ArticleMolecular therapy : the journal of the American Society of Gene Therapy2010
Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 39 citations in OpenAlex.
- Development of Engineered-U1 snRNA Therapies: Current Status.International journal of molecular sciences · 2023Review
- Exon Skipping Through Chimeric AntisenseNucleic acid therapeutics · 2022Article
- Trans-generational epigenetic regulation associated with the amelioration of Duchenne Muscular Dystrophy.EMBO molecular medicine · 2020Article
- Review
- Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations.Molecular therapy. Nucleic acids · 2016Article
- The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials.Journal of neurology, neurosurgery, and psychiatry · 2016Article
- The lack of the Celf2a splicing factor converts a Duchenne genotype into a Becker phenotype.Nature communications · 2016Article
- Novel long noncoding RNAs (lncRNAs) in myogenesis: a miR-31 overlapping lncRNA transcript controls myoblast differentiation.Molecular and cellular biology · 2015Article
- Development of therapeutic splice-switching oligonucleotides.Human gene therapy · 2014Review
- Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- mRNA transcript diversity creates new opportunities for pharmacological intervention.Molecular pharmacology · 2012Review
- Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- A long noncoding RNA controls muscle differentiation by functioning as a competing endogenous RNA.Cell · 2011Article
- Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.The American journal of pathology · 2011Review
- Activation of AKT signaling promotes cell growth and survival in α7β1 integrin-mediated alleviation of muscular dystrophy.Biochimica et biophysica acta · 2011Article
- miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy.EMBO reports · 2011Article
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
Abstract
One promising approach for the gene therapy of Duchenne muscular dystrophy (DMD) is exon skipping. When thinking of possible intervention on human, it is very crucial to identify the most appropriate antisense sequences able to provide the highest possible skipping efficiency. In this article, we compared the exon 51 skipping activity of 10 different antisense molecules, raised against splice junctions and/or exonic splicing enhancers (ESEs), expressed as part of the U1 small nuclear RNA (snRNA). The effectiveness of each construct was tested in human DMD myoblasts carrying the deletion of exons 48-50, which can be treated with skipping of exon 51. Our results show that the highest skipping activity and dystrophin rescue is achieved upon expression of a U1 snRNA-derived antisense molecule targeting exon 51 splice sites in combination with an internal exon sequence. The efficacy of this molecule was further proven on an exon 45-50 deletion background, utilizing patient's fibroblasts transdifferentiated into myoblasts. In this system, we showed that the selected antisense was able to produce 50% skipping of exon 51.
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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.