In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy

@inproceedings{Echigoya2015InSS,
  title={In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy},
  author={Yusuke Echigoya and Vincent Mouly and Luis Gonz{\'a}lez Garc{\'i}a and Toshifumi Yokota and William Duddy},
  booktitle={PloS one},
  year={2015}
}
The use of antisense 'splice-switching' oligonucleotides to induce exon skipping represents a potential therapeutic approach to various human genetic diseases. It has achieved greatest maturity in exon skipping of the dystrophin transcript in Duchenne muscular dystrophy (DMD), for which several clinical trials are completed or ongoing, and a large body of data exists describing tested oligonucleotides and their efficacy. The rational design of an exon skipping oligonucleotide involves the… CONTINUE READING
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