| 发明名称 |
MODIFIED U7 SNRNAS FOR TREATMENT OF NEUROMUSCULAR DISEASES |
| 摘要 |
<p>The present invention relates to a method to improve the activity of engineered U7 snRNAs used in the context of RNA-based therapeutics; particularly in exon skipping, exon inclusion, and mRNA eradication strategies. The resulting modified U7 snRNAs are useful for treating neuromuscular diseases, in particular Duchenne neuromuscular dystrophy, myotonic dystrophy DM1 and spinal muscular atrophy.</p> |
| 申请公布号 |
WO2011113889(A1) |
申请公布日期 |
2011.09.22 |
| 申请号 |
WO2011EP54026 |
申请日期 |
2011.03.17 |
| 申请人 |
ASSOCIATION INSTITUT DE MYOLOGIE;GARCIA, LUIS;FURLING, DENIS;BELEY, CYRIAQUE;VOIT, THOMAS |
发明人 |
GARCIA, LUIS;FURLING, DENIS;BELEY, CYRIAQUE;VOIT, THOMAS |
| 分类号 |
C12N15/11;A61K31/7088;A61K48/00 |
主分类号 |
C12N15/11 |
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