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Exosomes for targeted siRNA delivery across biological barriers

Advanced drug delivery reviews, 2013-03, Vol.65 (3), p.391-397

2013

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The acute inflammatory response to intranigral α-synuclein differs significantly from intranigral lipopolysaccharide and is exacerbated by peripheral inflammation

Journal of neuroinflammation, 2011-11, Vol.8 (1), p.166-166, Article 166

2011

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Alpha-synuclein induces the unfolded protein response in Parkinson's disease SNCA triplication iPSC-derived neurons

Human molecular genetics, 2017-11, Vol.26 (22), p.4441-4450

2017

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Exosomes mediate sensory hair cell protection in the inner ear

The Journal of clinical investigation, 2020-05, Vol.130 (5), p.2657-2672

2020

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Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle

Nucleic acids research, 2008-11, Vol.36 (20), p.6418-6428

2008

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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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PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice

EMBO molecular medicine, 2023-11, Vol.15 (11), p.e17683-e17683

2023

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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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Exosomes and the blood-brain barrier: implications for neurological diseases

Therapeutic delivery, 2011-09, Vol.2 (9), p.1095-1099

2011

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Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

Molecular therapy, 2010-10, Vol.18 (10), p.1822-1829

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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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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The role of long non-coding RNAs in neurodevelopment, brain function and neurological disease

Philosophical transactions of the Royal Society of London. Series B. Biological sciences, 2014-09, Vol.369 (1652), p.20130507

2014

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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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Cells release subpopulations of exosomes with distinct molecular and biological properties

Scientific reports, 2016-03, Vol.6 (1), p.22519-22519, Article 22519

2016

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Lysosomal dysfunction increases exosome-mediated alpha-synuclein release and transmission

Neurobiology of disease, 2011-06, Vol.42 (3), p.360-367

2011

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Profiling of Extracellular Small RNAs Highlights a Strong Bias towards Non-Vesicular Secretion

Cells (Basel, Switzerland), 2021-06, Vol.10 (6), p.1543

2021

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Functional Delivery of Lipid-Conjugated siRNA by Extracellular Vesicles

Molecular therapy, 2017-07, Vol.25 (7), p.1580-1587

2017

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Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

The Journal of clinical investigation, 2019-11, Vol.129 (11), p.4739-4744

2019

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Microvesicles and exosomes: Opportunities for cell-derived membrane vesicles in drug delivery

Journal of controlled release, 2012-07, Vol.161 (2), p.635-644

2012

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Therapeutic Potential of Multipotent Mesenchymal Stromal Cells and Their Extracellular Vesicles

Human gene therapy, 2015-08, Vol.26 (8), p.506-517

2015

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Targeting RNA to treat neuromuscular disease

Nature reviews. Drug discovery, 2011-08, Vol.10 (8), p.621-637

2011

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Genetic therapies for RNA mis-splicing diseases

Trends in genetics, 2011-05, Vol.27 (5), p.196-205

2011

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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Exosomes for targeted siRNA delivery across biological barriers

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

The acute inflammatory response to intranigral α-synuclein differs significantly from intranigral lipopolysaccharide and is exacerbated by peripheral inflammation

Alpha-synuclein induces the unfolded protein response in Parkinson's disease SNCA triplication iPSC-derived neurons

Exosomes mediate sensory hair cell protection in the inner ear

Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle

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

PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice

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

Exosomes and the blood-brain barrier: implications for neurological diseases

Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

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

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

The role of long non-coding RNAs in neurodevelopment, brain function and neurological disease

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

Cells release subpopulations of exosomes with distinct molecular and biological properties

Lysosomal dysfunction increases exosome-mediated alpha-synuclein release and transmission

Profiling of Extracellular Small RNAs Highlights a Strong Bias towards Non-Vesicular Secretion

Functional Delivery of Lipid-Conjugated siRNA by Extracellular Vesicles

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

Microvesicles and exosomes: Opportunities for cell-derived membrane vesicles in drug delivery

Therapeutic Potential of Multipotent Mesenchymal Stromal Cells and Their Extracellular Vesicles

Targeting RNA to treat neuromuscular disease

Genetic therapies for RNA mis-splicing diseases

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