Gene therapy for ocular disorders
US11564996B2 · kind B2 · utility
0Cited by
23References
22Claims
0Family size
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Key dates
| Filing date | Mar 1, 2018 |
| Grant date | Jan 31, 2023 |
| Priority date | — |
| Expiry date | May 9, 2039 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2800/22
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Compositions and methods are provided for treating Leber congenital amaurosis (LCA) in a subject. In one aspect, a recombinant adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding Lebercilin. In another aspect, Lebercilin has an amino acid sequence of SEQ ID NO: 1. In yet another aspect, the nucleic acid molecule has a sequence of SEQ ID NO: 3 or a variant thereof. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.