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

GSE262976 Homo sapiens Expression profiling by high throughput sequencing 15 samples 2024/05/30 GPL24676
Summary
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.
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