[Correspondence] Exon-skipping therapy for Duchenne muscular dystrophy – Authors' reply
Source: The Lancet
Satyakam Bhagavati questions the choice of biochemical outcome measures in our systemic phase 2 exon-skipping study of Duchenne muscular dystrophy. He suggests two RNA-based assays (quantitative RNAse protection and northern-blot analysis), which are theoretically feasible but difficult to do and limited in their scope. To assess exon skipping at the RNA level, we used the standard methods that are regularly used on both animal and human tissue, and in all the published clinical trials to date. We detailed the RNA results in the original webappendix and, although we acknowledge that the methods of RNA detection used are not fully quantitative, the main aim of the study was that of increasing protein expression in these boys.