Oligonucleotide compositions and methods of use thereof
US11597927B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Jun 1, 2018 |
| Grant date | Mar 7, 2023 |
| Priority date | — |
| Expiry date | Oct 6, 2038 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y301/01003
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Among other things, the present disclosure provides designed PNPLA3 oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or N patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.