Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo.

@article{Moulton2007CellpenetratingPC,
  title={Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo.},
  author={Hong M. Moulton and Susan Fletcher and Benjamin William Neuman and Graham McClorey and David A. Stein and Sa{\"i}d Abes and Stephen D. Wilton and Michael J Buchmeier and Bernard Lebleu and Patrick L. Iversen},
  journal={Biochemical Society transactions},
  year={2007},
  volume={35 Pt 4},
  pages={826-8}
}
The cellular uptake of PMOs (phosphorodiamidate morpholino oligomers) can be enhanced by their conjugation to arginine-rich CPPs (cell-penetrating peptides). Here, we discuss our recent findings regarding (R-Ahx-R)(4)AhxB (Ahx is 6-aminohexanoic acid and B is beta-alanine) CPP-PMO conjugates in DMD (Duchenne muscular dystrophy) and murine coronavirus research. An (R-Ahx-R)(4)AhxB-PMO conjugate was the most effective compound in inducing the correction of mutant dystrophin transcripts in… CONTINUE READING