Deletion of Exon 23 in Mouse iPSCs with Two Guide RNAs (gRNAs) coupled with Cas9
5:25
Identification of iPSC Colonies with Exon23 Deletion
6:33
Results: CRISPR/Cas9-mediated Exon23 Deletion
6:57
Conclusion
TRANSCRIÇÃO
Duchenne is a muscular dystrophy causing premature exhausting of muscle progenitor cells. It is one of the most severe Muscular Dystrophies. To promote a functional muscle regeneration, we use CRISPR/Cas9-mediated gene editing to restore dystrophi
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Here, we present a Cas9-based exon23 deletion protocol to restore dystrophin expression in iPSC from Dmdmdx mouse-derived skin fibroblasts and directly differentiate iPSCs into myogenic progenitor cells (MPC) using the Tet-on MyoD activation system.