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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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Detalles Bibliográficos
Autores principales:	Verhaart, Ingrid E. C., Aartsma-Rus, Annemieke
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2012
Materias:	Muscular Dystrophy
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3523663/ https://www.ncbi.nlm.nih.gov/pubmed/23259153 http://dx.doi.org/10.1371/currents.md.597d700f92eaa70de261ea0d91821377

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