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Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of exon skipping therapy

Journal of human genetics, 2017-04, Vol.62 (4), p.459-463

2017

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LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts

Scientific reports, 2017-06, Vol.7 (1), p.3672-9, Article 3672

2017

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Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides

Nucleic acid therapeutics, 2014-02, Vol.24 (1), p.57

2014

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In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophy

PloS one, 2015-03, Vol.10 (3), p.e0120058-e0120058

2015

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Antiviral Efficacy of RNase H-Dependent Gapmer Antisense Oligonucleotides against Japanese Encephalitis Virus

International journal of molecular sciences, 2023-10, Vol.24 (19), p.14846

2023

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Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy

Proceedings of the National Academy of Sciences - PNAS, 2022-03, Vol.119 (9), p.1

2022

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Deletion of exons 3-9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy

Journal of human genetics, 2016-07, Vol.61 (7), p.663-667

2016

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Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges

Journal of personalized medicine, 2018-12, Vol.8 (4), p.41

2018

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Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts

PloS one, 2018-05, Vol.13 (5), p.e0197084-e0197084

2018

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Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy

Molecular therapy, 2017-11, Vol.25 (11), p.2561-2572

2017

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Efficacy of Multi-exon Skipping Treatment in Duchenne Muscular Dystrophy Dog Model Neonates

Molecular therapy, 2019-01, Vol.27 (1), p.76-86

2019

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Amelioration of intracellular Ca2+ regulation by exon-45 skipping in Duchenne muscular dystrophy-induced pluripotent stem cell-derived cardiomyocytes

Biochemical and biophysical research communications, 2019-11, Vol.520 (1), p.179-185

2019

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A fatal case of a captive snowy owl (Bubo scandiacus) with Haemoproteus infection in Japan

Parasitology research (1987), 2021, Vol.120 (1), p.277-288

2021

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Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of Duchenne Muscular Dystrophy

Methods in molecular biology (Clifton, N.J.), 2017-01, Vol.1565, p.201

2017

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A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping

International journal of molecular sciences, 2021-12, Vol.22 (23), p.13065

2021

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Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

Molecular therapy. Nucleic acids, 2015-02, Vol.4 (2), p.e225-e225, Article e225

2015

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Mutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice

PloS one, 2013-07, Vol.8 (7), p.e69194-e69194

2013

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Current Translational Research and Murine Models For Duchenne Muscular Dystrophy

Journal of neuromuscular diseases, 2016-03, Vol.3 (1), p.29-48

2016

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Molecular characterization and expression pattern of the equine lactate dehydrogenase A and B genes

Gene, 2009-11, Vol.447 (1), p.40-50

2009

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Molecular cloning and expression of bottlenose dolphin CD34

Veterinary immunology and immunopathology, 2011-02, Vol.139 (2), p.303-307

2011

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Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy

Proceedings of the National Academy of Sciences - PNAS, 2020-07, Vol.117 (28), p.16509-16515

2020

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Molecular characterization and expression of the equine M1 and M2-pyruvate kinase gene

Comparative Biochemistry and Physiology Part B: Biochemistry and Molecular Biology, , Vol.151 (1), p.125-132

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Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy

Proceedings of the National Academy of Sciences - PNAS, 2017-04, Vol.114 (16), p.4213-4218

2017

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DUX4 Transcript Knockdown with Antisense 2′-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy

Molecular therapy, 2021-02, Vol.29 (2), p.848-858

2021

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Dystrophin-deficient large animal models: translational research and exon skipping

American journal of translational research, 2015-01, Vol.7 (8), p.1314-1331

2015

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Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of exon skipping therapy

LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts

Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides

In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophy

Antiviral Efficacy of RNase H-Dependent Gapmer Antisense Oligonucleotides against Japanese Encephalitis Virus

Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy

Deletion of exons 3-9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy

Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges

Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts

Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy

Efficacy of Multi-exon Skipping Treatment in Duchenne Muscular Dystrophy Dog Model Neonates

Amelioration of intracellular Ca2+ regulation by exon-45 skipping in Duchenne muscular dystrophy-induced pluripotent stem cell-derived cardiomyocytes

A fatal case of a captive snowy owl (Bubo scandiacus) with Haemoproteus infection in Japan

Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of Duchenne Muscular Dystrophy

A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping

Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

Mutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice

Current Translational Research and Murine Models For Duchenne Muscular Dystrophy

Molecular characterization and expression pattern of the equine lactate dehydrogenase A and B genes

Molecular cloning and expression of bottlenose dolphin CD34

Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy

Molecular characterization and expression of the equine M1 and M2-pyruvate kinase gene

Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy

DUX4 Transcript Knockdown with Antisense 2′-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy

Dystrophin-deficient large animal models: translational research and exon skipping

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