Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells

@inproceedings{Lehto2014CellularTD,
  title={Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells},
  author={Taavi Lehto and Alejandra Castillo Alvarez and Sarah Gauck and Michael J. Gait and Thibault Coursindel and Matthew J. Wood and Bernard Lebleu and Prisca Boisguerin},
  booktitle={Nucleic acids research},
  year={2014}
}
Cell-penetrating peptide-mediated delivery of phosphorodiamidate morpholino oligomers (PMOs) has shown great promise for exon-skipping therapy of Duchenne Muscular Dystrophy (DMD). Pip6a-PMO, a recently developed conjugate, is particularly efficient in a murine DMD model, although mechanisms responsible for its increased biological activity have not been studied. Here, we evaluate the cellular trafficking and the biological activity of Pip6a-PMO in skeletal muscle cells and primary… CONTINUE READING

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