AAV-mediated delivery of ATP1A3 genes to central nervous system
US11738093B2 · kind B2 · utility
Assignees
Inventors
Key dates
| Filing date | Apr 9, 2019 |
| Grant date | Aug 29, 2023 |
| Priority date | — |
| Expiry date | Dec 6, 2040 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14143
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
This invention relates to a transgenic vector for transducing cells of a mammal's CNS and transgenically expressing a protein in the mammal's CNS. The transgenic vector comprises a virus-derived vector, a nucleic acid sequence encoding the protein, and an endogenous ATP1A3 promoter sequence. This invention also relates to a composition comprising a recombinant AAV vector comprising a nucleic acid sequence encoding a ATP1A3 protein, in a form compatible with administration into the CNS. The invention also relates to a method for treating a subject having a neurological disorder associated with mutations in the ATP1A3 gene and a method for delivering a transgenic ATP1A3 DNA to the central nervous system of a mammal by administering the recombinant AAV vector into the mammal's CNS.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.