Systemic delivery of a Peptide-linked morpholino oligonucleotide neutralizes mutant RNA toxicity in a mouse model of myotonic dystrophy.

@article{Leger2013SystemicDO,
  title={Systemic delivery of a Peptide-linked morpholino oligonucleotide neutralizes mutant RNA toxicity in a mouse model of myotonic dystrophy.},
  author={Andrew J. Leger and Leocadia M. Mosquea and Nicholas P Clayton and I-Huan Wu and Timothy Weeden and Carol A. Nelson and Lucy Phillips and Errin Roberts and Peter A. Piepenhagen and Seng H. Cheng and B. M. Wentworth},
  journal={Nucleic acid therapeutics},
  year={2013},
  volume={23 2},
  pages={109-17}
}
Expansions of CUG trinucleotide sequences in RNA transcripts provide the basis for toxic RNA gain-of-function that leads to detrimental changes in RNA metabolism. A CTG repeat element normally resides in the 3' untranslated region of the dystrophia myotonica-protein kinase (DMPK) gene, but when expanded it is the genetic lesion of myotonic dystrophy type 1 (DM1), a hereditary neuromuscular disease. The pathogenic DMPK transcript containing the CUG expansion is retained in ribonuclear foci as… CONTINUE READING
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