CRISPR/Cas9 method reduces DMD pathology

In a paper published in Nature Communications, researchers from the University of Washington showed that muscle-specific CRISPR/Cas9 can be used to insert a "mutation corrected" DNA template into myogenic cells in a mouse model of Duchenne muscular dystrophy and reduce DMD pathophysiology. The model expressed a nonsense mutation in exon 53, which leads to loss of dystrophin expression.

The researchers evaluated two different CRISPR strategies. The first strategy involved deletion of the nonsense mutation by excising exons 52 and 53. The second strategy used CRISPR to insert a corrected DNA template at exon 53. In the latter, insertion of the template was performed so that if it were not successful, exon 53 would still be disrupted...