Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy

@article{Dzierlega2020OptimizationOA,
  title={Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy},
  author={Kasia Dzierlega and Toshifumi Yokota},
  journal={Gene Therapy},
  year={2020},
  pages={1-10}
}
Duchenne muscular dystrophy (DMD) is one of the most common lethal muscle-wasting disorders affecting young boys caused by mutations in the DMD gene. Exon skipping has emerged as a promising therapy for DMD. Antisense oligonucleotides (AONs) are designed to induce the skipping of exon(s), in order to restore the reading frame, and therefore, allow for dystrophin expression. Eteplirsen and golodirsen, AONs for DMD exons 51 and 53 skipping, have been recently approved by the FDA. Viltolarsen, an… 

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