Article

Clinical trials of exon skipping in Duchenne muscular dystrophy

2017-08-10

Abstract excerpt

Introduction: DMD is most common form of severe childhood muscular dystrophy. The large size of the DMD gene challenges DNA repair or replacement. However, experimental in vitro and in vivo studies using antisense oligonucleotides (AONs) showed that exon skipping could result in production of a truncated dystrophin protein with potential to modify the dystrophic phenotype. This provided the impetus for clinical te...

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Literature Corpus work: 90eff783-e7e8-5ab1-b42d-3e2e2c728dea
DOI: 10.1080/21678707.2017.1366310

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