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The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice

Antisense-mediated exon skipping is currently in clinical development for Duchenne muscular dystrophy (DMD) to amend the consequences of the underlying genetic defect and restore dystrophin expression. Due to turnover of compound, transcript, and protein, chronic treatment with effector molecules (a...

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Detalles Bibliográficos
Autores principales: Verhaart, Ingrid E C, van Vliet-van den Dool, Laura, Sipkens, Jessica A, de Kimpe, Sjef J, Kolfschoten, Ingrid G M, van Deutekom, Judith C T, Liefaard, Lia, Ridings, Jim E, Hood, Steve R, Aartsma-Rus, Annemieke
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3950770/
https://www.ncbi.nlm.nih.gov/pubmed/24549299
http://dx.doi.org/10.1038/mtna.2014.1