Dataset Information


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

ABSTRACT: [This corrects the article DOI: 10.1371/journal.pone.0111079.].


PROVIDER: S-EPMC6237414 | BioStudies | 2018-01-01

REPOSITORIES: biostudies

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Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in Duchenne muscular dystrophy.

Cao Limin L   Han Gang G   Gu Ben B   Yin HaiFang H  

PloS one 20141103 11

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 the most commonly used model for antisense oligonucleotide (AO)-mediated exon skipping pre-clinical studies, with a mild phenotype. However, the accessibility of mdx mouse colonies particularly in dev  ...[more]

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