Abstract
The primary molecular endpoint for many DMD clinical trials and the proof of concept for therapeutic approaches is the induction or increase in the production of de novo dystrophin. However, it is important to not only quantify levels of restored dystrophin following treatment, but to determine its molecular functionality using unbiased methodologies. We assessed if induced dystrophin following 48 weeks treatment with an exon 53 skipping morpholino (golodirsen), which we previously demonstrated to be capable of inducing dystrophin restoration, also results in enhanced levels of dystrophin associated proteins (DAPs) and a reduction in regenerating myofibres in post treatment biopsies.
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