ArticleMolecular therapy : the journal of the American Society of Gene Therapy2015
Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 1 of them a synthesis that pooled it.
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Who cites it
20 citing papers in PubMed, 1 synthesis or guideline pooled it, 27 citations in OpenAlex.
- Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future.Pharmacological reports : PR · 2020Pooled it
- Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation.Methods in molecular biology (Clifton, N.J.) · 2025Article
- Myostatin inhibition with orally administeredFrontiers in neurology · 2025Article
- Targeting the Skin: The Study of a Bottlebrush Polymer-Antisense Oligonucleotide Conjugate in a Psoriasis Mouse Model.Small (Weinheim an der Bergstrasse, Germany) · 2024Article
- The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy.Neurology and therapy · 2024Article
- Inhalable Bottlebrush Polymer Bioconjugates as Vectors for Efficient Pulmonary Delivery of Oligonucleotides.ACS nano · 2024Article
- The effect of modification of DNA interference on myostatin gene expression in mice.Journal of genetics · 2024Article
- A New Method of Myostatin Inhibition in Mice via Oral Administration ofInternational journal of molecular sciences · 2022Article
- Use of Small Animal Models for Duchenne and Parameters to Assess Efficiency upon Antisense Treatment.Methods in molecular biology (Clifton, N.J.) · 2022Article
- Inhibition of Myostatin Reduces Collagen Deposition in a Mouse Model of Oculopharyngeal Muscular Dystrophy (OPMD) With Established Disease.Frontiers in physiology · 2020Article
- Mechanisms Underlying Metabolic Syndrome-Related Sarcopenia and Possible Therapeutic Measures.International journal of molecular sciences · 2019Review
- Review
- Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches.Nature communications · 2017Article
- Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice.Molecular therapy. Nucleic acids · 2017Article
- Splice-switching antisense oligonucleotides as therapeutic drugs.Nucleic acids research · 2016Review
- Welcome to the splice age: antisense oligonucleotide-mediated exon skipping gains wider applicability.The Journal of clinical investigation · 2016Article
- Nitric Oxide Donor Molsidomine Positively Modulates Myogenic Differentiation of Embryonic Endothelial Progenitors.PloS one · 2016Article
- Anti-inflammatory drugs for Duchenne muscular dystrophy: focus on skeletal muscle-releasing factors.Drug design, development and therapy · 2016Review
- Advances in gene therapy for muscular dystrophies.F1000Research · 2016Review
- Assessment and management of respiratory function in patients with Duchenne muscular dystrophy: current and emerging options.Therapeutics and clinical risk management · 2015Review
Corrections and comments
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Authors and funding
6 authors at 3 institutions in 1 country.
Funding
Abstract
The fatal X-linked Duchenne muscular dystrophy (DMD), characterized by progressive muscle wasting and muscle weakness, is caused by mutations within the DMD gene. The use of antisense oligonucleotides (AOs) modulating pre-mRNA splicing to restore the disrupted dystrophin reading frame, subsequently generating a shortened but functional protein has emerged as a potential strategy in DMD treatment. AO therapy has recently been applied to induce out-of-frame exon skipping of myostatin pre-mRNA, knocking-down expression of myostatin protein, and such an approach is suggested to enhance muscle hypertrophy/hyperplasia and to reduce muscle necrosis. Within this study, we investigated dual exon skipping of dystrophin and myostatin pre-mRNAs using phosphorodiamidate morpholino oligomers conjugated with an arginine-rich peptide (B-PMOs). Intraperitoneal administration of B-PMOs was performed in neonatal mdx males on the day of birth, and at weeks 3 and 6. At week 9, we observed in treated mice (as compared to age-matched, saline-injected controls) normalization of muscle mass, a recovery in dystrophin expression, and a decrease in muscle necrosis, particularly in the diaphragm. Our data provide a proof of concept for antisense therapy combining dystrophin restoration and myostatin inhibition for the treatment of DMD.
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Registered trials
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