ArticleMolecular therapy. Nucleic acids2012
Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy.
Article in Molecular therapy. Nucleic acids, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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Who cites it
24 citing papers in PubMed, 40 citations in OpenAlex.
- Targeting Drug Delivery System to Skeletal Muscles: A Comprehensive Review of Different Approaches.Journal of cachexia, sarcopenia and muscle · 2025Review
- Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation.Methods in molecular biology (Clifton, N.J.) · 2025Article
- Therapeutic approaches for Duchenne muscular dystrophy.Nature reviews. Drug discovery · 2023Review
- Systemic Delivery of a Monoclonal Antibody to Immunologically Block Myostatin in the A17 Mouse Model of OPMD.Methods in molecular biology (Clifton, N.J.) · 2023Article
- Use of Small Animal Models for Duchenne and Parameters to Assess Efficiency upon Antisense Treatment.Methods in molecular biology (Clifton, N.J.) · 2022Article
- Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model.Human molecular genetics · 2021Article
- Antimyostatin Treatment in Health and Disease: The Story of Great Expectations and Limited Success.Cells · 2021Review
- Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases.Journal of neuromuscular diseases · 2021Review
- Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy.Journal of cachexia, sarcopenia and muscle · 2020Article
- Dystrophin R16/17 protein therapy restores sarcolemmal nNOS in trans and improves muscle perfusion and function.Molecular medicine (Cambridge, Mass.) · 2019Article
- Review
- Cell-Penetrating Peptides to Enhance Delivery of Oligonucleotide-Based Therapeutics.Biomedicines · 2018Review
- Article
- Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy.Nucleic acid therapeutics · 2017Article
- Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice.Molecular therapy. Nucleic acids · 2017Article
- Investigating Synthetic Oligonucleotide Targeting of Mir31 in Duchenne Muscular Dystrophy.PLoS currents · 2016Article
- Targeted mutations in myostatin by zinc-finger nucleases result in double-muscled phenotype in Meishan pigs.Scientific reports · 2015Article
- Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Article
- Peptide nanoparticle delivery of charge-neutral splice-switching morpholino oligonucleotides.Nucleic acid therapeutics · 2015Article
- Targeted excision of VCP R155H mutation by Cre-LoxP technology as a promising therapeutic strategy for valosin-containing protein disease.Human gene therapy methods · 2015Article
Corrections and comments
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Authors and funding
8 authors at 4 institutions in 1 country.
Funding
Abstract
The knockdown of myostatin, a negative regulator of skeletal muscle mass may have important implications in disease conditions accompanied by muscle mass loss like cancer, HIV/AIDS, sarcopenia, muscle atrophy, and Duchenne muscular dystrophy (DMD). In DMD patients, where major muscle loss has occurred due to a lack of dystrophin, the therapeutic restoration of dystrophin expression alone in older patients may not be sufficient to restore the functionality of the muscles. We recently demonstrated that phosphorodiamidate morpholino oligomers (PMOs) can be used to re-direct myostatin splicing and promote the expression of an out-of-frame transcript so reducing the amount of the synthesized myostatin protein. Furthermore, the systemic administration of the same PMO conjugated to an octaguanidine moiety (Vivo-PMO) led to a significant increase in the mass of soleus muscle of treated mice. Here, we have further optimized the use of Vivo-PMO in normal mice and also tested the efficacy of the same PMO conjugated to an arginine-rich cell-penetrating peptide (B-PMO). Similar experiments conducted in mdx dystrophic mice showed that B-PMO targeting myostatin is able to significantly increase the tibialis anterior (TA) muscle weight and when coadministered with a B-PMO targeting the dystrophin exon 23, it does not have a detrimental interaction. This study confirms that myostatin knockdown by exon skipping is a potential therapeutic strategy to counteract muscle wasting conditions and dual myostatin and dystrophin skipping has potential as a therapy for DMD.Molecular Therapy - Nucleic Acids (2012) 1, e62; doi:10.1038/mtna.2012.54; published online 18 December 2012.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.