Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
US11306329B2 · kind B2 · utility
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18Claims
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Key dates
| Filing date | Feb 19, 2019 |
| Grant date | Apr 19, 2022 |
| Priority date | — |
| Expiry date | Dec 9, 2039 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14143
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Provided herein are adeno-associated virus (AAV) compositions that can restore F8 gene function in a cell without co-transducing or co-administering an exogenous nuclease or a nucleotide sequence that encodes an exogenous nuclease. Also provided are methods of using the AAV compositions to correct an F8 gene mutation and/or treat a disease or disorder associated with an F8 gene mutation. Packaging systems for making the adeno-associated virus compositions are also provided.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.