Compositions for modulating C9ORF72 expression
US10221414B2 · kind B2 · utility
9Cited by
21References
34Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Oct 11, 2014 |
| Grant date | Mar 5, 2019 |
| Priority date | — |
| Expiry date | Oct 11, 2034 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2310/341
- WIPO fieldMeasurement
- WIPO sectorInstruments
Abstract
Disclosed herein are compositions and methods for reducing expression of C90RF72 mRNA and protein in an animal with C90RF72 specific inhibitors. Also disclosed herein are compositions and methods of selectively inhibiting a C90RF72 pathogenic associated mRNA variant by administering an antisense compound targeting the region beginning at the start site of exon 1A to the start site of exon 1B of a C90RF72 pre-mRNA. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C90RF72 specific inhibitors include antisense compounds.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.