecular therapy, 2007-07, Vol.15 (7), p.1288-1296

2007

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An overview of the clinical application of antisense oligonucleotides for RNA-targeting therapies

Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment

An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides

Modulating the expression of disease genes with RNA-based therapy

Cell-Penetrating Peptides to Enhance Delivery of Oligonucleotide-Based Therapeutics

Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies

Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration

Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles

Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy

Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics

Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice

Uniform sarcolemmal dystrophin expression is required to prevent extracellular microRNA release and improve dystrophic pathology

Expression Analysis in Multiple Muscle Groups and Serum Reveals Complexity in the MicroRNA Transcriptome of the mdx Mouse with Implications for Therapy

Fine Tuning of Phosphorothioate Inclusion in 2′-O-Methyl Oligonucleotides Contributes to Specific Cell Targeting for Splice-Switching Modulation

Splice-Switching Therapy for Spinal Muscular Atrophy

Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy

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

Small RNA-Mediated Epigenetic Myostatin Silencing

Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse

Engineered extracellular vesicle decoy receptor-mediated modulation of the IL6 trans-signalling pathway in muscle

Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides

Extracellular microRNAs are dynamic non-vesicular biomarkers of muscle turnover

Control of backbone chemistry and chirality boost oligonucleotide splice switching activity

Antisense Oligonucleotide-induced Exon Skipping Across the Human Dystrophin Gene Transcript

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