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Optimizing exon skipping therapies for DMD

Exon skipping is one of the more promising therapeutic options for Duchenne Muscular Dystrophy (DMD). The idea is to use antisense oligonucleotides to splice out selected exons from the pre-mRNA, at or next to the mutation site, so as to generate a translatable transcript from the mutant dystrophin...

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Bibliografiske detaljer
Main Authors: Yokota, T, Duddy, W, Partridge, T
Format: Artigo
Sprog:Inglês
Udgivet: Pacini Editore SpA 2007
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2949311/
https://ncbi.nlm.nih.gov/pubmed/18646569
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