Ultrasound-enhanced delivery of Morpholino with Bubble liposomes ameliorates the myotonia of myotonic dystrophy model mice

@inproceedings{Koebis2013UltrasoundenhancedDO,
  title={Ultrasound-enhanced delivery of Morpholino with Bubble liposomes ameliorates the myotonia of myotonic dystrophy model mice},
  author={Michinori Koebis and Tamami Kiyatake and Hiroshi Yamaura and Kanako Nagano and Mana Higashihara and Masahiro Sonoo and Yukiko K. Hayashi and Yoichi Negishi and Yoko Endo-Takahashi and Dai Yanagihara and Ryoichi Matsuda and Masanori P. Takahashi and Ichizo Nishino and Shoichi Ishiura},
  booktitle={Scientific reports},
  year={2013}
}
Phosphorodiamidate morpholino oligonucleotide (PMO)-mediated control of the alternative splicing of the chloride channel 1 (CLCN1) gene is a promising treatment for myotonic dystrophy type 1 (DM1) because the abnormal splicing of this gene causes myotonia in patients with DM1. In this study, we optimised a PMO sequence to correct Clcn1 alternative splicing and successfully remedied the myotonic phenotype of a DM1 mouse model, the HSALR mouse. To enhance the efficiency of delivery of PMO into… CONTINUE READING
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