CRISPR Fixes Multiple Dystrophin Duplications in DMD Patient Cells
CRISPR-Cas9 gene editing in France targets intronic regions of the DMD gene, restoring the open reading frame and dystrophin expression in myogenic cells with exon 2, 2–9, or 8–9 duplications, offering a potential therapeutic avenue for DMD patients not covered by existing exon-skipping therapies.
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CRISPR Fixes Multiple Dystrophin Duplications in DMD Patient Cells
CRISPR-Cas9 gene editing in France targets intronic regions of the DMD gene, restoring the open reading frame and dystrophin expression in myogenic cells with exon 2, 2–9, or 8–9 duplications, offering a potential therapeutic avenue for DMD patients not covered by existing exon-skipping therapies.