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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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Autors principals: Adkin, Carl F., Meloni, Penelope L., Fletcher, Susan, Adams, Abbie M., Muntoni, Francesco, Wong, Brenda, Wilton, Steve D.
Format: Artigo
Idioma:Inglês
Publicat: Pergamon Press 2012
Matèries:
Accés en línia: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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