Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

@inproceedings{Betts2012Pip6PMOAN,
  title={Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment},
  author={Corinne A Betts and Amer F Saleh and Andrey A. Arzumanov and Suzan M Hammond and Caroline R Godfrey and Thibault Coursindel and Michael J. Gait and Matthew J. Wood},
  booktitle={Molecular therapy. Nucleic acids},
  year={2012}
}
Antisense oligonucleotides (AOs) are currently the most promising therapeutic intervention for Duchenne muscular dystrophy (DMD). AOs modulate dystrophin pre-mRNA splicing, thereby specifically restoring the dystrophin reading frame and generating a truncated but semifunctional dystrophin protein. Challenges in the development of this approach are the relatively poor systemic AO delivery and inefficient dystrophin correction in affected non-skeletal muscle tissues, including the heart. We have… CONTINUE READING
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Wood , Expression analysis in multiple muscle groups and serum reveals complexity in the microRNA transcriptome of the mdx mouse with implications for therapy

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