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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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Egile Nagusiak: Yokota, T, Duddy, W, Partridge, T
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Pacini Editore SpA 2007
Gaiak:
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC2949311/
https://ncbi.nlm.nih.gov/pubmed/18646569
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