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Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations

Antisense oligomer induced exon skipping is showing promise as a therapy to reduce the severity of Duchenne Muscular Dystrophy. To date, the focus has been on excluding single exons flanking frame-shifting deletions in the dystrophin gene, however, a third of all Duchenne Muscular Dystrophy causing...

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Hlavní autoři: Forrest, Sarah, Meloni, Penny L, Muntoni, Francesco, Kim, Jihee, Fletcher, Sue, Wilton, Steve D
Médium: Artigo
Jazyk:Inglês
Vydáno: 2010
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2991584/
https://ncbi.nlm.nih.gov/pubmed/20817455
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nmd.2010.07.276
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