ArticleMolecular therapy : the journal of the American Society of Gene Therapy2009
Rational design of antisense oligomers to induce dystrophin exon skipping.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.
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Who cites it
29 citing papers in PubMed, 43 citations in OpenAlex.
- Antisense oligonucleotides and their applications in rare neurological diseases.Frontiers in neuroscience · 2024Review
- Networking to OptimizeBiomedicines · 2023Article
- Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing ofInternational journal of molecular sciences · 2022Article
- Review
- Induction of cryptic pre-mRNA splice-switching by antisense oligonucleotides.Scientific reports · 2021Article
- TargetedBiomedicines · 2021Article
- Delivery of oligonucleotide-based therapeutics: challenges and opportunities.EMBO molecular medicine · 2021Review
- A Splice Intervention Therapy for Autosomal Recessive Juvenile Parkinson's Disease Arising from Parkin Mutations.International journal of molecular sciences · 2020Article
- Trans-generational epigenetic regulation associated with the amelioration of Duchenne Muscular Dystrophy.EMBO molecular medicine · 2020Article
- Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing.International journal of molecular sciences · 2019Article
- Systematic Approach to Developing Splice Modulating Antisense Oligonucleotides.International journal of molecular sciences · 2019Article
- Consequences of Making the Inactive Active Through Changes in Antisense Oligonucleotide Chemistries.Frontiers in genetics · 2019Article
- The genetic basis for individual differences in mRNA splicing and APOBEC1 editing activity in murine macrophages.Genome research · 2014Article
- The era of genomic medicine.Clinical medicine (London, England) · 2013Review
- Antisense suppression of donor splice site mutations in the dystrophin gene transcript.Molecular genetics & genomic medicine · 2013Article
- Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches.Nature reviews. Genetics · 2013Review
- Article
- Article
- A prospective study in the rational design of efficient antisense oligonucleotides for exon skipping in the DMD gene.Human gene therapy · 2012Article
- Multiple exon skipping strategies to by-pass dystrophin mutations.Neuromuscular disorders : NMD · 2012Article
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Authors and funding
6 authors at 3 institutions in 3 countries.
Funding
Abstract
Duchenne muscular dystrophy (DMD), one of the most severe neuromuscular disorders of childhood, is caused by the absence of a functional dystrophin. Antisense oligomer (AO) induced exon skipping is being investigated to restore functional dystrophin expression in models of muscular dystrophy and DMD patients. One of the major challenges will be in the development of clinically relevant oligomers and exon skipping strategies to address many different mutations. Various models, including cell-free extracts, cells transfected with artificial constructs, or mice with a human transgene, have been proposed as tools to facilitate oligomer design. Despite strong sequence homology between the human and mouse dystrophin genes, directing an oligomer to the same motifs in both species does not always induce comparable exon skipping. We report substantially different levels of exon skipping induced in normal and dystrophic human myogenic cell lines and propose that animal models or artificial assay systems useful in initial studies may be of limited relevance in designing the most efficient compounds to induce targeted skipping of human dystrophin exons for therapeutic outcomes.
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