Accurate quantification of dystrophin mRNA and exon skipping levels in Duchenne Muscular Dystrophy

@article{Spitali2010AccurateQO,
  title={Accurate quantification of dystrophin mRNA and exon skipping levels in Duchenne Muscular Dystrophy},
  author={Pietro Spitali and Hans Heemskerk and Rolf H. A. M. Vossen and Alessandra Ferlini and Johan T. den Dunnen and Peter A. C. 't Hoen and Annemieke Aartsma-Rus},
  journal={Laboratory Investigation},
  year={2010},
  volume={90},
  pages={1396-1402}
}
Antisense oligonucleotide (AON)-mediated exon skipping aimed at restoring the reading frame is a promising therapeutic approach for Duchenne muscular dystrophy that is currently tested in clinical trials. Numerous AONs have been tested in (patient-derived) cultured muscle cells and the mdx mouse model. The main outcome to measure AON efficiency is usually the exon-skipping percentage, though different groups use different methods to assess these percentages. Here, we compare a series of… CONTINUE READING
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