Modified U7 snRNAs for treatment of neuromuscular diseases
US9080170B2 · kind B2 · utility
2Cited by
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24Claims
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Key dates
| Filing date | Mar 17, 2011 |
| Grant date | Jul 14, 2015 |
| Priority date | — |
| Expiry date | Mar 17, 2031 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2330/51
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
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.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.