Papers, explained in our own words

1. A single liver-sparing myotropic AAV ameliorates muscular dystrophy in humanized mice by base editing

Pien Yc, Li H, Bulliard G, Liu H, Durbacz Mz, Xu Z, Et Al.

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, most of which are large exon deletions that result in loss of dystrophin protein. Adenine base editing (ABE) enables permanent gene correction by modifying splice sites to cause exon skipping and restoration of the DMD open reading frame. We…

• Genetics
• Muscle biology
• Skeletal muscle