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Multiple exon skipping strategies to by-pass dystrophin mutations

Manipulation of dystrophin pre-mRNA processing offers the potential to overcome mutations in the dystrophin gene that would otherwise lead to Duchenne muscular dystrophy. Dystrophin mutations will require the removal of one or more exons to restore the reading frame and in some cases, multiple exon...

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Main Authors: Adkin, Carl F., Meloni, Penelope L., Fletcher, Susan, Adams, Abbie M., Muntoni, Francesco, Wong, Brenda, Wilton, Steve D.
格式: Artigo
語言:Inglês
出版: Pergamon Press 2012
主題:
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC3488593/
https://ncbi.nlm.nih.gov/pubmed/22182525
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nmd.2011.10.007
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