GARCIA, LUIS,FURLING, DENIS,BELEY, CYRIAQUE,VOIT, THOMAS
申请号：
CA2792696
公开号：
CA2792696C
申请日:
2011.03.17
申请国别(地区):
CA
年份:
2020
代理人:
摘要：
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.
