A novel CRISPR-Cas9 strategy to target DYSTROPHIN mutations downstream of exon 44 in patient-specific DMD iPSCs

Here, we performed CRISPR-cas9 mediated genetic correction in DMD patient derived iPSC and then differentiated them into in vitro myotubes. Pairing bulk RNA sequencing between Wild-type (WT), DMD and the corrected counterparts we were able to differentiate the molecular changes that occurs during the absence of dystrophin. Overall design: We performed gene expression profiling for data obtained through bulk RNA-sequencing of 15 uniques samples. Samples included WT, DMD and DMD corrected iPSC derived myotubes.