ArticleMethods in molecular biology (Clifton, N.J.)2023
Restoring Dystrophin Expression with Exon 44 and 53 Skipping in the DMD Gene in Immortalized Myotubes.
Article in Methods in molecular biology (Clifton, N.J.), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
2 citing papers in PubMed, 2 citations in OpenAlex.
- Incidental finding of aFrontiers in pediatrics · 2025Article
- Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide.Molecular therapy. Nucleic acids · 2023Article
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Authors and funding
2 authors at 2 institutions in 2 countries.
Funding
Abstract
Phosphorodiamidate morpholino oligomer (PMO)-mediated exon skipping is a therapeutic approach that applies to many Duchenne muscular dystrophy (DMD) patients harboring out-of-frame deletion mutations in the DMD gene. In particular, PMOs for skipping exon 44 have been developing in clinical trials, such as the drug NS-089/NCNP-02. Two exon 53 skipping PMOs, golodirsen and viltolarsen, have received conditional approval for treating patients due to their ability to restore dystrophin protein expression. Although promising, further development of exon-skipping technology is needed for patients to have more therapeutic benefit. This chapter describes evaluation methods of exon 44 and 53 skipping PMOs in immortalized DMD patient-derived skeletal muscle cells. We introduce how to quantify exon-skipping efficiencies and dystrophin rescue levels represented by RT-PCR and western blotting, respectively. The screening methods using immortalized patient myotubes can serve to find exon-skipping PMO drug candidates.
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