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...

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Detalles Bibliográficos
Autores principales: Shabanpoor, Fazel, McClorey, Graham, Saleh, Amer F., Järver, Peter, Wood, Matthew J.A., Gait, Michael J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2015
Materias:
Chemical Biology and Nucleic Acid Chemistry
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

Internet

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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