A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.

@article{Yin2009AFP,
  title={A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.},
  author={HaiFang Yin and Hong M. Moulton and Corinne A Betts and Yiqi Seow and Jordan Boutilier and Patrick L Iverson and Matthew J. Wood},
  journal={Human molecular genetics},
  year={2009},
  volume={18 22},
  pages={4405-14}
}
Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene that abolish the synthesis of dystrophin protein. Antisense oligonucleotides (AOs) targeted to trigger excision of an exon bearing a mutant premature stop codon in the DMD transcript have been shown to skip the mutated exon and partially restore functional dystrophin protein in dystrophin-deficient mdx mice. To fully exploit the therapeutic potential of this method requires highly efficient systemic AO delivery to multiple… CONTINUE READING