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

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 opti...

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Detalles Bibliográficos
Autores principales: Koebis, Michinori, Kiyatake, Tamami, Yamaura, Hiroshi, Nagano, Kanako, Higashihara, Mana, Sonoo, Masahiro, Hayashi, Yukiko, Negishi, Yoichi, Endo-Takahashi, Yoko, Yanagihara, Dai, Matsuda, Ryoichi, Takahashi, Masanori P., Nishino, Ichizo, Ishiura, Shoichi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2013
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3718203/
https://www.ncbi.nlm.nih.gov/pubmed/23873129
http://dx.doi.org/10.1038/srep02242

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