3 Result(s)
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Veronica Pini
Science, Humanities and Social Sciences, multidisciplinary
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Article
Open AccessTransiently expressed CRISPR/Cas9 induces wild-type dystrophin in vitro in DMD patient myoblasts carrying duplications
Among the mutations arising in the DMD gene and causing Duchenne Muscular Dystrophy (DMD), 10–15% are multi-exon duplications. There are no current therapeutic approaches with the ability to excise large multi-ex...
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Article
Open AccessPublisher Correction: Necroptosis mediates myofibre death in dystrophin-deficient mice
The original version of this article contained an error in Fig. 3. In panel c, the labels ‘mdx’ and ‘mdx Ripk3-/-‘ were inadvertently inverted. This has now been corrected in the PDF and HTML versions of the A...
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Article
Open AccessNecroptosis mediates myofibre death in dystrophin-deficient mice
Duchenne muscular dystrophy (DMD) is a severe degenerative disorder caused by mutations in the dystrophin gene. Dystrophin-deficient muscles are characterised by progressive myofibre necrosis in which inflamma...
