RNA interference for the treatment of gain-of-function disorders
US11299734B2 · kind B2 · utility
1Cited by
18References
9Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | May 17, 2019 |
| Grant date | Apr 12, 2022 |
| Priority date | — |
| Expiry date | Jun 5, 2039 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/34
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.