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Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

Proceedings of the National Academy of Sciences - PNAS, 2016-09, Vol.113 (39), p.10962-10967

2016

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A chemical view of oligonucleotides for exon skipping and related drug applications

Nucleic acid therapeutics, 2014-02, Vol.24 (1), p.37

2014

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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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Efficient inhibition of miR-155 function in vivo by peptide nucleic acids

Nucleic acids research, 2010-07, Vol.38 (13), p.4466

2010

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Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells

Nucleic acids research, 2014-03, Vol.42 (5), p.3207

2014

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Pip5 Transduction Peptides Direct High Efficiency Oligonucleotide-mediated Dystrophin Exon Skipping in Heart and Phenotypic Correction in mdx Mice

Molecular therapy, 2011-07, Vol.19 (7), p.1295-1303

2011

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Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

Molecular therapy. Nucleic acids, 2012-08, Vol.1 (8), p.e38-e38, Article e38

2012

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Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy

Nucleic acid therapeutics, 2017-06, Vol.27 (3), p.130

2017

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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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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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Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide

Nucleic acids research, 2007-07, Vol.35 (13), p.4495

2007

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Assessing the Delivery of Molecules to the Mitochondrial Matrix Using Click Chemistry

Chembiochem : a European journal of chemical biology, 2016-07, Vol.17 (14), p.1312-1316

2016

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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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Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA

Molecular therapy. Nucleic acids, 2012, Vol.1 (6), p.e27-e27, Article e27

2012

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Modulation of Pro-Inflammatory IL-6 Trans-Signaling Axis by Splice Switching Oligonucleotides as a Therapeutic Modality in Inflammation

Cells (Basel, Switzerland), 2023-09, Vol.12 (18), p.2285

2023

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Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy

JCI insight, 2022-12, Vol.7 (24)

2022

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Endosome trapping limits the efficiency of splicing correction by PNA-oligolysine conjugates

Journal of controlled release, 2006-02, Vol.110 (3), p.595-604

2006

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Introduction and History of the Chemistry of Nucleic Acids Therapeutics

Methods in molecular biology (Clifton, N.J.), 2022, Vol.2434, p.3

2022

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Synthesis and Splice-Redirecting Activity of Branched, Arginine-Rich Peptide Dendrimer Conjugates of Peptide Nucleic Acid Oligonucleotides

Bioconjugate chemistry, 2010-10, Vol.21 (10), p.1902-1911

2010

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Optimization of Peptide Nucleic Acid Antisense Oligonucleotides for Local and Systemic Dystrophin Splice Correction in the mdx Mouse

Molecular therapy, 2010-04, Vol.18 (4), p.819-827

2010

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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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Delivery of therapeutic oligonucleotides with cell penetrating peptides

Advanced drug delivery reviews, 2015-06, Vol.87, p.52-67

2015

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Exploiting cell surface thiols to enhance cellular uptake

Trends in biotechnology (Regular ed.), 2012-04, Vol.30 (4), p.185-190

2012

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Parallel synthesis and splicing redirection activity of cell-penetrating peptide conjugate libraries of a PNA cargo

Organic & biomolecular chemistry, 2013-11, Vol.11 (43), p.7621-7630

2013

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MALDI-TOF mass spectral analysis of siRNA degradation in serum confirms an RNAse A-like activity

Molecular bioSystems, 2007-01, Vol.3 (1), p.43-50

2007

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Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

A chemical view of oligonucleotides for exon skipping and related drug applications

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

Efficient inhibition of miR-155 function in vivo by peptide nucleic acids

Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells

Pip5 Transduction Peptides Direct High Efficiency Oligonucleotide-mediated Dystrophin Exon Skipping in Heart and Phenotypic Correction in mdx Mice

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy

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

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

Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide

Assessing the Delivery of Molecules to the Mitochondrial Matrix Using Click Chemistry

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

Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA

Modulation of Pro-Inflammatory IL-6 Trans-Signaling Axis by Splice Switching Oligonucleotides as a Therapeutic Modality in Inflammation

Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy

Endosome trapping limits the efficiency of splicing correction by PNA-oligolysine conjugates

Introduction and History of the Chemistry of Nucleic Acids Therapeutics

Synthesis and Splice-Redirecting Activity of Branched, Arginine-Rich Peptide Dendrimer Conjugates of Peptide Nucleic Acid Oligonucleotides

Optimization of Peptide Nucleic Acid Antisense Oligonucleotides for Local and Systemic Dystrophin Splice Correction in the mdx Mouse

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

Delivery of therapeutic oligonucleotides with cell penetrating peptides

Exploiting cell surface thiols to enhance cellular uptake

Parallel synthesis and splicing redirection activity of cell-penetrating peptide conjugate libraries of a PNA cargo

MALDI-TOF mass spectral analysis of siRNA degradation in serum confirms an RNAse A-like activity

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