Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino.

@article{Wu2012LongtermRO,
  title={Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino.},
  author={Bo Wu and Peijuan Lu and Caryn Cloer and Mona Shaban and Snimar Grewal and Stephanie Milazi and Sapana N. Shah and Hong M. Moulton and Qi Long Lu},
  journal={The American journal of pathology},
  year={2012},
  volume={181 2},
  pages={
          392-400
        }
}
Exon skipping is capable of correcting frameshift and nonsense mutations in Duchenne muscular dystrophy. Phase 2 clinical trials in the United Kingdom and the Netherlands have reported induction of dystrophin expression in muscle of Duchenne muscular dystrophy patients by systemic administration of both phosphorodiamidate morpholino oligomers (PMO) and 2'-O-methyl phosphorothioate. Peptide-conjugated phosphorodiamidate morpholino offers significantly higher efficiency than phosphorodiamidate… CONTINUE READING
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