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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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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 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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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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Selection of Fluorescent, Bioluminescent, and Radioactive Tracers to Accurately Reflect Extracellular Vesicle Biodistribution in Vivo

ACS nano, 2021-02, Vol.15 (2), p.3212-3227

2021

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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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Re-evaluation of the Mutagenic Response to Phosphorothioate Nucleotides in Human Lymphoblastoid TK6 Cells

Toxicological sciences, 2015-05, Vol.145 (1), p.169-176

2015

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Extracellular vesicles induce minimal hepatotoxicity and immunogenicity

Nanoscale, 2019-04, Vol.11 (14), p.6990-7001

2019

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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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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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In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy

The journal of gene medicine, 2010-04, Vol.12 (4), p.354-364

2010

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Improved Tat-mediated plasmid DNA transfer by fusion to LK15 peptide

Journal of controlled release, 2010-04, Vol.143 (2), p.233-242

2010

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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 research, 2015-01, Vol.43 (1), p.29-39

2015

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Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice

Molecular therapy, 2015-08, Vol.23 (8), p.1341-1348

2015

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2′-O-(2-Methoxyethyl) Nucleosides Are Not Phosphorylated or Incorporated Into the Genome of Human Lymphoblastoid TK6 Cells

Toxicological sciences, 2018-05, Vol.163 (1), p.70-78

2018

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The Lack of Mutagenic Potential of a Guanine-Rich Triplex Forming Oligonucleotide in Physiological Conditions

Toxicological sciences, 2017-01, Vol.155 (1), p.101-111

2017

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Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model

The Journal of clinical investigation, 2014-09, Vol.124 (9), p.4067-4081

2014

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YOYO as a Dye to Track Penetration of LK15 DNA Complexes in Spheroids: Use and Limits

Journal of fluorescence, 2008-01, Vol.18 (1), p.155-161

2008

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Diaphragm rescue alone prevents heart dysfunction in dystrophic mice

Human molecular genetics, 2011-02, Vol.20 (3), p.413-421

2011

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Dystrophin involvement in peripheral circadian SRF signalling

Life science alliance, 2021-10, Vol.4 (10), p.e202101014

2021

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Peptide nanoparticle delivery of charge-neutral splice-switching morpholino oligonucleotides

Nucleic acid therapeutics, 2015-04, Vol.25 (2), p.65-77

2015

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Enhancement of gene transfer using YIGSR analog of Tat-derived peptide

Biopolymers, 2008-01, Vol.89 (1), p.62-71

2008

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

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 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

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

Selection of Fluorescent, Bioluminescent, and Radioactive Tracers to Accurately Reflect Extracellular Vesicle Biodistribution in Vivo

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

Re-evaluation of the Mutagenic Response to Phosphorothioate Nucleotides in Human Lymphoblastoid TK6 Cells

Extracellular vesicles induce minimal hepatotoxicity and immunogenicity

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

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

In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy

Improved Tat-mediated plasmid DNA transfer by fusion to LK15 peptide

Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy

Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice

2′-O-(2-Methoxyethyl) Nucleosides Are Not Phosphorylated or Incorporated Into the Genome of Human Lymphoblastoid TK6 Cells

The Lack of Mutagenic Potential of a Guanine-Rich Triplex Forming Oligonucleotide in Physiological Conditions

Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model

YOYO as a Dye to Track Penetration of LK15 DNA Complexes in Spheroids: Use and Limits

Diaphragm rescue alone prevents heart dysfunction in dystrophic mice

Dystrophin involvement in peripheral circadian SRF signalling

Peptide nanoparticle delivery of charge-neutral splice-switching morpholino oligonucleotides

Enhancement of gene transfer using YIGSR analog of Tat-derived peptide

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