Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector
US11723988B2 · kind B2 · utility
Assignees
Inventors
Key dates
| Filing date | Oct 22, 2018 |
| Grant date | Aug 15, 2023 |
| Priority date | — |
| Expiry date | Nov 23, 2039 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2830/008
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Several new vector-promoter combinations to overcome the limitations associated with AAV-mediated cone transduction in the fovea are provided. The delivery modality relies on a cone-specific promoter and result in high-level transgene expression compatible with optogenetic vision restoration. Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector comprising a VP1 capsid protein as set forth in SEQ ID NO: 11 and the polynucleotide of interest under the control of the pR1.7 promoter as set forth in SEQ ID NO: 12 are also provided.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.