Compositions and methods for treating CEP290 associated disease
US11566263B2 · kind B2 · utility
1Cited by
29References
28Claims
0Family size
Assignee
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Key dates
| Filing date | Aug 2, 2017 |
| Grant date | Jan 31, 2023 |
| Priority date | — |
| Expiry date | Jun 16, 2039 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2800/80
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Nucleic acids and viral vectors, particularly adeno-associated virus (AAV) vectors are provided that encode Cas9 and paired guide RNAs. The nucleic acids and vectors, and compositions that comprise them, can be used in methods to treat subjects, to alter cells in subjects who may suffer from an inherited retinal dystrophy such as CEP290 associated disease or who may be in need of alteration of a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as the CEP290 gene, and/or to treat inherited retinal dystrophies including CEP290 associated disease.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.