In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy

The use of antisense ‘splice-switching’ oligonucleotides to induce exon skipping represents a potential therapeutic approach to various human genetic diseases. It has achieved greatest maturity in exon skipping of the dystrophin transcript in Duchenne muscular dystrophy (DMD), for which several clin...

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Detalles Bibliográficos
Autores principales: Echigoya, Yusuke, Mouly, Vincent, Garcia, Luis, Yokota, Toshifumi, Duddy, William
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2015
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4376395/
https://www.ncbi.nlm.nih.gov/pubmed/25816009
http://dx.doi.org/10.1371/journal.pone.0120058

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