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A Cell-Based High-Throughput Screening Assay for Posttranscriptional Utrophin Upregulation

31 Oct 2012

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a devastating muscle-wasting disease caused by mutations in the dystrophin gene. Utrophin is a homologue of dystrophin that can compensate for its absence when overexpressed in DMD animal models. Utrophin upregulation is therefore a promising therapeutic approach for DMD. Utrophin is regulated at both transcriptional and posttranscriptional levels. Transcriptional regulation...

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PMID: 23112083
DOI: 10.1177/1087057112465648

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