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Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy

A readily available animal model is essential for rapidly identifying effective treatments for Duchenne muscular dystrophy (DMD), a devastating neuromuscular disorder caused by the lack of dystrophin protein, which results from frame-disrupting mutations in the DMD gene. Currently, the mdx mouse is...

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Autors principals: Cao, Limin, Han, Gang, Gu, Ben, Yin, HaiFang
Format: Artigo
Idioma:Inglês
Publicat: Public Library of Science 2014
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4217760/
https://ncbi.nlm.nih.gov/pubmed/25365558
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0111079
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