ArticleBiochimica et biophysica acta2009
Differential stabilities of alternative exon-skipped rod motifs of dystrophin.
Article in Biochimica et biophysica acta, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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Who cites it
14 citing papers in PubMed, 27 citations in OpenAlex.
- Four new mouse models of Duchenne muscular dystrophy with clinically relevant exon deletions in the human DMD gene.Disease models & mechanisms · 2026Article
- In-Frame Deletion of Dystrophin Exons 8-50 Results in DMD Phenotype.International journal of molecular sciences · 2023Article
- Cardiac Myoediting Attenuates Cardiac Abnormalities in Human and Mouse Models of Duchenne Muscular Dystrophy.Circulation research · 2021Article
- Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping inMolecular therapy. Methods & clinical development · 2020Article
- The Dystrophin Complex: Structure, Function, and Implications for Therapy.Comprehensive Physiology · 2015Review
- Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.Annals of neurology · 2015Article
- In vitro stability of therapeutically relevant, internally truncated dystrophins.Skeletal muscle · 2015Article
- Article
- Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2013Article
- Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Thermodynamic stability, unfolding kinetics, and aggregation of the N-terminal actin-binding domains of utrophin and dystrophin.Proteins · 2012Article
- Internal deletion compromises the stability of dystrophin.Human molecular genetics · 2011Article
- Exon-skipped dystrophins for treatment of Duchenne muscular dystrophy: mass spectrometry mapping of most exons and cooperative domain designs based on single molecule mechanics.Cytoskeleton (Hoboken, N.J.) · 2010Article
- Disease-causing missense mutations in actin binding domain 1 of dystrophin induce thermodynamic instability and protein aggregation.Proceedings of the National Academy of Sciences of the United States of America · 2010Article
Corrections and comments
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Authors and funding
3 authors at 2 institutions in 1 country.
Funding
Abstract
Exon skipping repair is a strategy being investigated in early stage clinical trials to treat Duchenne muscular dystrophy. This is most applicable to the majority of cases which arise when genetic defects cause frame shift mutations, and induced exon skipping of out-of-phase exons restores the reading frame. However, the consequences to the edited protein so produced have not been considered. In many cases alternative routes to restoring the reading frame are possible, and we show in a test case involving exon 44 that the resulting differently edited proteins greatly vary in stability, with one of them very similar to normal unskipped dystrophin, and the other much less stable as assessed by the thermodynamics of folding as well as resistance to proteolysis. This has implications for the design of optimal therapeutic exon skipping strategies, which presumably wish to result repairs with as much fidelity to normal dystrophin as possible.
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Registered trials
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