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Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy
The potential for therapeutic application of splice-switching oligonucleotides (SSOs) to modulate pre-mRNA splicing is increasingly evident in a number of diseases. However, the primary drawback of this approach is poor cell and in vivo oligonucleotide uptake efficacy. Biological activities can be s...
Autores principales: | Shabanpoor, Fazel, McClorey, Graham, Saleh, Amer F., Järver, Peter, Wood, Matthew J.A., Gait, Michael J. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4288157/ https://www.ncbi.nlm.nih.gov/pubmed/25468897 http://dx.doi.org/10.1093/nar/gku1256 |
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