Nanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophy.

@article{Falzarano2014NanoparticleDO,
  title={Nanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophy.},
  author={Maria Sofia Falzarano and Chiara Passarelli and Alessandra Ferlini},
  journal={Nucleic acid therapeutics},
  year={2014},
  volume={24 1},
  pages={87-100}
}
Antisense therapy is a powerful tool for inducing post-transcriptional modifications and thereby regulating target genes associated with disease. There are several classes of antisense oligonucleotides (AONs) with therapeutic use, such as double-stranded RNAs (interfering RNAs, utilized for gene silencing, and single-stranded AONs with various chemistries, which are useful for antisense targeting of micro-RNAs and mRNAs. In particular, the use of AONs for exon skipping, by targeting pre-mRNA… CONTINUE READING
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