ArticlePloS one2009
Identification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screening.
Article in PloS one, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
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Who cites it
18 citing papers in PubMed, 41 citations in OpenAlex.
- Evolution and Breakthroughs in Exon Skipping and Splice Modulation: From Inception to Clinical Success.Methods in molecular biology (Clifton, N.J.) · 2025Article
- Molecular and Biochemical Therapeutic Strategies for Duchenne Muscular Dystrophy.Neurology international · 2024Review
- Current Strategies of Muscular Dystrophy Therapeutics: An Overview.Methods in molecular biology (Clifton, N.J.) · 2023Review
- STAU2 protein level is controlled by caspases and the CHK1 pathway and regulates cell cycle progression in the non-transformed hTERT-RPE1 cells.BMC molecular and cell biology · 2021Article
- Dual Fluorescence Splicing Reporter Minigene Identifies an Antisense Oligonucleotide to Skip Exon v8 of theInternational journal of molecular sciences · 2020Article
- Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models.Molecular therapy. Nucleic acids · 2019Article
- Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse.Molecular therapy. Nucleic acids · 2018Article
- The M-phase specific hyperphosphorylation of Staufen2 involved the cyclin-dependent kinase CDK1.BMC cell biology · 2017Article
- The downregulation of the RNA-binding protein Staufen2 in response to DNA damage promotes apoptosis.Nucleic acids research · 2016Article
- Post-transcriptional regulation of Rbm5 expression in undifferentiated H9c2 myoblasts.In vitro cellular & developmental biology. Animal · 2016Article
- Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cells.Skeletal muscle · 2015Article
- High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics.Biology · 2014Review
- PubChem applications in drug discovery: a bibliometric analysis.Drug discovery today · 2014Review
- Differential downregulation of Rbm5 and Rbm10 during skeletal and cardiac differentiation.In vitro cellular & developmental biology. Animal · 2014Article
- High-content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC-independent pathway in myotonic dystrophy cell lines.Human molecular genetics · 2014Article
- Recent advances in Duchenne muscular dystrophy.Degenerative neurological and neuromuscular disease · 2012Review
- Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.The American journal of pathology · 2011Review
- Targeting RNA splicing for disease therapy.Wiley interdisciplinary reviews. RNAReview
Corrections and comments
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
One therapeutic approach to Duchenne Muscular Dystrophy (DMD) recently entering clinical trials aims to convert DMD phenotypes to that of a milder disease variant, Becker Muscular Dystrophy (BMD), by employing antisense oligonucleotides (AONs) targeting splice sites, to induce exon skipping and restore partial dystrophin function. In order to search for small molecule and genetic modulators of AON-dependent and independent exon skipping, we screened approximately 10,000 known small molecule drugs, >17,000 cDNA clones, and >2,000 kinase- targeted siRNAs against a 5.6 kb luciferase minigene construct, encompassing exon 71 to exon 73 of human dystrophin. As a result, we identified several enhancers of exon skipping, acting on both the reporter construct as well as endogenous dystrophin in mdx cells. Multiple mechanisms of action were identified, including histone deacetylase inhibition, tubulin modulation and pre-mRNA processing. Among others, the nucleolar protein NOL8 and staufen RNA binding protein homolog 2 (Stau2) were found to induce endogenous exon skipping in mdx cells in an AON-dependent fashion. An unexpected but recurrent theme observed in our screening efforts was the apparent link between the inhibition of cell cycle progression and the induction of exon skipping.
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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.