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Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

EBioMedicine, 2019-07, Vol.45, p.630-645

2019

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Extracellular vesicles: emerging targets for cancer therapy

Trends in molecular medicine, 2014-07, Vol.20 (7), p.385-393

2014

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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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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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Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

Nature Medicine, 2015-03, Vol.21 (3), p.270-275

2015

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Biomarker Potential of Extracellular miRNAs in Duchenne Muscular Dystrophy

Trends in molecular medicine, 2017-11, Vol.23 (11), p.989-1001

2017

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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.1-12

2019

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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.130-143

2017

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EV-mediated promotion of myogenic differentiation is dependent on dose, collection medium, and isolation method

Molecular therapy. Nucleic acids, 2023-09, Vol.33, p.511-528

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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Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD

Molecular therapy. Nucleic acids, 2022-12, Vol.30, p.379-397

2022

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Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD

Molecular therapy. Nucleic acids, 2018-12, Vol.13, p.1-15

2018

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Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides

Nucleic acid therapeutics, 2021-04, Vol.31 (2), p.172-181

2021

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Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy

BioMed research international, 2013-01, Vol.2013 (2013), p.1-8

2013

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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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Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency

Molecular therapy. Nucleic acids, 2020-09, Vol.22, p.166-166

2020

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Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy

JCI insight, 2021-07, Vol.6 (13)

2021

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Neuroprotective autoimmunity-a double-edged sword?

Nature medicine, 2000-04, Vol.6 (4), p.383-385

2000

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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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Wrangling RNA: Antisense oligonucleotides for neurological disorders

Science translational medicine, 2019-09, Vol.11 (511)

2019

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Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1

Molecular therapy, 2020-12, Vol.28 (12), p.2527-2539

2020

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Tsc1 (hamartin) confers neuroprotection against ischemia by inducing autophagy

Nature medicine, 2013-03, Vol.19 (3), p.351-357

2013

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Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

Extracellular vesicles: emerging targets for cancer therapy

Functional Delivery of Lipid-Conjugated siRNA by Extracellular Vesicles

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

Exosomes mediate sensory hair cell protection in the inner ear

Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

Biomarker Potential of Extracellular miRNAs in Duchenne Muscular Dystrophy

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

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

EV-mediated promotion of myogenic differentiation is dependent on dose, collection medium, and isolation method

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

Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD

Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD

Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides

Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy

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

Interventions Targeting Glucocorticoid-Krüppel-like Factor 15-Branched-Chain Amino Acid Signaling Improve Disease Phenotypes in Spinal Muscular Atrophy Mice

Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency

Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy

Neuroprotective autoimmunity-a double-edged sword?

Therapeutic Potential of Multipotent Mesenchymal Stromal Cells and Their Extracellular Vesicles

Wrangling RNA: Antisense oligonucleotides for neurological disorders

Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1

Tsc1 (hamartin) confers neuroprotection against ischemia by inducing autophagy

Ultrafiltration with size-exclusion liquid chromatography for high yield isolation of extracellular vesicles preserving intact biophysical and functional properties

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