Efficient Skipping of Single Exon Duplications in <i>DMD</i> Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach

Abstract

This study provides another proof of concept for the feasibility of therapeutic skipping in patients carrying exon duplications in order to express wild-type, full-length mRNA, although careful evaluation of the skipping efficiency should be performed as some exons are easier to skip than others. Such a personalized strategy is expected to be highly beneficial for this subset of DMD patients, compared to inducing expression of an internally-deleted dystrophin.