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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Bibliographische Detailangaben
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2014
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3950770/
https://ncbi.nlm.nih.gov/pubmed/24549299
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2014.1
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