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Exon Skipping Approach to Duchenne Muscular Dystorphy
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- Takeda Shin’ichi
- Translational Medical Center, National Center of Neurology and Psychiatry
Bibliographic Information
- Other Title
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- デュシェンヌ型筋ジストロフィーに対するエクソン・スキップ誘導療法
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Description
Exon skipping therapy by antisense oligonucleotide is a promising approach to Duchenne muscular dystrophy (DMD). We have reported the proof-of-concept studies using morpholino on mice or dog DMD model and on patient derived cells. Based on these results, we had promoted collaborative research with a Japanese pharmaceutical company and encouraged development of DMD gene exon 53 skipping drug, then finally started an investigator-initiated clinical trial from 2013. Furthermore, we are addressing exploratory researches to expand the possibility of AON; such as, an application of AON to Fukuyama congenital muscular dystrophy, and an elucidation of AON uptake mechanism.
Journal
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- Rinsho Shinkeigaku
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Rinsho Shinkeigaku 54 (12), 1071-1073, 2014
Societas Neurologica Japonica
