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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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Dettagli Bibliografici
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2014
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Accesso online: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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