One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice.

@article{Wu2011OneyearTO,
  title={One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice.},
  author={Bo Wu and Bin Xiao and Caryn Cloer and Mona Shaban and Arpana Sali and Peijuan Lu and Juan Li and Kanneboyina Nagaraju and Xiao Xiao and Qi Long Lu},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2011},
  volume={19 3},
  pages={
          576-83
        }
}
Antisense therapy has been successful to skip targeted dystrophin exon with correction of frameshift and nonsense mutations of Duchenne muscular dystrophy (DMD). Systemic production of truncated but functional dystrophin proteins has been achieved in animal models. Furthermore, phase I/II clinical trials in United Kingdom and the Netherlands have demonstrated dystrophin induction by local and systemic administrations of antisense oligomers. However, long-term efficacy and potential toxicity… CONTINUE READING
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Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy.

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