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The Effect of 6-Thioguanine on Alternative Splicing and Antisense-Mediated Exon Skipping Treatment for Duchenne Muscular Dystrophy

The severe muscle wasting disorder Duchenne muscular dystrophy (DMD) is caused by genetic defects in the DMD gene, leading to a complete absence of dystrophin protein. Of the therapeutic approaches addressing the underlying genetic defect, exon skipping through antisense oligonucleotides (AONs) is t...

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Bibliografiske detaljer
Main Authors: Verhaart, Ingrid E. C., Aartsma-Rus, Annemieke
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science 2012
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3523663/
https://ncbi.nlm.nih.gov/pubmed/23259153
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/currents.md.597d700f92eaa70de261ea0d91821377
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