Recombinant virus products and methods for inhibition of expression of DUX4
US11802291B2 · kind B2 · utility
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Key dates
| Filing date | Mar 12, 2019 |
| Grant date | Oct 31, 2023 |
| Priority date | — |
| Expiry date | Mar 12, 2039 |
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.