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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...
Autores principales: | Adkin, Carl F., Meloni, Penelope L., Fletcher, Susan, Adams, Abbie M., Muntoni, Francesco, Wong, Brenda, Wilton, Steve D. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Pergamon Press
2012
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3488593/ https://www.ncbi.nlm.nih.gov/pubmed/22182525 http://dx.doi.org/10.1016/j.nmd.2011.10.007 |
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