Allele selective inhibition of mutant C9orf72 foci expression by duplex RNAs targeting the expanded hexanucleotide repeat
US11840690B2 · kind B2 · utility
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Key dates
| Filing date | Nov 17, 2021 |
| Grant date | Dec 12, 2023 |
| Priority date | — |
| Expiry date | Nov 17, 2041 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2310/3521
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Provided herein are compositions and methods for reducing expression of C9orf72 transcripts in cells containing expanded intronic GGGGCC regions, including those in subjects having or at risk of developing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.