Recombinant virus products and methods for inhibition of expression of DUX4
US9469851B2 · kind B2 · utility
7Cited by
6References
5Claims
0Family size
Assignee
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Key dates
| Filing date | Jul 24, 2012 |
| Grant date | Oct 18, 2016 |
| Priority date | — |
| Expiry date | Oct 26, 2032 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14143
- WIPO fieldBiotechnology
- WIPO sectorChemistry
Abstract
The present invention relates to RNA interference-based methods for inhibiting the expression of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.