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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Bibliografische gegevens
Hoofdauteurs: 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
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Nature Publishing Group 2014
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Original Article
Online toegang: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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