Article

Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping

2023-05-09

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a progressive muscle disease caused by the absence of dystrophin protein. One current DMD therapeutic strategy, exon skipping, produces a truncated dystrophin isoform using phosphorodiamidate morpholino oligomers (PMOs). However, the potential of exon skipping therapeutics has not been fully realized as increases in dystrophin protein have been minimal in clinical trials. Here,...

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Literature Corpus work: 29c8ca11-84ac-57a3-8991-5ca00d82f910
DOI: 10.1101/2023.05.09.540042

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