Gene therapy for lysosomal storage diseases
US8796236B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Dec 4, 2009 |
| Grant date | Aug 5, 2014 |
| Priority date | — |
| Expiry date | Jan 5, 2031 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y302/01045
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.