Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy

@inproceedings{Nichols2016MultiexonSU,
  title={Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy},
  author={Bailey Miskew Nichols and Yoshitsugu Aoki and Masatsugu Kuraoka and Joshua J.A. Lee and Shin'ichi Takeda and Toshifumi Yokota},
  booktitle={Journal of visualized experiments : JoVE},
  year={2016}
}
Duchenne muscular dystrophy (DMD) is one of the most common lethal genetic diseases worldwide, caused by mutations in the dystrophin (DMD) gene. Exon skipping employs short DNA/RNA-like molecules called antisense oligonucleotides (AONs) that restore the reading frame and produce shorter but functional proteins. However, exon skipping therapy faces two major hurdles: limited applicability (up to only 13% of patients can be treated with a single AON drug), and uncertain function of truncated… CONTINUE READING
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