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Therapeutic exon skipping for dysferlinopathies?

✍ Scribed by Aartsma-Rus, Annemieke; Singh, Kavita H K; Fokkema, Ivo F A C; Ginjaar, Ieke B; van Ommen, Gert-Jan; Dunnen, Johan T den; van der Maarel, Silvère M

Book ID
109849384
Publisher
Nature Publishing Group
Year
2010
Tongue
English
Weight
413 KB
Volume
18
Category
Article
ISSN
1018-4813

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Therapeutic exon skipping for dysferlino
Therapeutic exon skipping for dysferlinopathies?
✍ Aartsma-Rus, Annemieke; Singh, Kavita HK; Fokkema, Ivo FAC; Ginjaar, Ieke B; van 📂 Article 📅 2010 🏛 Nature Publishing Group 🌐 English ⚖ 153 KB
Exon skipping-mediated dystrophin readin
Exon skipping-mediated dystrophin reading frame restoration for small mutations
✍ Pietro Spitali; Paola Rimessi; Marina Fabris; Daniela Perrone; Sofia Falzarano; 📂 Article 📅 2009 🏛 John Wiley and Sons 🌐 English ⚖ 318 KB

Exon skipping using antisense oligonucleotides (AONs) has successfully been used to reframe the mRNA in various Duchenne muscular dystrophy patients carrying deletions in the DMD gene. In this study we tested the feasibility of the exon skipping approach for patients with small mutations in in-frame