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Identification of Small Molecule and Genetic Modulators of AON-Induced Dystrophin Exon Skipping by High-Throughput Screening

One therapeutic approach to Duchenne Muscular Dystrophy (DMD) recently entering clinical trials aims to convert DMD phenotypes to that of a milder disease variant, Becker Muscular Dystrophy (BMD), by employing antisense oligonucleotides (AONs) targeting splice sites, to induce exon skipping and rest...

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Detalles Bibliográficos
Autores principales:	O'Leary, Debra A., Sharif, Orzala, Anderson, Paul, Tu, Buu, Welch, Genevieve, Zhou, Yingyao, Caldwell, Jeremy S., Engels, Ingo H., Brinker, Achim
Formato:	Texto
Lenguaje:	English
Publicado:	Public Library of Science 2009
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2791862/ https://www.ncbi.nlm.nih.gov/pubmed/20020055 http://dx.doi.org/10.1371/journal.pone.0008348

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