Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice.

@article{Yin2008EffectiveES,
  title={Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice.},
  author={HaiFang Yin and Qilong Lu and Matthew W. Wood},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2008},
  volume={16 1},
  pages={38-45}
}
Duchenne muscular dystrophy (DMD) is the most common and severe form of muscular dystrophy, arising from mutations in the dystrophin gene that preclude the synthesis of functional protein. Antisense oligonucleotides (AOs) have been shown to induce specific exon skipping and thereby restore the reading frame and expression of functional dystrophin. In this report, we examine the effects of peptide nucleic acid (PNA) oligonucleotides and PNAs conjugated with peptides including TAT, muscle… CONTINUE READING

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