Gene therapy for neurometabolic disorders
US10913956B2 · kind B2 · utility
0Cited by
5References
4Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Jul 1, 2016 |
| Grant date | Feb 9, 2021 |
| Priority date | — |
| Expiry date | Jul 1, 2036 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14143
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The disclosure pertains to methods and compositions for treating disorders affecting the central nervous system (CNS). These disorders include neurometabolic disorders such as lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. They also include disorders such as Alzheimer's disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.