Cas3-Mediated DMD Exon Skipping (IMAGE)

Osaka University

Cas3-Mediated DMD Exon Skipping

Caption

Cas3-mediated DMD exon skipping in induced pluripotent stem cells. (Left) RT-PCR analysis of the DMD exon 45 skipping in skeletal muscle cells differentiated from subclones of DMD patient-derived iPSCs treated with Cas3. (Right) The restored DMD protein detected in skeletal muscle cells differentiated from the exon skipping subclones of DMD-iPSCs.

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Osaka University

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