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Eteplirsen in the treatment of Duchenne muscular dystrophy

Drug design, development and therapy, 2017-01, Vol.11, p.533-545

2017

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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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Exonic mutations and exon skipping: Lessons learned from DFNA5

Human mutation, 2018-03, Vol.39 (3), p.433-440

2018

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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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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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Alternative Splicing Increases Sirtuin Gene Family Diversity and Modulates Their Subcellular Localization and Function

International journal of molecular sciences, 2021-01, Vol.22 (2), p.473

2021

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Advances in the Treatment of Duchenne Muscular Dystrophy: New and Emerging Pharmacotherapies

Pharmacotherapy, 2017-04, Vol.37 (4), p.492-499

2017

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Antisense Oligonucleotide Modified with Disulfide Units Induces Efficient Exon Skipping in mdx Myotubes through Enhanced Membrane Permeability and Nucleus Internalization

Chembiochem : a European journal of chemical biology, 2021-12, Vol.22 (24), p.3437-3442

2021

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Evaluation of CRISPR/Cas9 exon‐skipping vector for choroideremia using human induced pluripotent stem cell‐derived RPE

The journal of gene medicine, 2023-02, Vol.25 (2), p.e3464-n/a

2023

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Zebrafish as a Model to Evaluate a CRISPR/Cas9-Based Exon Excision Approach as a Future Treatment Option for EYS-Associated Retinitis Pigmentosa

International journal of molecular sciences, 2021-09, Vol.22 (17), p.9154

2021

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Trimerization profile of type IV collagen COL4A5 exon deletion in X-linked Alport syndrome

Clinical and experimental nephrology, 2024-09, Vol.28 (9), p.874-881

2024

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Cardio‐respiratory and phenotypic rescue of dystrophin/utrophin‐deficient mice by combination therapy

EMBO reports, 2022-06, Vol.23 (6), p.e53955-n/a

2022

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MOTS‐c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice

EMBO molecular medicine, 2021-02, Vol.13 (2), p.e12993-n/a

2021

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Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

Nucleic acid therapeutics, 2017-10, Vol.27 (5), p.251-259

2017

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Synthesis and Exon-Skipping Properties of a 3'-Ursodeoxycholic Acid-Conjugated Oligonucleotide Targeting DMD Pre-mRNA: Pre-Synthetic versus Post-Synthetic Approach

Molecules (Basel, Switzerland), 2021-12, Vol.26 (24), p.7662

2021

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Trans‐generational epigenetic regulation associated with the amelioration of Duchenne Muscular Dystrophy

EMBO molecular medicine, 2020-08, Vol.12 (8), p.e12063-n/a

2020

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Oxytocin accelerates tight junction formation and impairs cellular migration in 3D spheroids: evidence from Gapmer-induced exon skipping

Frontiers in cellular neuroscience, 2022-10, Vol.16, p.1000538-1000538

2022

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Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy

Journal of personalized medicine, 2018-11, Vol.8 (4), p.38

2018

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Gene modification strategies using AO‐mediated exon skipping and CRISPR/Cas9 as potential therapies for Duchenne muscular dystrophy patients

Engineering biology, 2020-12, Vol.4 (3), p.37-42

2020

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Natural Disease Course in Usher Syndrome Patients Harboring USH2A Variant p.Cys870 in Exon 13, Amenable to Exon Skipping Therapy

Genes, 2023-03, Vol.14 (3), p.652

2023

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Description of Two Siblings with Apparently Severe CEP290 Mutations and Unusually Mild Retinal Disease Unrelated to Basal Exon Skipping or Nonsense-Associated Altered Splicing

Advances in experimental medicine and biology, 2019, Vol.1185, p.189-195

2019

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Pathological Issues in Dystrophinopathy in the Age of Genetic Therapies

Annual review of pathology, 2019-01, Vol.14 (1), p.105-126

2019

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Analysis and Prediction of Exon Skipping Events from RNA-Seq with Sequence Information Using Rotation Forest

International journal of molecular sciences, 2017-12, Vol.18 (12), p.2691

2017

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Understanding Neuromuscular Health and Disease: Advances in Genetics, Omics, and Molecular Function

2021

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Eteplirsen in the treatment of Duchenne muscular dystrophy

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

Exonic mutations and exon skipping: Lessons learned from DFNA5

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

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

Alternative Splicing Increases Sirtuin Gene Family Diversity and Modulates Their Subcellular Localization and Function

Advances in the Treatment of Duchenne Muscular Dystrophy: New and Emerging Pharmacotherapies

Antisense Oligonucleotide Modified with Disulfide Units Induces Efficient Exon Skipping in mdx Myotubes through Enhanced Membrane Permeability and Nucleus Internalization

Evaluation of CRISPR/Cas9 exon‐skipping vector for choroideremia using human induced pluripotent stem cell‐derived RPE

Zebrafish as a Model to Evaluate a CRISPR/Cas9-Based Exon Excision Approach as a Future Treatment Option for EYS-Associated Retinitis Pigmentosa

Trimerization profile of type IV collagen COL4A5 exon deletion in X-linked Alport syndrome

Cardio‐respiratory and phenotypic rescue of dystrophin/utrophin‐deficient mice by combination therapy

MOTS‐c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

Synthesis and Exon-Skipping Properties of a 3'-Ursodeoxycholic Acid-Conjugated Oligonucleotide Targeting DMD Pre-mRNA: Pre-Synthetic versus Post-Synthetic Approach

Trans‐generational epigenetic regulation associated with the amelioration of Duchenne Muscular Dystrophy

Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy

Oxytocin accelerates tight junction formation and impairs cellular migration in 3D spheroids: evidence from Gapmer-induced exon skipping

Applications of CRISPR/Cas9 for the Treatment of Duchenne Muscular Dystrophy

Gene modification strategies using AO‐mediated exon skipping and CRISPR/Cas9 as potential therapies for Duchenne muscular dystrophy patients

Natural Disease Course in Usher Syndrome Patients Harboring USH2A Variant p.Cys870 in Exon 13, Amenable to Exon Skipping Therapy

Description of Two Siblings with Apparently Severe CEP290 Mutations and Unusually Mild Retinal Disease Unrelated to Basal Exon Skipping or Nonsense-Associated Altered Splicing

Pathological Issues in Dystrophinopathy in the Age of Genetic Therapies

Analysis and Prediction of Exon Skipping Events from RNA-Seq with Sequence Information Using Rotation Forest

Understanding Neuromuscular Health and Disease: Advances in Genetics, Omics, and Molecular Function

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